Latest news with #Precigen
Yahoo
17 hours ago
- Health
- Yahoo
Precigen wins immunotherapy approval; Pfizer sickle cell drug fails trial
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. Today, a brief rundown of news involving Precigen and Pfizer, as well as updates from the Institute for Clinical and Economic Review, Superluminal Medicines and Generation Bio that you may have missed. The Food and Drug Administration granted full approval to a first-of-its-kind treatment for recurrent respiratory papillomatosis, a rare and potentially life-threatening condition caused by persistent HPV infections. Thursday's clearance of Precigen's Papzimeos, an immunotherapy that helps clear HPV-infected cells, was based on study results showing a little more than half of drug recipients didn't need surgery within a year of therapy. Center for Biologics Evaluation and Research director Vinay Prasad, who rejoined the FDA this week, described the approval as proof 'randomized trials are not always needed to approve medical products.' That statement should be 'reassuring' to biotech investors concerned about stricter regulatory standards under Prasad, wrote Cantor Fitzgerald analyst Jennifer Kim. Precigen shares rose higher Friday on the news. — Ben Fidler An experimental Pfizer drug for sickle cell disease failed to meet its goal in a Phase 3 study, the company said Friday. Testing showed that treatment with inclacumab, a drug Pfizer acquired via its 2022 buyout of Global Blood Therapeutics, failed to significantly reduce versus placebo the pain crises people with sickle cell often experience. Pfizer said it would share analyses of the data with the scientific and patient community in 'due course.' Last year, the company pulled from market another sickle cell drug, Oxbryta, that it gained from Global Blood, citing safety concerns. The company plans to provide updates on Oxbryta and a third Global Blood drug, the experimental osivelotor, when they become available. — Ned Pagliarulo Eli Lilly will collaborate with biotechnology startup Superluminal Medicines to develop new drugs for cardiometabolic diseases and obesity. Through the alliance, the two intend to discover and advance small molecule medicines aimed at undisclosed G protein-coupled receptor, or GPCR, targets 'relevant' to those conditions. Lilly will receive exclusive rights to the compounds emerging from the deal, while Superluminal could get up to $1.3 billion in total payouts, including an unspecified upfront payment as well as an equity investment, the companies said Thursday. — Ben Fidler Autolus Therapeutics is delaying launching its leukemia cell therapy Aucatzyl in Europe following approval there as the company 'evaluates potential pricing and feasibility of market entry opportunities' in some countries. Launch in Germany is on hold and Autolus 'does not anticipate any EU sales of Aucatzyl in 2025 and 2026,' the company said in its second quarter earnings report. In the U.K., where Aucatzyl has also been approved, a government cost-effectiveness monitor has initially decided not to pay for it. Autolus said it 'will continue to work towards a pathway for patient access to therapy in the U.K.' Approved by the Food and Drug Administration in November 2024, Aucatzyl earned just shy of $30 million in sales in the first six months of 2025, all from the U.S. — Jonathan Gardner Generation Bio revealed preclinical results suggesting a delivery technology it's developing can effectively send nucleic acid payloads into T cells. But the company also said this week that it may not be able to raise the funds to prove that approach works in humans and, as a result, began a strategic review that could end in a sale or merger. Generation will lay off roughly 90% of its workforce, including all of its research and development staff, by the end of October. Company shares climbed 60%, though they've lost much of their value since the company's initial public offering in 2020. — Ben Fidler Recommended Reading Eli Lilly's Julie Gilmore on finding up-and-coming companies and backing small biotechs Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
a day ago
- Health
- Business Insider
Precigen Stock (PGEN) Roars Over 60% Higher on FDA Green Light for Papzimeos Therapy
Shares in biopharmaceutical company Precigen (PGEN) rocketed higher today after the U.S. Food and Drug Administration gave the green light to a therapy to treat a rare respiratory disease. Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. The approval of the therapy, called Papzimeos, is the first treatment for the condition to receive the health regulator's nod. Fatal Tumors Papzimeos has been approved to treat recurrent respiratory papillomatosis (RRP) — a condition that causes growth of wart-like tumors in the respiratory tract due to human papillomavirus (HPV) infection. RRP can lead to severe voice disturbance, a compromised airway, and recurrent post-obstructive pneumonias. Precigen estimates that there are about 27,000 adult RRP patients in the U.S. RRP can be fatal as there is no cure and the current standard-of-care is repeated surgeries. However, despite being removed in these surgeries, the growth has a tendency to return. 'For more than a century, since RRP was first recognized as a distinct disease, patients have had to rely on repeated surgeries to manage this relentless condition. Today marks a historic turning point. With the landmark FDA approval all adult RRP patients are now eligible for access to the first and only approved therapy that targets the root cause of the disease,' said Helen Sabzevari, PhD, President and CEO of Precigen. Analyst Upgrade Simon Best, associate professor of Otolaryngology at Johns Hopkins Hospital, added: 'Everybody is anxiously awaiting a new treatment for this disease. The patients are and the surgeons are. There's nothing more frustrating than doing a surgery and then having the patient come back six months later.' The approval was based on an early-to-mid-stage study data that showed 51% of patients required no surgeries in the 12 months after the treatment. upgraded Precigen to Neutral from Underweight on the news. It cited the removal of Precigen's 'major regulatory overhang' and its revenue potential for the upgrade. It estimates that peak sales for the drug in the U.S. to be about $250 million. Precigen is certainly making strides when it comes to its financials – as can be seen below. Precigen recently reported a Q2 net loss of $26.6 million, a significant improvement from the $58.8 million loss in the same period last year. Total revenues for the quarter were $856,000, up from $717,000 in the previous year, driven by increased service revenues. Is PGEN a Good Stock to Buy Now? On TipRanks, PGEN has a Hold consensus based on 2 Buy, 1 Hold and 1 Sell ratings. Its highest price target is $6. PGEN stock's consensus price target is $4.50, implying a 143.24% upside.
Reuters
2 days ago
- Health
- Reuters
Precigen shares jump after US FDA approves first therapy for rare respiratory disease
Aug 15 (Reuters) - Precigen's (PGEN.O), opens new tab immunotherapy for a rare respiratory disease has become the first treatment to win U.S. regulatory approval for the condition, which typically requires frequent surgeries, sending the company's shares soaring 83% on Friday. The U.S. Food and Drug Administration has approved the therapy, Papzimeos, to treat adults with recurrent respiratory papillomatosis (RRP) — a condition that causes growth of benign tumors in the respiratory tract due to human papillomavirus (HPV) infection. A distinguishing aspect of RRP is the tendency for the tumors to return even after removing them through surgical procedures. The disease can be fatal as there is no cure. The FDA approval was based on an early-to-mid-stage study data that showed 51% of patients required no surgeries in the 12 months after the treatment. "Randomized trials are not always needed to approve medical products and this approval is proof of that philosophy," said Vinay Prasad, who recently returned to the FDA to oversee vaccine, gene therapy and blood product regulation. Papzimeos is designed to stimulate an immune response against cells infected with HPV types 6 and 11 — the strains that cause the disease. H.C. Wainwright analysts estimate peak sales of the drug to reach $1.1 billion in 2033. "We may finally be able to say no more surgery," said Kim McClellan, president of the Recurrent Respiratory Papillomatosis Foundation. McClellan herself was diagnosed with RRP at the age of five and has since then had more than 250 surgeries. Precigen estimates about 27,000 adult RRP patients in the U.S. It did not immediately respond to a Reuters request for comment on the treatment's pricing. Simon Best, associate professor of Otolaryngology at Johns Hopkins Hospital, said patients were eagerly awaiting a new treatment. "There's nothing more frustrating than doing a surgery and then having the patient come back six months later."
Business Wire
2 days ago
- Health
- Business Wire
Hope Realized: FDA Approves Papzimeos for Adults with Recurrent Respiratory Papillomatosis (RRP)
FORT WORTH, Texas--(BUSINESS WIRE)--The Recurrent Respiratory Papillomatosis Foundation (RRPF) is excited to share the news that the U.S. Food and Drug Administration (FDA) has granted full approval to Precigen's Papzimeos (zopapogene imadenovec-drba) under Priority Review for the treatment of adults with Recurrent Respiratory Papillomatosis (RRP). Papzimeos is the first and only FDA-approved treatment for RRP. (See FDA's announcement here) A moment of profound healing for the RRP Community This is truly a remarkable day – the culmination of decades of advocacy, resilience, and unwavering commitment from patients, families, researchers, and supporters. The approval of the first non-surgical treatment for adults with RRP marks not only a scientific milestone, but an inflection point for the entire RRP community. We extend our deepest gratitude to the many individuals and institutions who helped make this breakthrough possible – from the investigators, NIH collaborators, and clinicians who advanced the science, to the industry partners, whose investment and commitment to this community helped bring this treatment to approval. The FDA's announcement is also a deeply personal victory for every voice that has spoken up, every surgery endured, and every hope held steadfast. At RRPF, we are honored to walk alongside this community, championing awareness, supporting research, and amplifying the lived experiences of those affected by this rare disease. We celebrate the light this approval brings and recommit ourselves to the journey ahead, ensuring that patients of every age and living beyond the U.S. can one day access safe, effective and nonsurgical treatments. 'For decades, our community has persevered through an isolating journey of countless surgeries, silence, and uncertainty. Yet while we continued to breathe, we continued to hope. Today, our hope is realized. Today, with the approval of Papzimeos our hope is no longer a dream, it's a reality,' said Kim McClellan, President of RRPF. 'This is a moment of profound healing, not just for the body, but for the spirit of every adult living with RRP. We finally have an FDA-approved treatment that targets the root cause of RRP and with it, the chance for many members of our community to reclaim lives unencumbered by RRP.' Quick facts about Papzimeos (zopapogene imadenovec-drba): The first and only FDA-approved treatment for RRP Approved for adults with RRP The only treatment to target the underlying cause of RRP Papzimeos is a non-replicating adenoviral vector-based immunotherapy designed to generate an immune response directed against papilloma cells expressing HPV 6/11 Delivered via four subcutaneous injections over a 12-week interval Patients can access Papzimeos SUPPORT for personalized services including insurance navigation, financial assistance, and ongoing access support, by calling (866) 827-8180. For Precigen's full release here: For those interested in learning more about resources available to individuals affected by RRP, visit Renewed hope and continued commitment The FDA full approval of Papzimeos is a monumental step forward for the RRP community, but it is not the final destination. RRPF remains deeply committed to advancing research for all patients – including pediatric patients, who continue to face burdens of this disease without an approved therapeutic option, and patients around the world. This milestone fuels our resolve and fortifies our determination to see a future where all people living with RRP have the same hope realized. With strengthened movement, we will continue to advocate, collaborate, and push for the science that brings healing at every age and to every corner of the world. About RRPF The Recurrent Respiratory Papillomatosis Foundation (RRPF) was born from a parent's urgent need to understand and confront a rare, chronic, and potentially life-threatening disease affecting their child. Since 1991, RRPF has been a lifeline for patients and families navigating the isolating journey of RRP – offering connection, advocacy, and hope. What began as a grassroots effort has grown into a mission-driven organization united by the voices of parents, patients, and researchers. RRPF provides vital resources including access to the latest research, updates on clinical trials and medical meetings, and a supportive community for those affected by RRP. The foundation has played a pivotal role in advancing scientific understanding of RRP – contributing to research as authors, funders, and collaborators across multiple studies and initiatives. RRPF honors the legacy of those like Jennifer Woo, a former president of RRPF whose life and career were dedicated to curing RRP, and continues to drive forward research, awareness, and support for all those impacted. While we breathe, we hope. Learn more here:
Epoch Times
2 days ago
- Health
- Epoch Times
FDA Approves Therapy for Rare Disease Without Randomized Trial Data
The Food and Drug Administration on Aug. 14 approved a therapy for a rare disease called recurrent respiratory papillomatosis (RRP). Regulators cleared Papzimeos, an immunotherapy made by Precigen, citing data from a single-arm, open-label trial that looked at how it performed in adults with RRP and who needed at least three surgeries a year.
