Barely a week after Wall Street hailed interim data from a RNA-targeting small-molecule candidate in spinal muscular atrophy (SMA) candidate, startup Skyhawk Therapeutics Inc., which also is taking a small-molecule approach to correct RNA mutations, disclosed a $40 million equity investment round and a neurology-focused option deal with Celgene Corp. that brings $60 million up front.

With one biopharma set to price its IPO this week, three others – neurology-focused Aptinyx Inc., stem cell gene therapy firm Avrobio Inc. and specialty pharma Xeris Pharmaceuticals Inc. – have set terms for their anticipated offerings, putting the sector on track to match last year's IPO activity, at least in terms of number of offerings. In terms of money raised, however, the first half of 2018 looks like it might top the amount raised in global IPOs priced in the first half of 2017.

BOSTON – When Novartis AG's Kymriah (tisagenlecleucel) became the first CAR T therapy on the market after winning FDA approval last year for B-cell precursor acute lymphoblastic leukemia (ALL), the $475,000 price tag was mitigated somewhat by news that the Swiss pharma offered an outcome-based plan in which treatment would be reimbursed only when patients responded to therapy at the end of the first month. While the move ramped up discussions on how to shift the U.S. health care economics from volume-based pricing to a value-based or outcomes-based model – a change that will become increasingly necessary as more life-changing and potentially curative gene and cell therapies reach market – cost alone marks only one of the challenges in getting CAR T treatments to patients. 

As Zymeworks Inc. prepares to unveil new phase I data from its lead bispecific antibody candidate at the upcoming American Society of Clinical Oncology meeting, its platform technology scored another win in the validation column. Less than two years after licensing rights to Zymeworks' Azymetric and EFECT technology platforms to develop one bispecific candidate, Daiichi Sankyo Co. Ltd. returned to the negotiating table for more.

Despite the rise of biologics – and biosimilars – in the ulcerative colitis (UC) space, investors backed Dublin-based Sublimity Therapeutics Ltd. in a $64 million round to advance STI-0529, its orally delivered soluble version of cyclosporine, into a phase IIb study.

Heading toward the May 21 PDUFA date for its thrombocytopenia drug, avatrombopag, Dova Pharmaceuticals Inc. was determined to have its sales and marketing team ready to go for a June launch, optimism that appeared justified Monday afternoon when the FDA gave the nod to the second-generation, once-daily, oral thrombopoietin (TPO) receptor agonist following a priority review.

As Zymeworks Inc. prepares to unveil new phase I data from its lead bispecific antibody candidate at the upcoming American Society of Clinical Oncology meeting, its platform technology scored another win in the validation column. Less than two years after licensing rights to Zymeworks' Azymetric and EFECT technology platforms to develop one bispecific candidate, Daiichi Sankyo Co. Ltd. returned to the negotiating table for more.

The possibility of offering the first treatment in the U.S. for neurological muscle spasms that bears no warning of sedative side effects in the label attracted the interest of Sofinnova Ventures, which led to a $60 million series A round for Neurana Pharmaceuticals Inc. Funds will be used to support an upcoming phase II study testing a highly purified version of muscle relaxant tolperisone in neuromuscular spasms of the neck and back.

Coming off a strong first quarter, in which sales of cabozantinib solidly bested Street expectations, Exelixis Inc. made another move to expand its early stage pipeline via a collaboration with multispecific antibody firm Invenra Inc. The two will work together using Invenra's B-Body technology platform to identify candidates for treating cancer.