PHARMA TECH

GNS and Servier Announce AI Collaboration to Advance Multiple Myeloma Drug Discovery and Clinical Development

Servier Group and GNS | August 25, 2022 | Read time : 03:00 min

GNS and Servier
GNS, the leader in the use of "Digital Twins" and Causal AI & biosimulation technology for biopharmaceutical drug discovery and development, and Servier, a global pharmaceutical group, today announced a collaboration to advance drug discovery, translational, and clinical development efforts in Multiple Myeloma (MM).

"This collaboration furthers Servier's goal to accelerate its drug discovery and clinical development efforts through AI and biosimulation and other digital initiatives. We believe GNS' Digital Twin and unique causal AI models will help our research and early development group improve our understanding of MM disease biology and, ultimately, transform our drug discovery and clinical development process".

Claude Bertrand, EVP of Research and Development at Servier

"We are delighted to collaborate with Servier to help advance research in MM and leverage our Gemini Digital Twin models to help uncover novel insights and reveal the hidden biological circuity of this disease," said Joe Donahue, Chief Business Officer at GNS. "Working together with the outstanding Servier team, we hope to make a difference in the lives of patients with MM."

The collaboration will address key questions about how MM progresses in different patient subpopulations, including discovering what are the most synergistic pathways for new combination therapies and will help Servier gain insights that can advance their translational and clinical development efforts. This work will initially leverage the GNS Gemini Digital Twin for MM developed using the rich clinico-genomic data from the Multiple Myeloma Research Foundation CoMMpass study.

GNS' MM Digital Twin leverages the rich patient multi-modal data generated by MMRF to create a transparent, accurate, quantified representation of complex biological mechanisms, revealing new circuitry of human disease. By conducting biosimulations, which are essentially computational experiments, one can discover new drug targets and disease mechanisms, and can also determine which drug candidates are more effective than others and why certain molecular profiles of patients lead to better and more durable response. Ultimately, the MM Digital Twins will radically change how Pharma R&D is conducted by helping to accelerate the identification of new targets and target combinations, new biomarkers, and enabling the in silico design of better trials.

About Servier Group
Servier is a global pharmaceutical group governed by a Foundation. With a strong international presence in 150 countries and a total revenue of 4.7 billion euros in 2021, Servier employs 21,800 people worldwide. Servier is an independent group that invests over 20% of its brand-name revenue in Research and Development every year. To accelerate therapeutic innovation for the benefit of patients, the Group is committed to open and collaborative innovation with academic partners, pharmaceutical groups, and biotech companies. It also integrates the patient's voice at the heart of its activities.

A leader in cardiology, the ambition of the Servier Group is to become a renowned and innovative player in oncology. Its growth is based on a sustained commitment to cardiovascular and metabolic diseases, oncology, neuroscience and immuno-inflammatory diseases. To promote access to healthcare for all, the Servier Group also offers a range of quality generic drugs covering most pathologies.

About GNS
GNS is the leader in the application of Causal AI and simulation technology to discover and validate novel drug targets, simulate clinical trials, and help pharmaceutical and biotech companies discover and develop new medicines faster. GNS' patented AI uncovers new insights from multi-omics and real-world data leading to the discovery and prioritization of novel biological targets, and more efficient clinical trials with patients who are likely to respond to therapies. The Gemini Digital Twin models across oncology, auto-immune diseases, and neurology enable the simulation of disease progression and drug response at the individual patient level to simulate clinical trials in diverse patient cohorts.

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PHARMACY MARKET, PHARMA TECH

MTTI Obtains FDA Allowance of Investigational New Drug (IND) for Hürthle Cell Thyroid Cancer

Molecular Targeting Technologies, Inc | December 09, 2022

Molecular Targeting Technologies, Inc. a clinical stage radiopharmaceutical therapy company focused on therapies for rare diseases, announced today the allowance of an Investigational New Drug application by the U.S. Food and Drug Administration. It is now waiting for Institutional Review Board approval. Once approved by IRB, it will enable a Phase I/II clinical study of the Safety, Dosimetry and Efficacy of EBTATE in adult patients with metastatic, radioactive iodine non-responsive Hürthle cell thyroid cancer.” EBTATE is a new generation of peptide receptor radiotherapeutic drug that has demonstrated potential clinical superiority over standard of care. It selectively binds to somatostatin receptor 2 on neuroendocrine and other tumors, which are then killed by the radionuclide. EvaThera platform products were designed to bind to serum albumin, due to the Evans blue moiety, extending in vivo residence time, enabling lower, less frequent dosing of the radiopharmaceutical and reducing risk of renal injury vs. the current standard of care. Dr. Joanna Klubo-Gwiezdzinska, MD, Ph.D, MHSc, acting section chief of Thyroid Tumors and Functional Thyroid Disorders in the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health (NIH) and study author commented: “Hürthle cell thyroid cancer, which currently lacks effective treatment options, is characterized by a particularly high expression of SSTR2*. With this phase I/II clinical trial, we hope to implement individualized dosimetry-based dosing for each patient with Hürthle cell thyroid cancer that spread outside of the thyroid gland and analyze how much of an active agent is being accumulated in the tumor tissue to establish a threshold associated with the best efficacy and safety.” “The clearance of our IND is an important milestone for MTTI. Having solidified our clinical trial preparedness and manufacturing readiness, we are well-positioned to advance EBTATE to target Hürthle cell thyroid cancer.” Dr. Chris Pak, President & CEO of MTTI About Molecular Targeting Technologies, Inc. (MTTI) Molecular Targeting Technologies, Inc., is a privately held, rapidly growing, well financed, clinical-stage biotech company developing next-generation targeted radiotherapeutics and diagnostics for rare cancers. Evathera platform technology product has shown application for glioblastoma multiforme (GBM) cancer patients overexpressing integrin. MTTI is committed to building value by acquiring and translating innovative imaging, radiopharmaceutical and theranostics assets to improve human health, reduce healthcare costs and reward stakeholders. MTTI expects to be orchestrating multiple clinical trials in 2023. The content in this release is the sole responsibility of the authors and does not necessarily represent the official views or imply endorsement of the National Institutes of Health.

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BUSINESS INSIGHTS

Ex-Biocon team Symbio Generrics completes capital raise from Ascent Capital

Symbio Generrics | November 25, 2022

Symbio Generrics India Private Limited a specialized Active Pharmaceutical Ingredients & Intermediates manufacturing and marketing organization, announced that it has completed an equity capital raise from Ascent Capital, a leading growth-capital focused private equity fund. Symbio was founded by pharmaceutical professionals, Salim Shaikh and Abhijit Kale. They were soon joined by the third promoter, Akash Puranik; all of whom possess decades of experience in the global pharmaceutical industry. Symbio's journey from a sales and marketing organization to a full-fledged vertically integrated manufacturer of specialized APIs has been exemplary. Through a well-developed customer ecosystem, the highly experienced & skilled team at Symbio has established a global footprint for its APIs. Ascent Capital's sizeable investment in Symbio will further accelerate the company's expansion plans and sets the company enroute to emerge as a top-10 manufacturer of APIs in India. The funds raised from Ascent will be utilized towards undertaking strategic acquisitions, establishing a best-in-class R&D facility and ramping up manufacturing capacity. The company's R&D Centre is housed in Bengaluru while the manufacturing plant is located at Dobbaspet, Bengaluru. The Company is in the process of acquiring another manufacturing facility in India. The team is focused on building a high-potential generics API portfolio for the global markets and to this effect, will work towards getting US FDA and other SRA approvals for their recently acquired facilities. "We are glad to be associated with Ascent Capital which has a reputation of partnering with entrepreneurs to build leading businesses in India. The sizeable investment from Ascent Capital will add power to our next growth spurt and enable us to emerge as a top-10 API company in India. We are setting ourselves a medium-term goal of growing rapidly into a sizeable company by leveraging the strength of our team and expanding our horizons to actively serve the global API market." Commenting on the fund raise, Salim Shaikh, Executive Chairman & Founder of Symbio Generrics Akash Puranik, Promoter, MD& CEO of Symbio Generrics, says, "The latest feather in our cap is the influx of resources from Ascent Capital. We have carved strategic alliances and relationships spanning over two decades with leading pharmaceutical entities in regulated and semi-regulated markets and are now keen to expand these synergies further in the global arena. With a well-developed infrastructure, both on the manufacturing and R&D front, we are keen to engage with potential partners for CDMO Business." Mr. Raja Kumar, Founder and MD of Ascent Capital, said, "Ascent Capital has been known to support highly qualified professional teams in niche domains. Continuing with this tradition, we are delighted to partner with the first-generation technocrat founders at Symbio. Collectively, the team at Symbio is one of the best multi-disciplinary teams in the Indian pharma space and has shown an exemplary track record of building global relationships. We look forward to working collaboratively with the Company to ensure that we have another category leader from the Ascent stable, akin to BigBasket, Sequent, Skanray, to name a few." IC Universal Legal's Chennai team led by Senior Partner, Sameena Chatrapathy represented Symbio as the legal counsel for the fund raise from Ascent Capital as well as the follow-on acquisitions. About Symbio Symbio Generrics India Private Limited founded in the year 2010 is a specialized Active Pharmaceutical Ingredients & Intermediates manufacturing and marketing organization with headquarters in Bengaluru. The Company was founded by first generation entrepreneurs coming from a leading pharmaceutical organization, with a combined experience of over 40 years. About Ascent Capital Ascent Capital is a leading India-focused Independent Private Equity Fund Manager. The team at Ascent is one of the most experienced investment teams in India with 150+ years of collective experience in Indian PE and capital markets. Thus far, Ascent has helped more than 60 entrepreneurs build leading businesses across diverse sectors such as Technology, Healthcare, Financial Services, Consumer Brands, etc. It typically invests USD 10-30 MM in fast growing businesses, led by outstanding entrepreneurs, that have the potential to emerge as leaders in its segment thereby delivering exceptional exit outcomes. Ascent is backed by marquee investors, who include long term institutional investors like pension funds, foundations, endowments, fund of funds and large corporations - a vote of confidence in the team's ability to excel and deliver results.

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PHARMACY MARKET

A novel azapeptide editing method provides potential for new drug development, Feinstein Institutes research shows

Feinstein Institutes | November 29, 2022

Peptides, short sections of proteins made up of chains of amino acids, are an important area of new drug development due to their potential for increased specificity and fewer side effects. Their main drawback is the rapid degradation by the body’s own enzymes, which has led to the need to develop longer-lasting peptide alternatives. Azapeptides are one type of peptide alternative that has shown great potential as therapeutics, exemplified by the HIV drug, Atazanavir. A new paper published in the journal Nature Communications by researchers at The Feinstein Institutes for Medical Research details proprietary reagents and methods that can selectively replace only those amino acids known to be targeted by enzymes with more stable versions to create azapeptides. The paper, authored by Yousef Al-Abed, PhD, co-director of the Institute of Bioelectronic Medicine at the Feinstein Institutes, describes the proprietary building blocks and method used to conduct systematic and robust peptide editing, replacing targeted amino acids with aza-amino acids to form more stable and efficient azapeptides. The azapeptides created could expedite the novel therapies to treat many diseases and conditions, including metabolic disease, influenza, pulmonary arterial hypertension, Crohn’s disease, arthritis, and irritable bowel diseases, among others. The paper, titled “Thiocarbazate building blocks enable the construction of azapeptides for rapid development of therapeutic candidates,” provides multiple examples of azapeptide construction, screening, and selection using a known peptide inhibitor of inflammation and bradykinin. “Peptides as drug candidates are easy to discover yet difficult to develop as final drugs because of their fast degradation in our body. A technology that circumvents these problems inherent to native peptides will change the future landscape of drug discovery. Our technology provides a platform of tools that enable minimal modification of any peptide at highly prone degradation sites, thereby increasing its life span in our body, allowing it more time to find its target and neutralize it.” Dr. Al-Abed Through peptide editing, going from peptide to azapeptide, using methods that Dr. Al-Abed and his team detail in the new paper, scientists can create potential therapies that have enhanced characteristics compared to natural peptides, like longer stabilities in a more efficient manner. Dr. Al-Abed and his team have precisely engineered thiocarbazates (a novel functional group) in the proprietary process to achieve an almost universal and more efficient means to selectively replace amino acids in a peptide chain. “Peptide drugs are a crucial resource for patients and the global pharmaceutical industry,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “Dr. Al-Abed and his team invented a fully automated chemical strategy using novel chemistry to make new peptide drugs which is an important new avenue for peptide drug discovery.” About the Feinstein Institutes The Feinstein Institutes for Medical Research is the research arm of Northwell Health, the largest health care provider and private employer in New York State. Home to 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health innovations and outcomes, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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