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.

How to assure screening for patients in whom just-approved Vitrakvi (larotrectinib) most likely will help is something "we've been working very hard for the last nine months" to establish, Joseph Germino, vice president of medical affairs for oncology with Bayer AG, told BioWorld. "The drug is so effective – it doesn't work in everybody [but] it works in most people – that it would be a shame if somebody were not able to get it because nobody checked" for eligibility.

Edge Therapeutics Inc.'s hunt for a "strategic alternative" after the failure of its flagship candidate, EG-1962 (nimodipine microparticles), ended with a stock-for-stock merger deal with privately held immuno-oncology (I-O) specialist Protein Delivery Sciences Biotechnology Corp. (PDS).

With a "cosmetically elegant" topical therapy, Pellepharm Inc. CEO Sanuj Ravindran told BioWorld that his firm is hoping to exploit the upside of hedgehog inhibition demonstrated so well by Erivedge (vismodegib, Roche Holding AG) while avoiding the downside of a systemic therapy with that mechanism of action in basal cell carcinoma (BCC).