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Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX

Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX

Mataró, Barcelona, Spain, November 16, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the first patients have been enrolled in its US Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD), an orphan indication with no alternative therapeutic options.

Minoryx - 11/17/2023

Aura Biosciences Receives FDA Agreement Under Special Protocol Assessment (SPA) for CoMpass Phase 3 Clinical Trial of Belzupacap Sarotalocan (Bel-sar) in Early-stage Choroidal Melanoma

BOSTON--(BUSINESS WIRE)--Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced it has received agreement from the U.S. Food and Drug Administration (FDA) under an SPA for the design and planned analysis of CoMpass, the Company’s global Phase 3 clinical trial of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma (CM). The Company also announced the presentation of positive Phase 2 safety and efficacy data of bel-sar with 90% of patients at twelve months of follow-up evaluating two key clinical endpoints: tumor control and visual acuity preservation using suprachoroidal (SC) route of administration for the first-line treatment of adult patients with early-stage CM. The results were presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California.

Aura / Uncategorized - 11/07/2023

Nick Pappas Joins Pappas Capital as Managing Partner

RESEARCH TRIANGLE PARK, N.C., Sept. 19, 2023 -- Pappas Capital, a leading investor in next-generation life science and technology companies, announced today that Nick Pappas (no relation) has joined the firm as a Managing Partner of Pappas Ventures, the firm's life science venture business. In his role, Nick will oversee Pappas Ventures and lead fundraising and investment efforts for its current and future venture funds. Previously, Nick served as a Managing Partner and Head of Investments at Philips Ventures, where he led a global investment program that was recognized as a leader in corporate venture capital.

0-Homepage Feature / 0-Megaphone / Nick Pappas - 09/19/2023

Mirum Pharmaceuticals Enters Agreement to Acquire Bile Acid Product Portfolio for the Treatment of Rare Liver Diseases from Travere Therapeutics

FOSTER CITY, Calif. & SAN DIEGO--July 18, 2023--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) and Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that they have entered into a definitive agreement for the sale of Travere’s bile acid product portfolio that includes Cholbam® (cholic acid) and Chenodal® (chenodiol), two medications addressing rare diseases in high-need settings.

Mirum Pharmaceuticals - 07/18/2023

Veralox Secures $24MM Financing to Advance Development of First-in-Class Therapies for Immune-Mediated Diseases

FREDERICK, MD., June 20, 2023 -- Veralox Therapeutics, a clinical-stage biotechnology company developing a new class of therapies targeting the 12-lipoxygenase (12-LOX) pathway to address some of medicine’s most persistent and serious immune-mediated diseases, today announced the appointment of Jonathan Mow as the company’s new chief executive officer.

Mr. Mow’s appointment comes as Veralox secured $24 million in funding to advance VLX-1005 through a Phase 2a proof-of-concept study evaluating its impact on heparin-induced thromobcytopenia (HIT), a life-threatening rare disease caused by an aberrant immune response to heparin exposure. The investment round included new investors Pappas Capital and NYBC Ventures and existing investors Hatteras Venture Partners, Sanofi Ventures, JDRF T1D Fund and Genesys Capital, amongst others. In conjunction with the financing, the company welcomes Peter Young of Pappas Capital as a director and Meg Wood of NYBC Ventures as an observer.

Veralox Therapeutics - 06/20/2023

Minoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral Adrenoleukodystrophy

Mataró, Barcelona, Spain, May 31, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the US Food and Drug Administration (FDA) approval of its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

The CALYX protocol has received both FDA and central IRB approval. Significant preparations for the trial commencement have been completed and patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.

Minoryx - 05/31/2023

Tune Therapeutics Unveils Breakthrough Data Showing Stable and Durable Epigenetic Regulation in Non-Human Primates

DURHAM, N.C. & SEATTLE--(BUSINESS WIRE)--May 22, 2023--Last week, at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), leading epigenetic editing company Tune Therapeutics showcased the power and potential of its TEMPO TM genetic tuning platform, by presenting data showing the stable repression of the PCSK9 gene and the effective reduction of LDL-cholesterol levels in non-human primates (NHPs). This data marks a significant waypoint on the path to epigenetic medicine, in that it represents the first demonstration of durable epigenetic gene regulation, in a large animal model, following transient delivery of an epi-editor.

A common therapeutic target for the prevention of cardiovascular disease, it is well established that repressing the PCSK9 gene reduces elevated LDL-cholesterol levels, in turn associated with the formation of artery-clogging plaques. The non-human primate data presented at the ASGCT conference showed that epigenome editing (or genetic tuning) can drive the stable repression of PCSK9 (ongoing past four months) following a single treatment, and without cutting or nicking the DNA, or altering its coding sequence in any way.

Tune Therapeutics - 05/22/2023

Horizon Quantum Computing Raises USD 18.1 million Series A to Advance Quantum Software Development

SINGAPORE--March 31, 2023--Horizon Quantum Computing, a Singapore-based company building software development tools to unlock the potential of quantum computing hardware, announced today that it has raised USD 18.1 million Series A investment from Sequoia Capital India, Tencent, SGInnovate, Pappas Capital and Expeditions Fund.

The investment round will boost the growth of the company, allowing it to strengthen its science and engineering teams to accelerate product development, establish its new engineering centre in Europe and bring Horizon’s unique technology to the market. Horizon Quantum Computing’s total funding now stands at approximately USD 21.3 million.

Horizon - 03/31/2023

Mirum Announces Label Expansion for LIVMARLI in the United States to Include Infants Three Months of Age and Older

FOSTER CITY, Calif.-- March 14, 2023 -- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a reduction in age from one year to three months for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). The label expansion was based on data from the RISE study which characterized the safety and tolerability of LIVMARLI in infants under one year of age with ALGS.

Mirum Pharmaceuticals - 03/15/2023
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