Novo Nordisk Reports Positive Phase 3 Results for Etavopivat in Sickle Cell Disease; UCB to Buy Neurona Therapeutics; KEYTRUDA Regimens Secure FDA Priority Review in Muscle-Invasive Bladder Cancer; Savara Announces FDA Review Extension for Molgramostim in Autoimmune PAP; Terremoto Biosciences Completes $108 Million Series C Financing to Expand AKT1 Inhibitor Development

Novo Nordisk’s Etavopivat Meets Phase 3 Endpoints in Sickle Cell Disease

Novo Nordisk announced topline findings from HIBISCUS, a pivotal Phase 3 study evaluating once-daily oral etavopivat in adults and adolescents living with Sickle Cell Disease. The trial met both co-primary endpoints, with etavopivat showing significant reductions in vaso-occlusive crises (VOCs) and notable improvements in haemoglobin (Hb) response compared with placebo.

Etavopivat, a once-daily oral pyruvate kinase-R (PKR) activator, is being developed as a potential treatment for Sickle Cell Disease, a severe, life-threatening condition affecting an estimated 8 million people globally.

HIBISCUS was a randomised, double-blind, 52-week efficacy and safety study comparing etavopivat 400 mg with placebo in 385 patients aged 12 years and older with Sickle Cell Disease. Participants were permitted to continue standard-of-care therapies during the study.

Results showed that patients receiving etavopivat achieved a 27% greater reduction in the annualised VOC rate versus placebo. The median time to first VOC was also extended to 38.4 weeks, compared with 20.9 weeks for placebo recipients.

Etavopivat also delivered superior haemoglobin improvements, with 48.7% of treated patients achieving an Hb increase of more than 1 g/dL at week 24, versus 7.2% in the placebo group, representing an adjusted rate difference of 41.2%. Exploratory analyses further indicated a significant reduction in blood transfusion risk.

The therapy was generally well tolerated, with a safety profile consistent with earlier etavopivat studies. Novo Nordisk intends to seek the first regulatory approval for etavopivat in the second half of 2026, with full HIBISCUS data expected to be presented at a scientific meeting later that year.

UCB to Acquire Neurona Therapeutics, Strengthening Epilepsy Innovation Through Regenerative Science

UCB, a global biopharmaceutical company, announced today that it has signed a definitive agreement to acquire Neurona Therapeutics, including its lead candidate NRTX-1001, strengthening UCB’s epilepsy treatment portfolio. Neurona Therapeutics is focused on developing regenerative cell therapies for epilepsy and other neurological disorders.

This acquisition builds on UCB’s long-standing expertise in epilepsy and its mission to bring innovative therapies to patients with unmet needs. It represents a strategic move into regenerative medicine and advanced therapeutics, while reinforcing UCB’s commitment to growth through acquisitions and treatments that extend beyond symptom control. 

Neurona’s platform leverages pluripotent stem cell technology to deliver therapies designed to restore damaged neural circuits both structurally and functionally. Its lead asset, NRTX-1001, is currently in Phase I/II clinical trials assessing safety, tolerability, and seizure reduction in patients with drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (mTLE), with or without Mesial Temporal Sclerosis (MTS). Delivered as a minimally invasive, one-time dose directly into the brain, the therapy introduces cells that release gamma-aminobutyric acid (GABA), aiming to rebalance hyperactive neural networks and provide lasting seizure control.

Supported by promising early clinical results, NRTX-1001 has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration for drug-resistant mesial temporal lobe epilepsy, as well as PRIME (Priority Medicines) designation from the European Medicines Agency for adults with drug-resistant focal epilepsy. These recognitions highlight the therapy’s potential to address major unmet needs and benefit from accelerated development pathways.

Under the agreement, UCB will make an upfront payment of $650 million, with an additional $500 million tied to future milestone achievements. The transaction is subject to customary closing requirements, including antitrust approvals, and is expected to be completed by the end of Q2 2026.

FDA Grants Priority Review to KEYTRUDA-Based Regimens for Cisplatin-Eligible Patients with Muscle-Invasive Bladder Cancer

Merck & Co. announced that the U.S. Food and Drug Administration has accepted for priority review two supplemental Biologics License Applications (sBLAs) for KEYTRUDA and KEYTRUDA QLEX, both in combination with Padcev. The applications seek approval for treating patients with Muscle-Invasive Bladder Cancer who are eligible for cisplatin-based chemotherapy. The FDA has assigned a target decision date of August 17, 2026.

If cleared, the proposed indications would broaden the use of KEYTRUDA and KEYTRUDA QLEX, each paired with Padcev, making them the first perioperative treatment options for MIBC patients regardless of cisplatin eligibility. This would further extend the combination’s current approved use in MIBC patients who cannot receive cisplatin-based chemotherapy.

The submissions are supported by findings from the Phase 3 KEYNOTE-B15 Trial, conducted jointly with Pfizer and Astellas Pharma. Data from the study were recently presented at the ASCO Genitourinary Cancers Symposium. The companies also intend to submit these results to regulatory agencies globally for potential approvals.

Currently, KEYTRUDA plus Padcev is approved in the U.S., European Union, Japan, and several other markets for adults with locally advanced or metastatic urothelial cancer. In the U.S., the combination is additionally approved for adults with MIBC who are not eligible for cisplatin chemotherapy. KEYTRUDA alone is also approved across multiple regions for selected patients with locally advanced or metastatic urothelial cancer, as well as certain forms of Non-Muscle-Invasive Bladder Cancer.

Savara Reports FDA Extension of Review Period for Molgramostim Inhalation Solution BLA in Autoimmune Pulmonary Alveolar Proteinosis

Savara Inc. announced that the U.S. Food and Drug Administration (FDA) has extended the review timeline for the biologics license application (BLA) of molgramostim in autoimmune PAP by an additional three months. The application continues to be evaluated under Priority Review, with a revised PDUFA decision date of November 22, 2026.

The extension follows the FDA’s determination that the company’s recent responses to agency information requests qualified as a major amendment to the BLA. As a result, the review period has been lengthened to provide more time for assessment of the newly submitted materials. In its communication, the FDA did not raise any concerns related to safety, efficacy, or manufacturing.

Molgramostim has also received multiple regulatory incentives, including Fast Track, Breakthrough Therapy, and Orphan Drug Designations from the FDA, Orphan Drug Designation from the European Medicines Agency (EMA), and Innovation Passport along with Promising Innovative Medicine status from the Medicines and Healthcare products Regulatory Agency (MHRA).

Terremoto Biosciences Raises $108 Million in Series C Funding for Selective AKT1 Inhibitor Pipeline

Terremoto Biosciences has secured $108 million in a Series C financing round to accelerate the development of its selective AKT1 inhibitor pipeline. The funding was backed by new investors RA Capital Management, Deep Track Capital, Osage University Partners (OUP), and BeOne Medicines, alongside continued support from existing investors OrbiMed, Third Rock Ventures, Novo Holdings, and Cormorant Asset Management.

The company’s lead oncology candidate, TER-2013, is currently being evaluated in a Phase 1 clinical trial for solid tumors with genetic alterations in PIK3CA, AKT, or PTEN. These mutations are associated with a broad range of cancers, including over half of HR-positive breast cancer cases. Terremoto is also progressing TER-4480 for hereditary hemorrhagic telangiectasia (HHT), a rare inherited vascular disorder marked by abnormal blood vessel growth and serious complications. With no approved treatments currently available, TER-4480 is expected to begin clinical testing later this year.

AKT plays a central role in disease progression across both cancer and HHT. Of its three isoforms, AKT1, AKT2, and AKT3, preclinical data suggest AKT1 is the primary disease-driving target, while AKT2 inhibition has been associated with side effects such as rash and impaired glucose regulation. Although multiple PI3K/AKT pathway inhibitors have been developed, their clinical utility has often been constrained by toxicity linked to PI3Kα or AKT2 inhibition. Leveraging advanced medicinal chemistry expertise, Terremoto has created a new class of AKT1-selective inhibitors designed to deliver stronger, longer-lasting responses with improved tolerability.