As we entered a new decade, BioWorld writers took the opportunity to review the highs and lows of the past 12 months and they concluded that 2019 was a great year for the sector – save for a few bumps in the road. The financial markets finally broke out of a slump in the fourth-quarter flourishing; cash flowed to the sector and dealmaking reached historic proportions. New technologies like CRISPR and the long-awaited promise of genomics found their way to the front lines of health care. On the flip side, the drug pricing debate continued to overhang the sector and expand beyond the U.S., and the ongoing Brexit saga and the U.S./China trade war kept investors off balance.
It was yet another banner year for European biotechnology firms engaged in drug development. The sector took in $7.73 billion in equity funding in 2019, just edging past the previous high of $7.71 billion it generated in 2018.
Plenty of new medicines
After a flurry of activity in December, when the FDA approved seven new molecular entities (NMEs), the total of novel drugs that were given the green light this year reached 48, a number that ranks third behind the record 59 new medicines the agency approved last year and 53 in 1996. Over the past three decades, the FDA has approved 924 novel medicines, averaging out to approximately 30 per year. The present decade yielded an above average of 38 NMEs compared to an average of 23 in the 2000-2009 decade and 31 average in the 1990-1999 period.
In China, regulators granted the marketing nod to Beigene Ltd.’s PD-1 antibody, tislelizumab, for treating patients with classical Hodgkin lymphoma who have received at least two prior therapies. This is Beigene’s first drug to win approval in the country, following an FDA approval for its BTK inhibitor, Brukinsa (zanubrutinib) in 2019.
It was not all good news on the clinical front, with Incyte Corp.’s bid to tackle acute graft-vs.-host-disease (GVHD) with JAK1 inhibitor itacitinib coming up short in pivotal testing, leaving analysts skeptical about the drug's future. In addition, Novan Inc. reported a phase III failure of SB-206, a topical nitric oxide, in treating molluscum contagiosum for patients ages 6 months and older.
Two players in the gene sequencing space, Illumina Inc. and Pacific Biosciences, have scotched their planned $1.2 billion merger roughly two weeks after the U.S. Federal Trade Commission posted a 5-0 vote to seek an injunction against the merger.
Acceleron Pharma Inc. and Fulcrum Therapeutics Inc. agreed to an R&D collaboration and license agreement to identify small molecules for treating an undisclosed target in pulmonary disease. Fulcrum receives $10 million up front and will be reimbursed for its R&D. It is in line for potential milestones of up to $295 million for the first product commercialized and a maximum of $143.5 million in additional milestone payments for all subsequent products commercialized.
Leap Therapeutics Inc. and Beigene Ltd. have entered an exclusive option and license agreement for the clinical development and commercialization of DKN-01, Leap's anti-Dickkopf-1 (DKK1) antibody, in Asia (excluding Japan), Australia and New Zealand. Leap will retain exclusive rights for the rest of the world
The holiday-shortened week slowed the number of financial deals; however, those of note were:
Urovant Sciences Inc., which signed a $300 million term loan facility with Sumitomo Dainippon Pharma Co. Ltd., with plans to use proceeds to repay existing debt, fund ongoing development projects, fund the initial commercial launch of vibegron, if approved by the FDA, and for general corporate purposes.
CF Pharmtech Inc. raised a $90 million series E investment led by New Alliance Capital.
Word on the Street: BioWorld's favorite quotes of the week
“With six additional screens planned for 2020, we think [Fulcrum]'s platform may continue to crank out potential drug candidates across multiple indications – now including pulmonary – that aim to address the root cause of each disease. We believe [Fulcrum]'s proprietary discovery platform is a distinguishing and valuable asset.”
SVB Leerink analyst Joseph P. Schwartz, on Fulcrum Therapeutics Inc.’s R&D collaboration and license agreement with Acceleron Pharma Inc. to identify small molecules for treating an undisclosed target in pulmonary disease
“Combination therapies will be the future for PD-1/PD-L1 antibodies. PD-1/PD-L1 antibodies are getting less competitive in the Chinese market if only used as monotherapies.”
Kai Sun, an analyst from Shanghai-based Green River Investment
"Incyte isn’t exactly ringing in the new year with a bang."
J.P. Morgan analyst Cory Kasimov, on the trial failure of Incyte Corp.’s JAK1 inhibitor, itacitinib