Opexa Therapeutics is developing Tcelna, an autologous T-cell immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). They are currently conducting a Phase IIb clinical trial of Tcelna in SPMS patients. Preliminary data from earlier trials in both relapsing-remitting and SPMS patients showed signs of efficacy, including reduced disability progression, brain atrophy, and relapse rates. If successful, Tcelna has the potential to be the first approved treatment specifically for SPMS, an underserved market with no approved therapies. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen-specific T-cell immunotherapy that targets myelin-reactive T-cells that cause damage in MS. It is currently in a Phase IIb clinical trial called Abili-T for secondary progressive MS (SPMS), which has limited treatment options. Previous clinical trials of Tcelna showed reductions in brain atrophy, disability progression, and relapse rates in SPMS patients. If successful, Abili-T could support Tcelna becoming the first approved treatment for SPMS.
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen specific T-cell immunotherapy that targets myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa is currently conducting a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) called Abili-T. Previous clinical trials of Tcelna showed promising results in reducing brain atrophy, disability progression, and relapse rates in MS patients. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS, which could provide up to $220 million in payments if milestones are
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation march 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin reactive T-cell clones that are believed to trigger an immune response targeting pathogenic myelin reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa Therapeutics Corporate Presentation July 2014OpexaTherapeutics
Opexa Therapeutics is developing precision immunotherapy treatments, with its lead candidate Tcelna in Phase IIb clinical trials for secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are believed to trigger an immune response targeting pathogenic T-cells that destroy myelin. The Phase IIb trial in SPMS has fully enrolled 180 patients and top-line data is expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation june 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa has completed enrollment in its Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) patients and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and commercial milestones are achieved.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTC) that cause damage in MS. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and sales milestones are achieved.
Opexa therapeutics corporate presentation january 2OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy called Tcelna to treat Secondary Progressive Multiple Sclerosis (SPMS). Tcelna aims to specifically target myelin-reactive T-cells that cause damage in MS, without broadly suppressing the immune system. An ongoing Phase IIb trial called Abili-T is testing Tcelna in 180 patients with SPMS over 24 months. The primary endpoint is reduction in brain atrophy. Secondary endpoints include disability progression, relapse rate, and lesion measures. Positive results could support Tcelna as a novel treatment for the underserved SPMS population.
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen-specific T-cell immunotherapy that targets myelin-reactive T-cells that cause damage in MS. It is currently in a Phase IIb clinical trial called Abili-T for secondary progressive MS (SPMS), which has limited treatment options. Previous clinical trials of Tcelna showed reductions in brain atrophy, disability progression, and relapse rates in SPMS patients. If successful, Abili-T could support Tcelna becoming the first approved treatment for SPMS.
Opexa Therapeutics is developing Precision Immunotherapy using Tcelna to treat multiple sclerosis (MS). Tcelna is an antigen specific T-cell immunotherapy that targets myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa is currently conducting a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) called Abili-T. Previous clinical trials of Tcelna showed promising results in reducing brain atrophy, disability progression, and relapse rates in MS patients. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS, which could provide up to $220 million in payments if milestones are
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation march 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy, for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin reactive T-cell clones that are believed to trigger an immune response targeting pathogenic myelin reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa Therapeutics Corporate Presentation July 2014OpexaTherapeutics
Opexa Therapeutics is developing precision immunotherapy treatments, with its lead candidate Tcelna in Phase IIb clinical trials for secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are believed to trigger an immune response targeting pathogenic T-cells that destroy myelin. The Phase IIb trial in SPMS has fully enrolled 180 patients and top-line data is expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa therapeutics corporate presentation june 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa has completed enrollment in its Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) patients and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and commercial milestones are achieved.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTC) that cause damage in MS. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and sales milestones are achieved.
Opexa therapeutics corporate presentation january 2OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy called Tcelna to treat Secondary Progressive Multiple Sclerosis (SPMS). Tcelna aims to specifically target myelin-reactive T-cells that cause damage in MS, without broadly suppressing the immune system. An ongoing Phase IIb trial called Abili-T is testing Tcelna in 180 patients with SPMS over 24 months. The primary endpoint is reduction in brain atrophy. Secondary endpoints include disability progression, relapse rate, and lesion measures. Positive results could support Tcelna as a novel treatment for the underserved SPMS population.
Opexa therapeutics corporate presentation november 21OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis. Their lead product Tcelna is in Phase 2 clinical trials for secondary progressive multiple sclerosis, a later stage form of MS with limited treatment options. Tcelna works by specifically targeting and reducing myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna has the potential to be the first treatment approved for secondary progressive MS and generate over $7 billion in annual sales. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis (MS). Their lead product, Tcelna, is in Phase II clinical trials for secondary progressive MS (SPMS), which affects around 30-45% of MS patients. Tcelna works by programming the immune system to specifically recognize and inhibit myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna could be the first approved treatment for SPMS, representing a potential $7 billion market in North America alone. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa therapeutics corporate presentation december 16OpexaTherapeutics
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of secondary progressive multiple sclerosis (SPMS). It is currently being tested in a Phase IIb clinical trial called Abili-T that aims to enroll 180 patients to receive two annual courses of treatment. The primary endpoint is reduction in whole brain atrophy, with secondary endpoints including measures of disability progression, relapse rate, and lesion activity. Positive results from previous trials support Tcelna's mechanism of reducing myelin-reactive T-cells and stabilizing disease progression in SPMS patients. An option agreement with Merck Serono provides the potential for commercialization if successful.
Opexa Therapeutics provides a summary of its precision immunotherapy platform and clinical programs. Key points include:
- Tcelna in development for multiple sclerosis, with a Phase 2b trial ongoing in secondary progressive MS. Topline data expected in 2016.
- Pipeline expansion includes OPX-212 for neuromyelitis optica, with IND submission planned for mid-2015.
- Clinical trials show Tcelna reduces myelin reactive T-cells and improved outcomes for relapsing remitting MS patients versus placebo in Phase 2b trials.
- Agreement with Merck Serono provides potential for commercial partnership if Phase 3 is initiated for multiple sclerosis indications.
Opexa therapeutics corporate presentation october 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, an autologous T cell immunotherapy, for the treatment of multiple sclerosis (MS). A Phase 2b clinical trial of Tcelna in secondary progressive MS (SPMS) is ongoing, with top-line results expected in late 2016. Previous clinical trials of Tcelna in relapsing-remitting MS showed a 37% reduction in relapse rate compared to placebo and reversal of disability progression in more active patients. Opexa signed an agreement with Merck Serono for development and commercialization of Tcelna in MS worldwide, excluding Japan, which could provide over $220 million in milestones to Opexa.
Opexa therapeutics corporate presentation september 2016OpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their lead asset, Tcelna, is in Phase 2b development for secondary progressive multiple sclerosis and top-line data is expected in the next 1-2 months. They are also developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies. Opexa has secured an option agreement with Merck Serono for Tcelna in MS and could receive up to $25 million in milestones if Merck exercises their option after seeing Phase 2b data.
This document provides a summary of Galena Biopharma's Q3 2016 financial results and corporate update. It discusses interim results from the Phase 3 PRESENT trial of NeuVax showing a potential delay in disease-free survival and highlights the risk of pseudoprogression in cancer immunotherapy trials. It also reviews the company's immunotherapy development pipeline, including NeuVax programs in breast and gastric cancer and GALE-301/302 programs in ovarian and breast cancer. Finally, it discusses the company's cash position and expected milestones for the remainder of 2016.
Cytori Therapeutics is developing novel cell therapies including ECCS-50 for the treatment of hand dysfunction in scleroderma patients. Clinical trials show ECCS-50 improves patient reported outcomes like hand function and reduces symptoms over 24 months. Cytori is currently enrolling two phase 3 trials and plans to submit for FDA and EMA approval in 2018-2019. In 2016, Cytori will launch a managed access program in Europe to provide early access to ECCS-50 for patients while the company seeks full marketing authorization.
Critical Outcome Technologies Inc. is a bioinformatics company that uses its proprietary CHEMSAS platform to reduce the time and cost of drug discovery. Its lead compound, COTI-2, shows promise for treating cancers with p53 mutations and is nearly ready for Phase 1 clinical trials. If successful, COTI-2 could generate hundreds of millions in licensing deals. The company also has additional compounds in development and collaborations that could produce milestone payments in the coming years. Critical Outcome Technologies aims to revolutionize drug discovery through computational simulation and reduce the traditional multi-billion dollar and decade-long process.
Mimotopes greatly increases Imugene's pipeline and B-cell peptide cancer immunotherapy franchise. HER-Vaxx is re-entering the clinic targeting the same receptor as Roche's $8 billion breast cancer drugs with a robust intellectual property portfolio. The company has compelling science, commercially validated targets, leadership and numerous upcoming milestones that could drive valuation over the next 12-24 months.
The document summarizes key information from Pfizer's June 9, 2017 Oncology Analyst Call. It includes:
1) Forward-looking statements about Pfizer's oncology strategy, portfolio, and anticipated performance are subject to risks and uncertainties.
2) Pfizer Oncology has 18 assets in clinical development, 6 in regulatory review/planned submissions in 2017, and 11 immuno-oncology compounds in clinical trials. Key focus areas include supporting breast and prostate cancer communities and establishing avelumab as a backbone PD-L1 therapy.
3) At the 2017 ASCO conference, Pfizer presented data on investigational assets talazoparib and dacomitinib, as well as approved
- PTX has a deep clinical pipeline with 3 ongoing or planned clinical trials, including Phase Ib/II trials of PTX-200 in breast cancer and ovarian cancer, and a planned Phase Ib trial of PTX-100 in AML.
- PTX-200 and PTX-100 are novel cancer drugs targeting the Akt and Ras pathways that have potential to overcome chemotherapy resistance.
- The clinical trials are being conducted at prestigious cancer centers in the US including Moffitt Cancer Center and Albert Einstein College of Medicine.
This corporate presentation from Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius is a late-stage therapeutics development company focused on four development programs using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- The company has a strong balance sheet with $50 million in cash as of June 2018 and low operating costs, positioning it for near-term success.
- Multiple value-creating events are expected in the next 18 months, including regulatory and clinical trial milestones across the pipeline.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET) as a potential third line therapy. Phase 1 and 2 clinical trials showed that GALE-401 maintains platelet lowering effects while reducing peak plasma concentrations and adverse events compared to immediate release anagrelide. The company plans to initiate a Phase 3 trial in Q2 2017 to further evaluate GALE-401 in ET patients who are intolerant or refractory to first and second line therapies such as hydroxyurea and anagrelide.
Investor cytori presentation public website 9 9 15_finalcytoriIR
Cytori Therapeutics is developing Cytori Cell Therapy, which uses a patient's own adipose-derived regenerative cells, for several indications including scleroderma. Scleroderma causes fibrosis and impaired hand function, which is a major cause of disability. Cytori has completed early clinical trials for scleroderma showing good safety and sustained improvements in hand function, pain, and quality of life. Cytori is currently enrolling patients in a Phase 3 pivotal trial in the US and plans to initiate a Phase 2/3 trial in Europe. Preclinical studies demonstrate Cytori Cell Therapy's pleiotropic mechanisms of action in reducing fibrosis.
GALE-401 is a controlled release formulation of anagrelide targeting patients who are intolerant to immediate release anagrelide, the current standard of care for third line treatment of essential thrombocythemia. Phase 1 and 2 clinical trials demonstrated GALE-401's improved pharmacokinetic profile with lower Cmax and longer half-life, maintaining platelet lowering effects while showing better tolerability. A Phase 3 trial is planned to initiate in Q2 2017. NeuVax is an immunotherapy targeting HER2-expressing cancers by eliciting a CD8+ T-cell immune response. Current clinical trials are investigating NeuVax in combination with trastuzumab for the treatment of HER2 1+/2+ breast cancer
Cytori Therapeutics provides an overview of their cell therapy technology and clinical pipeline. Their lead indication is treating hand dysfunction associated with scleroderma using their Cytori Cell Therapy, which involves harvesting a patient's own adipose tissue, processing it to isolate regenerative cells, and delivering the cells back to affected areas. They have an ongoing phase III trial in Europe for scleroderma and anticipate European introduction in 2016 and potential US approval in 2018. Their clinical pipeline also includes trials for knee osteoarthritis, urinary incontinence, and burns. A pilot study of 12 patients with scleroderma found improvements in hand function, Raynaud's scores, and other measures out to 24 months follow up
PharmAust is a clinical-stage oncology company focused on drug repurposing. Its lead product PPL-1 (Monepantel-MPL) successfully completed Phase I/II trials and is now Phase II ready. MPL has potential as a new class of targeted anti-cancer therapy. PharmAust also has a profitable specialty chemicals business and options for veterinary cancer applications through partnerships. The company is significantly undervalued given its pipeline and clinical progress.
ROSALIND is a computer simulation program that uses a patient's tumor gene profile to evaluate all available cancer therapy options and predict the most effective treatments. It can simulate over 10,000 drug combinations in under 2 hours, allowing for a comprehensive and fast analysis. ROSALIND's predictions aim to personalize cancer treatment for each patient and help guide them to therapies with the highest chance of remission or available clinical trials when standard options are unlikely to work. The company envisions ROSALIND being widely adopted to achieve higher remission rates and lower healthcare costs through more personalized cancer care.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa Therapeutics is developing Precision Immunotherapy using attenuated myelin reactive T-cell clones (Tcelna) to treat multiple sclerosis. Tcelna is designed to program the immune system to target pathogenic myelin reactive T-cells that cause demyelination. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in 190 patients with secondary progressive multiple sclerosis, with top-line data expected in late 2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in multiple sclerosis that could provide up to $220 million in payments if milestones are achieved.
Opexa therapeutics corporate presentation november 21OpexaTherapeutics
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis. Their lead product Tcelna is in Phase 2 clinical trials for secondary progressive multiple sclerosis, a later stage form of MS with limited treatment options. Tcelna works by specifically targeting and reducing myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna has the potential to be the first treatment approved for secondary progressive MS and generate over $7 billion in annual sales. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa Therapeutics is developing Precision Immunotherapy using their T-cell platform to treat autoimmune diseases like multiple sclerosis (MS). Their lead product, Tcelna, is in Phase II clinical trials for secondary progressive MS (SPMS), which affects around 30-45% of MS patients. Tcelna works by programming the immune system to specifically recognize and inhibit myelin reactive T-cells that cause damage to the protective myelin sheath surrounding nerves. If successful, Tcelna could be the first approved treatment for SPMS, representing a potential $7 billion market in North America alone. Opexa has an option agreement with Merck for the development of Tcelna in MS.
Opexa therapeutics corporate presentation december 16OpexaTherapeutics
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of secondary progressive multiple sclerosis (SPMS). It is currently being tested in a Phase IIb clinical trial called Abili-T that aims to enroll 180 patients to receive two annual courses of treatment. The primary endpoint is reduction in whole brain atrophy, with secondary endpoints including measures of disability progression, relapse rate, and lesion activity. Positive results from previous trials support Tcelna's mechanism of reducing myelin-reactive T-cells and stabilizing disease progression in SPMS patients. An option agreement with Merck Serono provides the potential for commercialization if successful.
Opexa Therapeutics provides a summary of its precision immunotherapy platform and clinical programs. Key points include:
- Tcelna in development for multiple sclerosis, with a Phase 2b trial ongoing in secondary progressive MS. Topline data expected in 2016.
- Pipeline expansion includes OPX-212 for neuromyelitis optica, with IND submission planned for mid-2015.
- Clinical trials show Tcelna reduces myelin reactive T-cells and improved outcomes for relapsing remitting MS patients versus placebo in Phase 2b trials.
- Agreement with Merck Serono provides potential for commercial partnership if Phase 3 is initiated for multiple sclerosis indications.
Opexa therapeutics corporate presentation october 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, an autologous T cell immunotherapy, for the treatment of multiple sclerosis (MS). A Phase 2b clinical trial of Tcelna in secondary progressive MS (SPMS) is ongoing, with top-line results expected in late 2016. Previous clinical trials of Tcelna in relapsing-remitting MS showed a 37% reduction in relapse rate compared to placebo and reversal of disability progression in more active patients. Opexa signed an agreement with Merck Serono for development and commercialization of Tcelna in MS worldwide, excluding Japan, which could provide over $220 million in milestones to Opexa.
Opexa therapeutics corporate presentation september 2016OpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their lead asset, Tcelna, is in Phase 2b development for secondary progressive multiple sclerosis and top-line data is expected in the next 1-2 months. They are also developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies. Opexa has secured an option agreement with Merck Serono for Tcelna in MS and could receive up to $25 million in milestones if Merck exercises their option after seeing Phase 2b data.
This document provides a summary of Galena Biopharma's Q3 2016 financial results and corporate update. It discusses interim results from the Phase 3 PRESENT trial of NeuVax showing a potential delay in disease-free survival and highlights the risk of pseudoprogression in cancer immunotherapy trials. It also reviews the company's immunotherapy development pipeline, including NeuVax programs in breast and gastric cancer and GALE-301/302 programs in ovarian and breast cancer. Finally, it discusses the company's cash position and expected milestones for the remainder of 2016.
Cytori Therapeutics is developing novel cell therapies including ECCS-50 for the treatment of hand dysfunction in scleroderma patients. Clinical trials show ECCS-50 improves patient reported outcomes like hand function and reduces symptoms over 24 months. Cytori is currently enrolling two phase 3 trials and plans to submit for FDA and EMA approval in 2018-2019. In 2016, Cytori will launch a managed access program in Europe to provide early access to ECCS-50 for patients while the company seeks full marketing authorization.
Critical Outcome Technologies Inc. is a bioinformatics company that uses its proprietary CHEMSAS platform to reduce the time and cost of drug discovery. Its lead compound, COTI-2, shows promise for treating cancers with p53 mutations and is nearly ready for Phase 1 clinical trials. If successful, COTI-2 could generate hundreds of millions in licensing deals. The company also has additional compounds in development and collaborations that could produce milestone payments in the coming years. Critical Outcome Technologies aims to revolutionize drug discovery through computational simulation and reduce the traditional multi-billion dollar and decade-long process.
Mimotopes greatly increases Imugene's pipeline and B-cell peptide cancer immunotherapy franchise. HER-Vaxx is re-entering the clinic targeting the same receptor as Roche's $8 billion breast cancer drugs with a robust intellectual property portfolio. The company has compelling science, commercially validated targets, leadership and numerous upcoming milestones that could drive valuation over the next 12-24 months.
The document summarizes key information from Pfizer's June 9, 2017 Oncology Analyst Call. It includes:
1) Forward-looking statements about Pfizer's oncology strategy, portfolio, and anticipated performance are subject to risks and uncertainties.
2) Pfizer Oncology has 18 assets in clinical development, 6 in regulatory review/planned submissions in 2017, and 11 immuno-oncology compounds in clinical trials. Key focus areas include supporting breast and prostate cancer communities and establishing avelumab as a backbone PD-L1 therapy.
3) At the 2017 ASCO conference, Pfizer presented data on investigational assets talazoparib and dacomitinib, as well as approved
- PTX has a deep clinical pipeline with 3 ongoing or planned clinical trials, including Phase Ib/II trials of PTX-200 in breast cancer and ovarian cancer, and a planned Phase Ib trial of PTX-100 in AML.
- PTX-200 and PTX-100 are novel cancer drugs targeting the Akt and Ras pathways that have potential to overcome chemotherapy resistance.
- The clinical trials are being conducted at prestigious cancer centers in the US including Moffitt Cancer Center and Albert Einstein College of Medicine.
This corporate presentation from Caladrius Biosciences provides an overview of the company and its pipeline. Key points include:
- Caladrius is a late-stage therapeutics development company focused on four development programs using CD34+ cells for ischemic repair and T regulatory cells for immune modulation.
- The company has a strong balance sheet with $50 million in cash as of June 2018 and low operating costs, positioning it for near-term success.
- Multiple value-creating events are expected in the next 18 months, including regulatory and clinical trial milestones across the pipeline.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET) as a potential third line therapy. Phase 1 and 2 clinical trials showed that GALE-401 maintains platelet lowering effects while reducing peak plasma concentrations and adverse events compared to immediate release anagrelide. The company plans to initiate a Phase 3 trial in Q2 2017 to further evaluate GALE-401 in ET patients who are intolerant or refractory to first and second line therapies such as hydroxyurea and anagrelide.
Investor cytori presentation public website 9 9 15_finalcytoriIR
Cytori Therapeutics is developing Cytori Cell Therapy, which uses a patient's own adipose-derived regenerative cells, for several indications including scleroderma. Scleroderma causes fibrosis and impaired hand function, which is a major cause of disability. Cytori has completed early clinical trials for scleroderma showing good safety and sustained improvements in hand function, pain, and quality of life. Cytori is currently enrolling patients in a Phase 3 pivotal trial in the US and plans to initiate a Phase 2/3 trial in Europe. Preclinical studies demonstrate Cytori Cell Therapy's pleiotropic mechanisms of action in reducing fibrosis.
GALE-401 is a controlled release formulation of anagrelide targeting patients who are intolerant to immediate release anagrelide, the current standard of care for third line treatment of essential thrombocythemia. Phase 1 and 2 clinical trials demonstrated GALE-401's improved pharmacokinetic profile with lower Cmax and longer half-life, maintaining platelet lowering effects while showing better tolerability. A Phase 3 trial is planned to initiate in Q2 2017. NeuVax is an immunotherapy targeting HER2-expressing cancers by eliciting a CD8+ T-cell immune response. Current clinical trials are investigating NeuVax in combination with trastuzumab for the treatment of HER2 1+/2+ breast cancer
Cytori Therapeutics provides an overview of their cell therapy technology and clinical pipeline. Their lead indication is treating hand dysfunction associated with scleroderma using their Cytori Cell Therapy, which involves harvesting a patient's own adipose tissue, processing it to isolate regenerative cells, and delivering the cells back to affected areas. They have an ongoing phase III trial in Europe for scleroderma and anticipate European introduction in 2016 and potential US approval in 2018. Their clinical pipeline also includes trials for knee osteoarthritis, urinary incontinence, and burns. A pilot study of 12 patients with scleroderma found improvements in hand function, Raynaud's scores, and other measures out to 24 months follow up
PharmAust is a clinical-stage oncology company focused on drug repurposing. Its lead product PPL-1 (Monepantel-MPL) successfully completed Phase I/II trials and is now Phase II ready. MPL has potential as a new class of targeted anti-cancer therapy. PharmAust also has a profitable specialty chemicals business and options for veterinary cancer applications through partnerships. The company is significantly undervalued given its pipeline and clinical progress.
ROSALIND is a computer simulation program that uses a patient's tumor gene profile to evaluate all available cancer therapy options and predict the most effective treatments. It can simulate over 10,000 drug combinations in under 2 hours, allowing for a comprehensive and fast analysis. ROSALIND's predictions aim to personalize cancer treatment for each patient and help guide them to therapies with the highest chance of remission or available clinical trials when standard options are unlikely to work. The company envisions ROSALIND being widely adopted to achieve higher remission rates and lower healthcare costs through more personalized cancer care.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are designed to program the immune system to target pathogenic myelin-reactive T-cells. Opexa has an ongoing Phase IIb clinical trial of Tcelna in SPMS and expects to complete enrollment in mid-2014, with top-line data expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Opexa Therapeutics is developing Precision Immunotherapy using attenuated myelin reactive T-cell clones (Tcelna) to treat multiple sclerosis. Tcelna is designed to program the immune system to target pathogenic myelin reactive T-cells that cause demyelination. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in 190 patients with secondary progressive multiple sclerosis, with top-line data expected in late 2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in multiple sclerosis that could provide up to $220 million in payments if milestones are achieved.
Opexa Therapeutics Corporate Presentation - May 2013OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a personalized immunotherapy for the treatment of multiple sclerosis (MS). Tcelna aims to specifically target myelin-reactive T-cells (MRTCs) that cause damage to the myelin sheath in MS patients. Clinical trials have shown Tcelna reduces MRTCs and relapses in both relapsing-remitting and secondary progressive MS patients. Opexa has an agreement with Merck for the development of Tcelna in MS that could provide up to $220 million in milestones for Opexa.
Opexa Therapeutics is conducting a Phase IIb clinical trial of its T-cell immunotherapy Tcelna to treat Secondary Progressive Multiple Sclerosis (SPMS). The company recently signed an option agreement with Merck Serono that could provide up to $225 million in payments if Merck exercises its option to acquire rights to Tcelna in MS. Opexa is also exploring opportunities to develop Tcelna and its stem cell platform in other autoimmune diseases. The ongoing Phase IIb trial is evaluating Tcelna's ability to reduce brain atrophy and progression in 180 SPMS patients over two years.
Tcelna is a precision immunotherapy under development by Opexa Therapeutics for the treatment of multiple sclerosis (MS). Opexa is currently conducting a Phase IIb clinical trial of Tcelna in patients with secondary progressive MS (SPMS). Previous clinical trials of Tcelna in both relapsing-remitting and SPMS patients showed promising results, including a 37% reduction in annualized relapse rate in a Phase IIb RRMS trial. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in MS that could provide over $220 million in payments if milestones are achieved.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead program, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis (SPMS). If successful, Tcelna has the potential to address an unmet medical need as there is currently only one approved drug for SPMS that is not suitable for chronic use due to side effects. Opexa has an agreement with Merck Serono to develop Tcelna for MS indications worldwide, excluding Japan. Topline results from the Phase 2b SPMS trial are expected in late 2016.
Opexa Therapeutics December 2015 Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their T-cell immunotherapy Tcelna is in Phase 2b clinical trials for secondary progressive multiple sclerosis. Preclinical studies of OPX-212 showed a reduction of AQP4 reactive T-cells in a murine model of neuromyelitis optica, an orphan disease with no approved therapies. Opexa secured funding to advance OPX-212 into a Phase I/II clinical trial in 2016 pending regulatory approval. Their proprietary ImmPath platform allows for personalized T-cell therapies tailored for individual patient's disease profiles.
Opexa therapeutics corporate presentation july 2016OpexaTherapeutics
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead candidate, Tcelna, is in a Phase 2b clinical trial for secondary progressive multiple sclerosis, with top-line results expected in early Q4 2016. Their platform also supports OPX-212 for neuromyelitis optica, an orphan disease with no approved therapies. Opexa has secured an option agreement with Merck Serono for Tcelna in multiple sclerosis and has established preclinical validation of their approach in animal models of disease.
Opexa Therapeutics presented information on their Precision Immunotherapy platform and lead programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown promise in reducing reactive T-cells and relapse rates in prior studies. Their platform also supports development of OPX-212 for neuromyelitis optica, a rare disease with no approved therapies. Opexa has an option agreement with Merck Serono for Tcelna in MS and recently secured $5 million in funding to advance OPX-212 into clinical trials for NMO.
Opexa Therapeutics Corporate Presentation September 2015OpexaTherapeutics
Opexa Therapeutics presented information on their Precision Immunotherapy platform and lead programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown promise in reducing reactive T-cells and relapse rates in prior studies. Their platform also aims to develop OPX-212 as a potential first-in-class therapy for neuromyelitis optica, an orphan disease with no approved treatments. Opexa has an option agreement with Merck Serono for Tcelna in multiple sclerosis and recently secured $5 million in funding to advance OPX-212 into clinical studies.
Opexa therapeutics corporate presentation march 2016OpexaTherapeutics
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Its lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has potential partnerships with Merck Serono. Opexa is also developing OPX-212 for neuromyelitis optica, an orphan disease with no approved therapies. Preclinical studies show OPX-212 reduces pathogenic T-cells in animal models. Opexa's platform aims to selectively eliminate harmful immune cells while leaving the rest of the immune system intact.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead candidate, Tcelna, is in a Phase 2b clinical trial for secondary progressive multiple sclerosis (SPMS) with top-line results expected in early Q4 2016. Tcelna uses the proprietary ImmPath platform to prime the immune system to target disease-causing T-cells. Previous clinical trials of Tcelna showed a significant reduction in myelin reactive T-cells and a 37% improvement over placebo in annualized relapse rate for relapsing-remitting MS. Opexa has an option agreement with Merck Serono for worldwide rights to develop Tcelna for MS indications.
Opexa therapeutics corporate presentation february 2016 webOpexaTherapeutics
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead program, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has received Fast Track designation from the FDA. Opexa is also developing OPX-212 for neuromyelitis optica, an orphan disease with no approved therapies. Preclinical studies of OPX-212 showed a statistically significant reduction in pathogenic T-cells. Topline results for Tcelna in SPMS are expected in the second half of 2016, which would be a key inflection point if positive.
Opexa Therapeutics Corporate Presentation October 2015OpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy programs, Tcelna and OPX-212. Tcelna is in a Phase 2b clinical trial for secondary progressive multiple sclerosis and has shown a 37% reduction in relapses in prior trials. OPX-212 is being developed for neuromyelitis optica, an orphan disease with no approved therapies. Opexa expects to file an IND for OPX-212 by the end of 2015 after completing preclinical studies. The company highlighted their personalized T-cell immunotherapy platform, clinical progress, pipeline expansion opportunities, and management team experience in drug development.
Opexa Therapeutics August 2015 OPXA Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and key programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown signs of stabilizing disease progression. Tcelna works by reducing myelin reactive T-cells that damage the myelin sheath. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in multiple sclerosis. Additionally, Opexa is developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown signs of stabilizing disease progression in previous trials. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in multiple sclerosis. Recent milestones include completing enrollment in the Tcelna Phase 2b trial for SPMS and receiving a $3 million payment from Merck Serono. Opexa is also developing OPX-212 for neuromyelitis optica and expects to file an IND by the end of 2015.
This document provides an overview and summary of Opexa Therapeutics' rights offering. Key points include:
- Opexa is conducting a rights offering of up to 28.8 million units to fund its ongoing Phase 2b trial of Tcelna in secondary progressive multiple sclerosis and a potential Phase 1/2 trial of OPX-212 in neuromyelitis optica.
- Proceeds will also be used for general corporate purposes.
- Tcelna utilizes Opexa's proprietary precision immunotherapy platform and has received Fast Track designation from the FDA for secondary progressive MS. Prior clinical trials in both relapsing-remitting and secondary progressive MS showed encouraging results.
- The rights offering is
CLBS Corporate Slide Presentation March 2018Steve Sizer
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Cell Therapy Expansion and Challenges in Autoimmune DiseaseHealth Advances
There is increasing confidence that cell therapies will soon play a role in the treatment of autoimmune disorders, but the extent of this impact remains to be seen. Early readouts on autologous CAR-Ts in lupus are encouraging, but manufacturing and cost limitations are likely to restrict access to highly refractory patients. Allogeneic CAR-Ts have the potential to broaden access to earlier lines of treatment due to their inherent cost benefits, however they will need to demonstrate comparable or improved efficacy to established modalities.
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2. 2
Forward-Looking Statements
• This investor presentation contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of
the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Statements contained in this
presentation, other than statements of historical fact, constitute “forward-looking statements.” The words “expects,” “believes,”
“anticipates,” “estimates,” “may,” “could,” “intends,” and similar expressions are intended to identify forward-looking statements.
Forward-looking statements do not constitute guarantees of future performance. Investors are cautioned that statements which are not
strictly historical statements, including, without limitation, statements regarding the development of our product candidate, Tcelna
(imilecleucel-T), constitute forward-looking statements.
• Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially
from those anticipated. These risks and uncertainties include, but are not limited to, risks associated with: market conditions; our capital
position; the rights and preferences provided to the Series A convertible preferred stock and investors in the convertible secured notes we
issued in July 2012 (including a secured interest in all of our assets); our ability to compete with larger, better financed pharmaceutical
and biotechnology companies; new approaches to the treatment of our targeted diseases; our expectation of incurring continued losses;
our uncertainty of developing a marketable product; our ability to raise additional capital to continue our development programs
(including to undertake and complete any ongoing or further clinical studies for Tcelna); our ability to satisfy various conditions required
to access the financing potentially available under the purchase agreements with Lincoln Park Capital Fund, LLC (“Lincoln Park”) or sell
shares of our common stock to Lincoln Park or under our at-the-market (ATM) facility; our ability to maintain compliance with NASDAQ
listing standards; the success of our clinical trials (including the Phase IIb trial for Tcelna in Secondary Progressive Multiple Sclerosis
which, depending upon results, may determine whether Ares Trading SA (“Merck”) elects to exercise its option for an exclusive license to
Tcelna for the treatment of multiple sclerosis (the “Option”)); whether Merck exercises the Option and, if so, whether we receive any
development or commercialization milestone payments or royalties from Merck pursuant to the Option; our dependence (if Merck
exercises the Option) on the resources and abilities of Merck for the further development of Tcelna; the efficacy of Tcelna for any
particular indication; our ability to develop and commercialize products; our ability to obtain required regulatory approvals; our
compliance with all FDA regulations; our ability to obtain, maintain and protect intellectual property rights; the risk of litigation
regarding our intellectual property rights or the rights of third parties; the success of third party development and commercialization
efforts with respect to products covered by intellectual property rights that we may license or transfer; our limited manufacturing
capabilities; our dependence on third-party manufacturers; our ability to hire and retain skilled personnel; our volatile stock price; and
other risks detailed in our filings with the SEC.
• These forward-looking statements speak only as of the date made. We assume no obligation or undertaking to update any forward-
looking statements to reflect any changes in expectations with regard thereto or any change in events, conditions or circumstances on
which any such statement is based. You should, however, review additional disclosures we make in our Annual Reports on Form 10-K,
Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K filed with the SEC.
3. 3
Introduction to Opexa
• Antigen specific T-cell immunotherapy platform with potential in multiple
autoimmune diseases
• Strong Patent Estate: 50 patents issued on T-cell platform (US and ROW,
collectively)
• Precision Immunotherapy potentially optimizes benefit-risk profile
• Lead product, Tcelna®, under development for Multiple Sclerosis
• Currently conducting a Phase IIb clinical trial in Secondary Progressive MS (SPMS)
• Limited treatment options currently available for SPMS
• Potential SPMS market in North America alone could exceed $7 Billion
• Fast Track designation from the U. S. Food and Drug Administration (FDA) for the
treatment of SPMS
• Option Agreement with Merck Serono, a strong commercial partner
4. 4
Merck Serono Agreement for MS indication
• February 2013 option and license agreement with Merck Serono
– Up to $220 million in additional payments
• Option exercise $25 million for starting Phase III/ or $15 million if another Phase II
• $35 million FDA filing, approval and commercialized in US
• $30 million for EU filing, approval and commercialization in at least three countries
• RRMS development and commercialization of up to $40 million
– One time commercial milestones of up to $85 million
• Royalties ranging from 8% to 15% of annual net sales with step-ups
occurring when net sales exceed $500 million, $1 B & $2 B
• Opexa maintains key rights:
– Development and commercialization rights to Tcelna in Japan
– Certain manufacturing rights
– Co-development funding option in exchange for increased royalties
– Rights to all other disease indications
5. 5
Tcelna in Secondary Progressive Multiple Sclerosis
• Patients initially experience a relapsing-
remitting course then transition to SPMS
• SPMS patients experience worsening QOL /
disability with or without relapses
• Over 450,000 patients in North America
and 2 million world wide have MS
• Approximately 30-45% of MS patients can be classified as Secondary Progressive
• Tcelna is being pursued for this SPMS indication
• Potential SPMS market in the North America alone could exceed $7 billion [150,000
SPMS patients at average cost of $50,000 per year of treatment]
• No SPMS treatment approved by EMA; only one approved by FDA with limited use
due to toxicity
• Upon successful clinical development, Tcelna has the potential to be the treatment of
choice in SPMS
6. 6
Tcelna Addressing the Root Cause of Multiple Sclerosis
Preventing Demyelination and Enabling Remyelination
• Myelin Reactive T-cells (MRTC) cross the blood brain
barrier, enter the brain, bind to antigen presenting cells
(APC) causing release of pro-inflammatory cytokines
which lead to a two pronged attack through:
i. Activation of microglial cells leading to destruction of myelin
sheath, the protective coating of nerve fibers
ii. Destruction of oligodendroglial cells which are responsible for
producing myelin
Result Destruction of the myelin sheath with
barriers to prevent remyelination
• T cells play a vital role in our immune system. Only a very
small proportion develop into MRTC. Most competing
therapies non-specifically suppress all T-cells
(including beneficial T cells) and/or focus on preventing
their entry into the central nervous system, which may
lead to serious side effects.
Opexa’s Strategy
Tcelna programs the immune system to specifically
recognize MRTCs as pathogenic thereby inhibiting further
destruction of the myelin sheath and potentially enabling
remyelinationAdapted by permission from Macmillan Publishers Ltd: NATURE REVIEWS
IMMUNOLOGY 3, 483-492 (June 2003), copyright (2003)
7. 7
Single Cycle Manufacturing Process:
Annual course of treatment (5 doses) from one blood draw
Cryopreservation
Formulation/
Irradiation of each
dose as required
Administration: 5
subcutaneous
injections/year
Manufacturing and QC Dispensation
35 days
Epitope Profiling
1 day14 days
- Red Cross
- Blood Group Alliance
Epitope Profiling Expansion of antigen specific T-cells
8. 8
Tcelna Development Program
Broad Spectrum of MS patients treated
Phase Completion
Dates
Population Total N Treatment
Duration
(months)
Tcelna Placebo
Baylor 1998
RRMS, PPMS,
SPMS (26)
114 Up to 24 months 114
Phase I dose
escalation
2006
RRMS (5)
SPMS (6)
16 12 16 -
Phase I/II
Open label
retreatment
2007
RRMS (9)
SPMS (4) 13 12 13 -
Phase IIb
TERMS
2008 RRMS, CIS 150 12 100 50
Phase IIb
extension
OLTERMS
2008 RRMS, CIS 38
At least one dose
post TERMS
15 from
placebo
arm
Phase IIb
Abili-T
1st Half 2016* SPMS 180* 24* 90* 90*
* Expected upon completion of ongoing SPMS Abili-T trial
9. 9
TERMS Study - Prospective Analysis in
More Active or Progressive Patients
Sub-population of patients (n=50) with more progressed/active disease
profile (baseline ARR >1) most closely mirrors SPMS patients
2.4
2.23
2.2
2.39
2.1
2.15
2.2
2.25
2.3
2.35
2.4
2.45EDSSScore(Mean)
Baseline Week 52
Tcelna
Placebo
(p=0.045)
0.28
0.63
0
0.1
0.2
0.3
0.4
0.5
0.6
0.7
ARR
(relapses/patient/yr)
Tcelna
Placebo
55%
n=18n=32
-0.04
-0.32
-0.35
-0.3
-0.25
-0.2
-0.15
-0.1
-0.05
0
Brainvolumechange
(%)
Tcelna
Placebo88%
n=18n=32
88% Reduction in
Brain Atrophy
Percent Brain Volume Change
at Week 52
Statistically Significant
Improvement in Disability (p=0.045)
55% Reduction in ARR
Annualized Relapse Rate (ARR)
at Week 52
Change in Disability (EDSS)
at Week 52
These Data Support Phase IIb Program in SPMS
10. 10
Tcelna Stabilizes Disease in SPMS at 2 Years
80%
20%
40%
0%
20%
40%
60%
80%
100%
PercentofPatientsShowing
DiseaseStabilization
Stabilization vs. Historical Progression
Stable
Progressed
80% of subjects treated with Tcelna showed no
further disease progression by EDSS at 2 years
Historical Disease
Progression
Tcelna Open Label
(n=35)
*A small percentage of patients in pooled analysis showed an
improvement (i.e. decrease in progression)
**Historical control: ESIMS Study,
published Hommes Lancet 2004
11. 11
Abili-T : Landmark trial in SPMS
• Abili-T Phase IIb clinical trial in SPMS is ongoing
– Double-blind, 1:1 randomized, placebo-controlled
– Inclusion criteria: Secondary Progressive MS with EDSS of 3 to 6
– 68 patients enrolled as of August 8, 2013
– Immune Monitoring program conducted on a blinded basis
• Fast Track designation granted by FDA for Tcelna in SPMS
• 180 Patients expected to be enrolled
– SPMS population
– 30 sites in USA and Canada
• 2 annual courses of personalized therapy
• Top line Data in 1st Half of 2016
12. 12
Efficacy Assessments
• Primary Endpoint
– Whole-brain atrophy
• Secondary Endpoints
– Sustained progression measured by EDSS
– Time to sustained progression
– T2 lesions progressing to hypointense lesions (black holes)
– Change in EDSS
– Annualized Relapse Rate (ARR)
– Change in MSFC Assessment of disability
– Change in Symbol Digit Modality Test (SDMT)
• Exploratory Endpoints
– Quality of life assessment by MSQLI
– Gd-enhancing lesion volume with increasing MTR
– Gd-enhancing lesion volume with decreasing MTR
– Change in MTR in normal-appearing white matter
– Changes in T-regulatory cell repertoire and function
13. 13
Immune Monitoring Program
Phenotypical Analysis
(CD4 and CD8)
Pro-inflammatory Anti-inflammatory
TH1 Treg
TH17 Tr1
TH2
IL-2 IL-4
IL-6 IL-5
IL-12 IL-10
IL-17E, IL-17F
IL-23 IL-27
TNFα TGFβ
IFNϒ BDNF
Plausible indicators of
Tcelna efficacy
• Reduction in Th1/TH17
• Increase in Treg/Tr1 cells
• Reduction in proinflammatory
cytokines (IL-12, IL-23, IFNϒ,
TNFα)
• Increase in anti-inflammatory
cytokines (IL-10, IL-27)
• Loss of proinflammatory
monocyte markers (CD16/HLA-
DR, CCR5)
• Gain in anti-inflammatory
monocyte markers (PDL-1, HLA-
G, ICOS)
2013 2014 20152012
Trial initiation
Immuno vigilance
Whole blood analysis:
Absolute frequency of T-
cells, B-cells, Dendritic cells
and Monocytes
• Goal is to show no systemic
impact on immune
response, i.e. Tcelna is a
Precision Immunotherapy
• Tcelna differentiates by not
depleting a broad spectrum
of T-cells
90 patients at dose 5 180 patients at 10 doses40 patients at dose 5
2016
End of Study
14. 14
Financials
Cash and Cash Equivalents (MM) as of June 30, 2013 $5.0 (1)
Estimated Net Proceeds (MM) from August 2013 common stock offering $17
Convertible Debt (MM) (2) $3.2
Shares outstanding (MM) as of September 4, 2013 ~20.8
Warrants (MM) (3) ~3.1
Stock Options (MM) (4) ~1.1
(1) Excludes $500,000 of restricted cash subject to a deposit control agreement
(2) Principal outstanding on convertible secured promissory notes
(3) Weighted average exercise price =$4.12 as of June 30, 2013
(4) Weighted average exercise price = $4.46 as of June 30, 2013
15. 15
Experienced Management Team and Board of Directors
Neil Warma, President & CEO, Director
› 19+ years international healthcare experience with large Pharma and emerging
biotechnology companies
› Former Senior Management, Novartis Pharmaceuticals, Basel, Switzerland
› Former CEO, Viron Therapeutics, Inc.
› Co-founder and President of MedExact Inc., a company subsequently acquired
Karthik Radhakrishnan, Chief Financial Officer
› 10+ years of health care capital markets experience
› Formerly, Vice President at ING Investment Management
› MBA, MS in Engineering, CFA charter holder
Don Healey, Ph.D., Chief Scientific Officer
› 25+ years of experience in cellular immunology and immune regulation
› Former Director of Immunology, Argos Therapeutics
Donna Rill, Chief Development Officer
› 30 years in cell and gene therapy research and clinical application
› Designed and validated cGMP Cell & Gene Therapy Laboratories, Vector Production
facilities, and Translational Research Labs
Kenny Frazier, VP of Clinical Dev. and Regulatory Affairs
› 24 years of extensive clinical and regulatory experience
› Formerly, Head of Clinical Operations, Lexicon Pharmaceuticals and Tanox, Inc.
Board of Directors
Gail J. Maderis
CEO, BayBio, Former CEO
of Five Prime Therapeutics,
Founder of Genzyme
Molecular Oncology
Michael S. Richman
CEO, Amplimmune
Scott B. Seaman
Executive Director,
Alkek Foundation
David E. Jorden
Interim CEO, Nanospectra
Biosciences
Neil K. Warma
CEO, Opexa
16. 16
SPMS Scientific Advisory Board
Dawn McGuire, M.D., FAAN (Chair)
• Advisory Council of the Gill Heart Institute
• Former Vice President of Clinical Research at Elan Pharmaceuticals
Hans-Peter Hartung, M.D
• Chair of Neurology at Heinrich-Heine University, Düsseldorf
• President ECTRIMS, World Health Organization Advisory Board on MS
Mark S. Freedman, M.D.
• Director of the Multiple Sclerosis Research Unit at Ottawa Hospital
• Multiple Sclerosis Society of Canada, National MS Society (USA)
• ACTRIMS committee member
Clyde Markowitz, M.D.
• Director of MS Center at the University of Pennsylvania
Doug Arnold, M.D.
• James McGill Professor Neurology and Neurosurgery at the Montreal Neurological Institute
Edward Fox, M.D., Ph.D.
• Director of Multiple Sclerosis Clinic of Central Texas
• Advisory Committee, Lone Star Chapter of the National Multiple Sclerosis Society
17. 17
Investment Thesis
• T-cell platform company with Fast Track designation in SPMS
• Strong Intellectual property with 50 issued patents
• Esteemed Scientific Advisory Board
• Precision Immunotherapy potentially optimizes benefit-risk profile
• Targeting an unmet medical need in a potentially substantial market
• Option Agreement with Merck Serono, a strong commercial partner
• Replacement value of company is multiples of present market cap
• Attractive potential risk-reward profile for long term/value investors
• Goal-oriented management team focused on value creation
19. 19
Tcelna Manufacturing:
Personalization followed by Expansion Step
The Epitope Profiling Assay (EPA)
• Screen peripheral blood for Myelin-Reactive T-cells (MRTCs), and
mapping of immunodominant epitopes to MBP, MOG and PLP
– 109 overlapping peptides encompassing MBP, MOG and PLP
– Interferon gamma response to individual peptide pools defines positive response
in 7 day assay
ImmPathTM Process
• Procure unit of blood from which up to six T-cell lines reactive with
immunodominant myelin peptides are generated and pooled as a
patient-specific Tcelna product
– Manufacturing performed under GMP/GTPs in functionally closed system
– Process generates a year of Tcelna doses from a single unit of blood
20. 20
Year 2
Year 3
Tcelna Manufacturing: Precision Medicine
Proprietary Assay Enables Annual Personalized Treatments
Year 1
Conduct analysis of 109 peptides from all three key myelin proteins (MBP, MOG, PLP)
• Re-assess epitope profile annually
to identify epitope shift
• Develop newly personalized
formulation annually based on
evolved epitope profile