JPM Day 3: 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.

2:15 p.m.: Y-mAbs President and Interim CEO Thomas Gad’s JPM presentation came one week after the company announced plans to lay off 35% of its workforce in an attempt to extend its cash flow and reprioritize its pipeline.

This decision followed the November receipt of a Complete Response Letter regarding the company’s Biologics License Application for omburtamab as a treatment for CNS/leptomeningeal metastasis from neuroblastoma.

Gad opened the presentation by addressing the CRL and subsequent restructuring initiatives that will see Y-mAbs shift its focus to three areas:

• Danyelza, previously approved in the U.S. for r/r high-risk neuroblastoma. Danyelza was granted conditional approval in December for the treatment of pediatric patients one year of age and older in China.
• Additional label expansions and indications into osteosarcoma.
• Other adult indications and partnerships to develop the aforementioned indications.

Gad closed the presentation by saying the workforce reductions and pipeline restructuring will take effect over the course of the first quarter of 2023.

1:30 p.m.: Only 11 days into his new post, Teva CEO Richard Francis stepped into J.P. Morgan expressing confidence about the company’s ability to continue to service its ongoing debt and achieve growth.

The presentation was chiefly led by CFO Eli Kalif, who said as the “world’s largest leader”, the company’s most critical element is managing its footprint, including sizable debt, to have better access to medicines to serve patients.

“What we also did in the last five years, and as we committed, we serviced the debt,” Kalif said, noting that Teva looked at any assets it was able to monetize and anything it could optimize in order to contribute cash. In total, Teva was able to pay around $18 billion, he said.

Moving forward, Francis, who assumed the position from Kare Schultz as of 2023, cautioned against any rapid strategic shifts. Francis, too, touted the generics business the company is known for.

Additionally, he said he hoped biosimilars could create growth.

“Some of the major growth drivers around biosimilars and around our innovative portfolio … are a significant unmet need that can be addressed.”

12:00 p.m.: Arcturus Therapeutics spoke at JPM Wednesday on the cusp of its 10-year anniversary. In his presentation, Joseph Payne, president & CEO, provided updates on assets across both the Arcturus-owned and partnered portfolios.

Payne expressed particular excitement about a Phase III study of ARCT-154, a COVID-19 vaccine built on the company’s LUNAR lipid-mediated delivery platform. Working with Japan’s Meiji Pharma, Arcturus will compare its self-amplifying mRNA vaccine to Pfizer’s Comirnaty in a non-inferiority trial.

As the Japanese population is highly vaccinated and boosted, this will provide an opportunity to “directly compare self-amplifying mRNA with conventional [mRNA] and…put to bed all questions and concerns” about how the technology fits into this space, Payne said.

In December, Arcturus struck a global licensing and collaboration agreement with CSL Ltd. subsidiary CSL Seqirus for access to this vaccine. CSL paid Arcturus $200 million upfront.

Other Milestones for 2023 include:

• Interim data from a Phase II trial of ARCT-810 in ornithine transcarbamylase (OTC) deficiency, a genetic urea cycle disorder that causes too much ammonia to accumulate in the blood
• Initiation of a Phase I trial for ARCT-032 for cystic fibrosis expected in Q1
• 734 additional subjects slated to be vaccinated in the Japanese trial of ARCT-154

11:15 a.m.: Nektar Therapeutics’ President and CEO Howard Robin’s presentation highlighted the company’s new strategy following last year’s course-altering clinical failure of bempegaldesleukin (bempeg) and Bristol Myers Squibb’s Opdivo. As a refresher, the two companies announced disappointing clinical trial data for the combo therapy in melanoma, renal cell carcinoma and urothelial carcinoma in the first half of 2022.

Nektar’s comeback plan will reportedly rest on three key pillars:

• Robin plans to continue to focus on reducing expenses to maximize the company’s cash runway. At last count, Nektar had approximately $500 million in cash and cash investments, which should be sufficient to fund the drugmaker’s near-term pipeline efforts.
• The company plans to spotlight the Rezpeg autoimmune collaboration with Eli Lilly. Rezpeg is on track to produce Phase II topline data in lupus in the first half of 2023. Additionally, Lilly is expected to initiate another Phase II trial for the drug in atopic dermatitis later this year. Finally, Robin said Lilly is gearing up to reveal Rezpeg’s third autoimmune indication in the first half of 2023.
• Robin also expects the company’s immune booster, NKTR-255, to play a vital role in Nektar’s comeback story. During his presentation, he called the experimental compound “potentially the best IL15 molecule in development”. Nektar is trialing the compound across of range of solid and liquid tumor types. Nektar’s NTKR-255 program ought to yield four major data releases over the course of 2023, per today’s presentation.

10:30 a.m.: Brian Goff, CEO of Agios Pharmaceuticals, presented updates on the company’s pyruvate kinase (PK) activation growth portfolio, touting its first-in-class therapy for adults with PK deficiency, the first disease-modifying therapy for this rare anemia.

According to Agios’ presentation, PK activation may address a range of hemolytic and acquired anemias underpinned by a shared pathophysiology. Agios intends to use the underlying red blood cell malfunction in these diseases to develop the PK application for more conditions.

Highlights of the presentation included:

• Compelling and consistent data across connected diseases, particularly those with an underlying pathophysiology of red blood cell metabolic stress (including PK, thalassemia, sickle cell disease and lower-risk myelodysplastic syndromes)
• Meaningful commercial opportunities (potential for additional PK applications by 2026)
• Building a diverse pipeline leveraging Agios’ expertise in cellular metabolism
• Goff said the company finds itself in an “enviable cash position” going into the decade

“By 2026, we have the potential to meaningfully expand our rare disease portfolio across connected diseases that share a common underlying pathophysiology,” he said.

Further, Goff stated that by divesting Agios’ oncology portfolio, the company can now concentrate on the rare disease space – an area where he forecasted promising growth.

“Our pipeline has grown dramatically since we announced the divestiture of our oncology business, and focused exclusively on rare diseases just two years ago.”