Results from the trial indicated that 70% of patients in the OPDIVO arm achieved 18-month EFS, in contrast to just 50% in the comparator arm. Moreover, 25% of patients receiving OPDIVO achieved a pathologic complete response (pCR), compared to only 4.7% in the control group. “With this new OPDIVO-based regimen, we are reinforcing our commitment to improving patient outcomes and expanding our thoracic portfolio in early-stage disease,” stated Wendy Short Bartie, senior vice president of U.S. Oncology and Hematology at Bristol Myers Squibb.
OPDIVO is associated with several warnings, including severe immune-mediated adverse reactions, and is not recommended for certain patient combinations outside of clinical trials. The recommended dosage includes 360 mg of OPDIVO combined with platinum-doublet chemotherapy every three weeks for up to four cycles, followed by 480 mg of single-agent OPDIVO every four weeks for up to a year or until disease recurrence or unacceptable toxicity occurs. This approval builds on previous successes of OPDIVO in various cancer treatments, reinforcing its role in neoadjuvant, adjuvant, and perioperative settings across multiple cancers, including melanoma and bladder cancer.
Palvella Therapeutics Secures $2.6M FDA Grant for Phase III Trial in Microcystic Lymphatic Malformations
Palvella Therapeutics, Inc. has announced that it has received a significant grant of up to $2.6 million from the FDA Office of Orphan Products Development to support its ongoing Phase III SELVA trial. This trial focuses on QTORIN 3.9% rapamycin anhydrous gel, which is being investigated for treating microcystic lymphatic malformation. The funding highlights the urgent need for effective therapies for patients suffering from this serious and rare genetic skin disease, for which there are currently no FDA-approved treatments.
Wes Kaupinen, Founder and Chief Executive Officer of Palvella, expressed enthusiasm about the grant, stating, “We believe this grant underscores the high unmet medical need in this serious, rare, and chronically debilitating genetic disease, the scientific rationale for targeted inhibition of the causative PI3K/mTOR pathway, and QTORIN rapamycin’s potential to be the first approved therapy and standard of care in the U.S. for microcystic LMs.” The FDA’s Orphan Products Grants Program received 51 grant applications in fiscal year 2024, with Palvella’s trial being one of only seven selected for funding, reflecting the program’s commitment to supporting innovative treatments for rare diseases.
The FDA Orphan Products Grants Program aims to foster the development of safe and effective medical products for rare conditions by awarding grants based on scientific and technical merit, as evaluated by an independent panel of experts. Historically, these grants have contributed to approving over 85 products since the program’s inception. The ongoing Phase III trial for QTORIN™ rapamycin is notable for receiving both Breakthrough Therapy Designation and Fast Track Designation from the FDA, emphasizing its potential to address significant unmet medical needs in treating microcystic lymphatic malformations.
First Patient Dosed in Phase III UGN-103 Trial for Low-Grade Non-Muscle Invasive Bladder Cancer
UroGen Pharma Ltd. has officially announced the dosing of the first patient in its Phase III clinical trial for UGN-103, an investigational intravesical solution designed to treat low-grade intermediate-risk non-muscle invasive bladder cancer. This next-generation formulation of mitomycin represents a significant advancement in bladder cancer treatment and is designed to streamline the manufacturing process and simplify the reconstitution procedure for healthcare providers.
Liz Barrett, President and Chief Executive Officer of UroGen, remarked, “Reaching this Phase III trial milestone for UGN-103 highlights our drive to innovate and bring forward cutting-edge treatments for low-grade intermediate-risk non-muscle invasive bladder cancer. UGN-103 represents a significant step forward, offering potential improvements in manufacturing, convenience, and cost.” The drug utilizes UroGen’s proprietary RTGel® platform technology, which is a sustained-release hydrogel that aims to enhance the therapeutic profile of existing drugs.
The FDA accepted the Investigational New Drug Application for UGN-103 in April 2024, allowing its investigational use in adults with this highly recurrent form of bladder cancer. Following the anticipated FDA approval of UGN-102, UroGen is poised to advance UGN-103, which is set to undergo evaluation in the UTOPIA study. This single-arm, multicenter study aims to enroll 87 patients, who will receive 75 mg of UGN-103 weekly for six weeks. Efficacy will be measured through the complete response rate at the three-month follow-up, with participants monitored until disease recurrence or progression, ensuring ongoing evaluation of UGN-103’s effectiveness in managing LG-IR-NMIBC.
Italian Pharma Company Recordati Acquires ENJAYMO from Sanofi for $825 Million
Recordati S.p.A., an Italian pharmaceutical company, has announced a major acquisition. It has secured the global rights to ENJAYMO (sutimlimab) from Sanofi SA for an upfront payment of $825 million, along with additional commercial milestone payments that could reach up to $250 million. ENJAYMO, a humanized monoclonal antibody approved by the FDA in 2022, is currently the only targeted treatment for cold agglutinin disease (CAD), a rare autoimmune disorder characterized by the premature destruction of red blood cells.
“Acquiring ENJAYMO allows us to expand our rare diseases portfolio in the U.S., Japan, and Europe,” said Recordati CEO Rob Koremans. He added, “This acquisition not only addresses a significant unmet medical need for patients suffering from CAD but is also expected to positively impact our top and bottom lines.” The deal, anticipated to close by the end of 2024, is expected to have minimal revenue contribution in 2024, but Recordati projects a substantial revenue increase in fiscal 2025, with estimates exceeding 150 million euros.
ENJAYMO generated approximately €100 million ($109.7 million) in revenue over the last year and is projected to achieve peak sales potential of €250 million to €300 million. The acquisition will be funded through existing cash and new bank debt, with Recordati maintaining that its capital allocation and dividend policies will remain unaffected. The deal follows Sanofi’s recent strategic moves, including a $27 million investment in Ventyx Biosciences, signaling a shift in focus under CEO Paul Hudson.
As CAD affects about 11,000 patients in the U.S., Japan, and Europe, ENJAYMO works by selectively targeting the C1 complement protein, effectively blocking the complement-mediated destruction of red blood cells. Although sales for ENJAYMO have seen significant growth, with nearly $80 million in revenue generated in 2023 and a 73% increase in the first half of 2024, the decision to sell reflects Sanofi’s broader strategy to regain investor confidence amid operational challenges.
AIRSUPRA Shows Significant Reduction in Severe Asthma Exacerbations in BATURA Phase III Trial
Asthma treatment took a major leap forward with AstraZeneca’s AIRSUPRA (albuterol/budesonide) demonstrating significant efficacy in reducing severe exacerbations in the BATURA Phase IIIb trial. The trial, which focused on patients with intermittent or mild persistent asthma, showed that AIRSUPRA, when used as an as-needed rescue medication, led to a statistically significant and clinically meaningful reduction in severe asthma exacerbations compared to albuterol alone. Due to the overwhelming efficacy, the Independent Data Monitoring Committee recommended stopping the trial early, marking a breakthrough in the management of mild asthma, a condition that affects millions globally.
James Donohue, Chair of the Independent Data Monitoring Committee, noted, “People with asthma are at risk of severe exacerbations regardless of their disease severity. The BATURA trial results showcase the benefits of an anti-inflammatory rescue approach that tackles both symptoms and inflammation simultaneously.” Sharon Barr, Executive Vice-President of BioPharmaceuticals R&D at AstraZeneca, added, “These results solidify AIRSUPRA’s role as a first-in-class treatment, helping to reduce the risk of asthma exacerbations and the need for systemic corticosteroids.”
AIRSUPRA is already approved in the US for adults and is being studied in adolescents and other global regions. Its safety profile in the BATURA trial remained consistent with previous studies, and full data will be presented at the upcoming American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting.
