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European Commission grants marketing authorisation for NEZGLYAL® (leriglitazone), the first pharmacological treatment approved for cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease

European Commission grants marketing authorisation for NEZGLYAL® (leriglitazone), the first pharmacological treatment approved for cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease

BARCELONA, Spain and DÜSSELDORF, Germany, Sept. 25, 2026 -- Neuraxpharm Group and Minoryx Therapeutics today announce that the European Commission (EC) has granted marketing authorisation under exceptional circumstances for NEZGLYAL® (leriglitazone), an orally bioavailable, brain penetrating, selective PPAR gamma agonist, as a treatment for male cALD patients, aged 2-12 years with Gadolinium (Gd)-negative brain lesions. The therapy is the first approved pharmacological treatment for cALD in the European Union (EU). The approval is based on results from the Phase 2/3 NEXUS 1 study and additional real-world evidence from compassionate use programmes. The first European launch is expected in Germany by the end of the year, with additional launches in Europe anticipated pending completion of national reimbursement negotiations.

0-Homepage Feature / 0-Megaphone / Minoryx - 09/25/2026

FDA Grants Breakthrough Therapy Designation to Glycomine’s GLM101 for the Treatment of PMM2-CDG

SAN CARLOS, Calif.--September 23, 2026--Glycomine, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to GLM101 for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), a serious, multisystem disorder with no approved treatments. GLM101 is an investigational liposomal mannose-1-phosphate substrate replacement therapy designed to address the underlying deficiency in mannose-1-phosphate and resulting disruption of glycosylation in PMM2-CDG. The designation is based on clinical evidence from Glycomine’s open-label Phase 2a study, including improvements in ataxia and other clinical measures after 24 weeks of GLM101 treatment.

Glycomine - 09/23/2026

Minoryx and Neuraxpharm complete enrolment in leriglitazone pivotal Phase 3 trial (CALYX) in adult patients with cerebral Adrenoleukodystrophy (cALD)

Mataró, Barcelona, Spain, and Düsseldorf, Germany, 09 September 2026 - Minoryx Therapeutics and Neuraxpharm Group (Neuraxpharm), announce that the last patient has been enrolled in the global Phase 3 clinical trial (CALYX) evaluating leriglitazone (NEZGLYAL®) in adult male patients with cerebral Adrenoleukodystrophy (cALD), an orphan neurodegenerative disease with no pharmacological therapeutic options. In Europe, leriglitazone has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for the treatment of boys aged 2-12 with cALD and non-gadolinium enhancing lesions. Approval under exceptional circumstances from the European Commission (EC) is expected later this month and Neuraxpharm, a leading CNS specialist company and Minoryx’s strategic partner in Europe, is preparing for the commercial launch.

Minoryx - 09/09/2026

NEZGLYAL® (leriglitazone) receives a positive CHMP opinion for the treatment of cerebral Adrenoleukodystrophy (cALD), a rare neurodegenerative disease

BARCELONA, Spain and DÜSSELDORF, Germany, July 24, 2026 -- Minoryx Therapeutics and Neuraxpharm Group, announce that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorisation approval under exceptional circumstances for NEZGLYAL® (leriglitazone) as a treatment for male cALD patients, aged 2-12 years with Gadolinium (Gd) negative brain lesions. European Commission approval is expected by the end of September 2026.

Minoryx - 07/26/2026

Aura Biosciences Announces Enrollment Completion in Phase 3 CoMpass Trial of Bel-sar in Early Choroidal Melanoma

BOSTON, June 01, 2026 -- Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced it has completed enrollment of 108 patients in the Phase 3 trial evaluating belzupacap sarotalocan (bel-sar) as a frontline treatment for patients with early choroidal melanoma. Topline data for the 15-month primary endpoint are anticipated in the second half of 2027.

Aura - 06/02/2026

Aktis Oncology Reports First-in-Human Clinical Imaging and Dosimetry Data for AKY-2519 Demonstrating Robust Tumor Uptake and Limited Normal Tissue Exposures in Patients with B7-H3 Expressing Tumors

BOSTON, May 21, 2026 -- Aktis Oncology, Inc. (NASDAQ:AKTS) (Aktis or the Company), a clinical-stage oncology company focused on expanding the breakthrough potential of targeted radiopharmaceuticals to large populations, including those not addressed by existing platform technologies, today reported first-in-human clinical imaging and dosimetry data for AKY-2519, a miniprotein radioconjugate targeting B7-H3 expressing tumors. The data from two separate assessments of AKY-2519 – a clinical imaging and dosimetry assessment in patients with mCRPC and a clinical imaging assessment in patients with various solid tumor types – demonstrated robust tumor uptake and limited normal tissue exposure. These findings, which supported the advancement of a broad clinical development program for AKY-2519, will be presented in two poster presentations at the upcoming 2026 ASCO Annual Meeting, being held May 29 – June 2, 2026, in Chicago.

Aktis Oncology - 05/21/2026

Aktis Oncology Initiates Phase 1b Clinical Trial for AKY-2519, a B7-H3 Miniprotein Radioconjugate, in Metastatic Castration-Resistant Prostate Cancer (mCRPC)

BOSTON, May 04, 2026 -- Aktis Oncology, Inc. (NASDAQ:AKTS) (Aktis or the Company), a clinical-stage oncology company focused on expanding the breakthrough potential of targeted radiopharmaceuticals to large populations, today announced the initiation of a Phase 1b clinical trial of AKY-25191 in patients with metastatic castration-resistant prostate cancer (mCRPC). The mCRPC-dedicated Phase 1b trial is part of Aktis’ clinical development strategy to develop AKY-2519 broadly in B7-H3 expressing tumors. Aktis plans to initiate a Phase 1b basket trial in lung, colorectal, and other high B7-H3 expressing solid tumors in the second half of 2026.

Aktis Oncology - 05/04/2026

Aktis Oncology Announces FDA Clearance of Investigational New Drug Applications for AKY-2519

BOSTON, March 30, 2026 -- Aktis Oncology, Inc. (NASDAQ:AKTS) (the “Company”), a clinical-stage oncology company focused on expanding the breakthrough potential of targeted radiopharmaceuticals to large populations, including those not addressed by existing platform technologies, today announced the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) applications for the Company to proceed to a Phase 1b clinical trial with AKY-25191. AKY-2519 is a miniprotein radioconjugate targeting B7-H3, which is expressed in several solid tumor types including prostate and lung cancers, and is the second clinical stage miniprotein radioconjugate discovered using Aktis’ proprietary platform. The Company’s lead miniprotein radioconjugate, AKY-1189, targeting Nectin-4, is currently enrolling patients in a Phase 1b clinical study. Aktis’ miniprotein radioconjugates are designed to selectively deliver actinium-225 (225Ac), a highly potent alpha-emitting radioisotope, to target-expressing tumors.

Uncategorized - 04/15/2026

Horizon Quantum and IonQ Enter into Strategic Agreement to Unlock Quantum Potential

SINGAPORE, COLLEGE PARK, Md., April 9, 2026 — Horizon Quantum Holdings Ltd. (“Horizon Quantum”), a pioneer of software infrastructure for quantum applications and IonQ — the world’s leading quantum platform company — today announced a strategic agreement. Horizon Quantum will purchase one of IonQ’s first 6th-generation, chip-based 256-qubit trapped-ion systems, in furtherance of Horizon Quantum’s mission to unlock the full potential of quantum computing with its software platform. The acquisition of the 256-qubit system marks a further step in Horizon Quantum’s efforts to enable broad quantum advantage.

Horizon - 04/09/2026

Glycomine Completes Enrollment in Global Phase 2b POLAR Study of GLM101 for the Treatment of PMM2-CDG

SAN CARLOS, Calif.--April 2, 2026--Glycomine, Inc. announced today that enrollment has been completed in the Phase 2b POLAR study, a global, randomized, double-blind, placebo-controlled clinical trial evaluating GLM101 for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). PMM2-CDG is a rare genetic disorder that causes serious neurological and multisystem impairments. More than 90% of patients with PMM2-CDG have ataxia, a key neurological manifestation and driver of disease burden. There are currently no approved treatments for PMM2-CDG.

Glycomine - 04/02/2026
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