On May 5, 2025 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, AnaptysBio, MAY 5, 2025, View Source [SID1234652496]).

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"Our lead program, rosnilimab, delivered impressive three-month Phase 2b efficacy, safety and tolerability data in rheumatoid arthritis (RA), with data through six months surpassing those of competitor all-active, head-to-head trials. We will report updated clinical and translational RA data in the first week of June, as well as initial Phase 2 ulcerative colitis (UC) data in Q4 2025, further defining rosnilimab’s game-changing potential," said Daniel Faga, president and chief executive officer of Anaptys. "With ANB033 and ANB101 progressing through Phase 1 trials, our autoimmune portfolio promises multiple catalysts over the next couple of years. We remain well-capitalized as we execute on our broad development plan for all three programs, while concurrently executing our $75 million stock repurchase program which are both further supported by substantial royalties and milestone payments anticipated from our GSK financial collaboration."

PORTFOLIO UPDATES

Rosnilimab (PD-1 depleter and agonist)

Announced in February that subcutaneously administered rosnilimab, including two once-monthly doses, achieved positive results in 424-patient Phase 2b RA trial and highest-ever reported clinical disease activity index (CDAI) low disease activity (LDA) response over 6 months
Full press release can be found here
Anaptys to host an investor call featuring Anaptys management and key opinion leaders in the first week of June to present rosnilimab’s updated Phase 2b clinical and translational data

Enrollment ongoing for global Phase 2 trial in moderate-to-severe UC
132-patient trial assessing two dose levels of subcutaneously administered rosnilimab vs. placebo (randomized 1:1:1)
Primary statistical analysis at Week 12 on well-established endpoints, including the primary endpoint of change from baseline in modified Mayo score (mMS) and supportive secondary endpoints of clinical response on mMS, clinical remission on mMS and endoscopic remission
All patients in all three study arms treat-through to Week 24 and remain blinded to treatment arm. Placebo-treated patients who achieved clinical response on partial modified Mayo score (pmMS) at Week 12 remain on placebo, while placebo-treated patients who are non-responders are crossed over to the high-dose rosnilimab treatment arm
Patients who are in clinical response on pmMS at Week 24 are eligible for an additional 26-week (50 weeks of total treatment) blinded treatment extension period (TEP)
Initial Phase 2 data anticipated in Q4 2025
ANB033 (CD122 antagonist)

Enrollment ongoing for Phase 1a trial in healthy volunteers
Phase 1b indication to be disclosed at H2 2025 R&D event
ANB101 (BDCA2 modulator)

Enrollment initiated for Phase 1a trial in healthy volunteers
COLLABORATION UPDATES

GSK Immuno-Oncology Financial Collaboration

GSK announced strong commercial performance for Jemperli ($220 million in Q1 2025 sales) with >15% quarter-over-quarter growth
GSK announced the EMA approval of Jemperli plus chemotherapy for all adult patients with primary advanced or recurrent endometrial cancer in January 2025

Anticipate receipt of a $75 million commercial sales milestone payment from GSK in either 2025 or 2026 once Jemperli achieves $1 billion in worldwide net sales in a calendar year

GSK anticipates top-line data in mid-2025 from COSTAR Lung Phase 3 trial in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and platinum-based chemotherapy comparing docetaxel alone to cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel and to dostarlimab plus docetaxel

Recent data published in The New England Journal of Medicine (NEJM) and presented at American Association for Cancer Research (AACR) (Free AACR Whitepaper) demonstrated neoadjuvant treatment with dostarlimab resulted in organ preservation in a high proportion of patients (80% of 103 patients), including 100% (rectal; n=49), 100% (bladder; n=6), and 82% (colon; n=22) complete responses in April 2025
GSK anticipates top-line data in 2026 from AZUR-1 pivotal Phase 2 trial of dostarlimab monotherapy in patients with untreated stage II/III dMMR/MSI-H locally advanced rectal cancer
Jemperli received U.S. FDA Breakthrough Therapy Designation for this indication in December 2024
Vanda Imsidolimab Collaboration

Announced an exclusive $15 million global out-license agreement with Vanda Pharmaceuticals to develop and commercialize imsidolimab (IL-36R antagonist), with Anaptys eligible to receive up to $35 million for future regulatory approvals and sales milestones, in addition to 10% royalty on global net sales
FDA BLA submission for generalized pustular psoriasis (GPP) expected in 2025
Full press release can be found here
FINANCIAL UPDATES

Stock Repurchase Program and Cash Runway

Authorized a Stock Repurchase Program in March 2025 of $75.0 million of the Company’s outstanding common stock

Cash and investments of $383.0 million as of March 31, 2025, and reiterating cash runway through year-end 2027
First Quarter 2025 Financial Results

Cash, cash equivalents and investments totaled $383.0 million as of March 31, 2025, compared to $420.8 million as of December 31, 2024, for a decrease of $37.8 million due primarily to operating activities and $4.4 million in shares repurchased offset by $15.0 million received from Vanda Pharmaceuticals for the license of imsidolimab.

Collaboration revenue was $27.8 million for the three months ended March 31, 2025, compared to $7.2 million for the three months ended March 31, 2024. The increase is due to a $11.0 million increase in royalties recognized for sales of Jemperli and $9.6 million in revenue recognized for the Vanda license agreement.

Research and development expenses were $41.2 million for the three months ended March 31, 2025, compared to $37.0 million for the three months ended March 31, 2024. The increase was due primarily to development costs relating to the Phase 2 trials in RA and UC for rosnilimab, and the Phase 1 trials for ANB033 and ANB101, offset by a decrease in development costs for imsidolimab and ANB032. The R&D non-cash, stock-based compensation expense was $4.4 for the three months ended March 31, 2025 as compared to $3.5 million in the same period in 2024.

General and administrative expenses were $14.1 million for the three months ended March 31, 2025, compared to $12.3 million for the three months ended March 31, 2024. The increase was due primarily to transaction costs associated with the Vanda Pharmaceuticals license agreement. The G&A non-cash, stock-based compensation expense was $4.8 million for the three months ended March 31, 2025 as compared to $6.7 million in the same period in 2024.

Net loss was $39.3 million for the three months ended March 31, 2025, or a net loss per share of $1.28, compared to a net loss of $43.9 million for the three months ended March 31, 2024, or a net loss per share of $1.64.

On May 4, 2025 Nutshell Therapeutics ( Shanghai ) Co., LTD. reported to have received IND clearance from the FDA to initiate Phase 1 clinical trial in the United States for its NTS071, a novel small molecule allosteric reactivator targeting p53 Y220C mutation (Press release, Nutshell Therapeutics, MAY 4, 2025, View Source [SID1234652483]).

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NTS071 is an oral small molecule allosteric reactivator targeting p53 Y220C with a novel scaffold. It selectively binds to the p53 Y220C mutant protein, improving its thermal stability, thereby enhancing the mutant protein’s ability to bind with DNA and restoring its transcriptional activity as well as tumor-suppressing function.

NTS071 was discovered by leveraging Nutshell’s proprietary AI driven allosteric small molecule drug discovery platform ALLOSTAR. NTS071 demonstrated potential Best-in-Class preclinical properties for its target. NTS071 achieved a picomolar-level biochemical activity which is 20 folds more potent than the competitive compound PC14586. Additionally, NTS071 shows better stability in both liver microsomes and hepatocytes across different species and exhibits lower in vivo clearance rates and higher oral exposure s in preclinical PK studies across all tested species compared to PC14586. NTS071 has relatively lower plasma protein binding and higher free fraction than PC14586, which is beneficial for in vivo efficacy. NTS071 also addresses the CYP3A4 inhibition issue of PC14586, presenting lower risks for potential drug – drug interactions. NTS071 further displays a large safety window by exhibiting an overall good safety profile in non-clinical toxicology studies.

NTS071 exhibited dose-dependent in vivo anti-tumor activity in multiple CDX and PDX models harboring p53 Y220C mutation, spanning a number of different cancer types, including ovarian cancer, lung cancer, gastric cancer, breast cancer, head and neck cancer, esophageal cancer, pancreatic cancer, and bladder cancer, etc. Therefore, NTS071 has the potential to be a tumor-agnostic therapy for patients carrying p53 Y220C mutation. Compared to PC14586, NTS071 has shown significantly lower effective doses or better efficacy at the same dose level in all comparative preclinical in vivo studies, implying that NTS071 may overcome the limitation of its competitor that has a higher dose requirement, thereby potentially achieving better therapeutic effects. NTS071 is anticipated to initiate Phase 1 clinical trial in second half of 2025 and expected to benefit patients with solid tumors harboring this mutation.

p53 Y220C mutation is widespread in various solid tumors. Studies show that there are 125,000 to 150,000 new cases worldwide annually, representing a significant market potential. Enriched with years of accumulated experience with its proprietary computation-based allosteric drug development technology and breakthrough innovation capabilities at Nutshell Therapeutics, NTS071 has the potential to stand out among peer products and become the most competitive drug molecule for this target.

The NTS071 Poster presented at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) 2024 conference can be accessed here: View Source

On May 3, 2025 TAE Life Sciences (TLS) reported the signing of a Letter of Intent to collaborate with The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) in the development and evaluation of novel boron-based drug compounds—an essential component of Boron Neutron Capture Therapy (BNCT) (Press release, TAE Life Sciences, MAY 3, 2025, View Source [SID1234652484]).

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This partnership marks the first-of-its-kind U.S.-based academic-industry alliance dedicated to accelerating BNCT drug innovation and translational research. By optimizing boron delivery agents, TLS and OSUCCC aim to unlock BNCT’s full potential as a powerful, highly selective, and relatively non-toxic therapy and positioning it as a new modality in the next era of precision oncology.

"We are entering a pivotal moment in cancer therapy," said Dr. Arnab Chakravarti, Chair and Professor of Radiation Oncology, Klotz Family Chair of Cancer Research and Director of the Brain Tumor Program at Ohio State. "BNCT offers an unprecedented opportunity to treat malignancies that are otherwise unresponsive to conventional therapies, while sparing healthy tissue. Our laboratory is uniquely poised to lead this next wave of boron drug innovation, and we are excited to collaborate with TAE Life Sciences in accelerating this effort on a global scale."

The initial focus of the collaboration will be the preclinical evaluation of TLS’s proprietary boron-10 drugs in cellular and animal models, leveraging Ohio State’s specialized neutron source optimized for BNCT research. In parallel, the teams will pursue joint development of next-generation boron drugs for BNCT and adjacent applications—unlocking new possibilities in precision cancer treatment.

"This partnership marks a major leap forward for the future of BNCT," said Robert Hill, CEO of TAE Life Sciences. "We are deeply honored to collaborate with Dr. Chakravarti and his world-class team of research and clinical experts. Their pioneering track record of leadership and innovation will be instrumental not only in advancing the science behind novel boron drug compounds but also in paving the way toward potential regulatory approval. Together, we’re creating a dynamic force for innovation that has the potential to redefine cancer care."

Hill notes that this partnership establishes a key pillar for accelerator-based BNCT and significantly accelerate the path to breakthrough cancer therapies, positioning the United States to have a leading position in the next wave of innovation in precision oncology. Hill emphasized that the growing body of international clinical evidence—particularly from Japan, Taiwan, and Europe validates BNCT’s ability to deliver durable responses in patients with otherwise untreatable cancers. This collaboration will help position the United States in having a leading position in advanced drug development and establishing the next wave in precision medicine.

On May 2, 2025 Amneal Pharmaceuticals, Inc. (Nasdaq: AMRX) ("Amneal" or the "Company") reported its results for the first quarter ended March 31, 2025 (Press release, Amneal Pharmaceuticals, MAY 2, 2025, View Source [SID1234654251]).

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"Amneal delivered another strong quarter to start 2025, with broad-based growth across all three segments driven by our team’s outstanding execution. We are very pleased with the commercial uptake of CREXONT for Parkinson’s Disease and the momentum of our recently launched injectable products, which are delivering tremendous value to patients, caregivers and customers. As a leading U.S.-based biopharmaceutical company, Amneal is proud to provide millions of Americans with access to affordable and innovative treatments—and we believe we are just getting started. With our diverse portfolio, expansive footprint in the U.S. and globally, and a resilient management team, we are confident in our ability to deliver sustainable growth and value creation for our stakeholders in 2025 and beyond as we embark on our exciting next chapter of growth and success," said Chirag and Chintu Patel, Co-Chief Executive Officers.

First Quarter 2025 Results

Net revenue in the first quarter of 2025 was $695 million, an increase of 5% compared to $659 million in the first quarter of 2024. Affordable Medicines net revenue increased 6% driven by strong performance of our complex product portfolio and new product launches. Specialty net revenue increased 3% driven by key branded products, including CREXONT and UNITHROID. AvKARE net revenue increased 6% driven by growth in the government label sales channel.

Net income attributable to Amneal Pharmaceuticals, Inc. was $12 million in the first quarter of 2025 compared to a net loss of $92 million in the first quarter of 2024, reflecting higher revenue and gross profit, and a legal settlement charge of $94 million in the first quarter of 2024.

Adjusted EBITDA in the first quarter of 2025 was $170 million, an increase of 12% compared to the first quarter of 2024, reflective of strong revenue performance, higher gross margin and operating expense leverage.

Diluted income per share in the first quarter of 2025 was $0.04 compared to diluted loss per share of $0.30 for the first quarter of 2024, due to higher operating income and lower interest expense. Adjusted diluted earnings per share in the first quarter of 2025 was $0.21, an increase of 50%, compared to $0.14 for the first quarter of 2024.

The Company presents GAAP and adjusted (non-GAAP) quarterly results. Please refer to the "Non-GAAP Financial Measures" section and the accompanying GAAP to non-GAAP reconciliation tables for more information.

Affirming Full Year 2025 Financial Guidance

The Company is affirming its previously provided full year 2025 guidance.

On May 2, 2025 HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics that reprogram disease-driving cell states for rare, common, chronic and age-related diseases, reported the presentation of data at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in New Orleans from May 13-17, 2025 (Press release, Haya Therapeutics, MAY 2, 2025, View Source [SID1234652482]).

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The company will showcase its latest advances in an invited presentation focused on HAYA’s lead program, HTX-001, an antisense oligonucleotide designed to target the long non-coding RNA (lncRNA) Wisper, the master regulator of fibrosis in the heart, currently in Investigational New Drug-enabling studies and in development for the treatment of non-obstructive hypertrophic cardiomyopathy (nHCM). Additionally, HAYA will have a poster presentation on its innovative multimodal omics approach for profiling and designing antisense oligonucleotide-based therapeutics that enables a precise and potent silencing of novel lncRNA targets.

"Unlike traditional approaches that block single genes or proteins, we have built a platform to reprogram the fundamental biology that underlies disease," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "We are identifying and drugging novel regulatory genome-derived drivers of disease pathogenesis to reprogram sick cells into healthy ones."

"Over the past years, we’ve made significant progress on our lead program, HTX-001, targeting myocardial fibrosis and we anticipate initiating clinical trials in the near future. This forward momentum brings us closer to our ultimate mission: bringing disease-modifying medicines to patients," said Daniel Blessing, Ph.D., Co-founder and CTO of HAYA Therapeutics.

Poster Presentation:

Title: Harnessing Functional Genomics to Advance the Discovery and Development of Oligonucleotide-Based Therapies Targeting the Regulatory Genome
Presenter: Rudi Micheletti, HAYA’s Senior Director of Biology and Functional Genomics
Presentation Date/Time: Tuesday, May 13, 2025, 6:00 PM – 7:30 PM CDT
Location: Poster Hall I2
Abstract Number: 660

Scientific Symposium:

Title: A Wisper from the Heart: Targeting a Cardiac Myofibroblasts-specific LncRNA to Treat Myocardial Fibrosis
Presenter: Daniel Blessing, HAYA’s CTO and Co-founder
Session Title: Targeting Myocardium: To The Heart Of The Matter (Organized by the Cardiovascular CGT Committee)
Presentation Date/Time: Wednesday, May 14, 2025, 3:45 PM – 5:30 PM CDT
Location: Room 388-390

HAYA’s RNA-guided therapeutic platform represents a new approach to treating disease by targeting the root causes at the cellular level. At its core is HAYA’s Regulatory Genome Atlas, which maps the connection between noncoding regulatory genome elements, or lncRNAs, and disease-driving cell states by integrating multimodal functional genomics with a stack of proprietary computational tools and machine learning methodologies. This enables the development of genetic medicines that reprogram diseased cells back to healthy ones.