JPM23, Day 3: Ionis CEO concedes PCSK9 ship may have sailed; Nektar forges ahead after bempeg blowup

We're off for another day of the J.P. Morgan Healthcare Conference. The flood of licensing agreements may have slowed, but there's still plenty of action to come as companies make pitches to their peers, investors and industry watchers. 

Some notable highlights from yesterday included Sage Therapeutics setting out its stall for depression drug zuranolone, while Karuna Therapeutics suggested that it didn’t need to take advantage of swirling acquisition rumors to scale up its hotly anticipated schizophrenia drug.

And while M&A deals may have dried up on Day 2, we're still holding out hope for a JPM-defining blockbuster acquisition. If that happens, you'll hear about it right here. 

Our Day 1 conference tracker is available here, and all the Day 2 action is wrapped here. Fierce Pharma’s take on the action so far can be found here and here.


6:05 p.m. ET: It appears that Ionis’ PCSK9 hopes are dimming. CEO Brett Monia told Fierce Biotech in an exclusive interview that the recently returned drug does not fit the company’s plans. The med, ION449, was returned to Ionis from AstraZeneca following a phase 2 data readout last year. The prospects of the company’s factor XI med, fesomersen, are more promising, however. Story

 

1:08 p.m. ET: Annexon Biosciences CEO Douglas Love said the company is giving itself "two ways to win” in the upcoming pivotal phase 2/3 trial for Huntington’s disease therapy ANX005, during his remarks at the JPM Wednesday morning. Love previewed the year ahead, which includes initiating ANX1502 in phase 1 studies for autoimmune indications, completing phase 3 enrollment for ANX005 in Guillain-Barre syndrome (GBS) and initiating the phase 2/3 trial for Huntington’s disease. Story

 

12:50 p.m. ET: Nektar Therapeutics is forging ahead with its existing pipeline, months after the failure of bempeg crumbled a multi-billion dollar deal with Bristol Myers Squibb. The company’s comeback story hinges mainly on two clinical-stage assets, one of which is a part of a collaboration with Lilly. A readout for that drug, rezpeg, is expected from a phase 2 trial in patients with lupus within the first half of the year, with CEO Howard Robin saying at the J.P. Morgan Healthcare Conference that he hopes the data is ready “sooner rather than later.”  Nektar’s remaining cancer opportunity rests solely on NKTR-255, an IL-15 agonist being teed up as a combo med for a number of cancer treatments. Story

 

12:05 p.m. ET: Novartis’ deal with Molecular Partners at the end of 2021 not only secured the biotech a potential $560 million biobucks payday in the future, it also cemented the reputation of radioligands as a “truly hot” therapy area for investors. At least, that’s how Molecular Partners CEO Patrick Amstutz, Ph.D., tells it.

“It’s hot because companies like Novartis have presented data, especially in prostate [cancer],” Amstutz told a JPM session. “So amazing results, and really opening this field for additional treatment.”

With radioligand programs progressing, as well as moving its first T cell engager into the clinic “literally any day” now, it’s a good thing the biotech has plenty of cash in the bank. “We’re well capitalized until 2026, not something we should take for granted in these stormy conditions,” the CEO said.

One outlier is Abicipar, a late-stage eye drug that was initially licensed out to Allergan and ultimately returned to Molecular Partners a decade later. The Swiss biotech is still on the lookout for a suitable partner, said Amstutz, who claimed the drug produced “beautiful data” in a late-stage trial for wet age-related macular degeneration. FDA concerns around inflammation put the breaks on approval hopes, but the CEO said the company has since had a “breakthrough” that the inflammation was caused by the type of syringe used.

“It seems we have solved the problem to have less inflammation,” Amstutz said. “We will not pursue this in Molecular Partners but we are in discussions to see if there is a path forward with this drug, especially as it would only need one additional safety trial to get it approved.”

 

While the conference is notorious for its dealmaking and partnership announcements, Takeda’s head of GI therapeutics Chinwe Ukomadu, M.D., Ph.D., told Fierce Biotech on the sidelines that the Japanese pharma is always looking for potential collaborators.

“We don’t wait for JPM,” Ukomadu said. “We’re having conversations all along with the companies. We see them on our team meetings, Zoom meetings, but there’s something unique about getting to sit with them in person now.”

The moral of the story? To be successful, Big Pharmas can’t be too inward looking. Under the guidance of R&D head Andy Plump, M.D., Ph.D., Takeda has built out 90% of its pipeline assets in fewer than 10 years, a feat only possible via collaboration.