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FORMA Therapeutics’ Olutasidenib Demonstrates Positive Phase 1b/2 Results, including Blood-Brain Barrier Penetration and Stable Disease in Patients with IDH1-mutated Glioma

On November 21, 2019 FORMA Therapeutics, Inc., a clinical stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported positive Phase 1b/2 results from an ongoing study in patients with IDH1-mutated gliomas (Press release, Forma Therapeutics, NOV 21, 2019, View Source [SID1234551571]). The data, presented at the 2019 Society for NeuroOncology Annual Meeting (SNO), demonstrate blood-brain barrier penetrance, as exhibited by cerebrospinal fluid exposure, and disease response to olutasidenib, a next-generation IDH1m inhibitor.

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"The safety profile and clinical activity observed to date with olutasidenib in patients with recurrent and often life-threatening gliomas are exciting," said Patrick Kelly, M.D., chief medical officer at FORMA Therapeutics. "In patients with a predominantly enhancing relapsed/refractory glioma, we achieved a clinical response with half of the patients still on study treatment at four months. Particularly for patients with late-stage gliomas where the disease progresses quickly, maintaining disease control holds promise."

FORMA is conducting a Phase 1b/2 study evaluating the safety, efficacy and pharmacokinetics/pharmacodynamics of olutasidenib for patients with advanced solid tumors and gliomas. The trial, 2102-ONC-102, is an open-label, single-agent dose-confirmation study in up to five cohorts (glioma, hepatobiliary tumors, chondrosarcoma, intrahepatic cholangiocarcinoma and other IDH1 mutant solid tumors). Results announced today are based on continuous oral treatment for 24 patients with advanced IDH1 mutant glioma.

"These data create a compelling profile for olutasidenib as a next generation IDH1m inhibitor," said Frank Lee, chief executive officer of FORMA Therapeutics. "Our goal at FORMA is to offer patients first-in-class and best-in-class medicines, and the early results announced today bring us closer to this goal."

Phase 2 of FORMA’s study is ongoing, evaluating both olutasidenib monotherapy and olutasidenib in combination with azacitidine in patients with confirmed IDH1 gene-mutated advanced glioma. Full study results will be reported in 2020.

Presentation Overviews

Phase 1b/2 study of FT-2102, an inhibitor of mutant IDH1, in patients with relapsed/refractory IDH1 mutant gliomas: preliminary safety and clinical activity

E-talk Presentation by Macarena de la Fuente, M.D., Sylvester Cancer Center, University of Miami

Findings presented regarding olutasidenib monotherapy in the Phase 1 cohort of 24 patients with confirmed IDH1 gene-mutated advanced glioma, the majority of which were enhanced and high-grade glioma. Dr. de la Fuente discusses data that:

Demonstrate blood-brain barrier penetration as measured by cerebrospinal fluid exposure and disease control;
Confirm partial response in one patient and stable disease in 10 patients with enhancing glioma with a median duration of treatment of 3.7 months;
Indicate steady-state olutasidenib plasma concentrations in glioma patients within two weeks of initiation of dosing, which remained consistent over time;
Demonstrate acceptable safety profile in patients with relapsed/refractory IDH1-mutated glioma at 150 mg BID; and
Report no trial discontinuations due to treatment-emergent adverse events (TAEs). Grade 3-4 TAEs in greater than 10 percent of patients included increased ALT, increased AST, decreased platelet count, vomiting and hemiparesis. Transaminase elevations resolved without consequential impact in all patients.
FT-2102 – A Potent and Selective Brain Penetrant Inhibitor of Mutant Isocitrate Dehydrogenase

Oral presentation by Maria Ribadeneira, Ph.D., executive director of DMPK and clinical pharmacology at FORMA Therapeutics

Data demonstrate broad in vitro activity against the major IDH1-R132 mutations observed in glioma. Olutasidenib was observed to be more than 1000-fold selective in R132H, the more common mutation observed in glioma, as compared to IDH1 wild type, and showed potent in vivo suppression of tumor 2-HG in a mouse xenograft (cellular accumulation of 2-HG impairs cell differentiation and promotes tumorigenesis). Data also show olutasidenib to be highly brain penetrant in a rat model.

About Olutasidenib (FT-2102)

FORMA Therapeutics’ most advanced clinical asset, olutasidenib, is designed to be a potent and selective next generation inhibitor of mutated isocitrate dehydrogenase 1 (IDH1m) to treat patients with relapsed/refractory acute myeloid leukemia (R/R AML) or myelodysplastic syndrome (MDS), as well as patients with glioma and other solid tumors with an IDH1 mutation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells; when mutated, its activity can promote blood malignancies and solid tumors. IDH1 mutations are present in 7-14% of patients with AML and more than 70% of patients with gliomas. In gliomas, IDH1 mutations occur early in the tumor pathogenesis and persist throughout progression from a neural stem or progenitor cell. Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors, and high-grade gliomas are associated with poor long-term prognosis, with a median survival ranging from five to seven years. Treatment options for relapsed glioma are limited.

Immunitas Therapeutics Launches with $39 Million to Advance Lead Programs to Human Efficacy Studies Based on a Unique Immunology-Focused Drug Development Platform

On November 21, 2019 Immunitas Therapeutics ("Immunitas"), a single cell genomics-based drug discovery company founded by Longwood Fund, reported a $39 million Series A financing led by Leaps by Bayer and Novartis Venture Fund and joined by additional investors including Evotec, M Ventures, Alexandria Venture Investments, and other institutional investors (Press release, Immunitas Therapeutics, NOV 21, 2019, View Source [SID1234551570]). The company plans to use this funding to advance its first programs, monoclonal antibody therapeutics with single agent activity in preclinical models of oncology, to clinical studies.

Underlying the company’s programs is the unique drug development platform crafted by the Immunitas team along with Aviv Regev (Professor of Biology and Core Member of the Broad Institute and Investigator at the Howard Hughes Medical Institute) that dissects the microenvironment of human tumors using single cell genomics-based approaches to identify novel immune targets. The Immunitas platform has generated fully humanized antibodies that act on these targets, advancing to human efficacy studies driven by specific clinical biomarkers, and a breadth of promising druggable cancer targets.

The scientific founders of Immunitas are pioneers in studying the immunobiology of human tumors, including the use of single cell genomics-based techniques and antibody-development techniques:

Kai Wucherpfennig M.D. Ph.D., Professor and Chair of the Department of Cancer Immunology and Virology at the Dana-Farber Cancer Institute and Professor of Neurology at Harvard Medical School,
Mario Suvà M.D., Ph.D., an Associate Professor in the Department of Pathology at Massachusetts General Hospital and at the Broad Institute of MIT and Harvard, and
Dane Wittrup, Ph.D., the Carbon P. Dubbs Professor of Chemical Engineering and Biological Engineering at the Massachusetts Institute of Technology.
"Single cell genomic sequencing has tremendous promise to help unravel the interactions between immune cells and cancer cells in tumors to advance cancer drug development but focusing it appropriately to discover meaningful new targets based on human biology has been challenging," said Dr. Wucherpfennig. "The Immunitas platform is designed to reveal novel and important adaptive and innate immune interactions with tumor cells, which may open up new possibilities in cancer therapy. My scientific co-founders and I look forward to continuing to work with the Immunitas team as they advance this powerful science."

"Our scientific founders are pioneers in the field of single cell sequencing and analysis. They have extensive expertise in deep computational biology, which has enabled us to discover novel therapeutic targets directly from human immunology," said Lea Hachigian, Ph.D., co-founder, director and President of Immunitas Therapeutics. "The data from this platform have also provided us with biomarkers for patient selection, which has potential to accelerate our development plans and provides improved chances for efficacy for individual patients."

Immunitas was founded to directly address the challenge of translating findings from laboratory research in model organisms to meaningful clinical advances in humans. Immunitas focuses on human samples, allowing the company to start with and stay closer to the most relevant and translatable biology for patients.

"One of today’s biggest challenges in oncology is how to efficiently and effectively move preclinical research into human therapies while avoiding the false signals often seen in animal models," said Dr. Jürgen Eckhardt, Head of Leaps by Bayer, Bayer AG’s strategic venture capital unit. "The scientific founders of Immunitas have elegantly solved this problem by dissecting the biology of immune cells in human tumors directly. We are excited to support this approach which has the potential to significantly improve cancer drug development."

Longwood-founded Immunitas also announced key senior management appointments as well as the Board of Directors of the company. Dr. Lea Hachigian is co-founder, director and President of Immunitas as well as a Principal at Longwood Fund. She is also a co-founder and director of TScan Therapeutics. Tarek Samad, Ph.D. is the Chief Scientific Officer at Immunitas Therapeutics. He has over two decades of experience in academia and industry leading small molecule and antibody biologic programs into the clinic. Amanda Wagner joins the company as Vice President of Strategy and Operations with over ten years of biotech experience in similar roles. The Board of Directors includes Dr. Laura Brass, Dr. Jürgen Eckhardt, Dr. Lea Hachigian, Dr. Lucio Iannone, Dr. Christoph Westphal, and Dr. Vincent Xiang.

EMERGENT BIOSOLUTIONS ANNOUNCES GROWTH STRATEGY AND 2024 GOALS AT ANALYST AND INVESTOR DAY

On November 21, 2019 Emergent BioSolutions Inc. (NYSE: EBS) reported its growth strategy over the next five years and announced its 2024 financial and operational goals during the company’s Analyst and Investor Day held in New York City (Press release, Emergent BioSolutions, NOV 21, 2019, View Source [SID1234551569]). Emergent’s senior management shared their vision for continuing to build leadership positions in select public health threat markets and contract development and manufacturing (CDMO), leveraging the company’s unique set of assets, and realizing sustained financial performance and shareholder value creation.

Robert G. Kramer Sr., president and chief executive officer of Emergent BioSolutions, said, "Emergent has developed a growth strategy that supports our desire to make a significant impact on global public health in pursuit of our mission – to protect and enhance life. We are pleased to share this plan, which serves as a roadmap towards our long-term vision of becoming a Fortune 500 company recognized for protecting and enhancing life, driving innovation, and living our values. We are excited by the opportunities ahead and remain steadfastly committed to our patients, customers, employees, and communities."

2020-2024 Corporate Growth Strategy
The new five-year plan builds upon the successful execution of the previous 2016-2020 plan and is guided by a core strategy focused on five pillars:

Execute core business – Deliver core business in products and services;

Grow through M&A – Expand impact on patients and customers while profitably delivering incremental topline revenue;

Strengthen R&D portfolio – Ensure R&D becomes a more meaningful contributor to growth after 2024;

Build scalable capabilities – Invest in operational excellence and innovation to support a growing enterprise that will deliver greater impact; and

Evolve culture – Evolve the organization’s culture to support employee engagement and empowerment.

2024 Financial and Operational Goals
Under the plan, the company has established the following key financial and operational goals to be accomplished by year end 2024:

Generate total revenue of >$2 billion;

Achieve adjusted EBITDA margin of 27% to 30%;

Expand and build scalable leadership positions across current and new global public health threat markets; and

Invest in capabilities, innovation, and operational excellence.

Commenting on the 2024 goals, Richard S. Lindahl, executive vice president and chief financial officer, said, "Emergent has delivered consistent operational excellence and value creation for the past 21 years. Building on our strong financial foundation, we are committed to funding the company’s growth, outlined in our new five-year strategic plan, with continued focus on prudent capital deployment and increasing shareholder value. If successful, we would substantially expand our global impact on public health for the benefit of our patients, partners, and customers worldwide."

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The presentations delivered at the Analyst and Investor Day event today are available on the Company’s website at View Source A replay of the webcast of the event will be made available via the same link shortly after the event’s conclusion and will be accessible for the next six months.

Entry into a Material Definitive Agreement.

On November 21, 2019, BioCryst Pharmaceuticals, Inc. (the "Company") reported that completed its offering of pre-funded warrants to purchase up to an aggregate of 11,764,706 shares of the Company’s common stock ("Common Stock") at an offering price of $1.69 per share (the "Pre-Funded Warrants") pursuant to a Securities Purchase Agreement (the "Purchase Agreement") dated as of November 19, 2019 among the Company and 667, L.P. and Baker Brothers Life Sciences, L.P (Filing, 8-K, BioCryst Pharmaceuticals, NOV 21, 2019, View Source [SID1234551568]). The offering has been registered under the Securities Act of 1933 (the "Securities Act") pursuant to a registration statement on Form S-3 (Registration No. 333-221421) of the Company (as amended, the "Registration Statement"), and a prospectus supplement dated November 19, 2019, filed with the Securities and Exchange Commission pursuant to Rule 424(b) of the Securities Act on November 21, 2019. Gross proceeds to the Company, before expenses, were approximately $19.9 million.

The pre-funded warrants have an exercise price of $0.01 per share, which is subject to adjustment in the event of certain stock dividends and distributions, stock splits, stock combinations, reclassifications or similar events affecting the Company’s common stock and also upon any distributions of assets to the Company’s stockholders. Each pre-funded warrant is exercisable upon issuance. In the event of certain corporate transactions, the holders of the pre-funded warrants will be entitled to receive, upon exercise of the pre-funded warrants, the kind and amount of securities, cash or other property that the holders would have received had they exercised the pre-funded warrants immediately prior to such transaction. The pre-funded warrants do not contain voting rights or any of the other rights or privileges as a holder of the Company’s common stock.

The foregoing summary of the Pre-Funded Warrants does not purport to be complete and is subject to, and qualified in its entirety by, the form of Pre-Funded Warrant attached as Exhibit 4.1 to this Current Report on Form 8-K, which is incorporated herein by reference.

BIO-PATH HOLDINGS ANNOUNCES CLEARANCE OF INVESTIGATIONAL NEW DRUG APPLICATION FOR BP1002

On November 21, 2019 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that the U.S. Food and Drug Administration (FDA) has reviewed and cleared the Investigational New Drug (IND) application for BP1002 (liposomal Bcl-2), the Company’s second drug candidate (Press release, Bio-Path Holdings, NOV 21, 2019, View Source [SID1234551567]). An initial Phase 1 clinical trial will evaluate the ability of BP1002 to treat refractory/relapsed lymphoma and chronic lymphocytic leukemia patients.

BP1002 targets the protein Bcl-2, which is responsible for driving cell survival in up to 60% of all cancers. High expression of Bcl-2 has been correlated with adverse prognosis for patients diagnosed with relapsed, aggressive non-Hodgkin’s lymphoma. Preclinical studies have shown BP1002 to be a potent inhibitor against the Bcl-2 target, and its benign safety profile should enable BP1002 combination therapy with approved agents.

"With this IND submission now accepted by the FDA, the path is now cleared for us to advance our important first-in-human clinical work for BP1002 in cancers with unmet medical need," said Jorge Cortes, M.D., Director of the Georgia Cancer Center and Chairman of the Bio-Path Scientific Advisory Board. "Importantly, BP1002 activity is based on blocking the Bcl-2 messenger RNA and not the BH3 domain, as is the case with venetoclax. As a result, we believe BP1002 may provide an alternative for relapsed venetoclax patients."

The Phase 1 clinical trial is expected to be conducted at several leading cancer centers, including The University of Texas MD Anderson Cancer Center and the Georgia Cancer Center. Initially, a total of six evaluable patients are scheduled to be treated with BP1002 monotherapy in a standard 3+3 design, with a starting dose of 20 mg/m2. The approved treatment cycle is two doses per week over four weeks, resulting in eight doses administered over twenty-eight days.

William G. Wierda, M.D., Ph.D. will serve as Principal Investigator for the trial. Dr. Wierda is a Professor and Center Medical Director for the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Dr. Wierda also serves as Section Chief – Chronic Lymphocytic Leukemia in the Department of Leukemia at MD Anderson.

"This IND clearance for BP1002 marks an important regulatory milestone for Bio-Path, as we progress our second drug candidate into the clinic. Given the encouraging pre-clinical data and safety profile we have seen to-date, we are eager to begin this first-in-human study," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings.

The IND review process was performed by the FDA’s Office of Oncologic Diseases, Division of Hematologic Malignancies and involved a comprehensive review of data submitted by the Company covering pre-clinical studies, safety, chemistry, manufacturing and controls, and the protocol for the Phase 1 clinical trial.