JPM22, Day 4: Galapagos plots rebound with M&A, new CEO; Gritstone hopes for new cancer biomarker; Vertex's cystic fibrosis dominance

We're into the final scheduled day of the annual J.P. Morgan Healthcare Conference.

After a slate of research collaborations defined the first days of the conference, there's still plenty of action to come as companies make their final pitches to their peers, investors and industry-watchers, and look to set the stage for the year ahead.

Want more? Catch the latest news from Fierce Biotech, and follow all of Fierce Pharma's coverage.

Be sure to have a look back at our Day 1 conference tracker available here, plus all the action from Day 2 and Day 3. And perhaps next year we'll see you in San Francisco in person!

UPDATED: Thursday 4:55 p.m. ET

Pacific Biosciences announced it will work with machine learning researchers at Google to push the accuracy of the company’s long-read DNA sequencing hardware even higher. Meanwhile, PacBio CEO Christian Henry responded to news from earlier in the conference that the company’s main DNA sequencing competitor, Illumina, plans to add longer-read capabilities to its current line of short-read machines. Story

Thursday 4:26 p.m. ET

Galapagos thinks 2022 will be a rebound year, with potential acquisitions and new leadership in the mix. The biopharma went south last year when it canned a phase 3 asset, ziritaxestat, and ran into speed bumps with its JAK inhibitor, filgotinib, as the FDA tightened its stance on the drug class.

In his final presentation as CEO, Onno van de Stolpe said the company expects to announce a new CEO “in weeks rather than months.” The new leader “has a fantastic legacy,” he said during the annual J.P. Morgan Healthcare Conference on Thursday morning. And, to bolster its later-stage pipeline, Galapagos’ business development team has a “massive” effort in the works to in-license or acquire an “attractive molecule,” the outgoing CEO said. Story

Thursday 12:46 a.m. ET

Renalytix is predicting that “2022 is going to be a heavy year of milestones” according to CEO James McCullough. In addition to bringing its AI tech to at least 20 new health system partners and securing reimbursement from Medicare and private insurers, the company is marching toward an FDA clearance for KidneyIntelX, which merges blood-based biomarkers with data points from health records to predict a person’s risk of kidney failure or progressive disease. Story

Thursday 11:44 a.m. ET

The AI-powered molecule designer Insilico Medicine has put another international drug discovery partnership under its belt, with the Shanghai-based Fosun Pharma signing on for work on four disease targets. Fosun will also assist in co-developing Insilico’s QPCTL program, aimed at a checkpoint protein in immuno-oncology, in addition to making an equity investment in the company. Story

Thursday 9:49 a.m. ET

Gritstone bio has taken its individualized neoantigen vaccine into a phase 2/3 trial that could support FDA approval in colorectal cancer. Discussing the news at JPM, Gritstone CEO Andrew Allen highlighted the data that supported the progress, revealing that all patients who were alive as of the earlier August cutoff were still alive as of last week. Gritstone has seen the best responses in patients who have a molecular response, as shown by decreased circulating tumor DNA (ctDNA), and expects regulators to ultimately start accepting the biomarker as evidence of efficacy.

“As we get more and more validation data, I think then we’ll start to see regulatory acceptance of ctDNA as a surrogate for efficacy and the basis for approval. We're not there yet. FDA obviously is accepting ctDNA for patient selection, but not yet as a basis for approval. I do believe that's a matter of time, however,” Allen said.

Thursday 8:45 a.m. ET

Vertex Pharmaceuticals thinks it’ll be the leader in cystic fibrosis (CF) “well beyond” this decade and into the 2030s, said President and CEO Reshma Kewalramani at JPM Monday. The reason is threefold, the CEO said: 25,000 patients who are eligible for treatment haven’t received it yet; the drugmaker is in the early launch stage in newly reimbursed countries like the Netherlands and awaits reimbursement in regions like Belgium and Australia; and Vertex aims to reach younger patients, including 2- to 5-year-olds down the road. Aside from its approved drugs, Vertex aims to treat CF with a triple combo that is in phase 3 and is working on an mRNA-based treatment, which hasn’t entered the clinic yet.

Beyond the lung and digestive disorder, Vertex is also working on Type 1 diabetes. One patient, who has had diabetes for decades, was able to produce insulin after receiving Vertex’s cell therapy, dubbed VX880, the company said in October. Now, Vertex said the patient has seen a 92% reduction in daily insulin use as of Day 150, and the safety profile remains unchanged, Kewalramani said.