On May 13, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported business updates and announced financial results for the first quarter ended March 31, 2025 (Press release, Verastem, MAY 13, 2025, View Source [SID1234652992]).

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"In the first quarter of 2025, we continued to make progress with our pipeline programs by exercising our option early to license VS-7375 from our partner GenFleet Therapeutics, completing enrollment in the initial cohorts in our RAMP 205 clinical trial in first-line metastatic pancreatic cancer, and continuing enrollment in the triplet combination in our RAMP 203 clinical trial in advanced KRAS G12C mutant non-small cell lung cancer," said Dan Paterson, president and chief executive officer of Verastem Oncology. "With a strengthened financial position, we are looking forward to a transformational second quarter with the FDA approval and launch of AVMAPKI FAKZYNJA CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, our plans to initiate a Phase 1/2a study in the U.S. for VS-7375, our potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, in mid-2025, and share updated data for both VS-7375 and RAMP 205 at ASCO (Free ASCO Whitepaper)."

First Quarter 2025 and Recent Updates

Avutometinib and Defactinib Combination in Low-Grade Serous Ovarian Cancer (LGSOC)

Announced the U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025, in advance of PDUFA action date of June 30, 2025.
Initiation of the commercial execution of the AVMAPKI FAKZYNJA CO-PACK launch in the U.S.
AVMAPKI FAKZYNJA CO-PACK is now available through a specialty distribution network in the U.S.
A support program for patients prescribed AVMAPKI FAKZYNJA CO-PACK, called Verastem Cares, is now available.
Submitted request for NCCN guideline inclusion.
Shared multiple oral and poster presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 on April 25-30, highlighting the exploration of the mechanisms by which the Company’s FAK inhibitor increases the anti-tumor efficacy of avutometinib.
Multiple abstracts were selected for oral and poster presentations at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting on Women’s Cancer on March 14-17 in Seattle. These presentations included an oral presentation of additional analyses from the Phase 2 RAMP 201 trial of avutometinib and defactinib combination with recurrent LGSOC and an oral presentation of interim results from a Phase 2 Investigator-Sponsored Trial evaluating avutometinib plus defactinib in advanced or recurrent gynecologic mesonephric cancer.
Key Milestones Expected for 2025:

Primary analysis from both the FRAME and RAMP 201 clinical trials anticipated to be published in H1 2025.
Complete enrollment for the international Phase 3 confirmatory RAMP 301 clinical trial for patients with recurrent LGSOC regardless of KRAS mutation status by the end of 2025.
Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with JGOG in H2 2025.
Continue to advance the regulatory pathway in Japan and Europe.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic Pancreatic Cancer

Completed enrollment of 60 patients in the dose-level evaluation phase of RAMP 205 study in Q1; follow-up continues.
In March 2025, announced several updates to the trial, including the addition of a new dose level "0" to evaluate the doses of avutometinib and defactinib used in LGSOC and expanding all dose levels to 12 patients each, including six additional patients to dose level "1", where 5/6 patients reported an objective response (83% cORR) at the ASCO (Free ASCO Whitepaper) 2024 annual meeting.
Key Milestones Expected for 2025:

Plan to report additional data when ASCO (Free ASCO Whitepaper) abstracts are live on May 22, 2025.
Select the recommended Phase 2 Dose (RP2D) for trial expansion in H1 2025.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in Non-Small Cell Lung Cancer (NSCLC)

Completed enrollment in the KRAS G12C inhibitor prior-treated Stage 1 Part B doublet cohort in Q1 2025.
Completed enrollment in the planned dose level evaluation cohorts for the triplet combination in Q1 2025.
Key Milestones Expected for 2025:

Present an interim update of both doublet and triplet data at a medical meeting in H2 2025.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

Verastem announced in April 2025 that the FDA had cleared the Company’s Investigational New Drug (IND) application for VS-7375, enabling a Phase 1/2a trial in advanced solid tumors in the U.S.
Shared a presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2025, highlighting that VS-7375 was found to be more potent than other KRAS G12D inhibitors in preclinical models.
GenFleet announced on Feb. 28, 2025, that it had dosed the first patient in the Phase 2 portion of the trial in China.
Verastem announced on January 14, 2025, that it had exercised its option early to license GFH375 (VS-7375) from partner GenFleet Therapeutics. In addition, the Company announced preliminary clinical data from the Phase 1 dose-escalation study conducted by GenFleet in China. In the study, VS-7375 demonstrated oral bioavailability, with no DLTs across six dose levels, and partial responses were achieved among multiple patients with both pancreatic and lung cancers.
Key Milestones Expected for 2025:

Initiate a Phase 1/2a trial in the U.S. by mid-2025.
GenFleet to share clinical data from the Phase 1 study of VS-7375 in an oral presentation at ASCO (Free ASCO Whitepaper) on Monday, June 2, 2025.
Upcoming Presentations

ASCO Annual Meeting
The meeting will be held from May 30 to June 3, 2025, in Chicago, IL, and abstracts are under embargo until May 22, 2025, at 5:00 pm EDT.

Title: A First-in-Human Phase I/II Study of GFH375, a Highly Selective and Potent Oral KRAS G12D Inhibitor in Patients with KRAS G12D Mutant Advanced Solid Tumors

Abstract Number: 3013
Session: Rapid Oral Abstract Sessions: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Date/Time: Monday, June 2, 2025 from 8:00 am to 9:30 am CDT
Title: Avutometinib/defactinib and gemcitabine/nab-paclitaxel combination in first-line metastatic pancreatic ductal adenocarcinoma: Updated safety and efficacy of a phase 1b/2 study (RAMP 205)

Abstract Number: e16043
Accepted for inclusion in the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting Proceedings, Journal of Clinical Oncology supplement. The abstract is under embargo until May 22, and the Company will be reporting additional data then.
ESMO Gynaecological Cancers Congress 2025
The meeting will be held from June 19 to 21, 2025, in Vienna, Austria, and the abstract is under embargo until June 16, 2025.

Title: Blood ctDNA vs tumor tissue screening for the detection of KRAS mutations in low-grade serous ovarian cancer
Abstract Number: 276
Date/Time: Thursday, June 19, from 2:00 to 3:30 pm EDT
Corporate Updates

In April 2025, Verastem strengthened its balance sheet by raising gross proceeds of approximately $75 million in a private placement of 3.4 million shares of its common stock and 7.3 million pre-funded warrants to purchase 7.3 million shares of its common stock.
First Quarter 2025 Financial Results

Verastem Oncology ended the first quarter of 2025 with cash, cash equivalents and investments of $117.6 million. On a pro forma basis, taking into account the $75 million of gross proceeds raised in a private placement in April, cash and cash equivalents were $192.6 million as of March 31, 2025.

Total operating expenses for the three months ended March 31, 2025 (the "2025 Quarter") were $44.2 million, inclusive of $6.8 million of one-time charges, compared to $28.1 million for the three months ended March 31, 2024 (the "2024 Quarter").

Research & development expenses for the 2025 Quarter were $29.2 million, compared to $17.7 million for the 2024 Quarter. The increase of $11.5 million, or 65.0%, was primarily related to the option exercise fee related to the GenFleet G12D program, increased contract research organization costs, and increased drug substance and drug product costs.

Selling, general & administrative expenses for the 2025 Quarter were $15.0 million, compared to $10.4 million for the 2024 Quarter. The increase of $4.6 million, or 44.2%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in KRAS mt LGSOC, increased personnel costs, including non-cash stock compensation, and one-time financing costs associated with the note purchase agreement.

Net loss for the 2025 Quarter was $52.1 million, or $0.96 per share (basic and diluted), compared to $33.9 million, or $1.26 per share for the 2024 Quarter.

For the 2025 Quarter, non-GAAP adjusted net loss was $42.9 million, or $0.79 per share (diluted) compared to non-GAAP adjusted net loss of $26.2 million, or $0.98 per share (diluted), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over- period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2025 and 2024 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication
AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions
The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for VS-7375 was cleared and plans to initiate a Phase 1/2a clinical trial in mid-2025. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024.

On May 13, 2025 Azitra, Inc. (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported financial results for the quarter ended March 31, 2025, and provided a business update (Press release, Azitra, MAY 13, 2025, View Source [SID1234652956]).

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Q1 2025 and Recent Business Highlights

Announced acceptance of poster detailing the Phase 1/2 clinical trial of the ATR-04 program in EGFR inhibitor ("EGFRi")-associated rash at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Entered into Purchase Agreement for up to $20 Million in Partnership with institutional investor Alumni Capital LP, to fund clinical pipeline
Announced closing of two public offerings raising a total of $2.2M
"The start of 2025 has been a vital period for Azitra as we build towards key milestones expected by mid-year for our first-in-class, precision, live biotherapeutic candidates designed for major undertreated dermatological diseases," said Francisco Salva, CEO of Azitra. "For ATR-12, our lead program targeting the rare, chronic and devastating Netherton syndrome, we expect to provide initial safety data from our Phase 1b trial in the first half of 2025 with topline results expected by year-end 2025. There are no approved treatments for Netherton syndrome, and we believe this novel approach has potential to be life-changing for these patients."

Salva continued: "Also by mid-2025, we look forward to dosing the first patient in our Phase 1/2 trial with our ATR-04 program, which contains a live biotherapeutic product candidate containing an isolated, naturally derived S. epidermidis strain being developed for the treatment of EGFRi-associated rash. EGFRi-associated rash is a dermatologic toxicity that often accompanies EGFRi treatments for cancer, impacting approximately 150,000 patients in the United States annually. These severe skin conditions can interfere with cancer treatment efforts, often causing significant physical and psychological discomfort for patients. Given the importance of EGFRi therapies to the cancer industry, Azitra was invited to present an update of our ATR-04 Phase 1/2 trial at ASCO (Free ASCO Whitepaper) 2025, which is the most prestigious cancer research conference in the world."

Salva concluded: "The remainder of 2025 is expected to be a milestone-rich period for Azitra during which we look forward to showcasing the potential of ATR-12 and ATR-04, as well as our unique, proprietary platform for delivering engineered proteins using topical live biotherapeutic products."

Pipeline and Anticipated Milestones

1H 2025: Initial safety data from first set of Netherton syndrome patients in the ATR-12 Phase 1b trial
1H 2025: First patient dosed with for EGFRi-associated rash in a Phase 1/2 trial for ATR-04
YE 2025: Topline data of the Phase 1b trial with ATR-12 in Netherton syndrome patients
Financial Results for the Quarter Ended March 31, 2025

Research and Development (R&D) expenses: R&D expenses for the quarter ended March 31, 2025, were $1.3 million compared to $1.5 million for the comparable period in 2024.
General and Administrative (G&A) expenses: G&A expenses for the quarter ended March 31, 2025, were $1.9 million compared to $1.5 million for the comparable period in 2024.
Net Loss was $3.1 million for the quarter ended March 31, 2025, compared to $2.9 million for the comparable period in 2024.
Cash and cash equivalents: As of March 31, 2025, the Company had cash and cash equivalents of $3.2 million.

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]).

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"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.

On May 13, 2025 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Candel Therapeutics, MAY 13, 2025, View Source [SID1234652957]).

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"During the quarter, we continued our strong momentum of compelling clinical evidence, reinforcing our promising pipeline of pan solid tumor immunotherapies," said Paul Peter Tak, MD PhD FMedSci, President and CEO of Candel. "Our primary focus for 2025 remains on working toward CAN-2409’s BLA submission for prostate cancer, which we believe represents a very significant unmet medical need and opportunity for value creation. In this indication, we observed the ability of CAN-2409 to reduce the risk of prostate cancer recurrence compared to standard of care, through meeting the primary endpoint agreed with the FDA under a Special Protocol Assessment in a phase 3 clinical trial. We are now focusing on executing strategic preparations for potential commercialization, to ensure that, if approved, CAN-2409 is immediately available to patients with localized prostate cancer."

Dr. Tak continued, "CAN-2409, Candel’s most advanced multimodal biological immunotherapy candidate, continues to demonstrate meaningful overall survival benefits in patients with advanced NSCLC, non-responsive to immune checkpoint inhibitors, as well as in patients with borderline resectable PDAC. The totality of data showing notable extension of survival based on both phase 2a clinical trials in NSCLC and PDAC, respectively, suggests that CAN-2409 has the potential to represent a transformative treatment option for patients with difficult-to-treat solid tumors. The FDA Fast Track Designation for each indication further validates the potential of this novel approach to address significant unmet medical needs in oncology."

First Quarter 2025 & Recent Highlights

•
CAN-2409 – Prostate Cancer
•
Positive phase 3 results from the CAN-2409 clinical trial in intermediate-to-high risk localized prostate cancer have been selected for an oral presentation at the upcoming 2025 ASCO (Free ASCO Whitepaper) Annual Meeting, taking place May 30 to June 3, 2025, in Chicago, IL.

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This phase 3 study was conducted under a Special Protocol Assessment (SPA) agreed with the U.S. Food and Drug Administration (FDA), meaning that certain data generated from this study could be sufficient for the Company to seek regulatory approval for CAN-2409 in this indication.

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The Company continues to work toward a Q4 2026 BLA submission for prostate cancer, following the positive topline data from its multicenter, randomized, placebo-controlled phase 3 clinical trial evaluating CAN-2409 in intermediate-to-high-risk localized prostate cancer patients.

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The FDA previously granted Fast Track Designation for CAN-2409 for the treatment of localized primary prostate cancer.

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CAN-2409 – Pancreatic Cancer
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Positive final survival data from the randomized controlled phase 2a clinical trial of CAN-2409 in borderline resectable PDAC demonstrated notable improvement in overall survival compared to standard of care. Patients who had received experimental treatment with CAN-2409 and chemoradiotherapy achieved a mOS of 31.4 months versus 12.5 months observed in the control arm treated with chemoradiotherapy.

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Notably, three long-term survivors in the CAN-2409 arm remained alive at 66.0, 63.6, and 35.8-months post-treatment, whereas only one patient from the control arm was still alive at the time of data cut-off (February 20, 2025). Patients in the CAN-2409 arm were stable at the time of last follow up with minimal maintenance therapy and, despite previous recurrence, experienced extended and ongoing post-progression survival, further highlighting the sustained benefit of CAN-2409, even in metastatic disease.

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The FDA previously granted Orphan Drug Designation and Fast Track Designation for CAN-2409 in borderline resectable PDAC.

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CAN-2409 – Non-Small Cell Lung Cancer
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In March, the Company reported final survival data from its phase 2a clinical trial of CAN-2409 in patients with stage III/IV NSCLC, inadequately responding to ICI treatment.

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In patients with an inadequate response to ICI treatment (Cohort 1+2, n=46), mOS was 24.5 months.

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In patients with progressive disease, despite ICI treatment (Cohort 2, n=41), mOS was 21.5 months, which is markedly longer than the 9.8–11.8 months of survival reported in published literature1,2 in the same patient population receiving standard of care of docetaxel chemotherapy.

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37% of patients with progressive disease at enrollment were still alive > 24 months after CAN-2409 treatment at the time of the March 3, 2025 data cut, suggesting a long tail of survival. 14/15 patients with overall survival > 24 months and 9/9 patients with overall survival > 30 months had non-squamous NSCLC.

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In patients with non-squamous NSCLC and progressive disease despite ICI (cohort 2, n=33), observed mOS was 25.4 months after CAN-2409 treatment.

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A decrease in the size of uninjected tumors was observed in 69% of patients with multiple lesions (n=35), indicating that a local injection is associated with a systemic anti-tumor immune response.

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CAN-2409 maintained its generally favorable safety and tolerability profile throughout the extended follow-up period.

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The FDA previously granted Fast Track Designation for CAN-2409 for the treatment of NSCLC.

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Recent Corporate Events
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In March 2025, the Company entered a strategic, commercial collaboration with IDEA Pharma, a Division of SAI MedPartners (IDEA). Under this agreement, IDEA will provide strategic commercial input throughout the development and commercialization process for Candel’s lead asset, CAN-2409. Through this collaboration, Candel will gain access to a dedicated team of experts with extensive experience in oncology commercialization and go-to-market strategy optimization.

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In March 2025, the Company appointed Elizabeth M. Jaffee, M.D., to its Research Advisory Board (RAB). Dr. Jaffee, an internationally recognized expert in cancer immunology and pancreatic cancer, brings her extensive expertise to the RAB, which is important in light of the Company’s focus on borderline resectable pancreatic cancer.

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Publication
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Manuscript published in the March 2025 online edition of Neuro-Oncology, reporting results of a phase 1b clinical trial exploring safety and tolerability of the combination of CAN-2409 plus prodrug (valacyclovir) and nivolumab, in addition to standard of care (neurosurgery, radiotherapy, and temozolomide), in patients with newly diagnosed rHGG.

Anticipated Milestones

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Clinical and biomarker activity data from an ongoing phase 1b clinical trial evaluating repeat doses of CAN-3110 in patients with rHGG expected in Q4 2025.

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Submission of BLA for CAN-2409 in prostate cancer expected in Q4 2026.

Financial Results for the First Quarter Ended March 31, 2025

Research and Development Expenses: Research and development expenses were $4.0 million for the first quarter of 2025 compared to $4.1 million for the first quarter of 2024. The decrease was primarily due to a decrease in employee-related expenses, partially offset by an increase in manufacturing costs, in support of the Company’s CAN-2409 programs. Research and development expenses included a non-cash stock compensation expense of ($0.1) million for the first quarter of 2025, as compared to a non-cash stock compensation expense of $0.6 million for the first quarter of 2024.

General and Administrative Expenses: General and administrative expenses were $4.1 million for the first quarter of 2025, compared to $3.8 million for the first quarter of 2024. The increase was primarily due to higher professional and consulting fees. General and administrative expenses included non-cash stock compensation expense of $0.4 million for the first quarter of 2025, as compared to a non-cash stock compensation expense of $0.5 million for the first quarter of 2024.

Net Income/Loss: Net income for the first quarter of 2025 was $7.4 million compared to a net loss of $8.2 million for the first quarter of 2024 and included net other income of $15.5 million and net other expense of $0.3 million, respectively. The increase in net income was primarily related to the change in the fair value of the Company’s warrant liability.

Cash Position: Cash and cash equivalents, as of March 31, 2025, were $92.2 million, as compared to $102.7 million as of December 31, 2024. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its current operating plan into the first quarter of 2027.

About CAN-2409

CAN-2409 (aglatimagene besadenovec), Candel’s most advanced multimodal biological immunotherapy candidate, is an investigational, off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to a patient’s specific tumor and induce an individualized, systemic immune response against the tumor. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells. Together, this regimen is designed to induce an individualized and specific CD8+ T cell-mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity, based on in situ immunization against a variety of tumor antigens. Because of its versatility, CAN-2409 has the potential to treat a broad range of solid tumors. Encouraging monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and ICI have previously been shown in several preclinical and clinical settings. More than 1,000 patients have been dosed with CAN-2409 with a favorable tolerability profile to date, supporting the potential for combination with other therapeutic strategies.

Currently, Candel is evaluating CAN-2409 in NSCLC and borderline resectable PDAC and has recently completed a successful phase 3 clinical trial in localized prostate cancer. CAN-2409 plus prodrug (valacyclovir) has been granted Fast Track Designation by the FDA for the treatment of PDAC, stage III/IV NSCLC in patients who are resistant to first line PD-(L)1 inhibitor therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy, and localized primary prostate cancer. Candel’s pivotal phase 3 clinical trial in prostate cancer was conducted under a SPA agreed with the FDA. The FDA has also granted Orphan Drug Designation to CAN-2409 for the treatment of PDAC.

About CAN-3110

CAN-3110 is a first-in-class, replication-competent herpes simplex virus-1 (HSV-1) next-generation oncolytic viral, immunotherapy candidate designed for dual activity for oncolysis and immune activation in a single therapeutic. CAN-3110 is being evaluated in a phase 1b clinical trial in patients with rHGG. In October 2023, the Company announced that Nature published results from this ongoing clinical trial. CAN-3110 was well tolerated with no dose-limiting toxicity reported. In the clinical trial, the investigators observed improved median overall survival compared to historical controls after a single CAN-3110 injection in this therapy-resistant condition.3 The Company and academic collaborators are currently evaluating the effects of repeat CAN-3110 injections in rHGG, supported by the Break Through Cancer foundation. CAN-3110 has previously received FDA Fast Track Designation and Orphan Drug Designation for the treatment of rHGG.

About the enLIGHTEN Discovery Platform

The enLIGHTEN Discovery Platform is a systematic, iterative HSV-based discovery platform leveraging human biology and advanced analytics to create new multimodal biological immunotherapies for solid tumors. The enLIGHTEN Discovery Platform has been designed to deconvolute the characteristics of the tumor microenvironment related to clinical outcomes. These characteristics are rapidly translated into optimized multi-gene payloads of tumor modulators that can be delivered to the tumor microenvironment for specific indications, disease stages, and rationally designed therapeutic combinations. In 2022, the Company announced a discovery partnership with the University of Pennsylvania Center for Cellular Immunotherapies to create new viral immunotherapies that could enhance the efficacy of chimeric antigen receptor T cell (CAR-T) therapy in solid tumors. During the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting and the 2023 International Oncolytic Virotherapy Conference (IOVC) Meeting, Candel presented encouraging data on the first candidate from this platform, Alpha 201-macro-1, which was designed to interfere with the CD47/SIRP1α pathway, in mouse models of breast cancer and lung cancer. During the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, Candel presented preclinical data, unveiling the second candidate from the enLIGHTEN Discovery Platform, a first-in-class multimodal immunotherapy candidate to induce tertiary lymphoid structures, being developed as a novel therapeutic for solid tumors. Candel also presented data on a multimodal viral therapeutic candidate encoding IL-12 and IL-15 at the 2024 IOVC meeting.

On May 13, 2025 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases, reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Kezar Life Sciences, MAY 13, 2025, View Source [SID1234652974]).

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"We shared exciting results this quarter from the PORTOLA trial, the first successful randomized study in treatment-refractory AIH," said Chris Kirk, CEO and co-founder of Kezar. "There has been almost no change over the last three decades in the treatment of this serious chronic disease, and patients and physicians are in need of new and effective therapies. We are encouraged by the promising safety and efficacy data in this difficult-to-treat patient population, in particular the durable and steroid-sparing remissions observed in patients treated with zetomipzomib. We continue to respond to the FDA Division of Hepatology and Nutrition on the partial clinical hold and are working toward achieving alignment on an appropriate trial design to demonstrate the clinical benefit of zetomipzomib in AIH."
Zetomipzomib: Selective Immunoproteasome Inhibitor
PORTOLA – Phase 2a clinical trial of zetomipzomib in patients with AIH (ClinicalTrials.gov: NCT05569759)
•In March, Kezar reported topline results from the PORTOLA Phase 2a clinical trial evaluating zetomipzomib in patients with autoimmune hepatitis. In relapsed AIH patients who entered screening on steroid-based therapy, 36% (5 of 14) of zetomipzomib-treated patients achieved a complete biochemical response (CR) and clinically significant steroid taper to 5 mg/day or less, compared to 0 of 7 of placebo patients. In the intention-to-treat (ITT) population, 31% (5 of 16) of zetomipzomib patients achieved a CR and steroid taper (5 mg/day or less), compared to 1 of 8 placebo patients. The median duration of response in zetomipzomib patients achieving a CR was 27.6 weeks (including the ongoing open-label extension at the time of the data cutoff), and no disease flares were reported in any zetomipzomib-treated patient achieving CR during study. A favorable safety profile was observed during the 6-month blinded treatment period.

•Kezar is responding to the information request from the FDA Division of Hepatology and Nutrition to resolve the partial clinical hold on the PORTOLA Phase 2a clinical trial.

PALIZADE – Phase 2b clinical trial of zetomipzomib in patients with active lupus nephritis (LN) (ClinicalTrials.gov: NCT05781750)
•In February, Kezar presented an update from the PALIZADE Phase 2b clinical trial evaluating zetomipzomib in patients with active LN. At the time of study termination, no patients had completed the full 52 weeks of treatment. Of the 12 patients receiving 60 mg of zetomipzomib who reached week 25, 42% achieved a urine protein-to-creatinine ratio (UPCR) of 0.5 or less, and the median UPCR was reduced by 79% from baseline. Marked improvements in serologic markers were observed in patients who received a 60 mg dose of zetomipzomib.
•In October 2024, Kezar made the strategic decision following a clinical hold by the FDA to terminate PALIZADE and focus the clinical development of zetomipzomib in AIH.
Financial Results
•Cash, cash equivalents and marketable securities totaled $114.4 million as of March 31, 2025, compared to $132.2 million as of December 31, 2024. The decrease was primarily attributable to cash used in operations.
•Research and development (R&D) expenses for the first quarter of 2025 decreased by $5.0 million to $12.2 million, compared to $17.2 million in the first quarter of 2024. This decrease was primarily due to the decreased clinical activities resulting from the Company’s strategic decision to terminate the PALIZADE trial in October 2024, a decrease in manufacturing expense related to the timing of drug manufacturing runs and a decrease in facility related expenses.
•General and administrative (G&A) expenses for the first quarter of 2025 decreased by $1.1 million to $5.4 million compared to $6.5 million in the first quarter of 2024. The decrease was primarily due to a decrease in legal and professional service expenses and non-cash stock-based compensation.
•Net loss for the first quarter of 2025 was $16.6 million, or $2.27 per basic and diluted common share, compared to a net loss of $21.7 million, or $2.98 per basic and diluted common share, for the first quarter of 2024.
•Total shares of common stock outstanding were 7.3 million shares as of March 31, 2025.
About PORTOLA
PORTOLA is a placebo-controlled, randomized, double-blind Phase 2a clinical trial evaluating the efficacy and safety of zetomipzomib in patients with AIH that are insufficiently responding to standard of care or have relapsed from a previous CR. The trial enrolled 24 patients, randomized (2:1) to receive 60 mg of zetomipzomib or placebo in addition to background therapy for 24 weeks, with a protocol-suggested steroid taper. All patients were required to receive a starting daily steroid dose of 20-40 mg/day of prednisone (or budesonide equivalent) and physicians were encouraged to taper daily steroid usage to 5 mg/day consistent with AIH treatment guidelines established by the American Association for the Study of Liver Diseases (AASLD). The primary efficacy endpoint of PORTOLA, which was not powered for efficacy, evaluated the proportion of patients who achieved a CR by Week 24, measured as normalization of alanine aminotransferase (ALT), aspartate aminotransferase (AST) and Immunoglobulin G (IgG) values (if elevated at baseline), with steroid dose levels not higher than baseline. A key secondary endpoint was the proportion of patients who achieved a CR by Week 24 with a steroid dose of 5 mg/day or less.