Ruxolitinib Phosphate

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. 

Rising biotech deals involving Chinese drugs, Takeda's positive OX2R readouts, and LaNova's acquisition by Sino Biopharm made our news this week. Biotech deals involving assets developed in China are surging, a Jefferies report shows. Takeda is preparing its narcolepsy candidate oveporexton for an FDA filing after two positive phase 3 readouts. Merck & Co.'s PD-1xVEGF partner is being acquired. And more.1. China biotechs ‘reshaping’ US biopharma as outlicensing deals rise 11%: Jefferies reportAbout a third of biopharma’s outlicensing deal value in the first quarter of 2025 centered on Chinese assets, versus 21% during the same period in both 2024 and 2023, according to Jefferies. Since 2022, China biotechs have developed 639 first-in-class drug candidates, a sharp rise compared with prior years, Jefferies tallied.2. Takeda’s narcolepsy blockbuster hopeful secures double phase 3 wins, teeing up FDA filingTakeda’s OX2R agonist oveporexton has hit the mark in two phase 3 narcolepsy trials. The FirstLight and RadiantLight trials met their primary endpoints showing statistically significant improvements for the drug compared with placebo in excessive daytime sleepiness, as measured by the Maintenance of Wakefulness Test at week 12. The company is now preparing for an FDA approval filing.3. Sino Biopharm buys Merck's PD-1xVEGF bispecific partner LaNova for up to $951MLaNova Medicines, from which Merck & Co. recently licensed a PD-1xVEGF bispecific candidate, is being bought out by Chinese compatriot Sino Biopharmaceutical in a deal worth up to $951 million. In addition, LaNova also provided AstraZeneca with a GPRC5D-targeting antibody-drug conjugate. Before the LaNova transaction, very few, if any, Chinese pharmas have acquired innovative domestic biotechs.4. For Lundbeck and Otsuka's PTSD filing, FDA questions Rexulti efficacy after failed phase 3For an upcoming advisory committee meeting, the FDA flagged “discordant results” from two phase 3 trials that Lundbeck and Otsuka are using in their application for Rexulti, in combination with Viatris’ Zoloft, as a treatment for post-traumatic stress disorder. The FDA is asking experts to weigh in on whether an exploratory phase 2 study can overcome one negative phase 3.5. Otsuka inks $613M deal for Swedish biotech's autoimmune antibodiesOtsuka is paying $33 million upfront for Cantargia’s IL1RAP antibody, CAN10, as well as a backup candidate, against autoimmune and inflammatory disorders. The deal also includes up to $580 million in milestone payments and gives the Japanese drugmaker exclusive first rights of negotiation for Cantargia’s next-gen IL1RAP programs.6. Sun Pharma's alopecia med Leqselvi hits US market after Incyte patent settlementSun Pharma has reached a patent settlement with Incyte so the Indian company can launch its JAK inhibitor Leqselvi in the U.S. for severe alopecia areata. Sun will pay an undisclosed upfront payment plus royalty payments until the related patents expire. The deal ended a lawsuit in which Incyte claimed that Leqselvi would infringe on patents around its blockbuster ruxolitinib.7. FDA hands down citations to 3 drugmakers after inspections found contamination, testing concernsMeanwhile, a Sun Pharma plan in Gujarat, India was subject to a 19-page, eight-observation Form 483 following an FDA inspection in June. Some citations, such as “poor aseptic behavior and inadequate environmental monitoring,” had previously been flagged in an FDA warning letter in 2022. Separately, a Daewoo Pharm site in Busan, Korea, and a Glenmark facility in Madhya Pradesh, India, received FDA warning letters.Other News of Note:8. Astellas reports 22% sales bump for Izervay, expects trend to continue this year9. China sentences Astellas exec to 3.5 years in prison for espionage (Bloomberg)10. Hengrui's GLP-1/GIP agonist reports 18% weight loss in phase 3 trial, readies China push11. Acumen taps JCR’s blood-brain barrier tech for $555M Alzheimer’s deal12. GSK expands Medicines Patent Pool deal to allow generic production of cabotegravir for HIV treatment13. Kowa licenses Nicox’s intraocular pressure eye drop for up to €191.5M (release)14. Korea’s Illimis raises $42M series B to advance Alzheimer’s candidate (release)