Sodium channels

BRIGHTON, England--( BUSINESS WIRE )--Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of those suffering from respiratory disease, today announced its novel cystic fibrosis (CF) investigational therapy, ETD001, has been granted ‘rare pediatric disease designation’ (RPD) in the US by the Food and Drug Administration (FDA). 1 ETD001 is a low molecular weight compound with first-in-class potential, which targets the sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of mucus. Enterprise announced dosing of the first person with cystic fibrosis (pwCF) in its Phase 2a trial of ETD001 on 23 rd July 2024 2 . The trial aims to deliver clinical proof-of-concept and to assess the safety and efficacy of ETD001 in the ~10% of pwCF with the highest unmet medical need. The study will assess lung function (FEV1) in pwCF who are either ineligible for or are not receiving CFTR modulator therapy. The RPD designation was based on the assessment of CF as a serious or life-threatening disease, primarily affecting individuals aged from birth to 18 years. CF is estimated to affect over 100,000 people worldwide, with an average life expectancy of only 50 years. Failed mucociliary clearance and mucus congestion in the lungs leads to cycles of infection and inflammation and an ongoing decline in lung function. Increasing fluid volume in the lung by inhibiting ENaC with ETD001 will hydrate mucus, improve clearance, reduce mucus congestion, and is expected to drive substantial improvements in lung function. ETD001 has previously demonstrated a well-tolerated profile in healthy subjects in a Phase 1 trial and has been shown to be long-acting in pre-clinical studies 3 . Dr John Ford, CEO, Enterprise Therapeutics, said: “We’re delighted with the FDA’s decision and would like to thank the Office of Pediatric Therapeutics and the Office of Orphan Products Development for their consideration. As we progress though our Phase 2a trial of ETD001, this RPD designation will further support our mission to advance this novel approach for treating pwCF with the highest unmet medical need, as rapidly as possible.” Annabella Amatulli, Head of Regulatory Affairs, commented: “We are thrilled to be granted the RPD designation by the FDA, a regulatory framework intended to encourage and accelerate innovative therapies, in recognition of the significance of our programme in addressing an unmet medical need. The RPD designation will give Enterprise access to valuable incentives and support from the FDA during the development of ETD001, including the eligibility to request a Priority Review Voucher (PRV) at the time of marketing approval.” https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions https://enterprisetherapeutics.com/enterprise-therapeutics-doses-first-person-with-cystic-fibrosis-in-phase-2-trial-for-novel-therapy-etd001/ https://enterprisetherapeutics.com/enterprise-therapeutics-publishes-preclinical-profile-of-etd001-a-novel-inhaled-enac-blocker/

PARIS, Sept. 24, 2024 (GLOBE NEWSWIRE) -- AlgoTherapeutix (AlgoTx), a clinical stage biotechnology company developing a first-in-class therapeutic for peripheral neuropathic pain, ATX01, announced today the completion of the Last Patient Last Visit (LPLV) in its 276 patient Phase II trial known as 'ACT (ATX01 in ChemoTherapy-induced peripheral neuropathy)’. Conducted under the guidance of Professor Guido Cavaletti (Univ of Milano-Bicocca, Italy), ACT is an international, double-blind, placebo-controlled evaluation of ATX01’s efficacy and safety in patients suffering from CIPN, a common side effect of neurotoxic cancer treatments caused by nerve degeneration (1). AlgoTx’s Founder, President & CEO, Stéphane Thiroloix said, “We’re delighted with the solid progress of our ACT clinical trial and commend the patients, investigators and AlgoTx clinical team for their dedication in achieving this major milestone. The trial involved over 40 sites in the US, Belgium, Czechia, France, Italy, Poland and Spain, which is a significant undertaking, and commitment to making a difference, for any biotech company.” AlgoTx’s Chief Development Officer, Philippe Picaut added, “CIPN affects two thirds of chemotherapy patients, representing a high unmet need without any approved treatments. Any progress in this field is therefore very gratifying. We look forward to sharing the ACT data and progressing to the next stage.” The company’s high-concentration, non-opioid ATX01 selectively targets specific nociceptive sodium channels in the peripheral nervous system involved in pain signaling. Its twice-daily local application provides sufficient concentrations to alleviate extreme pain whilst minimizing toxicity through limited systemic exposure. Reference: (1) “Efficacy and Safety of ATX01 in Adult Patients With CIPN (Chemotherapy-induced Peripheral Neuropathy) (ACT)”. The purpose of this clinical trial is to compare the efficacy of twice daily applications of ATX01 (10% & 15%) versus placebo during a 12-week treatment period in treating CIPN in adult cancer survivor patients. Full details here: [link] ENDS Media Contacts AlgoTxCEO Stéphane Thiroloixstephane@algotx.com Scius CommunicationsKatja StoutKatja@sciuscommunications.com+44 778 943 5990 Daniel Goochdaniel@sciuscommunications.com +44 774 787 5479 About AlgoTherapeutix (AlgoTx) AlgoTherapeutix (AlgoTx) is a clinical stage biotechnology company developing a targeted, first in class therapeutic for peripheral neuropathic pain. Its lead asset, ATX01, is a proprietary topical formulation of amitriptyline for use in chemotherapy-induced peripheral neuropathy (CIPN), an area of high unmet need associated with unbearable pain in patients’ extremities and for which there is currently no viable treatment options. ATX01 targets the signaling pathways involved in the regulation of pain by inhibiting specific nociceptive sodium channel receptors. Its topical administration provides targeted relief from the excruciating pain in patients’ extremities from CIPN, which is caused by nerve degeneration and is a common side effect of cancer treatment, occurring in 68% of patients. ATX01 was granted FDA fast track development status in CIPN. The company has completed recruitment into an international Phase II clinical trial of 276 patients with CIPN (the ‘ACT’ study). This trial is due to provide its final data read early in 2025, and the company is making plans to expedite a Phase 3 trial. The company has seasoned leadership, with experience of bringing global products successfully to market, and is backed by an international board. AlgoTx raised a Series B round of EUR 20 million in 2023 and has raised EUR 35 million to date. AlgoTx has a consortium of investors including Relyens Innovation Santé / Turenne Capital, who led the Series B fund raise, along with UI Investissement, and angel investors. Historical investors include Bpifrance through its Innobio 2 fund and Omnes Capital. In 2023, the company won France Biotech’s prestigious ESG HealthTech Trophy in recognition of its early commitment to ESG initiatives. For more information, see the company’s website and follow AlgoTx on LinkedIn.

Vertex Pharmaceuticals’ breakthrough in cystic fibrosis was a medicine that treats just 4% of patients who have the lung disorder. Today, Vertex’s portfolio of blockbuster CF products spans multiple drugs and drug combinations that can treat 90% of CF patients. CEO Reshma Kewalramani says Vertex aims to eventually reach all CF patients. But as the company moves closer to this goal, it brings up another part of the company’s strategy: pipeline diversification. The lion’s share of Vertex’s revenue comes from its CF portfolio, so revenue growth will have to come from adding on new therapeutic areas. Kewalramani sees this effort as building on the company’s CF work. “The real drive for Vertex is to invest in serial innovation, not just do it once, but do it repeatedly,” Kewalramani said, speaking in a fireside chat Monday at the World Medical Innovation Forum in Boston. “The goal here is to learn from all the lessons we have from CF and to invest in other disease areas.” presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum While Vertex is known for CF drugs, its research in pain medicines actually began even earlier. Vertex began its CF work about 20 years ago, Kewalramani said. The pain program started 25 years ago. The same scientists who worked on CF are the exact ones who now work on the pain program, she said. The past two decades have not seen new medications for acute pain or chronic pain. With innovation lacking in this therapeutic area, opioids filed the gap. Vertex is trying to change pain treatment paradigm with a different approach. Opioids treat pain by working in the central nervous system (CNS) to alter the perception of pain, Kewalramani said. By developing drugs that act on the peripheral nervous system, Vertex aims to avoid the safety, tolerability, and addictive problems of drugs that act on the CNS. Vertex’s most advanced pain product candidate is Suzetrigine, formerly known as VX-458. This non-opioid drug is a small molecule that stops pain signals from reaching the brain by blocking NaV1.8, a sodium channel in peripheral nerves. Kewalramani acknowledges that other companies are also developing sodium channel-blocking drugs. But Vertex is in position to bring one to patients sooner. The FDA accepted Vertex’s new drug application for Suzetrigine in July, setting a Jan. 30, 2025 target date for a regulatory decision. Vertex separates the pain market into three segments: acute, neuropathic, and musculoskeletal. The latter two segments are what many people would call chronic pain, Kewalramani said. Vertex is aims to discover, develop, and commercialize drugs for acute and neuropathic pain. These medicines may also work on musculoskeletal pain, but Kewalramani said that if the company enters the market for such drugs, it will be through a partnership. Sponsored Post What Healthcare Can Learn from Costco The path forward is clear: employers must evaluate their health plan the same way they do their Saturday shopping excursion. By Jeff Bak, Imagine360 CEO and President When Vertex launched 35 years ago, the company’s focus was developing small molecule drugs. With that in mind, Kewalramani said it’s interesting to note that Vertex is now the first to bring patients a CRISPR-Cas9 therapy, Casgevy. This genetic medicine won FDA approval last winter for treating the rare blood disorders sickle cell disease and beta thalassemia. Vertex’s broadening scope includes cell therapies for type 1 diabetes. VX-880 is what Kewalramani describes as a “naked cell” program. This therapy involves taking stem cells and coaxing them into differentiating into pancreatic islet cells that are sense glucose levels and release insulin. But this therapy requires chronic immunosuppression to prevent the immune system from destroying these cells. The next Vertex type 1 diabetes cell therapy, VX-264, takes these same cells and encapsulates them in a proprietary device that shields them from the immune system so no immunosuppression is needed. Both programs are in the clinic. The third program type 1 diabetes program is still preclinical, but here, Vertex is using CRISPR technology to edit cells to avoid sparking an immune response, obviating the need for immunosuppressants. Kewalramani said the potential to move from chronic therapies to a potentially curative therapy makes this one of the programs she is most excited about. Rather than focusing on specific technologies, Vertex is a disease-first company, she said. “We use whatever modality is needed to transform if not cure those diseases of interest,” Kewalramani explained. “One of the advantages to having that approach is that we can combine modalities.”