JPM Day 2: Highlights

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Follow updates from the JPM Annual Healthcare Conference - BioSpace will be tracking key updates here all day. All times are Eastern Standard.

5:15 p.m.: The last time Relay Therapeutics CEO Sanjiv Patel presented at JP Morgan week, it was as the head of a preclinical company. On Tuesday, he showcased a pipeline of six candidates, three of them in early-to-mid clinical development. 2023, he said, is “all about execution.”

Patel first focused on the Phase I trial of PI3Kα Inhibitor RLY-2608 as monotherapy in advanced solid tumor patients and in combination with fulvestrant for patients with advanced breast cancer. Relay expects initial data from this trial during the first half of 2023.

Also in 2023:

• Relay projects being in the clinic with RLY-5836, a pan-mutant selective PI3Kα inhibitor, in Q2, 2023.
• Relay anticipates full dose escalation data from the REFOCUS study of FGFR2 inhibitor RLY-4008 in the first half of 2023. RLY-4008 is being assessed in patients with intrahepatic cholangiocarcinoma and other advanced solid tumors

• Relay expects to nominate a development candidate for its Estrogen Receptor Alpha (ERα) Degrader program in 2023. This program is also targeting breast cancer.

During his presentation, Patel emphasized the favorable interim tolerability profile of the clinical candidates, which he said minimized key dose-limiting off-target toxicities. The company also has five undisclosed programs in preclinical development.

4:30 p.m.: Just four months after becoming CEO of Scholar Rock, Jay Backstrom, M.D. touted some of the company’s recent achievements, emphasizing its “revolutionary platform” and “robust pipeline of promising products.”

By far, Scholar Rock’s most notable achievement in 2022 came from its investigational antibody, apitegromab. In the Phase II TOPAZ trial, the therapeutic improved quality of life in patients with types 2 and 3 spinal muscular atrophy.

The candidate is poised to be the first muscle-directed therapy in SMA, Backstrom said. He added that if successful, it “will be the catalyst for Scholar Rock to become a fully integrated commercial company.”

Though there are already three approved therapies for SMA, Backstrom said Scholar Rock’s candidate will “complement, not compete” with them, as it uses a completely different mechanism of action.

In 2023, all eyes will be on the Phase III SAPPHIRE trial, which, if positive, is expected to form the basis for regulatory submissions.

Backstrom also cited the November appointment of chief medical officer Jing Marantz, M.D. as a notable achievement over the past year.

1:30 p.m.: Sanofi CEO Paul Hudson presented the company’s outlook for the new year, highlighting upcoming milestones and achievements that mark what he called the “new Sanofi.”

He opened by touting Dupixent’s success in 2022, stating that over 500,000 patients are already being treated with the therapeutic. Hinting at expectations for the new year, he added that Sanofi hasn’t “touched the surface” yet in terms of Dupixent’s growth.

Hudson also emphasized the progress of Sanofi’s once-weekly hemophilia A treatment, which was granted breakthrough therapy designation by the FDA in June 2022. He anticipates the therapeutic will be approved in February, 2023.

Also in focus was the company’s RSV vaccine efforts. Days before the conference began, the FDA accepted Sanofi’s application for nirsevimab. If approved, it would become the first protective option against RSV disease for all infants.

Hudson said Sanofi hopes to launch the vaccine in time for RSV season later this year. He expressed his hope that an approval would prevent 80% of hospitalizations in this indication.

1:30 p.m.: Sage Therapeutics CEO Barry Greene began his presentation by stating, “We are in a brain health pandemic.” He went on to lay out his company’s answer to the crisis.
Greene touted the consistent data profile for zuranolone across major depressive disorder (MDD), postpartum depression (PPD), treatment-resistant depression, generalized anxiety disorder and bipolar depression.

Sage and development partner Biogen filed an NDA with the FDA for zuranolone, a positive allosteric modulator of the GABA-A receptor, in MDD and PPD in December. If approved, it would be the first oral medication specifically intended for PPD.

If approved, Greene emphasized zuranolone would provide a “completely new approach to treating depression,” and said Sage and Biogen would be prepared for an advisory committee meeting should the FDA choose to take that route.

Zuranolone is a rapid-acting, once-daily, 14-day oral short-course treatment. This differentiates it from most treatments currently on the market, which primarily treat depression as a chronic condition. Zuranolone would treat the disease episodically.

Earlier on in the pipeline, Greene announced that will be moving another asset, SAGE-319 for GABA hypofunction, into Phase I. GABA, an amino acid, functions as the inhibitory neurotransmitter in the brain. Among other indications, reduced levels of GABA have been shown to play a role in preclinical models of adolescent schizophrenia.

Noon: Karuna Therapeutics came into JPM with a new CEO as Bill Meury took over for Steve Paul, who remains with the company as chief scientific officer and president of R&D.

Much attention is currently focused on the Boston-based neuroscience company as it intends to submit schizophrenia-hopeful KarXT (xanomeline-trospium) for FDA approval by mid-2023.

If approved, KarXT would be the first treatment to address the symptoms of the disease, including withdrawal, social isolation, apathy and lack of motivation.

Karuna anticipates data from the ongoing Phase III EMERGENT-3 trial in the first quarter of 2023 – data Paul said would be a part of the New Drug Application.

Meury emphasized the potential of KarXT to be a platform consisting of schizophrenia, schizophrenia adjunctive treatment and Alzheimer’s disease. The presentation particularly highlighted plans to “execute a robust Phase III program” in psychosis in Alzheimer’s disease.

Meury highlighted three key strategic priorities for 2023:

1. Maximizing the value of KarXT – particularly securing approval in schizophrenia.

2. Building platform capabilities to commercialize KarXT.

3. Expanding the pipeline by identifying and developing new compounds - either validated targets and/or differentiated pharmacology

He laid out preparations for a potential launch of KarXT in schizophrenia, which he said will include strategic positioning of the therapy, working with medical advocacy groups to continue to destigmatize the disease and preparing deployment plans for the field sales team.

10:30 a.m.: GSK CEO Emma Walmsley highlighted the company’s pivot to a pure-play global biopharma company following the de-merger of its consumer healthcare unit Haleon.

As part of this effort, Walmsley pointed out the company’s rapid innovation in vaccines, including the development of a long-acting HIV injection, the strong commercial performance of the groundbreaking shingles vaccine Shingrix and the recent FDA acceptance of its experimental RSV vaccine. Commercially speaking, GSK’s leader expects the company’s vaccines business to be a stable, long-term contributor to its core value proposition.

Keeping with this theme, Walmsley said the company has 23 vaccine projects currently under development. GSK’s experimental RSV vaccine stands out in particular due to its potential to generate multi-billion dollar-per-year sales in the back half of the decade.

Lastly, GSK’s leader also reconfirmed the company’s commitment to bringing novel immunology, respiratory and oncology medicines to market. As a result of this commitment to innovation, the drugmaker is anticipating top-line growth of more than 5% on an annual basis over the period from 2021 to 2026.