Making good on a promise detailed during its earnings call with investors last month, Voyager Therapeutics Inc. kicked off Restore-1, a phase II, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of gene therapy VY-AADC for Parkinson's disease (PD) in patients with motor fluctuations refractory to medical management.

This year, about 55,440 people (29,200 men and 26,240 women) will be diagnosed with pancreatic cancer, according to the American Cancer Society, and about 44,330 people (23,020 men and 21,310 women) will die of this particularly virulent tumor type. For men, the average lifetime risk is about one in 63; for women, about one in 65. But risk factors – tobacco use, obesity, exposure to certain chemicals – play a role.

In attention deficit hyperactivity disorder (ADHD), doctors are "starving for something new, something refreshing, something that can really add value and is not only a formulation of an existing amphetamine or methylphenidate," said Supernus Pharmaceuticals Inc. CEO Jack Khattar – and in fast-onset SPN-812, they just might have it.

Orbimed Advisors portfolio firm Smartzyme Biopharma Inc. joined forces with Hemoshear Therapeutics Inc. to come up with protein-based drugs for rare metabolic diseases, spinning off the former's protein-engineering platform into a new enterprise called Carnot Biosciences.

Ocular Therapeutix Inc. said the therapeutic area has plenty of room for its newly approved Dextenza (dexamethasone ophthalmic insert) 0.4 mg for postoperative eye pain along with Eyepoint Pharmaceuticals Inc.'s single-dose, sustained-release, intracameral Dexycu (dexamethasone intraocular suspension) 9 percent, granted U.S. marketing clearance earlier this year for inflammation after surgery.

With dosing complete in its phase II trial testing Ra Pharmaceuticals Inc.'s RA-101495 in generalized myasthenia gravis (MG) and date likely to roll out near the end of this year, the world already has begun weighing odds of the compound in a space where the likes of Alexion Pharmaceuticals Inc. and Argenx SE are formidable players.

With dosing complete in its phase II trial testing Ra Pharmaceuticals Inc.'s RA-101495 in generalized myasthenia gravis (MG) and date likely to roll out near the end of this year, the world already has begun weighing odds of the compound in a space where the likes of Alexion Pharmaceuticals Inc. and Argenx SE are formidable players.

Full results are not due until the spring, but investors meanwhile didn't seem much enthused by Dublin-based Alkermes plc's positive top-line results from Enlighten-2, a pivotal phase III study of ALKS-3831 (olanzapine/samidorphan), a once-daily, oral atypical antipsychotic prospect for schizophrenia.

Cancer data typically steal the show at the American Society of Hematology (ASH) meeting, but hemophilia often gets plenty of play as well, and this time around could be especially compelling for investors in firms with therapies in the works for the clotting disorder.