Novartis sickle cell drug wins FDA priority review

Pharmatimes | July 18, 2019

The US Food and Drug Administration (FDA) has given Novartis investigational sickle cell medicine, crizanlizumab SEG101, Priority Review. The governing body accepted the company's Biologics License Application (BLA) which was submitted for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD) and was granted Breakthrough Therapy designation in December 2018. The submission is supported by Phase II results from the SUSTAIN study, which showed that crizanlizumab (5 mg/kg) reduced the median annual rate of VOCs leading to health care visits by 45.3% compared with placebo. Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. The designation is intended to shorten the FDA review period to six months from the standard ten months. "The FDA's decision to give crizanlizumab priority review reflects the impact that this medicine could have for the many thousands of US sickle cell adult patients who experience painful vaso-occlusive crises," said John Tsai, head of global drug development and chief medical officer, Novartis.

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In vitro tests are widely used, from R&D through to QC, to improve the efficacy of drug delivery and confirm product consistency. Ensuring a drug reaches its intended site of action in vivo, in anappropriate state, is a crucial first step towards meeting clinical performance goals. Drugs may bedelivered by the gastrointestinal tract; or via the rectal and vaginal mucosal membranes; through thesurface of the skin; or by inhalation via the nose or lungs. In each case the requirements for effective delivery are different and the tests applied to assess drug performance differ accordingly.


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EyePoint Pharmaceuticals Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

EyePoint Pharmaceuticals, Inc. | April 19, 2022

EyePoint Pharmaceuticals, Inc. a pharmaceutical company committed to developing and commercializing therapeutics to help improve the lives of patients with serious eye disorders, announced that the Company granted non-statutory stock options to new employees as inducement awards outside the Company’s 2016 Long-Term Incentive Plan in accordance with NASDAQ Listing Rule 5635(c)(4). The Company granted stock options to purchase up to an aggregate of 56,500 shares of EyePoint Pharmaceuticals common stock to four new employees. The stock options were granted on April 14, 2022. The grants were approved by the Compensation Committee and made as an inducement material to each employee entering into employment with EyePoint Pharmaceuticals in accordance with NASDAQ Listing Rule 5635(c)(4). The option awards have an exercise price of $12.69 per share, the closing price of EyePoint Pharmaceuticals’ common stock on April 14, 2022. The options have a ten-year term and vest over four years, with 25% of the original number of shares vesting on the first anniversary of the applicable employee’s date of grant and the remainder vesting in equal monthly installments over the following three years. Vesting of the options is subject to the employee’s continued service with EyePoint Pharmaceuticals through the applicable vesting dates. About EyePoint Pharmaceuticals EyePoint Pharmaceuticals is a pharmaceutical company committed to developing and commercializing therapeutics to help improve the lives of patients with serious eye disorders. The Company's pipeline leverages its proprietary Durasert® technology for sustained intraocular drug delivery including EYP-1901, a potential six-month intravitreal anti-VEGF treatment initially targeting wet age-related macular degeneration. Durasert’s proven intravitreal drug delivery platform has been safely administered to thousands of patients' eyes across four U.S. FDA approved products, including YUTIQ® for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye, which is currently marketed by the Company. EyePoint Pharmaceuticals is headquartered in Watertown, Massachusetts.

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Stallergenes Greer Strengthens Precision Medicine Approach in Allergen Immunotherapy With Imperial College London Research Collaboration

Stallergenes Greer | January 17, 2022

Stallergenes Greer, a global healthcare company specialising in allergen immunotherapy (AIT), today announced that the company has entered into a research collaboration on the discovery of biomarkers of AIT efficacy with Imperial College London, a global top ten university with a world-class reputation in science, engineering, business and medicine. This long-term research collaboration, which combines the long-standing expertise of Stallergenes Greer in AIT with the prestigious research capabilities of Imperial College London, aims to push the boundaries of allergic patient care by identifying biomarkers of AIT efficacy and expanding the knowledge of the pathophysiology of allergic diseases and their treatment with AIT. “We are thrilled about this collaboration with Imperial College London which will contribute to both deciphering the mode of action of AIT and identifying key markers that can be used in the routine practice of allergology for the benefit of patients and healthcare practitioners. Based on the understanding of specific phenotypes and the responses of patients with allergies, allergologists will be able to tailor treatment modalities with the right dosing at the right time for each individual. This underscores our commitment to advancing precision medicine for allergy sufferers”, Amer Jaber, Executive VP Operations Europe and International and President of Stallergenes SAS “This research collaboration is a remarkable opportunity for a fruitful partnership between Imperial College London, a pioneer and world-leading university in the biomarker field, and Stallergenes Greer. It will build on the recent advances of biomarker research to further develop precision medicine in AIT for the benefit of patients with allergic diseases,” said Dr Mohamed Shamji, Head of the Immunomodulation and Tolerance Group, Imperial College London. The results of this research collaboration will be shared in international congresses and peer review publications. About Allergen Immunotherapy Allergies are the most prevalent and fastest growing chronic diseases in the industrialised world, affecting over one billion people worldwide. Allergen immunotherapy uniquely alters the natural course of respiratory allergies. It is the only therapeutic class capable of modifying disease progression and potentially preventing the onset of the disease by inducing tolerance in the immune system. About Stallergenes Greer Headquartered in London (UK), Stallergenes Greer Ltd is a global healthcare company specialising in the diagnosis and treatment of allergies through the development and commercialisation of allergen immunotherapy products and services. Stallergenes Greer Ltd is the parent company of Greer Laboratories, Inc. About Imperial College London Imperial College London is one of the world's leading universities. The College's 20,000 students and 8,000 staff are working to solve the biggest challenges in science, medicine, engineering, and business. Imperial is University of the Year 2022 in the Times and Sunday Times Good University Guide. It is the world’s fifth most international university, according to Times Higher Education, with academic ties to more than 150 countries. Reuters named the College as the UK's most innovative university because of its exceptional entrepreneurial culture and ties to industry.

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invoX Pharma to Acquire F-star Therapeutics, Inc. a Pioneering Next Generation Bispecific Discovery Platform and Clinical Programs

F-star Therapeutics, Inc. | June 24, 2022

invoX Pharma a wholly owned subsidiary of Sino Biopharmaceutical Limited focused on research and development and business development activities outside of China, and F-star Therapeutics, Inc. a clinical-stage biopharmaceutical company pioneering bispecifics in immunotherapy so more people with cancer can live longer and improved lives, today announced that the companies have entered into a definitive agreement whereby invoX will acquire all of the issued and outstanding shares of F-star common stock for $7.12 per share. The proposed acquisition values F-star at approximately $161 million. The transaction has been unanimously approved by the invoX and F-star Boards of Directors and is expected to close in the second half of 2022. F-star’s proprietary platform technology pioneers the use of tetravalent (2+2) bispecific antibodies that enable the simultaneous targeting of two different antigens and a unique set of pharmacology to deliver focused, potent and safe immune activation in the tumor microenvironment. Four programs are progressing in clinic, three based on F-star’s tetravalent platform and one next generation STING agonist, with multiple further undisclosed programs in development. These programs represent potentially first- and best-in-class drug candidates for many areas of unmet medical need, including patients with cancer and other serious diseases who have few other options available. Leveraging its modular antibody technology, F-star has forged collaborations with major international biopharma companies across a wide range of therapeutic areas including oncology, immunology and neurology. invoX, established in 2021 in the United Kingdom, is Sino Biopharm’s international expansion platform, focusing on R&D and business development activities outside of China, with a core focus on oncology and respiratory therapeutics. F-star will form a key element of invoX’s strategy to accelerate Sino Biopharm’s development of innovative medicines to transform the lives of patients worldwide, complementing its existing R&D platforms and pipeline. “Today’s proposed acquisition is aligned with invoX’s strategy to become a fully integrated biopharmaceutical company with an advancing pipeline of innovative products addressing unmet healthcare needs, worldwide. We are excited to welcome F-star employees and look forward to working with them as we invest in the company to progress and grow its clinical pipeline to realize the full potential of the platform.” Ben Toogood, Chief Executive Officer of invoX Eliot Forster, Chief Executive Officer of F-star said: “We believe our tetravalent bispecifics offer the best approach to tackle hard-to-treat cancers and other serious diseases, with the ambition to deliver longer and improved lives for patients. Today’s announcement is good news for F-star, for our shareholders and, of course, for patients. This transaction enables greater and longer-term opportunities to develop the F-star platform and accelerate delivery of our novel medicines as we work together towards a future free from cancer and other serious diseases. I’d like to thank the fantastic team at F-star as well as our partners for all their hard work, support and dedication and I’m delighted to share this exciting development.” About invoX Pharma invoX Pharma was incorporated in March 2021 and is a wholly owned subsidiary of Sino Biopharm, a global top 40 pharmaceutical company with more than 24,000 employees. United Kingdom-based invoX is Sino Biopharm’s international expansion platform, focusing on R&D and business development activities outside of China, with a core focus on oncology and respiratory therapeutics. At its core, the Company aspires to improve patients’ lives by creating access to innovative medicine. About Sino Biopharm Sino Biopharm, together with its subsidiaries, is a leading, innovative research and development driven pharmaceutical conglomerate in China, with a business scope that is vertically integrated including research and development, manufacturing and sales and marketing infrastructure. The Company’s product offerings include a variety of biologics and small molecule drugs, and in therapy areas that include hepatology, oncology, cardiovascular and cerebrovascular diseases, orthopaedics, digestive and immune and respiratory diseases. About F-star Therapeutics, Inc. F-star Therapeutics, Inc. is a clinical-stage biopharmaceutical company pioneering bispecifics in immunotherapy so more people with cancer can live longer and improved lives. F-star is committed to working towards a future free from cancer and other serious diseases, through the use of tetravalent (2+2) bispecific antibodies to create a paradigm shift in treatments. The Company has four second-generation immuno-oncology therapeutics in the clinic, each directed against some of the most promising IO targets in drug development, including LAG-3 and CD137. F-star’s proprietary antibody discovery platform is protected by an extensive intellectual property estate. F-star has over 500 granted patents and pending patent applications relating to its platform technology and product pipeline. The Company has attracted multiple partnerships with biopharma targeting significant unmet needs across several disease areas, including oncology, immunology, and CNS.

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FDA Grants Direct Biologics Regenerative Medicine Advanced Therapy (RMAT) Designation for the use of ExoFlo in COVID-19 Related ARDS

Direct Biologics | April 13, 2022

Direct Biologics, an innovative biotechnology company with a groundbreaking extracellular vesicle platform drug technology, announced that the U.S. Food and Drug Administration has awarded their EV drug product ExoFlo with a Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of Acute Respiratory Distress Syndrome associated with COVID-19. The RMAT program is designed to expedite the approval of promising regenerative medical products in the US that demonstrate clinical evidence indicating the ability to address an unmet medical need for a serious life-threatening disease or condition. Under the RMAT designation, the FDA provides intensive guidance on drug development and post-market requirements through early and frequent interactions. Additionally, an RMAT confers eligibility for accelerated approval and priority review of biologics licensing applications. "After intensively reviewing our preclinical data, manufacturing processes, and clinical data from our Phase II multicenter, double blinded, placebo controlled randomized clinical trial, the FDA has recognized ExoFlo as a lifesaving treatment for patients suffering from Acute Respiratory Distress Syndrome (ARDS) due to severe or critical COVID-19. The additional attention, resources, and regulatory benefits provided by an RMAT designation demonstrate that the FDA views ExoFlo as a product that can significantly enhance the standard of care for the thousands still dying from ARDS every week in the US," he said. Mark Adams, Chief Executive Officer "We are very pleased that the FDA has recognized the lifesaving potential of our platform drug technology ExoFlo. The RMAT has provided a pathway to expedite our drug development to achieve a BLA in the shortest possible time," said Joe Schmidt, President. "I am very proud of our team. Everyone has been working around the clock for years in our mission to save human lives taken by a disease that lacks treatment options, both in the US and abroad. We are grateful for the opportunity to accelerate development of ExoFlo under the RMAT designation as it leads us closer to our goal of bringing our life saving drug to patients who desperately need it." ExoFlo is an acellular human bone marrow mesenchymal stem cell derived extracellular vesicle product. These nanosized EVs deliver thousands of signals in the form of regulatory proteins, microRNA, and messenger RNA to cells in the body, harnessing the anti-inflammatory and regenerative properties of bone marrow MSCs without the cost, complexity and limitations of scalability associated with MSC transplantation. ExoFlo is produced using a proprietary EV platform technology by Direct Biologics, LLC. Physicians can learn more and may request information on becoming a study site at clinicaltrials.gov. For more information on Direct Biologics and regenerative medicine, visit: https://directbiologics.com. About Direct Biologics Direct Biologics, LLC, is headquartered in Austin, Texas, with an R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in San Antonio, Texas. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust EV platform technology. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving segment of next generation regenerative biotherapeutics. Direct Biologics has obtained and is pursuing multiple additional clinical indications for ExoFlo through the FDA's investigational new drug (IND) process.

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Spotlight

In vitro tests are widely used, from R&D through to QC, to improve the efficacy of drug delivery and confirm product consistency. Ensuring a drug reaches its intended site of action in vivo, in anappropriate state, is a crucial first step towards meeting clinical performance goals. Drugs may bedelivered by the gastrointestinal tract; or via the rectal and vaginal mucosal membranes; through thesurface of the skin; or by inhalation via the nose or lungs. In each case the requirements for effective delivery are different and the tests applied to assess drug performance differ accordingly.

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