Myqorzo’s success in “non-obstructive” hypertrophic cardiomyopathy separates it from Bristol Myers Squibb’s rival Camzyos and opens up a multibillion-dollar market opportunity.

Original title: “Cytokinetics heart drug scores in closely watched trial”

The new data could support a broader approval submission for a therapy that is already under review and set to be showcased at the year’s biggest meeting for cancer research.

Original title: “Celcuity strengthens case for ASCO-spotlighted breast cancer drug”

The advisory committee meeting—the FDA’s first drug-related adcomm in nine months—could have been a “more conceptual discussion” about the design of AstraZeneca’s Phase 3 trial of camizestrant in HER2-negative advanced breast cancer, former cancer regulator Harpreet Singh told BioSpace.

Original title: “First FDA ODAC meeting without Pazdur highlights conceptual challenges with trial”

QTORIN™ rapamycin was previously ... for the treatment of angiokeratomas, reflecting its potential to address an unmet medical need in a serious disease with no FDA-approved therapies. About Palvella Therapeutics Founded and led by rare disease biotech veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage ... QTORIN™ rapamycin was previously granted Fast Track Designation by FDA for the treatment of angiokeratomas, reflecting its potential to address an unmet medical need in a serious disease with no FDA-approved therapies. About Palvella Therapeutics Founded and led by rare disease biotech veterans, Palvella Therapeutics, Inc. (Nasdaq: PVLA) is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no FDA-approved therapies. Clinically significant angiokeratomas represent a rare, chronic and debilitating lymphatic malformation with no FDA-approved therapies and an estimated more than 50,000 diagnosed patients in the U.S. Phase 2 single-arm, baseline-controlled trial expected to enroll up to 15 subjects at leading vascular anomaly centers and high-volume dermatology centers in the U.S. (Palvella or “the Company”) (Nasdaq: PVLA), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. Food and Drug Administration (FDA)-approved therapies, today announced that the first patients have been dosed in LOTU, a multicenter Phase 2 clinical trial designed to evaluate the safety and efficacy of QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin) for the treatment of clinically significant angiokeratomas. Palvella is developing a broad pipeline of product candidates based on its patented QTORIN™ platform, with an initial focus on serious, rare skin diseases and vascular malformations, many of which are lifelong in nature. Palvella’s lead product candidate, QTORIN™ 3.9% rapamycin anhydrous gel (QTORIN™ rapamycin), is currently being developed for the treatment of microcystic lymphatic malformations, cutaneous venous malformations, and clinically significant angiokeratomas.

Original title: “Palvella Therapeutics Announces First Patients Dosed in Phase 2 LOTU Trial of Fast Track-Designated QTORIN™ Rapamycin for Clinically Significant Angiokeratomas”

Summit Therapeutics planned an early interim progression-free survival readout for HARMONi-3 in the hope of enabling earlier regulatory engagement—but the early analysis delivered disappointment for the company and shareholders.

Original title: “Summit shares descend as PD-1/VEGF asset misses early survival mark”

Veppanu, the first PROTAC therapy approved by the FDA, improved progression free survival by 43% versus AstraZeneca’s Faslodex but showed no such significant benefit in the intention-to-treat analysis.

Original title: “FDA clears Pfizer, Arvinas’ novel breast cancer therapy despite mixed data”

The FDA is looking at a slew of label expansions this month, including one that could open up home-based treatments for Alzheimer’s disease.

Original title: “FDA Action Alert: Argenx, AstraZeneca/Daiichi Sankyo, Biogen/Eisai and Cingulate”

Neoadjuvant therapy has become a cornerstone in the management of locally advanced rectal cancer (LARC). In this single-arm, open-label phase II study, we evaluated the efficacy and safety of total neoadjuvant chemotherapy (TNT) using a CapOX regimen combined with a programmed cell death protein ... Neoadjuvant therapy has become a cornerstone in the management of locally advanced rectal cancer (LARC). In this single-arm, open-label phase II study, we evaluated the efficacy and safety of total neoadjuvant chemotherapy (TNT) using a CapOX regimen combined with a programmed cell death protein 1 (PD‑1) antibody (sintilimab) and interleukin‑2 (IL‑2) in patients with microsatellite stable (MSS)/defining proficient mismatch repair (pMMR) LARC. Signal Transduction and Targeted Therapy - Total neoadjuvant chemotherapy combined with PD‑1 blockade and IL‑2 in MSS/pMMR locally advanced rectal cancer: short-term results of a... In summary, this study provides preliminary and encouraging results for patients with MSS/pMMR type LARC through the combination of total neoadjuvant chemotherapy with a PD‑1 antibody and IL‑2. The observed pCR rate of 42.4% represents a hypothesis-generating signal suggesting potential superiority over conventional therapy, which requires confirmation in future randomized controlled trials to establish clinical benefit. Besides, we also exhibited a controllable safety profile with no grade 4 or 5 AEs or treatment‑related deaths. Rahma, O. E. et al. Use of Total Neoadjuvant Therapy for Locally Advanced Rectal Cancer: Initial Results From the Pembrolizumab Arm of a Phase 2 Randomized Clinical Trial.

Original title: “Total neoadjuvant chemotherapy combined with PD‑1 blockade and IL‑2 in MSS/pMMR locally advanced rectal cancer: short-term results of a prospective, single-arm phase II study | Signal Transduction and Targeted Therapy”

Known as Veppanu, the drug is the first of its kind to get to market. But Arvinas and partner Pfizer are still searching for a new partner to sell it.

Original title: “Arvinas’ ‘Protac’ breast cancer drug cleared by FDA”

FDA is announcing that it issued a “safe to proceed” letter to Revolution Medicines, allowing the sponsor to initiate an expanded access treatment protocol (EAP) for its experimental pancreatic cancer drug, daraxonrasib.

Original title: “FDA Permits Expanded Access for Investigational Pancreatic Cancer Drug”

Members of the FDA’s Oncologic Drugs Advisory Committee questioned the design of AstraZeneca’s Phase 3 trial of camizestrant, which involved switching treatments at the point of mutation detection, as opposed to the current practice of changing regimens upon disease progression.

Original title: “AstraZeneca’s breast cancer drug fails to earn backing of FDA advisory committee”

Corcept Therapeutics’ ALS drug was linked to an 87% reduction in the risk of death, a result the biotech hopes to replicate in an upcoming Phase 3 trial.

Original title: “Corcept ties ALS drug to improved 2-year survival as Phase 3 start date nears”

Media round-up about the FDA taking major steps to advance the implementation of real-time clinical trials. Paradigm Health CEO, Kent Thoelke, via press release: “This collaboration represents an important step toward expediting how clinical trial data is submitted to and reviewed by the FDA…With Paradigm Health's platform, clinical trial data can be analyzed for key signals in near real time and shared with trial sponsors and the FDA in days, rather than months. This means that regulators, sponsors, and providers can have a continuous view of safety and efficacy. By modernizing the flow of information from sites to sponsors to regulators, we have an opportunity to accelerate the delivery of innovative therapies to patients across the country.” Triatomic Capital Co-Founder, Jeff Huber, via X: “Huge progress from the @US_FDA, to enable innovation with speed and quality with 'real-time clinical trials.' Many thanks to @JonathanHirsch & the Paradigm Health Team for helping show the way!” · InVitria Founder and CEO, Scott E. Deeter, via X: “This is smart!” · Tevogen Bio Founder and CEO, Ryan Saadi, via X: “Brilliant! Faster development of life-saving therapies, Improved patient affordability & U.S. Derya Unutmaz, M.D., via X: “This is a very big deal! Incredible to see this acceleration in clinical trials happening thanks to the amazing leadership at @US_FDA! Accelerating clinical trials via AI is a key milestone toward my bold claim that we can treat/cure most, or even all, diseases within a decade!” Matt Ripkey, healthcare technologist and investor via X: “This is fantastic progress! Clinical trials is a market that, implemented effectively, will dramatically improve public perception of AI. It shouldn't take 8-12 years to bring treatments to market.

Original title: “WTAS: FDA Announces Major Steps to Implement Real-Time Clinical Trials | HHS.gov”

After delaying a late-stage readout last year due to “irregularities” at certain study sites, pivotal data for Bristol Myers Squibb’s Cobenfy appear set to arrive later this year.

Original title: “BMS positions Cobenfy as safer alternative ahead of pivotal Alzheimer’s psychosis readout”

The patient death occurred outside the U.S. and was deemed unrelated to Newron Pharmaceuticals’ investigational schizophrenia drug.

Original title: “Patient death prompts FDA pause on Newron’s Phase 3 schizophrenia study”

Boehringer Ingelheim reports significant reductions in weight

Original title: “Survodutide delivers 16.6% weight loss in major phase 3 obesity trial”