SAN DIEGO, May 19, 2020 -- Amplyx Pharmaceuticals, a biotech company developing innovative therapies for debilitating and life-threatening diseases in patients with compromised immune systems, announced today that it has advanced development of its lead program, fosmanogepix (APX001). Enrollment has been completed in a Phase 2 trial evaluating the safety and efficacy of fosmanogepix for the treatment of infections caused by Candida. Top-line data from the trial is expected by July; the results of an initial analysis of the first 10 patients enrolled showed a high level of treatment success. The first patients have also completed treatment in two additional Phase 2 clinical trials evaluating the safety and efficacy of fosmanogepix (APX001) for the treatment of invasive mold infections caused by Aspergillus and rare molds, as well as a study of invasive candidiasis caused by Candida auris, an emerging, life-threatening, drug-resistant fungal pathogen.
Amplyx Pharmaceuticals Advances Development of Novel Antifungal, Fosmanogepix
HealthpointCapital Acquires Majority Stake in IlluminOss Medical, Inc.
NEW YORK and EAST PROVIDENCE, R.I., May 1, 2020 -- HealthpointCapital, the leading private equity firm focused exclusively on musculoskeletal healthcare, today announced the acquisition of a majority stake in IlluminOss Medical, Inc. ("IlluminOss" or the "Company").
IlluminOss offers a unique and disruptive technology for the orthopedic trauma market. The Company's Photodynamic Bone Stabilization System is a minimally invasive approach for fracture repair and stabilization through a patient-specific intramedullary implant. The system utilizes a light-curable liquid polymer, contained within an expandable balloon, to create a patient-conforming, rigid implant within the bone canal. The technology is particularly suited for the fast-growing elderly patient population. It enables increased stability for osteoporotic and compromised bone, as well as early mobilization. IlluminOss can be also used in conjunction with cleared plates, screws and nails for supplemental fixation in compromised bone.
Curzion Pharmaceuticals Acquired by Horizon Therapeutics
DUBLIN -- April 2, 2020 -- Horizon Therapeutics plc (Nasdaq: HZNP) announced today that it has acquired Curzion Pharmaceuticals, Inc., a privately held development-stage biopharma company, and its development-stage oral selective lysophosphatidic acid 1 receptor (LPAR1) antagonist, CZN001 (renamed HZN-825). Under terms of the agreement, Horizon acquired Curzion for a $45 million upfront cash payment with additional payments contingent on the achievement of development and regulatory milestones. CZN001 was originally discovered and developed by Sanofi, which is eligible to receive contingent payments upon the achievement of development and commercialization milestones and royalties based on revenue thresholds.
Pappas Capital Strengthens Specialized Fund Management Business With Appointment Of Karen LeVert As Venture Partner
RESEARCH TRIANGLE PARK, NC, Feb. 3, 2020 -- Pappas Capital, a leading investor in early-stage life sciences companies, has appointed Karen LeVert as Venture Partner to help lead the expansion of the firm's Specialized Fund Management business, with a focus on identifying attractive investment opportunities in agricultural and other related technologies. Ms. LeVert has a proven track record of progressing early stage technologies from university research labs to commercialization, and most recently co-founded two independent innovation labs, Southeast TechInventures, Inc. (STI) and Ag TechInventures, LLC (AgTI).
Liquidia Submits New Drug Application for LIQ861 (treprostinil) inhalation powder to U.S. Food and Drug Administration for the Treatment of Pulmonary Arterial Hypertension (PAH)
RESEARCH TRIANGLE PARK, N.C., Jan. 27, 2020 -- Liquidia Technologies, Inc. (Nasdaq:LQDA) (“Liquidia”), a late-stage clinical biopharmaceutical company focused on the development of products using its proprietary PRINT® technology, today announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for LIQ861. LIQ861 is an investigational, inhaled dry powder formulation of treprostinil designed using Liquidia’s PRINT® technology with the goal of enhancing deep-lung delivery using a convenient, palm-sized dry powder inhaler for the treatment of pulmonary arterial hypertension (PAH).
Mirum Pharmaceuticals Completes Successful Pre-NDA Meeting with FDA for Maralixibat
December 16, 2019, Foster City, CA, Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced the successful completion of its clinical pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of pruritus associated with Alagille syndrome. In addition, the company had a chemistry, manufacturing and controls (CMC) meeting with the FDA. The purpose of the meetings was to discuss and confirm the clinical, non-clinical and CMC requirements for the company’s proposed NDA submission. The company also today announced that Rare Pediatric Disease Designation has been granted for maralixibat for Alagille syndrome, and thus may qualify for the receipt of a priority review voucher if the NDA is approved by the FDA. Maralixibat was recently granted Breakthrough Therapy Designation by the FDA for Alagille syndrome.
Allievex Completes Series A Financing Led by Pappas Capital and Novo Holdings
MARBLEHEAD, Mass., Oct. 23, 2019 — Allievex Corporation (“Allievex”), a clinical-stage biotechnology company founded by Pappas Capital, today announced the completion of a Series A financing co-led by Novo Holdings and Pappas Capital. Allievex has also appointed Kyle Rasbach, Ph.D., PharmD., Managing Partner at Pappas Ventures and Tiba Aynechi, Ph.D., Partner at Novo Holdings, to its Board of Directors. Proceeds from the fundraise will be used to support registrational Phase 2 clinical studies of Allievex’s lead product candidate, tralesinidase alfa (formerly known as BMN 250), an investigational enzyme replacement therapy to treat Sanfilippo syndrome type B or mucopolysaccharidosis IIIB (MPS IIIB) for which Allievex has obtained an exclusive, worldwide license from BioMarin Pharmaceutical Inc. Under the terms of its agreement with BioMarin, Allievex will assume all financial obligations associated with the development and commercialization of tralesinidase alfa, while BioMarin will provide certain manufacturing services in support of a marketing authorization filing.
Former CoLucid Drug, Lasmiditan, Receives FDA Approval
INDIANAPOLIS, Oct. 11, 2019 -- Eli Lilly and Company (NYSE: LLY) announced today that the U.S. Food and Drug Administration (FDA) has approved REYVOW™ (lasmiditan) an oral medication for the acute treatment of migraine, with or without aura, in adults. REYVOW has a unique mechanism of action and is the first and only FDA-approved medicine in a new class of acute treatment for migraine (serotonin (5-HT)1F receptor agonists).
Vertex and Ribometrix Establish Strategic Collaboration to Discover and Develop RNA-Targeted Small Molecule Therapeutics
BOSTON & DURHAM, NC, September 30, 2019 -- Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and RNA therapeutics developer Ribometrix, Inc. announced today that the companies have entered into a strategic collaboration to discover and develop novel RNA-targeted small molecule therapeutic candidates for serious diseases. The collaboration will combine Ribometrix’s proprietary discovery platform for identifying and optimizing small molecule therapeutics that modulate RNA function by targeting three-dimensional (3D) RNA structures with Vertex’s scientific, clinical and regulatory capabilities for up to three therapeutic programs, including one ongoing discovery program from Ribometrix.
Ribometrix Announces $7.8 Million in New Funding to Advance RNA-Targeting Drug Discovery Platform
DURHAM, NC, September 27, 2019 -- Ribometrix, a biotechnology company developing small molecule therapeutics that directly target RNA to treat human diseases, today announced $7.8 million in additional funding from existing investors, the Dementia Discovery Fund and Illumina Ventures. Ribometrix also announced the appointment of Mike Clayman, M.D., as chairman of the board of directors. The new funding will support the continued development of the Ribometrix discovery platform, which uses sophisticated structural analysis and proprietary assays to identify ligands that bind to highly structured pockets in therapeutically compelling RNAs to modulate protein expression. Ribometrix’s approach is designed to enable the discovery of selective, orally bioavailable, RNA-binding small molecules with broad tissue distribution and bioavailability.
