Centro Ricerche Cliniche di Verona Srl

The Reblozyl trial flop adds to a concerning trend of clinical woes at Bristol Myers Squibb this year. Despite a phase 3 flop, Bristol Myers Squibb—encouraged by “clinically meaningful results” and bruised by three other recent pivotal trial failures—still plans to discuss a potential approval filing with the FDA for the company’s Reblozyl.Friday, the New Jersey pharma announced that a phase 3 study evaluating Reblozyl in patients with myelofibrosis-associated anemia who required red blood cell transfusions did not meet its main goal. The patients were also on background treatment with a JAK inhibitor such as Incyte’s Jakafi.The trial, coded Independence, failed to show a statistically significant difference for Reblozyl versus placebo in freeing patients of transfusions during a consecutive 12-week period, starting within the first 24 weeks of treatment.The P-value for the primary endpoint’s analysis landed at 0.0674, narrowly missing the maximum 0.05 threshold that’s typically considered statistically significant. The exact bar for this trial is unclear.Nevertheless, BMS noted that the data favored Reblozyl, with the drug showing a “numerical and clinically meaningful improvement” in transfusion independence, in line with previous findings from a phase 2 study.Several secondary measures also showed a “clinically meaningful” benefit for the BMS drug, according to the company. These include a higher number of patients who achieved at least a 50% reduction—and by at least four red blood cell units—in transfusion burden.However, the company’s July 18 release didn’t mention how Reblozyl fared on the trial’s key secondary endpoint, which is weighing the number of patients who became transfusion-independent over any consecutive 16-week period, beginning within 24 weeks of treatment. In response to a Fierce Pharma request for clarification, a BMS spokesperson said the company plans to share full data from the study in a future peer-reviewed setting. Based on the data, BMS said it will engage with the FDA and the European Medicines Agency about potentially applying for marketing approvals. The company believes “the totality of these results, including meaningful improvements in transfusion burden and hemoglobin levels, support the potential to address an unmet need in patients who have few treatment options,” Anne Kerber, BMS’ head of development in hematology, oncology and cell therapy, said in a July 18 release.The Reblozyl trial flop adds to a concerning trend of clinical woes at BMS this year. All four of the pivotal life-cycle management trials that have read out this year for BMS’s existing drugs have turned up negative.The losing streak started in February with the LAG-3/PD-1 combo Opdualag flunking as an adjuvant treatment for resected stage 3 or 4 melanoma. Then, the heart med Camzyos couldn’t move the needle in non-obstructive hypertrophic cardiomyopathy.A few days later, the company’s Cobenfy stumbled as an adjunctive treatment for inadequately controlled schizophrenia. That leaves the closely watched Adept-2 trial for Cobenfy in Alzheimer’s disease psychosis as the only planned phase 3 readout this year for an in-market BMS drug that has not reported outcomes.The pivotal success drought is troublesome because all those four drugs have been tasked with helping BMS navigate a huge patent cliff. During a presentation at the J.P. Morgan Healthcare Conference in January, BMS CEO Chris Boerner highlighted the four drugs, plus the CAR-T therapy Breyanzi, as five products key to the company’s growth portfolio, which was expected to exceed 50% of BMS’s revenues in 2025. Reblozyl is already approved by the FDA to treat two other types of anemia related to beta thalassemia and lower-risk myelodysplastic syndromes (MDS).  BMS has projected the drug, acquired in its Celgene buyout, can reach $4 billion or more in peak sales. In the first quarter of 2025, Reblozyl sales jumped 35% year over year, reaching $478 million.It’s not the end of the road for the first-in-class erythroid maturation agent. While BMS shoots for potential submissions in myelofibrosis-associated anemia, the phase 3 Element-MDS trial testing Reblozyl in nontransfusion-dependent MDS anemia is expected to read out in 2027. And the drug is also being evaluated in alpha thalassemia.Elsewhere in the myelofibrosis universe, Novartis recently took a hit as a safety signal has delayed—if not completely derailed—the Swiss pharma’s regulatory plan for its BET inhibitor candidate pelabresib, which was obtained from its $2.9 billion MorphoSys buyout.Incyte recently shared phase 1 data for its BET inhibitor INCB057643, used with or without Jakafi, in patients with relapsed or refractory myelofibrosis and other myeloid neoplasms. The company recently scrapped development of its ALK-2 inhibitor zilurgisertib in myelofibrosis-related anemia after a failed phase 1/2 trial.Editor's Note: The story was updated with a BMS response to Fierce's inquiry. 

JERSEY CITY, NJ, USA I July 02, 2025 I Organon (NYSE: OGN), a global healthcare company with a focus on women’s health, today announced that the Phase 2 ELENA proof-of-concept study evaluating the investigational candidate OG-6219 in endometriosis-related pain did not meet its primary efficacy endpoint. OG-6219 is an oral 17β-hydroxysteroid dehydrogenase type 1 (HSD17B1) inhibitor, which Organon acquired through its acquisition of Forendo Pharma in 2021. In the study, OG-6219 did not demonstrate improvement in moderate-to-severe endometriosis-related overall pelvic pain compared to placebo. Based on these results, the company plans to discontinue the OG-6219 clinical development program. “While these study results are disappointing, Organon remains committed to our long-term vision to create a better and healthier every day for all women including those living with endometriosis,” said Juan Camilo Arjona Ferreira, M.D., Head of Research & Development and Chief Medical Officer at Organon. “We are grateful to all the patients, caregivers, and investigators for their participation in and support of this study.” The randomized, double-blind, placebo-controlled, parallel-group, global multi-center Phase 2a/b study ( NCT05560646 ) aimed to evaluate the efficacy, safety, and tolerability of three dose levels of OG-6219 in pre-menopausal women 18 to 49 years of age who have been surgically diagnosed with endometriosis with moderate-to-severe endometriosis-related pain. Eligible patients were randomized to receive one of three doses of OG-6219 or placebo orally twice a day. The primary efficacy endpoint was the change from the baseline cycle (placebo run-in cycle) to the last planned treatment cycle (third month of treatment exposure), in the mean endometriosis-related overall pelvic pain score (measured using an eleven-point numerical rating scale). About Organon Organon (NYSE: OGN) is a global healthcare company with a mission to deliver impactful medicines and solutions for a healthier every day. With a portfolio of over 70 products across Women’s Health and General Medicines, which includes biosimilars, Organon focuses on addressing health needs that uniquely, disproportionately or differently affect women, while expanding access to essential treatments in over 140 markets. Headquartered in Jersey City, New Jersey, Organon is committed to advancing access, affordability, and innovation in healthcare. Learn more at www.organon.com and follow us on LinkedIn , Instagram , X , YouTube , TikTok and Facebook . SOURCE: Organon