Less than stellar Phase I results have triggered a pipeline cleanup at Syros Pharma, where one of its two lead drugs — a CDK7 inhibitor administered intravenously — is getting swapped out with an oral version.

The biotech said it maintains its belief in the CDK7 pathway for cancer, but thinks prioritizing SY-5609, which appears more selective and potent in preclinical models, is a wiser choice than holding onto an IV drug that didn’t quite embody the optimal balance between efficacy and tolerability. A human trial for SY-5609 is planned for early next year.

For a pharma company with about $5 billion in revenue, a couple of respectably sized blockbuster drugs on the market and some high-profile partners like Amgen, Belgium’s UCB has kept an unusually low profile on the pipeline side of things over the years.
Until now.
Just days after striking a $2.1 billion deal to buy Ra Pharmaceuticals and its C5 rival to Soliris, UCB is posting positive top-line Phase III results for a dual IL-17 inhibitor that it’s steering into one of the most competitive commercial spaces in the industry. And despite plenty of obvious challenges as they struggle to roll out Evenity with Amgen and patent expirations loom on its franchise drugs, including Cimzia, the company just may be ready to tackle some of the biggest players on the planet.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.
We don’t know the PASI90 and IGA scores — but UCB knows that with the kind of heavyweight competition it faces with Novartis and others, marginal gains for patients won’t stack up. So we’ll be watching for the hard numbers. And there’s another head-to-head with Cosentyx that will play a big role in pushing up analysts’ projections on peak sales, which currently fall well short of blockbuster status.
UCB hasn’t exactly been in the spotlight for the last few years, but it’s in a position now that the company has to win some respect in R&D, with blockbuster projects that can keep investors’ attention at a time the industry is experiencing booming R&D development efforts around the planet.
It hasn’t been easy. There was a setback on a lupus drug partnered with Biogen. But there have been some advances, with a deal to buy Proximagen’s NDA-ready nasal spray therapy USL261, designed as a rescue therapy for acute repetitive seizures, for $150 million in cash and another $220 million in sales and regulatory milestones. There was even a report that the company was kicking the deflated tires at Acorda, though nothing came of that.
Late last year UCB also committed to spend up to £200 million on a new R&D hub in the UK.
That may not translate into a lot of excitement right now, but they’re trying. And there’s a subtle promise that more deals may be in the works.

Five months after the FDA approved Incyte to expand its flagship Jakafi drug to patients with steroid-refractory acute graft-versus-host disease (GVHD), the company announced positive topline results from its Phase III study.

“This announcement, while a positive, is not surprising given the recent label expansion for Jakafi in the U.S. into this setting based on results from the single-arm REACH1 trial,” Baird’s Brian Skorney wrote, adding that it should allow Novartis, Incyte’s partner, to begin registering the drug abroad.

Roche $RHBBY unveiled a cheerful update on its performance in the first nine months of this year, with demand for its newer drugs — primarily in oncology/immunology — driving sales, particularly in China where cancer is on the rise. In its press release, the Swiss drugmaker mentioned its pending $4.3 billion acquisition of Spark Therapeutics $ONCE, promising the deal — which has seen delay after delay due to unrelenting scrutiny by competition regulators — will be consummated by the end of the year.

If alpha beta T-cells — the foundation of CAR-T cell therapy — are “killer cells,” trained in the biologically ancient art of executing intruders, think of their gamma delta cousins as armed guards, capable of engaging an interloper but also of sounding an alarm to kick the rest of the body’s defenses into action.

“The key role they play is to conduct immune surveillance,” Natalie Mount told Endpoints News.  And once they find an intruder “they can stimulate a whole immune response as well as be cytotoxic [cell-killing].”

Eight months after reeling in Clementia and their drug palovarotene in a $1.3 billion buyout deal, David Meek has another rare disease therapy to add to the pipeline at Ipsen.

The Paris-based biotech (EPA: $IPN) is fronting $25 million in cash to acquire worldwide rights to Blueprint’s BLU-782, an ALK2 inhibitor designed to fight an ultra-rare genetic condition called fibrodysplasia ossificans progressiva, or simply FOP. And there’s the full slate of $510 million more for milestones running through development, regulatory as well as commercial.
That didn’t help Blueprint’s share price, though. The stock was in the red ahead of the bell with investors likely a little edgy over the limited amount of cash involved and a lower likelihood of a buyout that would have jacked up their equity value considerably.
At the beginning of this year, BLU-782 had been teed up as the next drug to beef up the biotech’s pipeline, with plans to run a Phase I in healthy volunteers and then pivot to Phase II in the first half of next year.
Loxo Oncology $LOXO — acquired by Eli Lilly — and Blueprint Medicines $BPMC have been duking it out with their studies on rival RET inhibitors, with the Loxo drug typically coming out ahead in the comparisons. Back in June Blueprint also submitted an NDA for avapritinib, a treatment for adults with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST.
Ipsen, meanwhile, seems happy to pursue more rare disease drugs as Meek positions the company along some familiar lines in the industry. Rare diseases — with their small trials and accelerated reviews — has become an attractive space for a broad swath of biotechs around the world.