SNAPSHOT

Capricor’s Phase 3 results reignite Duchenne therapy momentum

Capricor's cell therapy deramiocel significantly slowed disease progression in Duchenne muscular dystrophy (DMD) patients, meeting key goals in a pivotal phase 3 trial and earning a 300% surge in its stock price.

Why it matters: The strong results revive Capricor’s push for FDA approval after a previous rejection, potentially bringing a much-needed treatment to patients with a devastating, progressive disease.

Backstory: In 2024, the FDA rejected deramiocel despite prior agreements on trial design. Capricor claimed the agency misinterpreted phase 2 data. The company is now using the new HOPE-3 results to support a resubmission.

Big picture: If approved, deramiocel could become a novel therapy for DMD, a rare and fatal muscle-wasting disease with limited treatment options, especially those targeting both muscle and heart function.

Zoom in: In the HOPE-3 trial with 105 boys and young men, deramiocel reduced upper limb function decline by 54% vs. placebo and in 83 patients, slowed heart function deterioration by 91%. Both outcomes were statistically significant, with a safety profile consistent with earlier trials. The therapy uses donor-derived cardiac cells to reprogram immune cells and reduce inflammation.

What’s next: Capricor plans to resubmit its biologics license application to the FDA, including the HOPE-3 data to address prior concerns raised in the agency’s complete response letter.

SNIPPETS

What’s happening in biotech today?

🏆 HAE win: Pharvaris has reported positive phase 3 trial results for deucrictibant, its oral treatment for hereditary angioedema (HAE), achieving its primary endpoint and paving the way for a regulatory filing in the first half of 2026. In the trial, patients treated with deucrictibant experienced symptom relief in 1.28 hours compared to over 12 hours with placebo, aligning with analyst expectations. The drug also showed promising secondary outcomes, including 83% of attacks resolved with a single dose and no serious treatment-related adverse events. These results position deucrictibant as a strong competitor to KalVista’s Ekterly, potentially offering faster and more complete relief for HAE attacks.

👁️ Dry eye deal: Laboratoires Théa has entered into a licensing deal worth up to $280 million with Iolyx Therapeutics to acquire global rights (excluding China, Japan, and select Asian countries) to develop and commercialize ILYX-002, a topical immunomodulator for moderate to severe dry eye disease (DED) linked to systemic autoimmune conditions. Although ILYX-002 did not meet its phase 2 primary endpoint, it showed statistically significant improvements in secondary measures, prompting Théa to describe the results as "highly encouraging." Théa will now lead phase 3 development, while Iolyx will receive milestone payments, tiered royalties up to 21%, and R&D reimbursement. Iolyx also announced a $15 million Series B financing to support its broader pipeline.

🚽 Leak relief: Muvon Therapeutics has reported promising phase 2 results for its muscle precursor cell (MPC) therapy for stress urinary incontinence (SUI), showing a 60% average reduction in incontinence episode frequency among 30 women unresponsive to conservative treatment. Patients received ultrasound-guided injections of MPCs into the urethral sphincter, with the high-dose group achieving a 71% reduction and both dose groups seeing at least a 50% drop in urine loss. The therapy met its primary and key secondary endpoints without any serious adverse events, and 87% of participants met treatment response criteria. Muvon positions the therapy as a potential alternative to synthetic implants like surgical mesh slings.

📈 Stock bump: Bayer has initiated the Phase III SUNFLOWER study to evaluate the efficacy and safety of its 52 mg levonorgestrel-releasing intrauterine system, Mirena, for treating nonatypical endometrial hyperplasia (NAEH), a condition characterized by abnormal thickening of the uterine lining due to hormonal imbalance. NAEH can lead to uterine cancer if left untreated and currently lacks approved medical therapies, highlighting a significant unmet need. Bayer's shares rose over 12% following the study’s announcement.

🚪NSCLC exit: Black Diamond Therapeutics is shifting the pivotal development of its lead EGFR inhibitor, silevertinib, from non-small cell lung cancer (NSCLC) to glioblastoma due to financial constraints, despite promising early data in first-line NSCLC with non-classical EGFR mutations. A 60% confirmed response rate was reported among 43 front-line patients, including 56% in those with PACC mutations. However, a large, costly phase 3 NSCLC trial remains out of reach without a licensing partner. Instead, Black Diamond will launch a smaller phase 2 trial in EGFRvIII-positive glioblastoma, aiming to enroll 150 patients and deliver initial data in 2028. Investor response was negative, with shares dropping 30%.  

SNAP AGAIN

Triana raises $120M to launch clinical trials for cancer molecular glue degrader

Triana Biomedicines secured a $120 million series B to advance its lead molecular glue degrader, TRI-611, into clinical trials for anaplastic lymphoma kinase–positive (ALK+) non-small cell lung cancer.

Why it matters: The funding accelerates Triana's push to develop a potentially transformative treatment for cancers resistant to current therapies, backed by major investors and the corporate investment arms from Pfizer and Regeneron.

Backstory: Triana also formed a partnership with Pfizer in 2024, receiving $49 million upfront to explore molecular glue degraders. This class of drugs enables targeted protein degradation, an emerging modality attracting Big Pharma’s interest. In 2025 alone, companies like Eli Lilly and Gilead committed over $100 million combined to secure access to molecular glue technology.

Big picture: Molecular glue degraders represent a promising frontier in cancer therapy, capable of tackling previously "undruggable" targets.

Zoom in: TRI-611 targets ALK+ NSCLC, aiming to overcome resistance seen with current ALK inhibitors. TRI-611 is not alone in the pipeline as Triana plans to advance a second candidate from its preclinical lineup.

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