Developmental delay

Oct. 08, 2024 -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced a regulatory update from the U.S. Food and Drug Administration (FDA). The FDA Review Division has determined that there does not appear to be a need for an advisory committee meeting at this time for the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS). However, the Division’s review team will continue to consider the potential need for an advisory committee meeting during their ongoing review. As announced previously, the DCCR NDA was designated for Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024. The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food, which can severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder. DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters. Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. An NDA for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS) is currently under review by the FDA and was granted Priority Review. For more information, please visit www.soleno.life. The content above comes from the network. if any infringement, please contact us to modify.

WARREN, NJ, USA I October 1, 2024 I PTC Therapeutics, Inc. (NASDAQ: PTCT ) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter. “The FDA filing acceptance for sepiapterin is a critical milestone toward bringing this potential important therapy to children and adults living with PKU in the United States,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “The clinical trial data, including the evidence of enabling diet liberalization, support that sepiapterin can provide significant benefit to PKU patients, helping to meet the persistent, significant unmet need in this community.” “NPKUA is deeply committed to improving the lives of individuals with PKU,” said Catherine Warren, Executive Director, National PKU Alliance. “Our families have been anxiously waiting for new treatments that provide an opportunity to lessen their dependance on costly and burdensome diets. We are excited by today’s news, as it brings us one step closer to providing another option for people of all ages with PKU to manage their condition.” The sepiapterin NDA is based on the highly statistically significant and clinically meaningful results from the phase 3 APHENITY trial. The results demonstrate a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU. The majority of subjects (84%) achieved Phe control in accordance with treatment guidelines of <360 µmol/L, and 22% of subjects showed normalization of Phe levels. The NDA also includes data from the APHENITY open-label extension study, which provides evidence of sepiapterin’s durability of effect as well as data from the Phe tolerance sub-study. The recent analysis from the Phe tolerance sub-study demonstrates that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while still maintaining Phe levels <360 μmol/L. The results from the Phe tolerance protocol support that sepiapterin could enable patients to liberalize their highly restrictive diets while still maintaining Phe control. About Sepiapterin Sepiapterin (formerly PTC923), an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, sepiapterin is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH. Sepiapterin also has an independent chaperone effect, protecting against PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, sepiapterin effectively reduces blood phenylalanine (Phe) levels and has the potential to treat the broad range of PKU patients. About Phenylketonuria Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine. If left untreated or poorly managed, phenylalanine – an essential amino acid found in all proteins and most foods – can build up to harmful levels in the body. This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with phenylketonuria initially don’t have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of phenylalanine in the first few years of life is irreversible. Diagnosis of phenylketonuria usually takes place during newborn screening programs. There are an estimated 58,000 people with phenylketonuria globally. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on X, Facebook, Instagram and LinkedIn. SOURCE: PTC Therapeutics

Genespire raises €46.6 million (~$52 million) in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic One of the largest private Italian Biotech company financings to date Financing co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital forming a strong syndicate with Indaco SGR Funds dedicated to advancing off-the-shelf pediatric gene therapy for Methylmalonic Acidemia (MMA) into the clinic, and to bolstering the Company’s gene therapy pipeline 25 September 2024 – Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, today announced the closing of a €46.6 million (~$52 million) Series B financing, co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital through its Large Venture Fund forming a strong syndicate with Indaco SGR. The financing will enable the development of GENE202, the company’s lead candidate, up to a Phase I/II clinical trial for the treatment of MMA, a devastating genetic disorder impairing the metabolism of certain amino acids and fats. MMA has onset in early infancy, is associated with significant mortality and morbidity, and is characterized by severe clinical manifestations including muscular weakness, seizures, developmental delays and organ damage. Currently, there are no disease-modifying treatments for MMA. GENE202 is a pioneering off-the-shelf gene therapy which harnesses the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLVs are designed to be used intravenously, enabling the patient's liver to produce the therapy throughout its lifetime. This unique mode of action makes Genespire’s approach optimally suited to address pediatric patients affected by genetic diseases who currently face the most pressing unmet medical needs, while also benefitting adult patients. The ISLV technology was developed by Genespire’s scientific co-founders and gene therapy leaders, Pr. Luigi Naldini and Dr. Alessio Cantore, at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget). This Series B financing round will also strengthen the Company’s pipeline by fostering discovery and preclinical work on candidate products targeting several additional genetic diseases. Karen Aiach-Pignet, Genespire’s CEO said, “Our highly innovative ISLV platform gives Genespire the unique ability to bring groundbreaking treatments that have the potential to transform the lives of children suffering from genetic disease. The support from our new and existing investors reflects the enthusiasm and confidence in our platform’s potential, with further validation achieved from the promising data generated to date. We extend a heartfelt gratitude to our previous CEO, Julia Berretta, for her instrumental role in this financing round, and we look forward to working closely with CDP Venture Capital, XGEN Venture and Indaco SGR, alongside our founding investor, Sofinnova Partners, as well as the SR-Tiget team, as we drive our first therapy in MMA towards the clinic.” “Since our initial investment, we’ve been deeply convinced by Genespire’s world-class team and its groundbreaking lentiviral technology.” said Lucia Faccio, Partner at Sofinnova Partners. “We remain firmly committed to supporting the company’s growth in collaboration with our colleagues from XGEN, CDP Venture Capital and Indaco, to pave the way for single treatment approaches for several genetic disorders in children.” “Gene therapy is starting to deliver results with several recent clinical successes and product approvals and we believe that Genespire’s approach offers huge opportunities. We look forward to working with fellow investors and the Management team to help realize this potential.” commented Paolo Fundarò, Founder and Managing Partner from XGEN Venture. “Health, particularly Biotechnology Research dedicated to therapeutic needs, is one of the strategic macro-areas prioritized in our 2024-2028 Business Plan,” says Agostino Scornajenchi, CEO and General Manager of CDP Venture Capital, “Genespire’s research started in the renowned laboratories of the San Raffaele Institute, and is being advanced by an ambitious team that, through advanced gene therapy technologies and gene therapies, aims to offer curative treatments for diseases that currently lack effective therapeutic options, such as MMA. We look forward to supporting the company to set a new international gold standard.” In connection with the Series B round, Marco Dieci, Senior Advisor at CDP Venture Capital and Chief Executive Officer of Simis S.r.l., will join Genespire’s Board of Directors as the representative for CDP Venture Capital, alongside Paolo Fundarò, Founder and Managing Partner from XGEN Venture. Genespire is a biotechnology company, developing off-the-shelf gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients affected by genetic diseases. ISLVs are designed to be used intravenously and allow the life-long production of the therapy directly from the patient’s liver. Genespire is initially advancing therapeutic programs in inherited metabolic diseases with high unmet medical need. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini and Dr. Alessio Cantore, at the Fondazione Telethon and Ospedale San Raffaele. Genespire is a spin-out of SR-Tiget, a world leading cell and gene therapy research institute. Find out more about us at www.genespire.com. Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London and Milan, the firm brings together a team of professionals from all over the world with strong scientific, medical and business expertise. Sofinnova Partners is a hands-on company builder across the entire value chain of life sciences investments, from seed to later-stage. The firm actively partners with ambitious entrepreneurs as a lead or cornerstone investor to develop transformative innovations that have the potential to positively impact our collective future. Founded in 1972, Sofinnova Partners is a deeply established venture capital firm in Europe, with 50 years of experience backing over 500 companies and creating market leaders around the globe. Today, Sofinnova Partners has over €2.8 billion under management. For more information, please visit: www.sofinnovapartners.com. For more information about the Sofinnova Telethon Strategy, visit: https://sofinnovapartners.com/strategy/telethon. XGEN Venture is a Milan-based, recently created life science venture capital firm. We work with a group of exceptional scientists and entrepreneurs to translate high level science into solutions for patients. Our company creation strategy identifies and supports the most convincing opportunities to build effective businesses and create value. Investing in technologies that truly improve severe health conditions is at the core of what XGEN does and is committed to doing so in a responsible and impactful way, promoting best ESG practices within the meaning of SFDR art 8. For more information, please visit www.xgenventure.com. CDP Venture Capital is an asset management company participated by CDP Equity (70%) and Invitalia (30%) with the goal of making venture capital a cornerstone of Italy's economic development and innovation, creating the conditions for the overall, sustainable growth of the entire innovation ecosystem. CDP Venture Capital manages 13 investment funds, amounting to over 3 billion euros in resources that support innovative enterprises at all stages of their life cycle, making both direct and indirect investments (funds of funds). Indaco Venture Partners SGR is one of the largest independent Italian venture capital asset management companies, with over €370 million in assets under management, focused on companies that innovate in electronics, robotics, new materials, medtech, biotech – pharma and digital. Indaco Bio Fund (around €100 million) is exclusively dedicated to investing in the development of innovative pharmacological therapies, across all therapeutic indications but with particular attention to Oncology, CNS, Ophthalmology, Metabolic diseases, Virology, and Infectious diseases. Indaco is focused on the Italian Biotech sector with select investments in other European countries, USA, Canada, and Israel. The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) was created in 1996 as a joint venture between the Fondazione Telethon and Ospedale San Raffaele, with the mission to perform cutting-edge research, towards the discovery of novel gene and cell therapy platforms and their development from preclinical models to first-in-human testing. The content above comes from the network. if any infringement, please contact us to modify.