Aerpio Pharmaceuticals, Inc.

近日，精准肿瘤公司Aadi Bioscience宣布将裁撤其研发人员的80%，Aadi本身也会聘请一家外部咨询公司寻求股东价值最大化。根据该公司公告，管线调整和R&D员工的人力资源减少将至少将其现金跑道延长至2026年下半年。 导致该公司做出这一决定的是该公司核心管线白蛋白结合型西罗莫司的II期临床数据。独立数据监测委员会的分析表明，该研究不太可能到达支持加速批准所需的疗效阈值。这使得该公司将暂停PRECISION1注册意向试验。 其实解雇的预兆在此前8月16日发布的SEC文件中就可以看到，当时该公司宣称将将解雇22名员工，占其员工总数的32%，而该公司共有48名研发人员，考虑到80%的裁撤，届时该公司可能不剩下多少研发人员了，被收购可能是这家公司未来的结局。 有趣的是，目前岌岌可危的Aadi本身也是收购其他公司的产物。在2021年时，已经走向破产道路的眼科治疗公司Aerpio Pharmaceuticals通过拍卖资产与Aadi Bioscience合并，合并后的Aadi Bioscience全力开始推动白蛋白结合型西罗莫司，尽管如此，这一举措仍未能拯救白蛋白结合型西罗莫司的颓势。 白蛋白结合型西罗莫司的失利 白蛋白结合型西罗莫司可以说是Aadi Bioscience公司的核心。机制机理来自于mTOR信号通路抑制。而白蛋白结合型药物能够利用白蛋白的自然属性，通过血液循环系统流经肿瘤组织血管时，通过EPR效应（增强渗透滞留效应）富集于肿瘤组织间液，实现药物的靶向递送，因此就能实现mTOR抑制剂对肿瘤的靶向。 该公司围绕白蛋白结合型西罗莫司打造了数条管线，也确实成功在一项罕见癌种中成功推动上市了一项产品（商品名：Fyarro） 罕见的小癌种是血管周围上皮样细胞瘤（PEComa），这一小癌种是软组织肉瘤中的罕见亚型，早先的研究发现，PEComa常常会伴有TSC1和/或TSC2基因突变，而TSC1和/或TSC2基因突变会导致下游mTOR信号通路激活。 那么能否证明在PEComa小癌种中的成功能够在同基因突变条件下的实体瘤中复刻，就成为了拓宽市场的重点。 毕竟PEComa这一小癌种患者人数较少，药品定价也不高，收入相当微薄，今年Q2一季度才卖出了620万美元。 但这次失利的临床恰巧就是TSC1或者TSC2基因突变的实体瘤患者，这也意味着拓宽适应症的尝试基本以失利告终，情势不容乐观。 现在还剩下子宫内膜癌(EEC)和神经内分泌肿瘤(NETs)的数据等待读出。 本周好文推荐 如需转载请联系佰傲谷并在醒目位置注明出处 · · · · · · · ·

Dhaval Desai, PharmD, named as Chief Development Officer; will oversee late-stage Product Development, Medical Affairs, Scientific Communications, Regulatory and Quality operations; most recently SVP & Chief Development Officer at Iveric Bio where he led development and approval of IZERVAYChristopher Simms named as Chief Commercial Officer, effective September 25, 2024; will oversee Pre-commercial and Commercial organizations, pre-launch preparations and development; most recently SVP & Chief Commercial Officer at Iveric Bio where he led commercial strategy and execution for the launch of IZERVAYCarlos Quezada-Ruiz, M.D., FASRS, named as SVP, Therapeutic Area Head, Ophthalmology; will lead the Ophthalmology franchise and oversee early- and late-stage clinical development; most recently Group Medical Director, Ophthalmology at Genentech where he led clinical development and approval of VABYSMO and SUSVIMOOphthalmology Advisory Board comprised of world-renowned retina specialists and thought leaders to support development strategy and registration for large market ophthalmology programs: Dr. Arshad Khanani (Chair), Dr. David Boyer, Dr. Frank Holz, Dr. Anat Loewenstein, Dr. Dante Pieramici EMERYVILLE, Calif., Aug. 05, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today strengthened the Company’s senior management team with the appointments of Dhaval Desai, PharmD, as Chief Development Officer, Christopher Simms as Chief Commercial Officer and Carlos Quezada-Ruiz, M.D., FASRS, as SVP, Therapeutic Area Head, Ophthalmology. Dr. Desai will lead 4DMT’s late-stage Product Development, Medical Affairs, Scientific Communications, Regulatory and Quality operations. Mr. Simms will lead 4DMT’s Pre-commercial and Commercial organizations, effective September 25, 2024. Both Dr. Desai and Mr. Simms will join 4DMT’s Executive Team, reporting to David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. Dr. Quezada-Ruiz will lead the Ophthalmology franchise and oversee early- and late-stage ophthalmology clinical development, reporting to Bob Kim, M.D., Chief Medical Officer of 4DMT. With the addition of Dr. Desai, An Song, Ph.D., will transition from Chief Development Officer to Chief Research & Translational Development Officer. “With the continued promise of 4D-150 as a pipeline-in-a-product, I am thrilled to have Dhaval, Chris and Carlos join the 4DMT team and bring their wealth of large market ophthalmology, regulatory, drug development and commercial experience to our company as we transition into Phase 3 development and pre-commercialization,” said Dr. Kirn. “Dhaval, Chris and Carlos’s leadership is vital to becoming a leading late-stage development genetic medicines company, with a focus on large market ophthalmic diseases. We believe the expansion of our senior leadership team with extensive ophthalmology experience will allow us to execute on our development objectives to design and rapidly enroll large global Phase 3 pivotal trials, and subsequently to make successful global regulatory submissions​.” “It is a privilege to join 4DMT at this stage of rapid progress and to be able to focus my development expertise to bring these important product candidates to patients as effectively and efficiently as possible,” said Dr. Desai. “My experience working on pivotal Phase 3 programs and U.S. Food and Drug Administration (FDA) submissions in multiple large market retinal indications will complement the already stellar team that David and 4DMT have built. I look forward to collaborating and partnering with the entire 4DMT team to increase the strength of our ophthalmology portfolio, progress 4D-150 through late-stage development, and prepare for potential BLA submissions in the coming years.” “Joining 4DMT at this time of growth is truly an incredible opportunity to contribute to the planning and potential introduction of transformational medicines in multiple large market ophthalmology indications like wet age-related macular degeneration (wet AMD), diabetic macular edema (DME), diabetic retinopathy (DR) and geographic atrophy (GA),” said Mr. Simms. “I look forward to building a leading commercial strategy and infrastructure in support of late-stage development, registration and ultimately, commercialization. My experience in large market ophthalmology commercial strategy and execution helps fortify and bring 4DMT’s exceptional product pipeline of potentially paradigm shifting treatments to patients who need them most.” “I am thrilled to join 4DMT and bring my drug development expertise, people and patient focus, to lead and further build 4DMT’s outstanding ophthalmology team ahead of the initiation of our first pivotal Phase 3 trial in wet AMD early next year, and to rapidly accelerate our development programs in additional large-market diseases including DME, DR and GA,” said Dr. Quezada-Ruiz. “As a medicines developer and practicing retina specialist, I am most excited about the potential of gene therapy to improve outcomes for our patients and believe that the greatest unmet need in treating wet AMD, DME, and now GA, is greater durability. Having played a leading role in the global development and approvals of VABYSMO and SUSVIMO, I have seen firsthand the positive impact that novel mechanisms of action and sustained durability have for patients, caregivers and physicians. Similarly, 4D-150’s potential to achieve sustained efficacy and extended durability should significantly reduce patients’ treatment burden while also maintaining vision through a safe, single intravitreally delivered gene therapy injection. I look forward to contributing to 4DMT’s mission to bring transformative genetic medicines to millions of patients in need around the world.” The Company also announced the formation of the Ophthalmology Advisory Board (OAB), comprised of world-renowned retina specialists and thought leaders. The OAB will be chaired by Arshad M. Khanani, M.D., M.A., FASRS, Director of Clinical Research at Sierra Eye Associates and Clinical Professor at University of Nevada, Reno, and will support development strategy and registration across large market ophthalmology indications including wet AMD, DME, DR and GA. “The wealth of clinical and global drug development experience that has been brought together under the OAB, in addition to our new senior leadership hires, provides 4DMT with unparalleled expertise in large market ophthalmology development,” added Dr. Kim. “As we prepare for our first Phase 3 clinical trial, the OAB will provide substantial support to maximize the value of our deep pipeline of retinal genetic medicines programs across multiple retinal disease indications. I look forward to working with our OAB members and new senior leaders to bring these important medicines to patients globally.” Senior Management Hires Dhaval Desai, PharmD, Chief Development OfficerDr. Desai has spent the past 20+ years working in all aspects of drug development with a focus on clinical development and medical affairs. Prior to joining 4DMT, Dr. Desai was SVP and Chief Development Officer at Iveric Bio (acquired by Astellas) where he led the clinical development, medical affairs and biostatistics departments responsible for the approval and launch of IZERVAY™, one of the first complement inhibitors approved for the treatment of GA. Before Iveric, Dr. Desai served as VP and Medical Unit Head of Ophthalmology at Novartis Pharmaceuticals, overseeing both the posterior and anterior segment therapeutic portfolios. In addition to these roles, Dr. Desai has also held leadership positions at other ophthalmology-focused biotech companies including Aerpio Therapeutics and ThromboGenics. Dr. Desai received his PharmD from and completed a post-doctoral industry fellowship at Rutgers University. Chris Simms, Chief Commercial OfficerMr. Simms is an accomplished healthcare leader with more than 20 years of diverse commercial leadership experience at Iveric Bio (acquired by Astellas), Johnson & Johnson, Genentech and Novartis, including focused experience in retina, ophthalmology and optometry. Most recently he was SVP and Chief Commercial Officer at Iveric where he built and led the commercial team from pre-commercialization through the successful U.S. launch of IZERVAY™. Prior to joining Iveric, Mr. Simms served as Vice President and Head of the Novartis U.S. Ophthalmics business unit which included the brands BEOVU® and Xiidra®. He joined Novartis in 2017 to build the commercial launch strategy for BEOVU® after leading commercial efforts on LUCENTIS® at Genentech. Prior to Genentech, he spent 16 years at Johnson & Johnson working with leading brands across their vision care, diabetes and consumer goods businesses in Canada, Japan and the U.S. Mr. Simms has a Bachelor of Commerce from Memorial University of Newfoundland and an MBA from York University, Toronto, Canada. Carlos Quezada-Ruiz, M.D., FASRS, SVP, Therapeutic Area Head, OphthalmologyDr. Quezada-Ruiz joins 4DMT from Genentech (a Roche company) where he served in the Ophthalmology management team as Group Medical Director, Clinical Science, Product Development. During his tenure at Genentech, he led the design, execution and readouts for U.S. and global registrational trials across multiple retinal diseases, including the wet AMD global clinical development program for VABYSMO, leading to U.S. and global approvals and the global clinical science team for SUSVIMO, supporting its initial FDA approval and launch in wet AMD, successfully leading his team through the U.S. voluntary recall and recent commercial relaunch. In addition, he led the DR and DME Phase 3 registrational programs. Dr. Quezada-Ruiz also oversaw the Medical Affairs team efforts for the U.S. launch of Lucentis Prefilled Syringe (PFS) and the Myopic Choroidal Neovascularization indication for LUCENTIS. Additionally, he played a pivotal role in the advancement of personalized healthcare in retina within Genentech by helping design and develop predictive models using machine learning and large language models to support drug development and clinical practice. Dr. Quezada-Ruiz is a practicing retina specialist and fellow of the American Society of Retina Specialists, with over 13 years of vitreoretinal clinical practice and research. Dr. Quezada-Ruiz received his M.D. from Universidad Autónoma de Coahuila, and completed fellowships in Vitreoretinal Surgery, Ocular Pathology-Research, and Retinal Disease and Vitreoretinal Surgery Research from Universidad Nacional Autónoma de México, McGill University, and the California Retina Research Foundation. He obtained a CIBE from Columbia Business School, Executive Education. Ophthalmology Advisory Board Arshad M. Khanani, M.D., M.A., FASRS (Chair): Managing Partner, Director of Clinical Research and Director of Fellowship at Sierra Eye Associates, and Clinical Professor at the University of Nevada, Reno School of Medicine. He has served as the principal investigator for more than 120 clinical trials and has over 150 scientific publications. Dr. Khanani is an elected member of the Macula Society and Retina Society. He has received numerous awards of distinction including the prestigious American Society of Retina Specialists (ASRS) Presidents’ Young Investigator Award and the ASRS Presidential Award. David S. Boyer, M.D.: Senior Partner at the Retina-Vitreous Associates Medical Group, and an Adjunct Clinical Professor of Ophthalmology at the University of Southern California/Keck School of Medicine in Los Angeles, California. He is a board-certified ophthalmologist specializing in the treatment of diseases of the retina and vitreous, and a leading clinical researcher for new treatments in macular degeneration and DME. Frank G. Holz, M.D., FEBO, FARVO: Professor and Chairman of the Department of Ophthalmology at the University of Bonn, Germany. He founded the GRADE Reading Center and the Medical Imaging Center Bonn, with a focus on innovative retinal imaging technologies and image analysis strategies. His research focuses on the pathogenesis, structural and functional biomarkers, and new therapies for macular and retinal diseases. He has published more than 600 articles in peer-reviewed journals and has received numerous awards including the Pro Retina Macular Degeneration Research Award, the Leonhard-Klein Award for Ocular Surgery, the Alcon Research Institute (ARI) Award, the Senior Achievement Award of the AAO and the Jules Gonin Award. Anat Loewenstein, M.D., MHA: Professor and Director, Division of Ophthalmology at the Tel Aviv Medical Center, VP Ambulatory Services at the Tel Aviv Medical Center, Sidney Fox Chair of Ophthalmology at the Sackler Faculty of Medicine at Tel Aviv University, Israel, and President of EURETINA. She has published more than 500 papers in peer reviewed journals and has contributed multiple chapters to ophthalmology textbooks. Her focus is the investigation of drug administration and toxicity to the retina, early detection of macular degeneration and home monitoring of retinal disease. Dante Pieramici, M.D.: Managing Partner at California Retina Consultants, President of the California Retina Research Foundation, a member of the Medical Leadership Board of the Retina Consultants of America. Currently, he is the Medical Director of Clinical Research at California Retina Consultants. Dr. Pieramici has served as the principal, sub, reading center investigator or advisor for over 100 clinical trials. His research has focused primarily on new surgical and pharmacologic treatments for age-related macular degeneration and diabetic-related eye diseases. About 4DMT 4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing six clinical-stage and one preclinical product candidate, each tailored to address rare and large market diseases in ophthalmology, pulmonology and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT. All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied. Learn more at www.4DMT.com and follow us on LinkedIn. Forward Looking Statements:This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits and market potential of 4DMT’s product candidates, as well as the plans, announcements, and related timing for the clinical development of, regulatory interactions regarding, and potential commercialization of 4D-150 and 4D-175 and 4D-150's potential to be a pipeline-in-a-product. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward looking statements. Contacts: Media:Katherine SmithInizio Evoke CommsKatherine.Smith@inizioevoke.com Investors:Julian PeiHead of Investor Relations and Corporate FinanceInvestor.Relations@4DMT.com

Palani Palaniappan, Ph.D., Stephen Hoffman, M.D., Ph.D., and Paul Westberg, MBA, bring deep industry experience to guide Company growth COLUMBUS, Ohio--(BUSINESS WIRE)-- Clarametyx Biosciences Inc. (“Clarametyx”), a clinical stage biotechnology company developing targeted, immune-enabling biologic therapies to counter persistent infections associated with biofilms, today announced the appointment of Palani Palaniappan, Ph.D., to its Scientific Advisory Board, as well as the appointments of Stephen Hoffman, M.D., Ph.D. and Paul Westberg, MBA as strategic advisors. “We are humbled to welcome Dr. Palaniappan to our scientific advisory board, as his extensive industry knowledge will not only augment our scientific focus but introduce critical development expertise that will enable us to pursue the full potential for our novel anti-biofilm technology,” said David Richards, Chief Executive Officer. “We are also pleased to engage Stephen Hoffman, MD, Ph.D. and Paul Westberg, MBA, who will further enhance our corporate and strategic development activities as we build momentum toward the next phase of the company’s growth.” Palani Palaniappan is an experienced business leader passionate about developing innovative and impactful medicines for patients. He is currently Chief Technology Officer of Pioneering Medicines at Flagship Pioneering. Dr. Palaniappan previously served as the Executive Vice President and Chief Technology Officer at Aruvant Sciences. His prior executive roles included Head of Global Technical Operations at Sarepta Therapeutics and Global Head of Biologics and New Modality Development at Takeda Pharmaceuticals. His early career included leadership positions at Millennium Pharmaceuticals (acquired by Takeda), Biogen, Nexstar Pharmaceuticals (acquired by Gilead), and Par Pharmaceuticals. Palani received his Ph.D. from the Indian Institute of Technology, Kanpur and completed his post-doctoral studies in biochemistry and biophysics at the University of California, Riverside and Virginia Commonwealth University. He holds a M.S. and B.S. from Annamalai University. Stephen Hoffman is a seasoned life sciences executive with over 30 years of experience building companies and leading entrepreneurial teams to create shareholder value. He is currently a Managing Partner and Co-Founder of Trekk Venture Partners and serves on multiple public and private company boards. He was previously the CEO of Aerpio Pharmaceuticals, Inc. and Allos Therapuetics, Inc. and served as Managing Director at Skyline Ventures, as well as General Partner at TVM Capital. Paul Westberg is a strategic business executive with more than 25 years of broad transactions, financing, market planning, and advisory experience focused on advancing transformational therapies in the life sciences sector. He was most recently the Chief Business Officer of Qpex Biopharma, a resistance-focused infectious disease company, which was acquired by Shionogi. Earlier in his career, Mr. Westberg was Chief Business Officer at Versartis and has previously served in numerous business development and finance roles for start-up companies since starting his career at Genentech. Mr. Westberg received his MBA from the University of California, Berkeley and his BA from the University of California, San Diego. About Clarametyx Biosciences Clarametyx Biosciences is combating the formidable challenge of persistent and recalcitrant infections through an innovative technology platform targeting the biofilm—a protective layer around bacteria—to enable a more effective immune response and antibiotic intervention. The Columbus, Ohio-based company is building a dynamic pipeline of immune-enabling therapies and vaccines for life-threatening bacterial infections associated with biofilms, with a near-term focus on challenging respiratory infections. For more information, visit us on the web or on LinkedIn.