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• For the first time, patients with abnormal cells detected in their bone marrow have a treatment option before the condition develops into multiple myeloma.

• The FDA approved Johnson & Johnson’s Darzalex subcutaneous formulation (daratumumab-hyaluronidase) to treat patients with smoldering multiple myeloma (SMM) that is at a high risk of developing into multiple myeloma.

• Before this approval, doctors could only monitor patients with SMM, which is an asymptomatic intermediate disease state, in hopes of catching early signs of the condition’s development into cancerous MM. (Fierce Pharma)

• Today, the FDA approved Komzifti (ziftomenib) to treat patients with relapsed or refractory acute myeloid leukemia, or AML, caused by a mutation in a gene called NPM1.

  • The mutation, which makes AML more difficult to treat, is a driver of approximately 30% of all AML cases.

• AML is an aggressive blood cancer that occurs when immature bone marrow cells fail to mature into healthy blood cells and instead start dividing uncontrollably. While AML can be treated with chemotherapy, targeted medicines, and bone marrow transplants, roughly half of patients relapse within five years.

• Syndax Pharmaceuticals secured FDA approval in October for a similar drug, called Revuforj, for the same type of genetically defined AML. (STAT)

• Cogent Biosciences has cleared another phase 3 hurdle for lead asset bezuclastinib, teeing up an approval submission for a form of gastrointestinal cancer in the first half of next year.

• The tyrosine kinase inhibitor, in combination with approved cancer med sunitinib, was tied to an objective response rate (ORR) of 46% in patients with gastrointestinal stromal tumors who had not or could not benefit from Novartis' Gleevec.

  • This ORR was significantly higher than the 26% rate for patients on sunitinib alone.

• Patients given the investigational combo also had a significantly higher median progression-free survival (mPFS) of 16.5 months compared to 9.2 months for the monotherapy, Cogent announced. (Fierce Biotech)

• Gilead Sciences has developed an industry-leading HIV portfolio in recent years with its megablockbuster daily treatment Biktarvy and its new long-acting pre-exposure prophylaxis (PrEP) medicine Yeztugo.

• Now, the company is finding success in combining two of the active ingredients in those products.

• Gilead's investigational single-tablet HIV regimen of bictegravir 75 mg/lenacapavir 50 mg (BIC/LEN) has prevailed in a phase 3 trial, the company announced.

• The open-label trial assessed responses to the investigational regimen in adults with HIV who were virologically suppressed on a complex multi-tablet regimen. The trial randomized patients 2 to 1 to either switch to the investigational once-daily tablet or stay on their existing regimens.

• In the Artistry-1 study, the efficacy of switching to BIC/LEN was "found to be statistically non-inferior" compared with staying on baseline multi-tablet regimens, Gilead said. (Fierce Pharma)

• Roche’s BTK inhibitor has cleared two registrational studies in different types of multiple sclerosis, a surprise turn for a drug class that’s seen several trial failures and safety setbacks.

• The Swiss drugmaker said that fenebrutinib met the primary endpoint of both phase 3 studies that separately tested it in people with relapsing multiple sclerosis and primary progressive multiple sclerosis (PPMS). The trials together enrolled more than 1,700 patients, according to a company release.

CMO Levi Garraway described the wins as “unprecedented” and said they could pave the way for fenebrutinib to become a “best-in-disease medicine.”

• The relapsing MS success will be seen as a “positive surprise,” Jefferies analysts said in a note to investors.

• But the analysts said they weren’t sure if a hit in the primary endpoint chosen for the PPMS trial will be enough for a filing with regulators. (Endpoints)

• From high-stakes clinical breakthroughs to billion-dollar transactions to controversial policy shifts, the pharma and biotech world in 2025 proved dynamic.

• As science and strategy collided, key developments sparked industry-wide conversation and debate.

1. The year everyone chased the weight-loss boom: Deals included Pfizer’s $10 billion acquisition of obesity drug developer Metsera; Eli Lilly’s $1.3 million deal with Superliminal to leverage its AI/ML capabilities to generate new small molecule obesity medications; and Roche’s $5.3 billion deal with Zealand Pharma to co-develop and commercialize the weight-loss candidate petrelintide.

2. A comeback year for biopharma M&A? After a cautious 2024, dealmaking made significant inroads in 2025. Pharma giants poured billions into biotech partnerships and takeovers, from obesity, as noted above, to neurology, oncology, and rare diseases.

3. China’s emergence as major innovation hub: Formerly best known in the pharma supply chain as a hub of active pharmaceutical ingredient development (of which it still maintains), China made headlines in 2025 as a burgeoning center for drug development.

4. New modalities, real commercial momentum: Even as Sarepta Therapeutics’ Elevidys in Duchenne muscular dystrophy (DMD) faltered, 2025 still brought major advances in modalities and manufacturing.

5. Convergence of AI and biotech/pharma operations: AI is becoming integral to not only the drug discovery process but accelerating innovation across development and manufacturing as well.

• Now, leaders are planning to meet with the FDA to discuss “converting from an accelerated to traditional approval,” based on other clinically meaningful changes seen in the 6- to 13-year-old study subjects.

• Sarepta isn’t the first company to recognize that a failed trial isn’t necessarily a death sentence, and it’s already rescued one drug from the brink. Its DMD gene therapy Elevidys received full approval and a label expansion even though a late-stage trial didn’t meet the primary endpoint.

“There’s no wiggle room in my mind,” said Dr. Joseph Ross, professor of medicine and public health at the Yale School of Medicine.

“I cannot understand how a trial that’s designed to demonstrate whether a drug works or not, if it fails, how a drug could then get approved for use. Because what was the point of the trial?”