Lacerta Therapeutics, Inc.

Shares in Lexeo opened down 17%, deflating the stock to below $15. Lexeo Therapeutics has guided its Friedreich ataxia (FA) gene therapy through an early test, generating evidence of improvements in cardiac status in early-phase trials. But the biotech ran into problems when testing the effect of the candidate on fitness and was left with a gap in evidence. The analysis pooled data from Lexeo’s phase 1/2 study and an investigator-initiated phase 1a trial run at Weill Cornell Medicine. Lexeo struck a deal with Weill Cornell for the data in April. The studies are testing whether LX2006 can treat cardiac dysfunction in FA by increasing the expression of FXN, a protein that is involved in the function of heart muscle. Lexeo has at least six months of data on eight participants. Four people had elevated left ventricular mass index (LVMI), a predictor of cardiac morbidity and mortality, at the start of the study. Three of those subjects experienced a more than 10% reduction in LVMI at 12 months. Lexeo also saw declines in lateral wall thickening, an early indicator of an inefficient heart, and drops in a biomarker of myocardial injury. FXN expression increased in evaluable patients. Lexeo tried to assess LX2006’s effect on patients’ lives using V02 max, a measure of cardiorespiratory fitness. However, the researchers saw baseline peak VO2 levels were extremely low, a finding that Lexeo said likely reflects a combination of neurological impairment and reduced cardiac functional capacity.  Three of the eight participants were unable to reach the level of effort needed to reliably interpret peak VO2. Across the other five subjects, Lexeo tracked average improvements of 1% after six months and 4% after 12 months. The biotech will continue evaluating peak V02 in its studies and is looking into other cardiopulmonary exercise test measures that may be more useful in the patient population.  While the evidence supporting LX2006 comes from cardiac assessments, for now, Lexeo said there are precedents for drugs winning approval on the strength of LVMI or transgene expression. Eric Adler, chief medical officer and head of research at Lexeo, made the case for LVMI on a call with investors to discuss the data.  “It has regulatory precedent behind it, obviously we’ve seen recently, and we know specifically in FA increases in LVMI are associated with bad outcomes. A 10% increase in LVMI is associated with a 20% increased risk of death,” Adler said. “So, we have a high degree of confidence that that should be an excellent potential outcome moving forward that we should look at in our trials.”  Lexeo has moved into the third dose cohort. Nolan Townsend, CEO at Lexeo, told investors “we believe a higher dose cohort could achieve higher protein levels” and quoted preclinical data showing benefits at higher doses to make his case. The move into the higher dose is supported by the safety profile seen in the two trials, neither of which has reported any treatment-related serious adverse events.   The biotech declined to discuss the regulatory timeline on the call with investors but sees potential for accelerated approval. The belief LX2006 may benefit from accelerated approval reflects a lack of existing treatments. Reata Pharmaceuticals, which Biogen bought for $7.3 billion, won FDA approval for Skyclarys in 2023 but it only showed efficacy on neurological measures. Lexeo’s focus on cardiac measures differentiates LX2006 from Skyclarys but the market could become more competitive in the coming years. Voyager Therapeutics and Neurocrine Biosciences are working on an FXN gene therapy. Solid Biosciences and Lacerta Therapeutics have gene therapies in the works, too, while Prime Medicine and Tune Therapeutics have early stage gene-editing programs.  Shares in Lexeo opened down 17%, deflating the stock to below $15.

BioFlorida's Annual Conference Spotlights Industry Success & Fuels Collaboration MIAMI, Nov. 7, 2022 /PRNewswire/ -- BioFlorida's 25th Annual Conference united statewide leaders of Florida's life sciences industry representing biopharmaceutical, medical technology, and digital health companies, globally recognized research universities and research institutions, and the state's health systems to spotlight the continued momentum and success in science, business, and public policy and spur collaboration and future growth. "For 25 years, BioFlorida has convened our state's champions for the life sciences – those leaders from Florida-based companies, research institutions, and health systems – to spotlight success, to foster collaboration, and to explore policy solutions to fuel continued industry growth and innovation," said Nancy K. Bryan, President and CEO of BioFlorida. "The Conference provided an opportunity to reflect on the role we've played to collectively establish Florida as a leading life science destination that today represents 8,600 establishments and research organizations that employ nearly 107,000 Floridians. But our focus remains on the patient, and we're excited to continue the unprecedented collaboration to develop new, innovative treatments and cures over the next 25 years." The Conference explored topics ranging from emerging therapies and breakthrough research in cancer care, rare disease, neurology and movement disorders, and gene and cell therapy to innovations in manufacturing, financing strategies, commercialization considerations, and other emerging trends, like AI and machine learning applications and leveraging MedTech to improve the patient, physician, and clinical experience. All conversations were led by CEOs, executives and top scientists and researchers from Florida-based companies, universities, research organizations, and health systems. Panels also explored opportunities to fuel Florida's life sciences ecosystem by attracting companies and talent to the state and policy discussions on the future of healthcare in Florida with Senator Ana Maria Rodriguez and Representative Tom Fabricio. Speaker of the Florida House of Representatives Chris Sprowls shared the impact of the life sciences industry as a cancer survivor and the Florida Legislature's role in helping to grow the industry. The keynote speaker at the Conference was Fred Hassan, a director with the private equity firm, Warburg Pincus, and board member of Precigen, IntegraConnect, Prometheus Biosciences, and BridgeBio. The industry titan drew on his years of success leading companies like Schering-Plough Corporation, where he served as Chairman and CEO from 2003 until its merger with Merck & Co. in 2009, and Pharmacia Corporation, where we served as Chairman and CEO from 2001 to 2003 until its acquisition by Pfizer Inc. to share strategies for success in today's environment. Jeffrey Duerk, PhD, Executive Vice President for Academic Affairs and Provost for the University of Miami, spurred collaborative solutions to connect academics, research, and industry in Florida to spotlight state efforts on a global level. The Spotlight on Innovation session showcased four companies that have exhibited transformational growth: Veru, Inc. (Miami); Accelus (Palm Beach Gardens); Catalyst Pharmaceutical Inc. (Coral Gables); and ILiAD Biotechnologies (Weston). Seed and early-stage companies competed in BioPitch, an opportunity for some of the state's most innovative companies to pitch to 25+ life science investors. A record-breaking 55 companies entered BioPitch with InnoCare Urologics LLC (Miami), a company developing the InnoCare Egress Catheter, the only indwelling urinary catheter designed to rapidly deflate if accidentally removed, winning the competition and receiving $10,000. Runners-up included Andante Biologics (Gainesville); FoxSeq LLC (Alachua); and, VuEssence (Tampa). BioFlorida recognized individuals and organizations throughout six unique categories for their incredible achievements. This year's award recipients include: Researcher of the Year Award The Researcher of the Year award recognizes distinguished researchers whose achievements have significantly advanced scientific knowledge with a proven impact on innovation and public health. This years' award was presented to Dr. James J. Hickman, Chief Scientist and Chairman of the Board at Hesperos, Inc., a global contract research organization he co-founded that is providing safety and efficacy testing services for novel therapeutics using its Human-on-a-Chip® platform – the most advanced, multi-organ microphysiological system available today. By developing human-based, clinically relevant disease models, Hesperos is accelerating drug discovery for a range of indications by providing unprecedented visibility into how those diseases and potential therapeutics impact the body over time, especially for rare diseases. This includes neurodegenerative and rare diseases such as Alzheimer's and ALS, as well as investigating the effects of opioid overdose and recovery, among others. The exciting new technology continues gaining industry adoption from commercial partners and regulators alike. This was most recently evidenced by the FDA authorizing a Phase II clinical trial (#NCT04658472) for a repurposed drug for a rare neurodegenerative disease using safety data from another indication but using efficacy data only from the Hesperos platform. This was a first-of-its-kind achievement and a significant milestone in the evolution of drug discovery for human-on-a-chip systems. In addition to his role at Hesperos, Dr. Hickman is the Founding Director of the NanoScience Technology Center, and Professor of NanoScience Technology and Electrical Engineering at the University of Central Florida. Hesperos was initiated in the UCF incubator before moving to its current facility. Previously, he was the Hunter Endowed Chair in the Bioengineering Department at Clemson University where he and Tom Boland invented the bioprinting of cells. Dr. Hickman earned his Ph.D. in Chemistry from MIT and has spent the past 30 years studying the interaction of biological species with modified surfaces in both industry and academia, including stints at NSF and DARPA in the area of biological computation. He has more than 31 issued patents, 164 publications, and 20 book chapters. He is a Fellow of the American Institute of Medical and Biomedical Engineers, the American Vacuum Society, International Academy of Nanobiotechnology, and most recently, the National Academy of Inventors. Deal of the Year Award The Deal of the Year Award recognizes a deal, whether it be an equity investment, key license agreement, significant grant award, or other agreement, with a significant impact on innovation, public health, and the deal-flow ecosystem within Florida's life sciences industry. The Deal of the Year Award was presented to Dr. Edgardo Rodriguez-Lebron, Co-founder, President and Chief Executive Officer of Lacerta Therapeutics, a biotechnology company focused on adeno-associated virus (AAV) technologies and founded in 2017 in Alachua. The Deal of The Year is Lacerta's capsid licensing and collaborative research agreement with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company. The collaboration will leverage Lacerta's established expertise in AAV technologies and Prevail's development and clinical capabilities, with the goal of accelerating development of gene therapies for patients with neurodegenerative disorders. Lacerta will lead capsid discovery, screening, and validation, while Prevail will complete preclinical and investigational New Drug-enabling studies. Prevail will oversee manufacturing, clinical development, and commercialization. Dr. Rodriguez-Lebron has been a leader at Lacerta from its foundation and has over two decades of research experience in neurodegenerative diseases and adeno-associated viruses (AAV). An active contributor to the gene therapy community, he is an ASGCT member and serves as the CSO of NF2Biosolutions, a nonprofit focused on the development of a gene therapy approach to neurofibromatosis type 2 (NF2). He has co-founded and helped launch multiple startups in the Gainesville, Florida area. Weaver H. Gaines Entrepreneur of the Year Award The Weaver H. Gaines Entrepreneur of the Year Award honors an individual who has made extraordinary contributions to the growth of Florida's life sciences industry. The 2022 Weaver H. Gaines Entrepreneur of the Year Award was presented to Courtney A. Miller, Ph.D., Co-Founder and acting CEO of Myosin Therapeutics, a Jupiter-based biopharmaceutical company focused on developing first-in-class therapies for the treatment of cancer and psychiatric disorders, targeting an untapped superfamily of proteins known as molecular nanomotors. Together with her co-founders, Drs. Pat Griffin and Ted Kamenecka, the team developed nMOTOR, a high-throughput screening platform to identify selective modulators of any one of the hundred different nanomotors encoded by the human genome. Myosin is currently advancing the development of MT-110 and MT-125, two CNS-penetrant small molecule inhibitors with novel nanomotor targets. Myosin was born out of more than a decade of work by the team in their labs at UF Scripps Biomedical Research, with $15 million in NIH support to help bring their compounds to the clinic. Under Dr. Miller's leadership, the company was founded in August 2020 and awarded a $2.5 million-dollar Fast Track SBIR from NIH just six months later to support completion of IND-enabling studies and a Phase I clinical trial of MT-110 for stimulant use disorder, for which there are currently no FDA-approved treatments. In parallel, the team has been working with physician-scientist, Dr. Steven Rosenfeld, Director of Neuro-Oncology at the Mayo Clinic in Jacksonville, on development of MT-125 for the treatment of glioblastoma, an aggressive form of brain cancer with no cure. MT-125 is safe and effective in preclinical studies, blocking both growth of the tumor and its invasion of healthy brain tissue, and shows potentially curative effects when combined with existing FDA-approved therapies. David J. Gury Company of the Year Award The David J. Gury Company of the Year Award honors a company that has achieved significant milestones and has contributed to the growth of Florida's life sciences industry. The 2022 David J. Gury Company of the Year Award was presented to Patrick J. McEnany, CEO of Catalyst Pharmaceuticals, a commercial-stage biotechnology company dedicated to making a meaningful difference in the lives of patients suffering from rare diseases. Based in Coral Gables, Catalyst was granted breakthrough therapy designation by the FDA in treating the rare disease Lambert-Eaton Myasthenic Syndrome (LEMS) in 2013. The product was approved in November 2018 and successfully launched FIRDAPSE in 2019. In 2022, the company received an sNDA approval for FIRDAPSE extending the patient population to include pediatric patients six years and older for the treatment of LEMS, and was named by Forbes as one of the 2022 Top Small Companies in America and South Florida Business Journal's 2022 Top 50 Public Companies. With a proven track record in development and commercialization, Catalyst continues to attain several significant milestones while generating yearly organic revenue growth. The company has $220 million in cash and short-term investments as of June 30, 2022, and no funded debut. BioFlorida Leadership Award The BioFlorida Leadership Award recognizes the BioFlorida volunteer who has taken on a leadership role within the organization, dedicated significant time to BioFlorida's mission, and has acted as a true ambassador to BioFlorida and the industry. The 2022 recipient of the BioFlorida Leadership Award is Kathy Chiu, a Founding Partner of DeepWork Capital, which manages venture capital funds investing in Florida startups. This year DeepWork Capital was selected to be the investment manager of Florida Opportunity Fund (FOF), a series of the state's venture capital investment program. Chiu co-founded DeepWork Capital, an Orlando-based company, in 2015 to help close the funding gap in Florida's innovation economy. DeepWork Capital is a rare multi-disciplinary early-stage venture capital fund in the state with life science capabilities. Its selection as the investment manager enables FOF to process life science investments, a development welcomed by BioFlorida. Chiu was instrumental in building up DeepWork's Life Science capabilities and led the firm in its investment in HCW Biologics (NASD: HCWB), which went public in 2021. At the 2022 BioFlorida Annual Conference, Chiu served as of BioPitch, a pitch competition that connects life science companies to investors. Chiu's dedication helped the organization expand its network to include a record-breaking 25+ investors and 55 applicants, as well as using her experience to implement key improvements to the BioPitch competition to address the needs of participants. BioFlorida Lifetime Achievement Award The BioFlorida Lifetime Achievement Award recognizes an individual's outstanding career achievements, leadership and contributions that have significantly contributed to the growth of his or her respective industry. The 2022 recipient of the BioFlorida Lifetime Achievement Award is Dr. Leonard Pinchuk. Over a forty-year career in biomedical engineering, Dr. Pinchuk's achievements include 137 issued U.S. patents, more than 100 publications and the founding of 10 companies. His major inventions are the Nylon 12 angioplasty balloon (used by all interventional cardiology companies), the helical undulating wire stent (Johnson and Johnson/Cordis, FL and Medtronic, MN), the modular stent-graft for bypassing aortic aneurysms (Boston Scientific Corporation, MA), a drug-eluting coronary stent (TAXUS®, Boston Scientific, MA), radiation oncology catheters (TriSalus, CO), a novel glaucoma treatment device (the PRESERFLO® MicroShunt, InnFocus/Santen, Osaka, JP), the next generation intraocular lens (Eyedeal, Xi'an China), a pull-out injury safe urinary drainage catheter (InnoCare, Miami, FL) and two important biomaterials; Bionate® polycarbonate urethane (Covestro AG, Germany) and SIBS® (Innovia LLC, Miami, FL), used in hundreds of medical devices world-wide. Dr. Pinchuk's inventions are used in over a billion patients worldwide with a financial impact of well over $100 billion. He was inducted into the U.S. National Academy of Engineering (NAE) in 2012 and, among other prestigious awards, received the National Academy of Engineering 2019 Fritz J. and Dolores H. Russ Prize, the world's top prize in bioengineering, for innovations in medical devices that enable minimally invasive angioplasty treatment of advanced coronary artery disease. About BioFlorida BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in BioPharma, MedTech, Digital Health, and Health Systems that collectively employ nearly 107,000 Floridians (Source: TEConomy/BIO). BioFlorida's member driven initiatives provide a strong business climate for the advancement of innovative products and technology that improve lives and promote economic benefits to the state. SOURCE BioFlorida

ALACHUA, Fla.--(BUSINESS WIRE)-- Lacerta Therapeutics, a leader in the development of adeno-associated virus (AAV) technologies for the treatment of central nervous system (CNS) diseases, today announced a new AAV capsid licensing and research collaboration agreement with Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company. The collaboration will focus on the discovery and development of novel AAV capsids for CNS diseases. “We are eager to initiate this exciting collaborative work with our new partners at Prevail. The goal is to broaden the AAV medicines cabinet by improving the safety, potency, and manufacturability of novel AAV capsid variants for CNS indications,” said Edgardo Rodriguez-Lebron, Chief Executive Officer of Lacerta Therapeutics. “With this agreement, Lacerta gains its third clinical and commercial partner as we continue to push towards our shared goal of improving the life of patients with intractable neurodegenerative diseases.” As a part of the collaboration, Prevail will receive rights to utilize one of Lacerta’s novel AAV capsids for select undisclosed CNS targets. In addition, Lacerta’s proprietary AAV capsid platform will be utilized to discover and develop novel AAV capsids that are optimized to target desired CNS tissues and cell types. The collaboration will leverage Lacerta’s established expertise in AAV technologies and Prevail’s development and clinical capabilities, with the goal of accelerating development of gene therapies for patients with neurodegenerative disorders. “We are excited to be collaborating with Lacerta on the discovery of novel AAV capsids with improved CNS delivery to advance the development of new medicines by Prevail and the Lilly Institute for Genetic Medicine,” said Mansuo Shannon, Chief Scientific Officer of Prevail Therapeutics. “We believe this will allow us to move promising therapeutics more quickly to the clinic to study novel genetic medicines for diseases with high unmet need.” Lacerta will lead capsid discovery, screening, and validation, while Prevail will complete preclinical and Investigational New Drug-enabling studies with therapeutic payloads. Prevail will be responsible for all manufacturing, clinical development, and commercialization. Under the terms of the agreement, Lacerta will receive an upfront payment and will be eligible for development and commercial milestones, as well as tiered royalties on future net sales of products that incorporate capsids covered under the collaboration. Lilly will also provide Lacerta with new financing under the terms of a convertible promissory note agreement. About Lacerta Therapeutics Lacerta Therapeutics is a fully-integrated gene therapy company that is focused on leveraging its proprietary capsid technology and scalable manufacturing platforms to advance adeno-associated virus (AAV) based therapies via three primary approaches: gene replacement, gene modulation and novel vectorization of antibodies. View source version on businesswire.com: Contacts Amy Griffin Lacerta Therapeutics, Inc. info@lacertatx.com Source: Lacerta Therapeutics View this news release online at: