Nasdaq has suspended trading in Epizyme (NASDAQ:EPZM) pending the release of news, in this case, the outcome of today’s FDA advisory committee meeting on its application for tazemetostat for epithelioid sarcoma.
Wednesday, December 18, 2019
FDA clears Medtronic guidance system for brain surgery
The FDA has granted 510(k) clearance to Medtronic’s (NYSE:MDT) Stealth Autoguide, a cranial robotic guidance system used for spatial positioning and orientation of instrument holders or tool guides during neurosurgical procedures. The device is part of the company’s Surgical Synergy platform.
Cytokinetics’ reldesemtiv an Orphan Drug in U.S.
The FDA has granted Orphan Drug designation to Cytokinetics’ (NASDAQ:CYTK) reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS).
In collaboration with Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal muscle troponin activator, as a treatment for people with ALS, SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.
Among the benefits of Orphan Drug status in the U.S. is a seven-year period of market exclusivity for the indication, if approved.
Regenxbio gene therapy shows encouraging action in MSP II study
Preliminary results from a Phase 1/2 clinical trial evaluating REGENXBIO’s (NASDAQ:RGNX) gene therapy RGX-121 in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter’s syndrome, showed a treatment effect.
Three patients in Cohort 1 received an intercisternal dose (administered directly into a part of the brain) of MGX-121. Levels of heparan sulfate, high levels of which are associated with neurocognitive decline, dropped an average of 33% from baseline to week 8.
Two patients who progressed beyond week 24 showed stable neurocognitive development, one with normal development at week 48 and the other, who was diagnosed with neurocognitive decline prior to dosing, was stable (based on preliminary assessments).
The estimated completion date is December 2022.
Neon to start cell therapy trial in Europe
Neon Therapeutics (NASDAQ:NTGN) has filed a Clinical Trial Authorization (CTA) with the Dutch Health Authority for the evaluation of NEO-PTC-01 in patients with metastatic melanoma who are refractory to checkpoint inhibitors.
NEO-PTC-01 is a personal neoantigen-targeted T cell therapy candidate derived from patients’ peripheral blood mononuclear cells.
The company plans to initiate a Phase 1 dose escalation trial in metastatic melanoma in collaboration with the Netherlands Cancer Institute in H1 2020.
Shares are up 3% premarket.
Ultragenyx sells Crysvitaj royalty rights in Europe for $320M
Ultragenyx (NASDAQ:RARE) inks an agreement with Royalty Pharma, agreeing to sell its royalty right in Europe from Kyowa Kirin Co., Ltd. from net sales of Crysvita (burosumab) for $320M.
The deal will automatically expire when Royalty Pharma receives payments equal to or greater than 1.9x the purchase price prior to December 31, 2030, or in the event aggregate payments are equal to or greater than 2.5x the purchase price if the prior threshold is not met by the end of 2030.
Crysvita is conditionally approved in the EU for X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons. An application for use in adults with XLH is currently under EMA review.
ANI Pharma launches bretylium tosylate injection in U.S.
ANI Pharmaceuticals (NASDAQ:ANIP) has launched Bretylium Tosylate Injection USP, 50 mg/mL, a class III antiarrhythmic medication approved for the treatment of ventricular fibrillation and ventricular arrhythmias such as ventricular tachycardia.
The estimated annual U.S. antiarrhythmic market is ~$915M, based on data from IQVIA.
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