On May 13, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported the presentation of six posters, including one oral presentation, at the 2025 annual meeting of the Professional Society for Health Economics and Outcomes Research (ISPOR), taking place May 13-16 in Montreal, Canada (Press release, Tempus, MAY 13, 2025, View Source [SID1234652998]). Tempus researchers are showcasing scientific and clinical studies highlighting the impact of AI and real-world data on health economics and outcomes research.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The research we’re presenting at ISPOR 2025 underscores the powerful potential of integrating clinical, molecular, and claims data to unlock actionable insights that drive more personalized and effective cancer care," said Emilie Scherrer, Senior Director and Head of Outcomes Research, at Tempus. "At Tempus, we share ISPOR’s deep focus on empowering providers and health systems with the real-world data they need to optimize treatment strategies and improve outcomes for their patients."

Research highlights include:

Oral Presentation: Oncology Trial Emulation Using Real-World Electronic Health Record Data: Results of the Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation (CARE) Initiative
Date/Time: Thursday, May 15; 10:15 AM – 11:15 AM ET

Overview: The Coalition to Advance Real-World Evidence through Randomized Controlled Trial (RCT) Emulation (CARE) Initiative seeks to advance understanding of when real-world data (RWD) can generate valid treatment effectiveness estimates by emulating RCTs. This study presents findings from three oncology emulations. The KEYNOTE-189 (metastatic NSCLC) and PALOMA-2 (advanced breast cancer) trials were emulated using electronic health record datasets. Trial entry criteria were applied, and treatment status was based on first-line medications. Inverse probability of treatment weighting controlled for baseline confounding, and Kaplan-Meier and Cox models estimated primary outcomes. In the KEYNOTE-189 emulation, the real-world progression-free survival (rwPFS) hazard ratio (HR) in one dataset was similar to the RCT finding, while the other was closer to the null. PALOMA-2’s rwPFS HR was also closer to the null. Real-world overall survival estimates in KEYNOTE-189 also varied across datasets. The researchers conclude that RWD oncology emulation conclusions depend on dataset features, route of administration, and real-world follow-up characteristics.
Poster Presentation: Impact of Adverse Event Definitions on Real-World Detection of Immune-Related Adverse Events
Date/Time: Thursday, May 15; 10:30 AM – 1:30 PM ET

Location: Exhibit Hall 220B-E, Poster #6044
Overview: Researchers investigated the impact of varying definitions on the identification of immune-related adverse events (irAEs) in real-world data (RWD) from non-small cell lung cancer (NSCLC) patients treated with immune checkpoint blockade (ICB). The research utilized Tempus clinico-genomic data linked to Komodo Health’s claims to analyze irAEs within one year of ICB treatment in patients with stage 3C+ NSCLC. Three peer-reviewed irAE definitions—differing in included irAEs, ICD-10 codes, and pre-treatment washout periods—were applied to the cohort of 4,831 patients. The overall prevalence of irAEs varied significantly across definitions: 41.0% (n=1,981) for Study A (9 irAEs), 75.4% (n=3,849) for Study B (10 irAEs), and 5.4% (n=264) for Study C (3 irAEs). This study demonstrates that irAE identification in RWD varies based on the definitions used, which can affect post-market surveillance, clinical practice guidelines, and patient care. The authors emphasize the need for researchers to accurately communicate the definitions used and conduct sensitivity analyses.
Poster Presentation: Comparison of Demographics and Clinical Characteristics using Real World Data from Tempus Multimodal Database and SEER Cancer Registry Across 17 Solid Cancer Cohorts
Date/Time: Thursday, May 15; 4:00 PM – 7:00 PM ET
Location: Exhibit Hall 220B-E, Poster #6019

Overview: This study benchmarked the Tempus multimodal database — a real-world data source with clinical and biomarker data from cancer patients — against the SEER cancer registry. Analyzing data from 63,520 patients with solid tumors diagnosed between 2016 and 2021, researchers compared baseline demographic, clinical, and treatment characteristics against the SEER database. The proportion of cancer types of new cancer cases were similar among the two databases. The Tempus database showed a larger representation of patients from the Midwest, whereas the SEER database had a heavy representation from the West. Tempus patients were younger on average but had more advanced cancer staging. Additionally, the Tempus database had more racial diversity based on self-reported race and more complete treatment data. In conclusion, the Tempus and SEER databases show general comparability in demographics and clinical characteristics, but the Tempus database provides greater treatment data granularity and captures more late-stage disease, attributable to sequencing patterns in clinical care.
Poster Presentation: Assessing the Completeness of Oncology Treatment Data from Administrative Claims: A Benchmarking Study Against Abstracted EHRs Using Patient-Level Linkages
Date/Time: Thursday, May 15; 4:00 PM – 7:00 PM ET
Location: Exhibit Hall 220B-E, Poster #6041

Overview: This study benchmarked oncology treatment data from administrative claims against abstracted electronic health records (EHR) for 6,487 stage 4 lung adenocarcinoma patients diagnosed between 2020 and 2023. Claims data (open and closed) were linked using de-identified patient tokens, with EHR data considered the ground truth. Sensitivities and positive predictive values (PPVs) were calculated for 13 infusional and 3 oral medications. Closed claims showed greater sensitivities (50.0-95.3%) than open claims (14.3-54.8%), with infusions having higher sensitivities than orals. PPVs were high for both infusions (closed: 79.1-98.3%; open: 61.5-99.1%) and orals (closed: 84.5-94.2%; open: 91.8-96.8%). Exact matches for abstracted infusion start dates in claims ranged from 45.5-82.5% for closed claims, while 27.6-65.9% of oral start dates matched within 7 days. The team concludes that while EHR remains the gold standard, individual claims may be sufficient for identifying patients receiving specific treatments, and closed claims may be suitable for constructing comprehensive treatment journeys.
Poster Presentation: Impact of NGS Testing Timing on Treatment Patterns and Clinical Outcomes in Colorectal Cancer
Date/Time: Thursday, May 15; 4:00 PM – 7:00 PM ET
Location: Exhibit Hall 220B-E, Poster #6013

Overview: Researchers evaluated the impact of next-generation sequencing (NGS) timing on real-world overall survival (rwOS) in 2,293 colorectal cancer (CRC) patients using the Tempus real-world multimodal database. The median age at diagnosis was 58.4 years, with most patients having stage 3 (23%) or stage 4 (68%) disease and receiving first-line (1L) chemotherapy without NGS-informed therapy. Time from biopsy to NGS test order was analyzed, revealing a notable delay in ordering NGS tests post-biopsy. A random forest classifier identified the timeline from biopsy to NGS results receipt as key in determining 1L treatment. Notably, stage 4 patients receiving NGS results within approximately two months of biopsy had a significant survival advantage. The study demonstrates that NGS testing may be associated with increased rwOS in CRC, highlighting the importance of timely NGS for guiding treatment decisions and improving outcomes.
Poster Presentation: Integrating Next Generation Sequencing, EHR, and Claims Data to Extend Follow-Up in a Real-World Advanced Lung Adenocarcinoma Biomarker-Treatment Landscape
Date/Time: Friday, May 16; 9:00 AM – 11:30 AM ET
Location: Exhibit Hall 220B-E, Poster #6046

Overview: The research team explored the use of closed claims data to enhance electronic health record (EHR)-derived treatment histories for stage 4 lung adenocarcinoma patients with comprehensive genomic profiling (CGP) and a diagnosis between 2020 and 2023. By linking closed claims data to EHRs, researchers extended abstracted lines of therapy (LOTs), defining new LOTs based on treatment gaps, persistent treatments, and follow-up duration. Integrating claims data increased the number of patients in LOT1, LOT2, and LOT3 and extended LOTs previously lost to follow-up. The integrated LOTs reflected NCCN guidelines, with EGFR inhibitors frequently used in EGFR-mutated patients and immunotherapy and KRAS inhibitors used in KRAS p.G12C patients. The study concludes that using closed claims to extend EHR-abstracted treatment data is valuable for real-world treatment pattern and outcome analyses.

On May 13, 2025 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported that new data from a matching-adjusted indirect comparison study evaluating taletrectinib versus entrectinib in ROS1-positive (ROS1+) non-small cell lung cancer (NSCLC) will be presented in a poster session at ISPOR 2025, the Professional Society for Health Economics and Outcomes Research’s annual conference, taking place May 13-16, 2025 in Montreal, QC, Canada (Press release, Nuvation Bio, MAY 13, 2025, View Source [SID1234652997]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Presentation Overview:

Title: Taletrectinib vs Entrectinib in ROS1-Positive (ROS1+) Non-Small Cell Lung Cancer (NSCLC): A Matching-Adjusted Indirect Comparison (MAIC)
Presenter: Misako Nagasaka, M.D., Ph.D., Associate Professor – Division of Hematology and Oncology, UCI School of Medicine
Date: Wednesday, May 14
Session Time: 10:30 a.m. – 1:30 p.m. ET
Session: Poster Session 1
Presentation Number: CO151
The materials will be made available in the Publications section of Nuvation Bio’s website after the presentation.

About Taletrectinib

Taletrectinib is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor specifically designed for the treatment of patients with advanced ROS1+ NSCLC. Taletrectinib is being evaluated for the treatment of patients with advanced ROS1+ NSCLC in two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, and TRUST-II (NCT04919811), a global study.

Based on results of the TRUST-I and TRUST-II clinical studies, the U.S. FDA has accepted and granted Priority Review to Nuvation Bio’s NDA for taletrectinib for advanced ROS1+ NSCLC (line agnostic, full approval) and assigned a PDUFA goal date of June 23, 2025. The U.S. FDA previously granted taletrectinib Breakthrough Therapy Designation for the treatment of patients with locally advanced or metastatic ROS1+ NSCLC who either have or have not previously been treated with ROS1 TKIs, and Orphan Drug Designation for the treatment of patients with ROS1+ NSCLC and other NSCLC indications. In January 2025, China’s NMPA approved taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1+ NSCLC.

On May 13, 2025 Atossa Therapeutics, Inc. (Nasdaq: ATOS) (Atossa or the Company), a clinical-stage biopharmaceutical company developing innovative medicines for breast cancer, reported its financial results for the first quarter ended March 31, 2025 and provided an update on recent company developments (Press release, Atossa Therapeutics, MAY 13, 2025, View Source [SID1234652996]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

First Quarter 2025 Highlights:

Announced Strategic Decision to Pursue Metastatic Breast Cancer Indication: Atossa announced plans to target metastatic breast cancer as its lead program for (Z)-endoxifen. The decision reflects its commitment to addressing the persistent unmet medical need in metastatic breast cancer and the potential for a more streamlined regulatory pathway to deliver (Z)-endoxifen to these patients. Current treatment options for metastatic breast cancer often provide limited durability of response and substantial side effects. In previous clinical trials, (Z)-endoxifen has been shown to be well-tolerated as a selective estrogen receptor modulator (SERM), which Atossa believes supports its potential to fill this critical gap in treatment.
Significantly Strengthened (Z)-endoxifen Patent Portfolio with Three New U.S. Patents: Atossa continued to bolster the intellectual property portfolio of (Z)-endoxifen with the grant of three new U.S. patents covering 31 claims directed at: sustained release compositions of (Z)-endoxifen (U.S. Patent No. 12,201,591); enteric oral formulations of (Z)-endoxifen and salts thereof as well as their use in treating hormone-dependent breast and reproductive tract disorders (U.S. Patent No. 12,275,684); and 58 claims covering (Z)-endoxifen formulations, including various levels of purity and stability as well as methods of using those formulations (U.S. Patent No. 12,281,056). Atossa’s robust patent portfolio now encompasses more than 200 patent claims related to (Z)-endoxifen formulations and their clinical applications.
"Our focus remains firmly on advancing (Z)-endoxifen as a next-generation therapy for breast cancer patients across the full spectrum of care—including a strategic emphasis on metastatic breast cancer, where therapeutic innovation is urgently needed," said Steven Quay, M.D., Ph.D., President and Chief Executive Officer of Atossa. "Across multiple clinical trials involving hundreds of patients, (Z)-endoxifen has consistently demonstrated strong tolerability and therapeutic versatility, which we believe shows its potential as a therapy for breast cancer from early-stage disease to more advanced stages. We are committed to unlocking the full potential of (Z)-endoxifen for patients while delivering value to our shareholders. A cornerstone of this strategy is the robust intellectual property portfolio we are building in an effort to protect our programs globally. As we look ahead to the remainder of 2025 and beyond, we are energized by the many opportunities to position (Z)-endoxifen as a potentially safer, more effective endocrine therapy for breast cancer patients worldwide."

Comparison of Three Months Ended March 31, 2025 and 2024

Revenue and Cost of Revenue. For the three months ended March 31, 2025 and 2024, we had no source of revenue and no associated cost of revenue.

Operating Expenses. Total operating expenses were $7.4 million for the three months ended March 31, 2025, which was an increase of $0.4 million, from the three months ended March 31, 2024 of $7.0 million. Factors contributing to the increased operating expenses in the three months ended March 31, 2025 are explained below.

Research & Development (R&D) Expenses. The following table provides a breakdown of major categories within R&D expenses for the three months ended March 31, 2025 and 2024, together with the dollar change and percentage change in those categories (dollars in thousands):

For the Three Months Ended March 31,

2025

2024

Increase (Decrease)

% Increase (Decrease)

Research and Development Expenses

Clinical and pre-clinical trials

$

2,747

$

2,884

$

(137)

(5) %

Compensation

880

626

254

41 %

Professional fees and other

530

238

292

123 %

Research and Development Expenses Total

$

4,157

$

3,748

$

409

11 %

Clinical and pre-clinical trial expenses decreased $0.1 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024, due to a slight decrease in spend related to our (Z)-endoxifen trials, including drug development costs.
The increase in R&D compensation expenses of $0.3 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024 was due to an increase in headcount.
The increase in R&D professional fees and other of $0.3 million was due to an increase in spending on regulatory consulting services.
General and Administrative (G&A) Expenses. The following table provides a breakdown of major categories within G&A expenses for the three months ended March 31, 2025 and 2024, together with the dollar change and percentage change in those categories (dollars in thousands):

For the Three Months Ended March 31,

2025

2024

Increase (Decrease)

% Increase (Decrease)

General and Administrative Expenses

Compensation

$

1,462

$

1,325

$

137

10 %

Professional fees and other

1,614

1,680

(66)

(4) %

Insurance

181

227

(46)

(20) %

General and Administrative Expenses Total

$

3,257

$

3,232

$

25

1 %

The increase in G&A compensation expenses of $0.1 million for the three months ended March 31, 2025, compared to the three months ended March 31, 2024 was due to an increase in headcount quarter over quarter.
Interest Income. Interest income was $0.7 million for the three months ended March 31, 2025, a decrease of $0.4 million from interest income of $1.1 million for the three months ended March 31, 2024. The decrease was due to a decrease in the balance in our money market account.

About (Z)-Endoxifen
(Z)-endoxifen is a highly potent SERM with demonstrated ability to inhibit—and potentially degrade—estrogen receptors. It has shown activity even in tumors that have developed resistance to other endocrine therapies. Beyond its anti-estrogenic properties, (Z)-endoxifen also targets protein kinase C beta 1 (PKCβ1), an oncogenic signaling protein, at clinically achievable blood levels. Importantly, (Z)-endoxifen seems to deliver comparable or superior bone-protective effects relative to tamoxifen, while exhibiting minimal or no endometrial proliferative activity—which we believe addresses key limitations of current standard-of-care therapies. Atossa is developing a proprietary oral formulation of (Z)-endoxifen that is enteric-coated to bypass stomach acid, which would otherwise convert the active (Z)-isomer to its inactive (E)-form. We believe this innovation allows for optimal bioavailability and therapeutic integrity. Clinical studies have shown Atossa’s (Z)-endoxifen to be well tolerated in both healthy women and those with breast cancer. Atossa is prioritizing the development of (Z)-endoxifen for the treatment of metastatic breast cancer, where novel therapeutic options are urgently needed. The compound is currently being evaluated in three Phase 2 trials: one in women with ductal carcinoma in situ (DCIS) and two in women with estrogen receptor positive (ER+) / human epidermal growth factor receptor 2 negative (HER2-) breast cancer, including the EVANGELINE study and an I-SPY study. Atossa’s (Z)-endoxifen program is supported by a growing global intellectual property portfolio, including three recently issued U.S. patents and numerous pending applications worldwide.

On May 13, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported the presentation of preclinical data demonstrating the potential and versatility of its proprietary RSwitch technology to enable the fine-tuning of transgene levels in gene therapy applications with orally administered small molecules specifically built to meet the needs of each indication with a range of pharmacokinetic properties (Press release, Rgenta Therapeutics, MAY 13, 2025, View Source [SID1234652995]). The data will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, which will be held from May 13 -17th, 2025, in New Orleans, LA.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The data presented at this year’s ASGCT (Free ASGCT Whitepaper) meeting provide evidence to support the advantages of our RSwitch technology which is designed to deliver precise temporal control of therapeutic transgene expression over a wide dynamic range and is applicable to a variety of gene and cell therapy applications," said Travis Wager, Ph.D., co-founder and chief scientific officer. "While there are existing methods to control transgene expression in gene therapy, they typically rely on engineered transcription factors and repurposed pharmacology, are not specific to the switching system, and consequently do not provide particularly tight control of the transgene’s expression. Rgenta has developed a platform for imparting gene therapy vectors with selective, dose-dependent regulation by orally bioavailable small molecules, RDrugs. Aided by massively parallel sequence library screening and deep learning-driven design, we have built RDrugs to meet the needs of each indication with a range of pharmacokinetic properties enabling direct control, via RSwitch, of specific pre-mRNA splicing events needed for the regulated production of the therapeutic payload."

In a poster titled RSwitch Enabled Gene Therapy to Fine Tune Frataxin Expression for the Treatment of Friedreich’s Ataxia, Rgenta scientists presented data demonstrating the ability of RSwitches to enable dose-dependent control with up to 4000-fold induction of gene expression by a specific RDrug. RSwitches were able to control gene expression in both high and low strength promotors. Data were also presented from an RSwitch-regulated AAV gene therapy designed to express frataxin (FXN) for the potential treatment of Friedreich’s Ataxia (FA). FA is a progressive neurodegenerative disease caused by a genetic deficiency of FXN, which is a small, nuclear-encoded mitochondrial protein believed to act as an iron chaperone or iron storage protein. While constitutive replacement of FXN by AAV vectors has shown promise in animal models, multiple studies have demonstrated the cardiotoxic effects of unregulated FXN overexpression making this gene therapy a good candidate for regulation of gene expression by a system such as RSwitch. The data demonstrate that an RSwitch-regulated FA expressing AAV-gene therapy achieves dose dependent expression of FXN in the heart with RDrug and human endogenous FXN levels in mice at low RDrug levels. A second test system demonstrated the ability of Rgenta’s RSwitch and corresponding brain penetrant RDrug to regulate expression of a transgene in the brain.

"Our proprietary RSwitch technology provides an example of the power of Rgenta’s RNA-targeted small molecule platform and represents a potentially game-changing tool for the development of regulatable gene and cell therapies that could improve the safety of these medicines," Simon Xi, Ph.D., cofounder and chief executive officer of Rgenta. "Rgenta’s internal pipeline is focused on the application of our platform to inhibit disease driving therapeutic targets, such as MYB for the treatment of solid tumors, including adenoid cystic carcinoma and colorectal cancer, and hematological cancers such as acute myeloid leukemia, and PMS1 for the potential treatment of repeat expansion diseases such as Huntington’s disease. We see the value of our RSwitch technology as a potential partnering opportunity with companies developing gene and cell therapies to improve the clinical success of these gene replacement therapies for difficult to treat diseases such as FA."

About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug (RDrug) that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.

On May 13, 2025 Altimmune, Inc. (Nasdaq: ALT), a late clinical-stage biopharmaceutical company developing novel peptide-based therapeutics for liver and cardiometabolic diseases, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Altimmune, MAY 13, 2025, View Source [SID1234652993]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The first quarter of 2025 was productive for Altimmune as the Company approaches a number of important milestones," said Vipin K. Garg, Ph.D., President and Chief Executive Officer of Altimmune. "The readout of our IMPACT Phase 2b trial of pemvidutide in MASH is on-track for the second quarter of 2025. We believe that achieving statistical significance on MASH resolution and fibrosis improvement at only 24 weeks, coupled with clinically meaningful weight loss, would position pemvidutide as the best-in-class therapeutic candidate for the treatment of MASH."

Dr. Garg continued, "At our recent R&D Day event we unveiled two additional indications for pemvidutide in AUD and ALD, and our intent to initiate Phase 2 clinical trials in these indications in Q2 and Q3, respectively. AUD and ALD are conditions of significant unmet medical need with limited treatment options. We remain committed to developing pemvidutide for treatment of liver and cardiometabolic diseases that leverage its differentiated clinical profile."

Recent Highlights and Anticipated Milestones

MASH

Top-line data from the IMPACT Phase 2b trial of pemvidutide in biopsy-confirmed F2/F3 MASH expected in Q2 2025
Top-line data is expected to include rates of MASH resolution and fibrosis improvement, weight loss, non-invasive tests, and data on safety and tolerability.
A total of 212 participants were randomized, exceeding the 190 originally planned.
If successful, pemvidutide would be the first investigational therapy in MASH to achieve statistical significance in both MASH resolution and fibrosis improvement, as well as demonstrate meaningful weight loss, after only 24 weeks of treatment.
Altimmune presented new data at the EASL International Liver Congress 2025, including a follow-on analysis of the Company’s Phase 1b trial in MASLD using the MASH Resolution Index (MASHResInd).
Developed by Dr. Rohit Loomba, Professor of Medicine and Chief of Gastroenterology and Hepatology at the University of California San Diego, MASHResInd is a non-invasive measure that has been highly predictive of MASH resolution.
The analyses indicated that after 24 weeks of treatment, the proportion of participants receiving pemvidutide achieving MASHResInd responses exceeded 90%. These findings indicate that high rates of MASH resolution may be observed in the upcoming IMPACT Phase 2b MASH trial readout.
Additional Indications for Pemvidutide: AUD and ALD

During Altimmune’s R&D Day, the Company announced the development of pemvidutide in two additional indications: AUD and ALD
AUD and ALD are characterized by large patient populations with significant unmet medical need and very few treatment options.
Investigational New Drug (IND) applications were cleared by the FDA in the first quarter of 2025. The Phase 2 trials in AUD and ALD are expected to initiate in the second and third quarters of 2025, respectively.
Preclinical data and data from other clinical trials support the potential of pemvidutide to reduce alcohol consumption, improve liver health, and provide the added benefit of meaningful weight loss.
Corporate Update

The Company entered into a $100 million credit facility with Hercules Capital, with an initial $15 million tranche funded at closing. An additional $25 million is available in 2025 at Altimmune’s option, subject to the achievement of certain clinical and financial milestones. The remaining $60 million is available beginning in 2026, with $15 million subject to the achievement of certain clinical and financial milestones and up to $45 million available subject to approval of Hercules. The credit facility significantly increases Altimmune’s financial strength and flexibility on attractive terms.
Financial Results for the Three Months Ended March 31, 2025

Altimmune reported cash, cash equivalents and short-term investments totaling $150 million on March 31, 2025.
Research and development expenses were $15.8 million for the three months ended March 31, 2025, compared to $21.5 million in the same period in 2024, the decrease resulting from timing of clinical trial costs. The expenses for the quarter ended March 31, 2025, included $9.2 million in direct costs related to pemvidutide development activities.
General and administrative expenses were $6.0 million for the three months ended March 31, 2025, compared to $5.3 million in the same period in 2024. The increase was primarily due to a $0.5 million increase in stock compensation and other labor-related expenses.
Interest income was $1.5 million for the three months ended March 31, 2025, compared to $2.4 million for the same period in 2024.
Net loss for the three months ended March 31, 2025, was $19.6 million, or $0.26 net loss per share, compared to a net loss of $24.4 million, or $0.34 net loss per share, in the same period in 2024.
Conference Call Information:

Date: May 13, 2025
Time: 8:30 a.m. Eastern Time
Webcast: To listen, the conference call will be webcast live on Altimmune’s Investor Relations website at View Source
Dial-in: To participate or dial-in, register here to receive the dial-in numbers and unique PIN to access the call.

Following the conclusion of the call, the webcast will be available for replay on the Investor Relations (IR) page of the Company’s website at www.altimmune.com. The Company has used, and intends to continue to use, the IR portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

About Pemvidutide

Pemvidutide is a novel, investigational, peptide-based 1:1 GLP-1/glucagon dual receptor agonist in development for the treatment of MASH, obesity, Alcohol Use Disorder (AUD) and Alcohol Liver Disease (ALD). Activation of the GLP-1 and glucagon receptors is believed to mimic the complementary effects of diet and exercise on weight loss, with GLP-1 suppressing appetite and glucagon increasing energy expenditure. Glucagon is also recognized as having direct effects on hepatic fat metabolism, which is believed to lead to rapid reductions in levels of liver fat and serum lipids. In clinical trials to date, once-weekly pemvidutide has demonstrated compelling weight loss with class-leading lean mass preservation, and robust reductions in triglycerides, LDL cholesterol, liver fat content and blood pressure. The U.S. FDA has granted Fast Track designation to pemvidutide for the treatment of MASH. Pemvidutide completed the MOMENTUM Phase 2 obesity trial in 2024 and is being studied in the ongoing IMPACT Phase 2b MASH trial with top line results expected in late June 2025. IND applications in AUD and ALD have received FDA clearance with Phase 2 trials scheduled to commence in Q2 and Q3 2025, respectively.