Zopapogene imadenovec-drba

Plus, news about SRT Therapeutics, Precigen, Enlivex, Tonix Pharmaceuticals and LakeShore Biopharma: Stealth BioTherapeutics resubmits its rare drug to the FDA: The privately held biotech’s experimental Barth syndrome drug was rejected by the regulator in May, and Stealth said it has since resolved manufacturing issues cited by the FDA. The agency had initially delayed its decision on the drug before issuing the CRL . Stealth is now seeking accelerated approval at the recommendation of the FDA. — Jaimy Lee Boehringer Ingelheim’s retinal pact: The German drug giant will work with Palatin Technologies on a melanocortin receptor agonist for diabetic retinopathy, paying up to €280 million (about $326 million). — Kyle LaHucik Former Prometheus Biosciences execs raise money for new biotech: Their startup, called SRT Therapeutics, raised $47 million, according to an SEC filing last week. The San Diego-based company was founded last year to go after “key pathways that not only modulate inflammatory pathways, but also promote tissue healing in IBD,” according to its LinkedIn page. SRT is led by Lauren Otsuki, a former operating chief at Prometheus, which Merck bought for $10 billion in 2023. Other Prometheus founders are also involved in SRT, including Cedars Sinai’s Stephan Targan and Janine Bilsborough. Several Prometheus employees followed CEO Mark McKenna to a new autoimmune biotech shortly after the acquisition. He started Mirador Therapeutics , which was unveiled in the spring of last year with $400 million for a still undisclosed pipeline . — Kyle LaHucik A course of Precigen’s therapy costs $460,000: The drugmaker announced Monday on an analyst call that the wholesale acquisition cost of its recurrent respiratory papillomatosis treatment is $115,000 per vial, and a course of treatment is four injections. The FDA approved the rare disease therapy, known as Papzimeos, on Thursday. Precigen’s shares $PGEN fell 5% premarket on Monday, though it’s up about 60% over the last five days. — Lei Lei Wu Enlivex Therapeutics’ cell therapy posts mid-stage data : In a Phase 1/2a trial in moderate-to-severe knee osteoarthritis, Allocetra showed a 24% reduction in knee pain and 26% improvement in knee function over placebo at three months. The company made no claim for statistical significance on these endpoints, and its stock $ENLV fell 25% on Monday morning after the data were announced. The therapy, composed of donor-derived mononuclear cells, also demonstrated a 72% reduction in knee pain and a 95% improvement in knee function compared with placebo among a subgroup of patients with age-related primary osteoarthritis. Enlivex called these “substantial, clinically meaningful and statistically significant” effects. There were no severe adverse events. The trial will continue to evaluate Allocetra’s effects at six and 12 months. — Elizabeth Cairns FDA approves fibromyalgia treatment: The agency on Friday greenlit Tonix Pharmaceuticals’ non-opioid analgesic for adults with the chronic pain condition. The sublingual formulation of cyclobenzaprine hydrochloride is designed for rapid absorption into the bloodstream. It will be branded as Tonmya. Tonix said it plans to launch Tonmya in the fourth quarter. It has not yet set a list price for the drug. — Ayisha Sharma LakeShore Biopharma gets an offer: Oceanpine Capital proposed a deal to buy the Beijing-based biotech for 86 cents per share $LSB , or about 8 cents per share above Friday’s closing price. LakeShore said the proposal is a preliminary, non-binding one, and the board still needs to “carefully review” Monday’s letter from Oceanpine, a life sciences and tech investment firm with about $3.5 billion in assets under management. The biotech, formerly known as YS Biopharma, is listed on the Nasdaq. It works on vaccines and biologics for infectious diseases and cancer, and it also markets a rabies vaccine in China. — Kyle LaHucik

The approval of Precigen’s Papzimeos (zopapogene imadenovec-drba) is in adults with RRP. Credit: grandbrothers / Shutterstock.com. The US Food and Drug Administration (FDA) has approved the first treatment for recurrent respiratory papillomatosis (RRP), a rare respiratory tract disease caused by human papillomavirus (HPV) that requires patients to undergo multiple surgeries every year. The approval of Precigen’s Papzimeos (zopapogene imadenovec-drba) by the FDA in adults with RRP means the need for repeated procedures to remove benign tumours caused by the disease could be reduced or eliminated. Papzimeos is a non-replicating adenoviral vector-based immunotherapy designed to help the immune system fight HPV types 6 and 11 – the two that cause RRP. The drug is administered as four subcutaneous injections over three months. According to Precigen, the immunotherapy targets the underlying pathology of RRP, meaning the benign tumours, also known as warts, that usually disturb the voice box and airway never develop. This was the case with Megan, a 23-year-old patient in the US with RRP, who required surgeries every eight months, as per a Recurrent Respiratory Papillomatosis Foundation (RRPF) patient story. Megan is just one of around 27,000 cases estimated in the US, many of whom rely on repeated surgeries to control the disease. RRPF president Kim McClellan said: “For the first time, adult patients with RRP have access to an FDA-approved therapy that offers the potential to reduce—or even eliminate—endless repeated surgeries.” GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Precigen CEO Helen Sabzevari said: “All adult RRP patients are now eligible for access to the first and only approved therapy that targets the root cause of the disease.” The FDA approval, which came in ahead of schedule of the agency’s Prescription Drug User Fee Act (PDUFA) date, represents a growing number of drugs approved with lower clinical data ceilings when intended for rare disease populations. Precigen completed submission of the rolling biologics licence application (BLA) in December 2024 under an accelerated approval pathway; however, the FDA granted Papzimeos full approval, which does not require a confirmatory clinical trial. The marketing decision was based on results from a single-arm, open-label pivotal trial (NCT04724980) conducted by clinicians at the National Institutes of Health (NIH). The trial evaluated Papzimeos in adult patients with RRP who required three or more surgeries per year. In patients who received Papzimeos over 12 weeks following surgical debulking procedures, 51.4% achieved a complete response – defined as not needing surgical intervention in the 12 months following treatment. Papzimeos showed a long-lasting effect, with follow-up data demonstrating responses were maintained in most patients through two years. Most treatment-emergent adverse events (AEs) were mild to moderate with no dose-limiting toxicities observed, and no reported treatment-related serious AEs. FDA’s Center for Biologics Evaluation and Research (CBER) director Vinay Prasad said: “Randomised trials are not always needed to approve medical products, and this approval is proof of that philosophy. “The FDA will always demand the correct clinical study for the specific medical product and disease. Our requirements for products given to tens of millions of healthy people will be different from products given to at most hundreds or thousands of patients with unique diseases.” The FDA approval validates Precigen’s decision to prioritise the drug in 2024. The strategic move involved cutting 20% of its workforce and pausing several other clinical programmes to target commercialisation of Papzimeos, formerly known as PRGN-2012. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now