Hutchinson-Gilford progeria syndrome

Pictured: A U.S. federal bankruptcy courthouse building/iStock, GussWilder Eiger BioPharmaceuticals on Monday announced that it has voluntarily filed for bankruptcy and has kicked off the process of selling its assets and winding down the company’s operations. The California-based biotech has filed for Chapter 11 protection with the Bankruptcy Court for the Northern District of Texas, and has also submitted customary “first day” motions. If granted, the motion will provide Eiger some relief allowing it to transition into bankruptcy while it meets the company’s commitments to its stakeholders without “material disruptions” to its ordinary operations, according to the announcement. Eiger will sell all its assets during the bankruptcy case as it prepares for “an orderly wind down” of its operations. As part of the process, the biotech has inked a “stalking horse” agreement with rare disease player Sentynl Therapeutics which has agreed to purchase Zokinvy (lonafarnib), a farnesyltransferase inhibitor approved to treat the rare and progressive genetic disease Hutchinson-Gilford progeria syndrome. Sentynl will pay up to $26 million for Zokinvy, with the final price subject to other adjustments including per diem reductions. Zokinvy is an oral small molecule drug that blocks the farnesyltransferase protein. It is also approved for use in processing-deficient progeroid laminopathies with specific genetic mutations. The companies expect to close the sale by April 24, 2024. As part of its bankruptcy filing, other bidders can also submit competing offers for Eiger’s assets through a court-supervised process. Eiger’s bankruptcy filing on Monday caps off a difficult year for the biotech. In September 2023, it announced that it was discontinuing the Phase III LIMT-2 study of its candidate peginterferon lambda for chronic hepatitis delta. A quarterly review by a Data Safety Monitoring Board (DSMB) found concerning safety signals associated with peginterferon lambda. Four patients who received the investigational therapy developed hepatobiliary events, which led to liver compensation, leading the DSMB to recommend the study’s discontinuation. In September 2022, the FDA also rejected Eiger’s application for an Emergency Use Authorization for peginterferon lambda, which the biotech was proposing for the treatment of mild-to-moderate COVID-19. Following this rejection, Eiger in June 2023 laid off 25% of its workers in a sweeping reprioritization and savings program. The initiative also included a pivot to its GLP-1 receptor antagonist avexitide, which at the time was being developed for post-bariatric hypoglycemia and congenital hyperinsulinism. Eiger expected the cost-cutting measures to support its operations through the fourth quarter of 2024. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

PRF's 10 Marathon Runners Raise Funds to cure Progeria, all connected to Sam Berns PEABODY, Mass., March 26, 2024 /PRNewswire/ -- The Progeria Research Foundation (PRF), the only nonprofit solely dedicated to finding treatments and the cure for Progeria, celebrates its first year as an Official Charity Partner of the Boston Athletic Association's 128th Bank of America Boston Marathon®. Represented by a team of 10 passionate runners, this partnership will bring vital funds for Progeria research endeavors, while casting a light on this ultra-rare disease and PRF's progress toward the cure. Continue Reading Progeria is an ultra-rare, fatal "rapid-aging" disease. Without treatment, children with Progeria die of heart disease at an average age of 14.5 years old. PRF is making extraordinary progress, including the discovery of one FDA-approved treatment, development of genetic and other drug therapies, and creation of healthcare guidelines - all of which are giving these special children longer, healthier lives while the work toward the cure diligently continues. All ten PRF runners are personally connected in some way to Sam Berns. Post this All ten PRF runners are personally connected in some way to Sam Berns, son of co-founders Drs. Leslie Gordon and Scott Berns and the inspiration behind the creation of PRF. "What is so amazing is that every runner has a connection to our son Sam, and that means the world to us," said Drs. Gordon and Berns. "From Foxboro neighbors, to middle school classmates, to athletes who got to know Sam at PRF's annual 5K Race for Research – it's heartwarming to know they're now representing The Progeria Research Foundation at the Boston Marathon, in Sam's honor." Sam Berns grew up in Foxboro, MA, and by his teenage years, he had inspired millions around the world with his resilience and positive attitude. In 2013, Sam starred in HBO's Emmy Award-winning documentary, "Life According to Sam," which features him realizing his dream of playing the snare drum in the Foxboro High School marching band. That same week, he delivered his iconic TEDx talk, "My Philosophy for a Happy Life," which is the 7th most viewed TED talk of all time and recently exceeded 100M views across the TED and TEDx platforms. Runner Paul Michienzie said, "I do this for the kids (and now young and thriving adults thanks to PRF) with Progeria and in memory and honor of Sam Berns, who was our Foxboro neighbor and close family friend. He inspires me every step of the way from the Hopkinton start to the finish line on Boylston Street, just near my office where Sam, his parents, Leslie and Scott, and our friends and families have watched other marathoners make their way to the finish line. When I cross that line again, I will be looking high and thinking of Sam!" "This is a tremendous honor to be invited to join the Bank of America's Official Charity Program in the 2024 Boston Marathon," said Audrey Gordon, President and Executive Director of The Progeria Research Foundation. "PRF's partnership with this prestigious organization will help us spotlight our mission to cure Progeria, while bringing in the necessary funds to propel our research endeavors to new heights." For more information on PRF's runners or to support their fundraising campaigns directly, click here. ABOUT THE PROGERIA RESEARCH FOUNDATION The Progeria Research Foundation (PRF) is a nonprofit organization established in 1999 by the family of Sam Berns, a child with Progeria. PRF funds and conducts research-related programs, including the clinical trials and studies that led to approval of the first-ever FDA-approved drug for Progeria, lonafarnib (Zokinvy®). PRF is the only organization in the world solely dedicated to finding treatments and the cure for Progeria and its aging related conditions, including heart disease. Due to the discovery of the biological connection between Progeria, heart disease and aging, finding the cure for one of the rarest diseases on earth could provide keys for treating millions of adults with heart disease and stroke associated with the natural aging process, as well as help the entire aging population. The organization fills a void, taking these children out of the background where they had been for more than 100 years and putting them and Progeria at the forefront of scientific efforts. For more information, and to support PRF's mission, please visit . SOURCE Progeria Research Foundation

Pictured: A green, blue and pink ribbon representing rare disease advocacy/iStock, itakdalee Yesterday was Rare Disease Day 2024. It was a reminder of the importance of raising awareness for rare diseases—defined as those that affect fewer than 200,000 people in the U.S., but collectively affecting some 30 million Americans. While much has been achieved in improving access to lifesaving and life-changing treatments, much more remains to be done for patients with these disorders. Despite recent investment and breakthroughs for rare diseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. Not surprisingly, patient advocacy groups (PAGs) have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia. BioSpace spoke with leaders of PAGs formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies and the sharing of resources and expertise to maximize the impact of R&D and clinical trials. BioMarin Pharmaceutical picked the wrong week to disclose that it has been hit with a Department of Justice subpoena in connection with its sponsored testing programs for the rare disease therapies Vivizim—an FDA-approved enzyme replacement therapy for the treatment of Morquio A syndrome—and Naglazyme, an enzyme replacement therapy approved for mucopolysaccharidosis VI. Vivizim was approved in 2014 and Naglazyme was approved in 2005. In one of 2023’s most highly anticipated FDA approvals, Sarepta Therapeutics’ Elevidys got approved in June as the first gene therapy for DMD targeting the underlying cause of the progressive genetic muscle-wasting disease, despite failing to meet the primary functional endpoint in a randomized trial. With a $3.2 million per patient price tag, Elevidys—developed and commercialized in partnership with Roche—also has the dubious distinction of being one of the world’s most expensive medicines. Sarepta CEO Doug Ingram told investors in Wednesday’s fourth quarter and full-year 2023 earnings call that the launch of Elevidys was “particularly impressive,” bringing in over $200 million in revenue since mid-2023, especially given that its label is “limited to four- and five-year-olds, representing only about 3% or so of the total Duchenne population.” The successful launch of Elevidys “shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne, and for all those with genetic conditions with unmet needs,” Sarepta Chief Customer Officer Dallan Murray said during the call. However, Murray cautioned analysts to “not use this current younger population as a frame of reference for our market potential in the overall population.” At the end of last year, Sarepta submitted a supplemental BLA seeking to expand the gene therapy’s label by removing age and ambulation restrictions, while transitioning Elevidys from an accelerated to a traditional approval. The FDA has set a PDUFA date of June 21, 2024, and will not convene an advisory committee meeting to discuss the efficacy supplement to the BLA. “Hopefully, by July, we will have a broader label and we will be able to serve a much greater percentage of the [DMD] population,” Ingram said. Next, Sarepta should work on lowering the hefty price of Elevidys and ensuring equitable access to as many patients as possible who will benefit from the therapy. Greg Slabodkin is the News Editor at BioSpace. You can reach him at greg.slabodkin@biospace.com. Follow him on LinkedIn.