PMP22

Pharnext hopes incoming data from a phase 3 trial run by its Chinese partner Tasly will offer some better news. Less than a year after Pharnext dropped its Alzheimer’s program over cost concerns, the French biotech has seen its lead program fail a phase 3 trial in a rare neurological disease. The company is developing PXT3003 in Charcot-Marie-Tooth disease type 1A, an inherited genetic disorder that affects the peripheral nerves, resulting in muscle weakness and potentially sensory loss. The 387-patient trial did not show that the drug was able to improve functional motor disability as measured by the Overall Neuropathy Limitation Scale (ONLS), the company revealed in a Dec. 11 release. Pharnext blamed an “unexpected” improvement in the disease among the placebo cohort, which it said “complicates the interpretation of the results.” “It also confirms what the medical literature as a whole has been saying for many years, namely that ONLS is undoubtedly a relevant endpoint over the long term, but not over such a short period of time as a clinical study,” the company claimed in the release. Trying to see the bright side, the biotech said there had been “no deterioration” of the condition among the patients who received the drug. “This suggests that PXT3003 might stabilize the condition of patients, which is an important consideration for a disease where progression is generally inevitable,” it concluded. PXT3003 is a combination of the approved meds baclofen, naltrexone and sorbitol. The oral drug is designed to downregulate the overexpression of the PMP22 protein in order to improve neuronal signaling in dysfunctional peripheral nerves. Pharnext hopes incoming data from a phase 3 trial run by its Chinese partner Tasly will offer some better news. In the meantime, the company “plans to continue analyzing the data, particularly in collaboration with potential partners for licensing or acquisition of PXT3003.” “Although the results we are sharing today are not exactly what we had hoped for, they are nonetheless very promising,” Pharnext’s Manager Hugo Brugière said in the release. “We are now going to make the most of all the data we have accumulated over the last 10 years, including our two phase 3 studies and our 6-year extension study, which tend to demonstrate a beneficial effect on patients.” “I remind that, today, PXT3003 is the only ray of hope for all CMT1A patients, and that no other drug candidate is currently in an advanced stage,” Brugière added. “It seems to me, therefore, that efficacy results for a risk-free drug could support an application for regulatory approval and market authorization. This is for us the final stage, which we will move forward with the patient community." Investors didn’t appear to see much cause for optimism, sending the company’s share price plunging 68% on Monday morning. However, with the company’s stock already sitting at below one euro cent, the difference was negligible in practicable terms. Pharnext’s stock crashed back in February, when the biotech walked away from its potential Alzheimer’s asset, dubbed PXT864, arguing that the encouraging data produced to date weren't enough to lead to commercialization in the “short or medium term.”  Instead, Brugière said at the time that the company would search for a partner to take the clinical baton.  It left Pharnext even more reliant on PXT3003. The company said in October that it had received potential international licensing offers worth over 250 million euros for the drug, although it remains to be seen whether these as-yet-undisclosed deals will be impacted by today’s results.

FIRST INVESTMENT FROM CMTA'S NEW VENTURE PHILANTHROPY ARM GLENOLDEN, PA / ACCESSWIRE / November 16, 2023 / The Charcot-Marie-Tooth Association (CMTA) today announced that it participated in the Seed Series Extension syndicate for Armatus Bio, Inc. (Armatus). Armatus is an emerging Biotechnology company that is advancing a unique gene therapy clinical candidate to target CMT type 1A, the most common form of the disease. Charcot-Marie-Tooth disease is an inherited peripheral neuropathy, principally affecting the long nerves of the hands and feet. Currently there are no FDA-approved therapies for any type of CMT. Thomas Dubensky, Ph.D., a CMTA board member commented "The CMTA is very excited to facilitate the advancement of novel therapies to be ultimately evaluated in patients living with all types of CMT. Armatus is the CMTA's first investment from its new venture philanthropy arm, which will make selected early-stage investments in Biotech companies developing unique and promising therapies against all types of CMT." Dr. Dubensky went on to say that "the venture philanthropy arm will work in tandem with STAR (Strategy to Accelerate Research), the arm of CMTA which is the largest private funder of CMT research in the world." Rachel Salzman, DVM, CEO of Armatus, commented, "Armatus is extremely grateful to the CMTA for its support, and we look forward to their continued advice and counsel as we advance ARM-101." Dr. Salzman went on to say, "Our ARM-101 development studies are particularly important for the CMT community because the data generated are designed to answer critical questions about how we can effectively deliver genetic medicines to the right parts of the body to meaningfully address this disease." She added, "As we accelerate development of ARM-101 and gain further evidence that our vectorized RNAi can normalize PMP22 in Schwann cells as a means of enabling human trials, we believe this therapy has the potential to provide benefit to the thousands of people living with CMT1A in the U.S. and around the world." Armatus Bio, based in Columbus, Ohio, is an emerging biotechnology company that is collaborating with renowned gene therapy experts to build a pipeline of novel therapeutic candidates designed to overcome the limitations of today's approaches and propel the next generation of genetic medicines. ABOUT THE CHARCOT-MARIE-TOOTH ASSOCIATION The Charcot-Marie-Tooth Association (CMTA) is the largest philanthropic funder of CMT research worldwide. The CMTA's Strategy to Accelerate Research (STAR) brings the best CMT researchers, clinicians, and experts in therapy development together with patients, pharmaceutical and biotechnology companies to expedite the development of treatments for CMT. Since 2008, the CMTA has invested more than $23.5 million via STAR. The CMTA also improves quality of life for CMT families by offering educational programs and materials, patient and professional conferences, support through youth programs, its branch system and global clinical Centers of Excellence. To learn more visit cmtausa.org and follow the CMTA on Instagram, Facebook, Twitter, TikTok, LinkedIn, and YouTube. Contact Information Marcia Semmes Media Contact, Charcot-Marie-Tooth Association marcia@cmtausa.org SOURCE: Charcot-Marie-Tooth Association View source version on accesswire.com:

Augustine Therapeutics announces a collaboration with the Charcot-Marie-Tooth Research Foundation (CMTRF) in the USA. CMTRF is a US-based, patient-led, non-profit focused on delivering treatments for Charcot-Marie-Tooth disease (CMT). With the financial support of CMTRF, Augustine Tx will investigate the efficacy and safety of their newly discovered class of selective HDAC6 inhibitors. Augustine Tx creates innovative medications that target the fundamental disease mechanisms of Charcot-Marie-Tooth - a category of rare, inherited chronic peripheral neuropathies causing progressive nerve and muscle deterioration. With almost three million individuals affected globally, there is currently no treatment approved for CMT. Augustine Tx’s pipeline is based on small molecule inhibitors of HDAC6, a class II histone deacetylase. This is a well-known molecular target in neurodegenerative disorders and neuropathies. Augustine’s third generation HDAC6 inhibitors do not exert known liabilities of earlier generations and therefore represent a very promising novel class of therapeutic molecules. Augustine Tx lead drug candidate AGT-216 has demonstrated significant potential in preclinical trials. In CMT1A C3 mice, modeling subtypes of the disease with the PMP22 underlying genetic mutation, the candidate has demonstrated HDAC6 target engagement and reversal of disease phenotypes. Effect in axonal damage biomarkers has been shown too, as well as halting disease progression after once-daily treatment for 4 weeks. Helped by the funding of CMTRF, Augustine Tx now aims to continue development of AGT-216. The objective is to gain an in-depth understanding of the compound by conducting pharmacological dose range finding, non-GLP toxicology and safety studies, as well as long-term efficacy tests in rodents. Conducting these studies is essential to advance the development of AGT-216 towards clinical studies in humans. Apart from its lead candidate, Augustine Tx will also continue assessments on other compound candidates identified using its drug discovery platform. Prof. dr. Ludo Van Den Bosch (VIB-KU Leuven), founding scientist at Augustine Tx and chairman of the Scientific Advisory Board says: “At Augustine Tx, we aim to deliver the first truly curative option for CMT patients. Following promising preclinical studies, we are on the right track to advance towards future clinical tests”. Cleary Simpson, CEO of CMTRF says: “HDAC6 inhibitors are promising as potential therapeutics for several forms of CMT. With funding from the CMT Research Foundation, Augustine Tx will be able to continue research on their promising lead candidate tackling CMT1A disease and to take the necessary steps towards first-in-human trials. We are hopeful that this collaboration will accelerate the progress to finding a cure for CMT1A patients who currently have no treatment options available.” Sylvain Celanire, CEO of Augustine Tx adds: “Our development progress will be greatly expedited thanks to the support provided by the CMT Research Foundation along with support by our historical investors as we move towards IND enabling studies”.