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Monday, July 1, 2019

Amicus Therapeutics inks deal with Brammer Bio for gene therapy programs

Amicus Therapeutics (NASDAQ:FOLD) has entered into a strategic manufacturing collaboration with Thermo Fisher Scientific (NYSE:TMO).
Thermo Fisher and Amicus will collaborate to develop platform manufacturing capabilities to support the broader portfolio of AAV gene therapy programs.
Current R&D production technologies and capabilities related to the Amicus preclinical and clinical-stage gene therapy programs for CLN6, CLN3, and other potential Batten disease programs are being transferred to and developed at Thermo Fisher’s viral vector services business.
FOLD shares are up 2% premarket.
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Bicycle Therapeutics up 10% premarket on positive THR-149 data

Thinly traded micro cap Bicycle Therapeutics (NASDAQ:BCYC) is up 10% premarket on light volume on the heels of encouraging data from an open-label Phase 1 clinical trial evaluating a single intravitreal injection of THR-149, a Bicycle-based plasma kallikrein inhibitor, in patients with diabetic macular edema (DME).
A rapid onset of action was observed from day 1 with an increasing improvement in best corrected visual acuity (BCVA) of up to 7.5 letters at day 14. The treatment effect was maintained (average improvement in BCVA of 6.5 letters) at day 90.
On the safety front, no dose-limiting toxicities or treatment-related adverse events were reported.
The study was conducted by licensee and collaboration partner Oxurion.
Development is ongoing.
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Alnylam files application in Europe for givosiran for rare of liver disorders

Alnylam Pharmaceuticals (NASDAQ:ALNY) has filed a marketing applicationin Europe seeking approval of RNAi therapeutic givosiran for the treatment of acute hepatic porphyria, a group of four rare inherited liver disorders characterized by intense abdominal pain over a period of one-to-two weeks.
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Cerecor’s CERC-301 shows encouraging action in early-stage hypotension study

Results from a Phase 1 dose-escalating clinical trial evaluating Cerecor’s (NASDAQ:CERC) CERC-301 in patients with neurogenic orthostatic hypotension (a sudden drop in blood pressure when the person stands up) showed a positive effect.
Participants receiving the 20 mg dose, the highest tested, showed clinically meaningful improvements in blood pressure throughout the study.
On the safety front, there were no serious adverse events reports and all doses were considered safe and well-tolerated.
CERC-301 is an NR2B subunit-specific NMDA receptor antagonist. NMDA receptor overactivation is associated with a range of neurodegenerative diseases, including Parkinson’s.
Development is ongoing.
Shares are up 10% premarket on light volume.
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Atara’s ATA188 shows favorable safety profile in early-stage MS study

Preliminary results from a Phase 1 study assessing Atara Biotherapeutics’ (NASDAQ:ATRA) ATA188 in patients with progressive multiple sclerosis (MS) demonstrated an encouraging safety profile. The data were presented at the European Academy of Neurology Annual Congress in Oslo.
Results from the first three dose cohorts showed no dose-limiting toxicities and no serious treatment-emergent adverse events.
The study is ongoing. The primary objective is to identify the optimal dose for a Phase 2 trial.
ATA188 is an allogeneic Epstein-Barr virus (EBV) cytotoxic T lymphocyte (CTL) immunotherapy that targets EBV antigens that the company believes play a key role in the treatment of MS.
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Pfizer’s crisaborole ointment safe in infants and toddlers

Results from a Phase 4 study, CrisADe CARE 1, evaluating Pfizer’s (NYSE:PFE) crisaborole ointment 2% in young children aged three months to less than 24 months with mild-to-moderate atopic dermatitis (AD) showed a safety profile consistent with earlier studies.
Detailed data will be submitted for presentation at future medical conference.
Crisaborole ointment, branded as Eucrisa, is currently approved in select countries, including the U.S., for the treatment of mild-to-moderate AD in patients at least two years old.
Shares are up 1% premarket on light volume.
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Biogen’s Spinraza shows long-term effect in mid-stage SMA study

Results from an open-label Phase 2 clinical trial, NURTURE, evaluating Biogen’s (NASDAQ:BIIB) Spinraza (nusinersen) in 25 presymptomatic patients with spinal muscular atrophy (SMA) showed a long-term treatment benefit. The data are being presented at the Cure SMA Annual SMA Conference in Anaheim, CA and the European Academy of Neurology Annual Congress in Oslo.
After 45.1 months of analysis, all treated patients were alive without the need for permanent ventilation and all were sitting independently. 88% were walking independently.
No new safety signals were reported.
The FDA approved the survival motor neuron-2-directed antisense oligonucleotide for the treatment of SMA in December 2016.
Shares are up 1% premarket on light volume.
Related ticker: Ionis Pharmaceuticals (NASDAQ:IONS)
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