Partnology

Please join us in recognizing Beth J Hoffman, Founder and CEO of Origami Therapeutics, Inc., in this weeks Biotech Leader Spotlight Series! Beth brings more than 25 years of #CNS drug discovery experience and has developed over 30 assets that have advanced into the clinic in cystic #fibrosis, #metabolic diseases, #neurology, #pain and #psychiatry. Prior to Origami, she was Vice President, Discovery Biology for Vertex Pharmaceuticals, where she generated and launched clinical candidates for #respiratory and #neurological diseases, including first-in-class blockbuster drugs in cystic fibrosis (Kalydeco, Orkambi, Symdeko, Trikafta). She expanded the market for Orkambi and increased sales to over $1 billion, and also generated a novel non-opioid pain #therapeutic and other protein folding programs at Vertex. Dr. Hoffman received an A.B. in Molecular Biology from Wellesley College and a Ph.D. in Cell Biology from The Johns Hopkins University. She is a member of the Board of Trustees for the Huntington's Disease Society of America as well as a Scientific Advisor for the Tau Consortium of the The Rainwater Charitable Foundation, focusing on alleviating diseases associated with tau #pathology, such as Alzheimer’s Disease. She is also on the Board of Biofrontera Inc. #biotechleader #womeninbio #womeninscience #femaleleaders #biotechexecutive #biotechfounder #biotech #biotechnology #drugdevelopment #drugdiscovery #therapeutics #biopharma

FDA Approvals: The FDA is preparing for fall vaccination season, approving two updated COVID-19 #vaccines, including Moderna’s Spikevax. The new formulation of Moderna’s #mRNA-based vaccine targets the Omicron KP.2 variant of the SARS-CoV-2 virus and is intended to help prevent COVID-19 in people six months and older. The new formulation is expected to be available in the U.S. in the coming days. Pfizer and BioNTech SE announced FDA approval for their updated version of COVID-19 vaccine Comirnaty. As with Spikevax, Pfizer and BioNTech’s vaccine is tailored to the KP.2 strain of the SARS-CoV-2 Omicron JN.1 lineage, based on guidance from the FDA. It is also fully approved for individuals 12 years and older and approved under emergency use authorization for those six months to 11 years of age. Recent Layoffs:  Aadi Bioscience, Inc (LA), a precision #oncology company, is laying off 80% of its R&D staff as it focuses on preserving cash while maximizing its commercial business. Aadi shared that it’s halting its PRECISION1 trial of nab-sirolimus in patients with solid tumors harboring TSC1- or TSC2-inactivating alterations, as the trial is unlikely to meet the efficacy threshold needed to support an accelerated approval. It will also pause new enrollment in two ongoing Phase 2 trials of nab-sirolimus for advanced or recurrent endometrioid-type endometrial cancer and #neuroendocrine tumors. Other Interesting News: BridgeBio Pharma (Palo Alto) has transferred several preclinical and early clinical-stage drugs to a new company, #GondolaBio, that’s drawn $300 million in promised investment from a syndicate of blue-chip life sciences investors. Viking Global Investors, Patient Square Capital and Frazier Life Sciences are among the investors backing GondolaBio. Also involved is an entity owned by BridgeBio CEO Neil Kumar. The CA biotech will initially hold a 45% stake in GondolaBio, although that will go down as additional capital commitments are funded. The new company will inherit drug programs aimed at the rare diseases erythropoietic protoporphyria, alpha-1 antitrypsin deficiency and tuberous sclerosis complex. Biopharma VC hit highest quarterly level since 2022, but challenges in exit landscape remain. There’s been a balanced split between IPOs and M&A for the year so far. Overall, the M&A cycle has slowed down, according to PitchBook. The data firm cited depleted cash, full pipelines or a move toward advancing startups versus selling them as possible reasons for the change. Meanwhile, it’s a “mixed picture” when looking at #IPOs, with high-quality companies still debuting on the public markets, just in decreased numbers, demonstrating a continued preference for companies with mature clinical data. While overall venture funding is rising, obstacles such as slow M&A activity and unfavorable public valuations make it hard to find suitable exit opportunities.

Please join us in recognizing Andrew Rudd, Cofounder and CEO of Palm Therapeutics, in this weeks Biotech Leader Spotlight Series! Dr. Andrew Rudd is the Cofounder and CEO of Palm Therapeutics. His career has centered around developing chemical tools to interrogate #biological lipids and their roles in health and disease. He received his Ph.D. in #chemistry from UC San Diego where he authored eight high-impact papers and was the recipient of several awards, including the Inamori Fellowship, which recognizes one chemistry Ph.D. student each year based on outstanding research performance. During his Ph.D. work, he developed the first small molecule probes for directly inhibiting protein #palmitoylation in cells. In January 2021, he launched Palm Therapeutics at JLABS San Diego with the mission of developing the first palmitoylation-targeted therapies. He has secured over one million dollars in non-dilutive funding from the NIH and serves as the lead PI on two active SBIR grants at Palm. He is also the recent recipient of the 2023 Servier FAST Discovery Award from Servier Laboratories and California Life Sciences. #biotechfounder #biotechleader #biotechexecutive #biotech #biotechnology #biopharma #drugdevelopment #therapeutics

Recent Funding: Avidity Biosciences, Inc. (SD) raised an additional $345 million by selling north of 8.4 million common shares at $41 apiece. The #biotech went public in 2020, and earlier this year raked in around $460 million through another stock offering. Avidity plans to use the fresh funds to further develop #RNA medicines that are designed to combine the advantages of monoclonal #antibodies and #oligonucleotide therapies. The most advanced of these medicines is in late-stage testing for a rare muscle-wasting disease. Recent Layoffs:  The psychedelic biotech, Lykos Therapeutics, is cutting 75% of staff (equivalent to about 75 people) following an FDA rejection and the retraction of three research papers. Viracta Therapeutics, Inc. is going all-in on its lymphoma program, hitting pause on its solid tumor program and putting 23% of its staff out of a job in the process. As part of a pivot to Tepezza competitor lonigutamab, ACELYRIN, INC. is waving goodbye to about 40 of its 135 employees, equivalent to 33% of its workforce. FDA Approvals: Gilead Sciences has won #FDA approval to sell Livdelzi, a new medicine for a rare liver disease known as primary biliary cholangitis. The FDA clearance covers patients who can’t tolerate a commonly used treatment called ursodeoxycholic acid or those whose disease doesn’t respond well enough to the older medicine alone. Johnson & Johnson’s Rybrevant is now cleared by the FDA for use with another drug called Lazcluze in people with previously untreated non-small cell lung #cancer that’s metastasized or advanced locally. Only people with specific mutations in a gene known as EGFR are eligible for treatment. J&J described the new treatment as the first chemotherapy-free regimen that’s demonstrated superiority to AstraZeneca’s lung cancer drug Tagrisso in this setting. Other Interesting News: Arrowhead Pharmaceuticals (Pasadena), an RNA interference (#RNAi) biotech company, presented two next-gen candidates dubbed ARO-INHBE and ARO-ALK7 that are designed to treat #obesity and #metabolic diseases (both set to enter the clinic in early 2025). In preclinical studies, the candidates have both shown potential reducing body weight and fat mass using a novel mechanism of action that might help preserve lean muscle mass, compared to currently marketed therapies. Denali Therapeutics (SF) reports new method to get antisense #oligonucleotides across blood-brain barrier. ASOs, which bind to RNA and prevent them from being translated into proteins, have gained popularity in recent years as a way to treat #neurodegenerative diseases. However, getting these drugs into the brain currently requires invasive infusion directly into the cerebrospinal fluid. Now, Denali has developed a way to smuggle them across the blood-brain barrier. In studies with mice and macaques, the biotech combined ASOs with their transferrin-targeting transport vehicle platform and successfully knocks down certain gene activity across the brain.

Please join us in recognizing Brooks Leitner, Cofounder of EpiTET Therapeutics, in this weeks Biotech Leader Spotlight Series! Brooks Leitner Co-founded EpiTET Therapeutics in 2023. He has also been a Venture Fellow at Canaan, BrightEdge - American Cancer Society , and Foresite Capital. Prior to that Brooks enrolled in Yale University's MD/PhD Program in 2018, and completed his PhD in Cellular & Molecular #Physiology in 2022 in the lab of Dr. Rachel J. Perry, investigating the intersection of #metabolic fitness and #cancer and #sepsis. Prior to Yale, he attended the National Institutes of Health in Bethesda, MD for a Postbaccalaureate position in the #Diabetes, #Endocrinology, and #Obesity branch of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). There, he studied human brown adipose tissue anatomy and function, and its role in human energy metabolism in the labs of Drs. Kong Chen and Aaron Cypess. Brooks earned his bachelor's degree in Kinesiology, with Honors in 2015 from the University of Maryland, College Park. There, he earned undergraduate researcher of the year in 2015 for his work on the effects of exercise on brain function in Dr. J. Carson Smith's lab. #biotechfounder #venturefellow #biotech #biotechnology #biopharma #drugdevelopment #pharmaceuticals #therapeutics #biotechleader

FDA Approvals: Actio Biosciences, Inc. (San Diego), a biotech leveraging a novel platform approach to #genetics and precision medicine to develop new therapeutics that target shared underlying biology in both rare and common diseases, announced the U.S. FDA has granted both orphan drug designation (ODD) and rare pediatric disease designation (RPDD) for ABS-0871, a TRPV4 inhibitor, for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (#CMT2C). TRPV4 mutations cause CMT2C, a devastating disease that leads to debilitating and life-threatening symptoms, such as severe muscle weakness, vocal cord paresis and respiratory complications. In tone-setting verdict, FDA rejects MDMA as a therapy aid for PTSD. The decision to turn down an application from Lykos Therapeutics (San Jose, CA) comes at a pivotal time for #psychedelics research, which, after decades of dismissal, has recently gained momentum. According to Lykos, the FDA concluded that it couldn’t approve the therapy based on the data submitted, and requested the company run another Phase 3 trial to further study the safety and efficacy of MDMA. Lykos said it plans to ask for the agency to reconsider its decision. The thinking goes that MDMA makes patients more emotionally open and able to connect with their therapist during psychotherapy sessions, which research suggests leads to better outcomes. Recent Layoffs:  Boundless Bio (San Diego): After an IPO in March, the cancer company has slimmed down its discovery work and "modestly reduced its workforce." The study for Boundless' first clinical-stage asset has been enrolling slower than expected, prompting the biotech to streamline operations. AN2 Therapeutics (Menlo Park): As part of a pivot to early-stage projects, AN2 is laying off half its employees and stopping a phase 3 study. The biotech had 41 full-time employees at the end of February. Chief Medical Officer Paul Eckburg, M.D., is among the people leaving. Other Interesting News: Avidity Biosciences, Inc. (San Diego) announces positive AOC 1044 data demonstrated significant increase of 25% in dystrophin production and reduction of creatine kinase levels to near normal in people living with Duchenne Muscular Dystrophy (#DMD) amenable to Exon 44 skipping in the Phase 1/2 EXPLORE44™ Trial. "This is an exciting moment as these data suggest del-zota has the potential to change the treatment paradigm...We have not seen this level of dystrophin production and reduction in creatine kinase with other PMO exon-skipping treatments," said Diana Castro, M.D., Board Certified #Neurologist and #Neuromuscular Physician.

Please join us in recognizing Sam Jackson, Chief Medical Officer of Neuron23, in this weeks Biotech Leader Spotlight Series! Check out our interview with Sam to learn from his years of leadership experience and biotech industry insights. Sam joined Neuron23 as Chief Medical Officer in 2022. A strategic leader and clinical development expert, Sam is a board-certified emergency #physician with fellowship training in medical #toxicology. He has a history of successful leadership roles in the #biopharmaceutical industry. Before joining Neuron23, Sam served as Chief Medical Officer (Interim) at Alector, where he oversaw clinical development and operations while leading programs in Alzheimer’s Disease and Frontotemporal #Dementia. Sam led the #clinical due-diligence discussions that resulted in a transformational alliance with GSK in one of the largest deals in the neurodegeneration space. Prior to Alector, Sam held the role of Chief Medical Officer at two other biotech companies after working in positions of increasing responsibility at Amgen, Genentech, and Dynavax Technologies. Sam graduated from Stanford University and received his MD and MBA degrees from the University of Pennsylvania. #biotechleader #biotechexecutive #biotech #biotechnology #biopharma #clinicaldevelopment #CMO #drugdevelopment #therapeutics

Please join us in recognizing Chief Scientific Officer of SOFIE, Sherly Mosessian in this weeks Biotech Leader Spotlight Series! Check out our interview with Sherly to learn from her biomedical research and executive leadership expertise. Dr. Mosessian earned her bachelor’s degree in Biochemistry, followed by her Ph.D. from UCLA Department of Molecular and Medical Pharmacology, with publications in tumor signaling, molecular imaging targets and clinical trials of Positron Emission Tomography (PET) probes. She joined the SOFIE team in 2021 as the Vice President of Clinical and Regulatory Affairs leading the clinical development of FAPI diagnostic. She was promoted to Chief Scientific Officer in 2022. Prior to joining SOFIE, she served as a Chief Administrative Officer at UCLA and oversaw the establishment of a current Good Manufacturing Process (cGMP) facility in support of PET radiopharmaceutical production for research and clinical service. She successfully established a pathway at the UCLA Department of Pharmacology to translate PET imaging agents from bench to the clinic in a time and cost-efficient manner. In addition to her expertise in biomedical research, she has served in an executive leadership capacity in technology, having co-led the establishment of DGIT, the integrated information technology organization in support of the research and education mission of the UCLA School of Medicine. Dr. Mosessian is Armenian and having immigrated to the United States at a young age, values the importance of diversity/inclusion, seeking opportunities and having a strong work ethic. #biotechleaders #womeninbio #womeninscience #biotech #biotechnology