CELL AND GENE THERAPY

Discovery Life Sciences Launches GMP Compliant Discovery Leukopaks™ to Accelerate Research and Development of Cell and Gene Therapies

Discovery Life Sciences | October 28, 2020

Discovery Life Sciences™ (Discovery), a global leader in biospecimen, genomic sequencing, cell, and immunohistochemistry (IHC) services, announces the launch of GMP-compliant Discovery Leukopaks™ and other cell-based products for use in human cell and gene therapy research, development and manufacturing programs. Cell and gene therapies have revolutionized treatment approaches to cancer and other complex diseases. Acceleration of the manufacturing and development of these novel therapies requires fast and reliable access to high quality, well-characterized human cellular raw materials.  Discovery's reputation for speed and quality will be a boon to researchers around the globe. Discovery's launch of GMP-compliant Discovery Leukopaks and derivatives adds to Discovery's existing RUO (research-use-only) fresh and cryopreserved leukopaks and isolated immune cell populations.  Discovery's next-generation short and long read sequencing and flow cytometry services can be incorporated to provide highly characterized, reliable human cellular materials for research, development, and GMP manufacturing.

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CELL AND GENE THERAPY

Shoreline Biosciences Announces $140M Financing To Advance Pipeline Of Next Generation Cell Immunotherapies

Shoreline Biosciences | November 02, 2021

Shoreline Biosciences, Inc. (Shoreline), a biotechnology company developing intelligently designed allogeneic off-the-shelf, standardized, and targeted induced pluripotent stem cells (iPSC) derived natural killer (NK) and macrophage cellular immunotherapies, today announced the completion of a $140M financing, led by Ally Bridge Group, with participation from new investors Eventide Asset Management, BeiGene, Irving Investors, Kingdon, NS Investment, Piper Heartland Healthcare Capital, and Superstring. Existing investors Boxer Capital, BVF Partners, L.P., Commodore Capital, Cormorant Asset Management, Janus Henderson Investors, Kite, a Gilead Company, Stork Capital, Wedbush Healthcare Partners and an undisclosed leading global investment firm, also participated. In connection with the financing, Frank Yu, Founder, CEO and CIO of Ally Bridge Group, joined the Shoreline Board of Directors. "We are proud of Shoreline's tremendous progress over the past year, including next generation, more persistent and more effective iPSC-derived NK cells, enhancing our core technologies, advancing our preclinical programs, establishing transformative and validating partnerships with Kite and BeiGene, creating smart manufacturing methods, and attracting exceptional talent to support our next phase of development and growthWith this financing and our corporate partnerships, Shoreline has now more than $300 million to continue building our pipeline of safe, effective and affordable cellular immunotherapies for both solid tumors and hematologic malignancies." Kleanthis G. Xanthopoulos, Ph.D., Shoreline's co-founder, Chairman and Chief Executive Officer "Having invested in – and followed closely for several years -- the iPSC-derived NK cells-based therapeutics field which has made highly encouraging progress in the clinic, we are convinced of the true differentiation demonstrated by the Shoreline technology platform. Ally Bridge Group, a leading investor in best-in-class cell therapy companies from oncology to autoimmune diseases, expects Shoreline to be a new category leader," said Frank Yu, Founder, CEO and CIO of Ally Bridge Group. The proceeds from the financing will allow Shoreline to continue the advancement of its proprietary iPSC platform focused on developing next generation natural killer (NK) cell and macrophage-cell therapies, create potent and persistent NK cell-specific Chimeric Antigen Receptors (CARs) as well as switchable CAR-NK cell engagers and macrophage-specific CARs to treat blood cancers, solid tumors, and other health conditions. With the close of this financing, Shoreline is well-capitalized with greater than $300 million, including committed partnership R&D funding, to execute on its goals and advance its pipeline. About Shoreline Biosciences Shoreline is dedicated to creating next-generation cellular immunotherapies for cancer that overcome the current limitations of first-generation cell therapy products. Shoreline is building a pipeline of natural killer cell and macrophage-cell therapy candidates derived from its deep expertise in iPSC differentiation methods and genetic reprogramming of disease relevant pathways. Shoreline has strategic partnerships with Kite, a Gilead Company, and BeiGene, a global biotechnology company, Contract Development and Manufacturing Organization partnerships with well-established organizations, and is supported by high-quality investors. Shoreline Biosciences is headquartered in San Diego, CA.

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CELL AND GENE THERAPY

Imara Announces Primary Endpoint Change in the Ardent Phase 2b Clinical Trial of Tovinontrine (IMR-687) in Sickle Cell Disease

Imara | November 22, 2021

Imara Inc. a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases, today announced a change to the primary endpoint for the Ardent clinical trial, a Phase 2b study of tovinontrine (IMR-687) in patients with sickle cell disease (SCD), based on the recommendation of the U.S. Food and Drug Administration (FDA). Imara requested feedback from the FDA on the draft statistical analysis plan (SAP) for the Ardent trial in which fetal hemoglobin (HbF) response was the primary endpoint and annualized rate of vaso-occlusive crises (VOCs) was the key secondary endpoint. In reviewing the Ardent draft SAP and prior to any database lock for analysis, the FDA recommended that Imara change the primary endpoint to be annualized rate of VOCs. HbF response will continue to be evaluated as a key secondary endpoint. The endpoint revisions do not affect the conduct of the trial or operational aspects of the study. As part of its recommendation, the FDA suggested further interactions regarding the revised SAP and engagement on the potential of the current program for regulatory decision-making. “We welcome the FDA’s recommendations and are in the process of changing the primary endpoint of the Ardent trial to be annualized rate of VOCs and moving HbF response to be a key secondary endpoint. A reduction in VOC rate is an established approval endpoint, and we are engaging the FDA further on this and related topics, including possible streamlined paths to registration.” Rahul Ballal, Ph.D., President and Chief Executive Officer of Imara Dr. Ballal continued, “In light of this endpoint revision, the previously planned fourth quarter interim analysis will no longer occur. That interim analysis had been designed to have a focus on safety and pharmacodynamic biomarkers, including HbF, but did not include a review of VOCs. The first review of data from the Ardent trial, including annualized VOC rate, will be conducted when all subjects have completed assessment at Week 24 or terminated early, and is planned for the first quarter of 2022, subject to our upcoming discussions with the FDA. Final data analysis from the Ardent trial remains on track for the second half of 2022. In June 2021, we reported promising data from our Phase 2a and open label extension clinical trials in SCD that demonstrated reduced annualized rates of VOCs in patients treated with tovinontrine versus placebo. We expect to present updated 12-month VOC data from our ongoing Phase 2a open label extension clinical trial at the American Society of Hematology Annual Meeting in December 2021.” About the Ardent Phase 2b Clinical Trial The Ardent Phase 2b clinical trial is a fully-enrolled, global, randomized, double-blind, placebo-controlled, multicenter study with approximately 115 adult patients with sickle cell disease (SCD) enrolled. The planned primary efficacy objective will be to evaluate the annualized rate of vaso-occlusive crises (VOCs) in patients dosed with tovinontrine (IMR-687) as compared to placebo. A key secondary endpoint will be to evaluate the proportion of all patients with fetal hemoglobin (HbF) response, defined as an absolute increase from baseline of at least 3% in HbF, as compared to placebo. Additional endpoints include the evaluation of the effect of tovinontrine versus placebo on other VOC-related outcome measures, HbF-associated biomarkers, markers of red blood cell hemolysis, white blood cell adhesion markers and quality of life measures over the course of a one-year treatment period. The FDA has granted Orphan Drug, Fast Track and Rare Pediatric Disease designations and the European Commission has granted Orphan Drug designation for tovinontrine for the treatment of SCD. About Tovinontrine (IMR-687) Tovinontrine is a highly selective and potent small molecule inhibitor of phosphodiesterase-9 (PDE9). PDE9 selectively degrades cyclic guanosine monophosphate (cGMP), an active signaling molecule that plays a role in vascular biology and hemoglobin production in red blood cells. Lower levels of cGMP are found in people with sickle cell disease (SCD) and beta-thalassemia and are associated with reduced blood flow, increased inflammation, greater cell adhesion and reduced nitric oxide mediated vasodilation. Blocking PDE9 acts to increase cGMP levels, which is associated with several benefits including the potential reactivation of fetal hemoglobin (HbF), a natural hemoglobin produced during fetal development. Increased levels of HbF in RBCs have been demonstrated to improve symptomology and substantially lower disease burden in both patients with SCD and patients with beta-thalassemia. About Sickle Cell Disease Sickle cell disease (SCD), a hemoglobinopathy, is a rare inherited red blood cell disorder. The disease causes structural abnormalities in hemoglobin that cause red blood cells to become inflexible and elongated, ultimately blocking blood flow to organs, which can lead to vaso-occlusive crises (VOCs). SCD is characterized by debilitating pain, progressive multi-organ damage and early death. The global prevalence of SCD is estimated to be approximately 4.4 million patients, including an estimated 100,000 patients in the United States and 134,000 patients in the European Union. About Imara Imara Inc. is a clinical-stage biotechnology company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of hemoglobin and other serious diseases. Imara is advancing tovinontrine (IMR-687), a highly selective, potent small molecule inhibitor of PDE9 that is an oral, potentially disease-modifying treatment currently in clinical development for sickle cell disease and beta-thalassemia and preclinical development for heart failure with preserved ejection fraction, or HFpEF. Imara is also advancing IMR-261, an oral activator of nuclear factor erythroid 2–related factor 2, or Nrf2. Cautionary Note Regarding Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to (i) the Company’s plans to change the primary and secondary endpoints for the Ardent Phase 2b clinical trial of tovinontrine (IMR-687), (ii) the timing for reporting of additional data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in patients with sickle cell disease and (iii) the Company’s planned discussions with the FDA regarding the regulatory pathway for tovinontrine. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the impact of extraordinary external events, such as the risks and uncertainties resulting from the impact of the COVID-19 pandemic on the Company’s business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities and ability to readout data from the Ardent Phase 2b and open label extension clinical trials of tovinontrine in sickle cell disease; the Company’s ability to advance the development of tovinontrine under the timelines it projects in current and future clinical trials, demonstrate in any current and future clinical trials the requisite safety and efficacy of tovinontrine; and other factors discussed in the “Risk Factors” section of the Company’s most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

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Bolder BioTechnology Announces Positive Preclinical Data for Use of BBT-032 to Inhibit Growth of the SARS-CoV-2 Virus

PR Newswire | August 26, 2020

Bolder BioTechnology, Inc. announced today that preclinical studies have shown that its proprietary long-acting interferon beta analog, BBT-032, strongly inhibits growth of the SARS-CoV-2 virus in culture at concentrations expected to be readily achievable in patients. SARS-CoV-2 is the coronavirus responsible for the Covid-19 pandemic.Based upon these promising results, Bolder BioTechnology intends to file an Investigational New Drug Application with the U.S. Food and Drug Administration to begin testing BBT-032 in humans. BBT-032 may prove useful for accelerating clearance of the SARS-CoV-2 virus in infected patients and for preventing infections in uninfected individuals.Interferon beta is a critical component of the host innate immune system that provides broad-based protection against viruses by stimulating a patient's cells to make proteins and other molecules that allow the cells to resist infection by viruses. By stimulating host innate immunity, BBT-032 has the potential to be an effective treatment not only against SARS-CoV-2, but also against other coronaviruses such as SARS-CoV and MERS-CoV, and future viral pandemic threats.

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