On May 15, 2025 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, reported financial results and recent corporate highlights for the first quarter ended March 31, 2025 (Press release, Fortress Biotech, MAY 15, 2025, View Source [SID1234653167]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress entered 2025 with strong momentum following a transformational fourth quarter marked by the U.S. Food and Drug Administration ("FDA") approvals of Emrosi and UNLOXCYT, and the acceptance of the New Drug Application ("NDA") for CUTX-101. In the first quarter 2025, our Fortress-founded partner company, Checkpoint Therapeutics, Inc., ("Checkpoint"), signed a merger agreement with Sun Pharma providing for Checkpoint’s acquisition by Sun Pharma, which we believe will enable broader patient access for Checkpoint’s UNLOXCYT (cosibelimab-ipdl) product and trigger a significant monetization event for Fortress — including an expected ~$28 million at closing, future potential royalties, and a potential CVR payment. These outcomes continue to validate Fortress’ business model — identifying, developing, and advancing innovative therapies with strategic optionality for value creation."

Dr. Rosenwald continued, "Looking ahead, we are focused on key value drivers, including the September 30, 2025 Prescription Drug User Fee Act ("PDUFA") action date for CUTX-101, which may also result in a Priority Review Voucher for our subsidiary, Cyprium Therapeutics, upon approval. Commercial launch of Emrosi is also underway with initial prescriptions filled at the end of March. Fortress’ robust pipeline — including multiple late-stage programs and newly approved products — positions us for continued revenue growth, value-driving milestones, and additional monetization opportunities. We remain committed to delivering innovative therapies to patients while building long-term shareholder value."

Recent Corporate Highlights1:

Monetization Updates

● In March 2025, our subsidiary Checkpoint entered into an agreement to be acquired by Sun Pharmaceutical Industries Limited (together with its subsidiaries and/or associated companies, "Sun Pharma"). Fortress owns approximately 6.9 million shares (including Class A Common on an as-converted basis) of Checkpoint’s common stock and is eligible to receive a 2.5% royalty on future sales of UNLOXCYT, pursuant to a royalty agreement between Checkpoint, Sun Pharma and Fortress. Upon completion of the transaction, Sun Pharma will acquire all outstanding shares of Checkpoint, and Checkpoint stockholders will receive, for each share of common stock they hold, an upfront cash payment of $4.10, without interest, and a non-transferable contingent value right ("CVR") entitling the stockholder to receive up to an additional $0.70 in cash if cosibelimab is approved prior to certain deadlines in the European Union pursuant to the centralized approval procedure or in Germany, France, Italy, Spain or the United Kingdom, subject to the terms and conditions in the CVR agreement. The closing of the transaction is subject to various conditions including the approval by requisite majorities of holders of Checkpoint’s shares at a special meeting of Checkpoint’s stockholders on May 28, 2025. We expect the transaction to close shortly after the stockholder meeting, assuming the requisite votes are received, although there can be no assurance that the transaction closes in a timely manner, or at all.

Regulatory Updates

● In November 2024, the FDA approved Emrosi (Minocycline Hydrochloride Extended-Release Capsules, 40mg), also known as DFD-29. Emrosi has the potential to be the new treatment paradigm for the millions of patients suffering from inflammatory lesions of rosacea. In March 2025, we announced the launch of Emrosi by our partner company, Journey Medical Corporation ("Journey Medical") (Nasdaq: DERM).
● In December 2024, the FDA approved UNLOXCYT, also known as cosibelimab, our anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC") who are not candidates for curative surgery or radiation. UNLOXCYT was developed at our partner company, Checkpoint (Nasdaq: CKPT).
● The FDA accepted the NDA submission for CUTX-101 (copper histidinate for Menkes disease) for priority review with a PDUFA goal date of September 30, 2025. In December 2023, we completed the asset transfer of CUTX-101 to Sentynl Therapeutics ("Sentynl"), a wholly owned subsidiary of Zydus Lifesciences Ltd. Sentynl completed the rolling submission of the NDA for CUTX-101 in the fourth quarter of 2024. Cyprium Therapeutics, our subsidiary company that developed CUTX-101, will retain 100% ownership over any FDA Priority Review Voucher that may be issued at NDA approval.

Commercial Product Updates

● Journey Medical’s net product revenues for the first quarter ended March 31, 2025, were $13.1 million, compared to net product revenues of $13.0 million for the first quarter ended March 31, 2024.
● At the end of March 2025, Journey Medical announced the launch of, and the first prescriptions filled for, Emrosi for the treatment of inflammatory lesions of rosacea in adults. Emrosi is available by prescription at specialty pharmacy chains.

Clinical Updates

● In March 2025, we announced that full results from two Phase 3 multicenter, randomized, double-blind, parallel-group, active-comparator and placebo-controlled clinical trials, Minocycline Versus Oracea in Rosacea-1 ("MVOR-1") and Minocycline Versus Oracea in Rosacea-2 ("MVOR-2"), evaluating Emrosi for the treatment of moderate-to-severe papulopustular rosacea in adults, were published in the Journal of the American Medical Association – Dermatology. The results demonstrated the efficacy, safety and tolerability of oral DFD-29 in rosacea. The full publication is available at View Source Information on such website is not a part of this release.
● In January 2025, we announced that the first patient was dosed in a multicenter, placebo-controlled and randomized Phase 2 clinical trial to evaluate Triplex, a cytomegalovirus ("CMV") vaccine, when administered to human leukocyte antigen matched related stem cell donors to reduce CMV events in patients undergoing hematopoietic stem cell transplantation. Triplex is currently in development at our subsidiary company, Helocyte, Inc.

General Corporate:

● In March 2025, Fortress entered into a strategic collaboration with Partex NV to identify and evaluate biopharmaceutical compounds using artificial intelligence for potential acquisition or licensing by Fortress.
● In February 2025, our partner company Mustang Bio, Inc. ("Mustang") raised net proceeds of $6.8 million in a public offering.
● Also in February 2025, Mustang announced the exit of the lease for its manufacturing facility in Worcester, Massachusetts and concurrent divestment of certain fixed assets including furniture and equipment to AbbVie Bioresearch Center Inc. for $1.0 million.

Financial Results:

● As of March 31, 2025, Fortress’ consolidated cash and cash equivalents totaled $91.3 million, compared to $57.3 million as of December 31, 2024, an increase of $34.0 million during the quarter.
● Fortress’ consolidated cash and cash equivalents totaling $91.3 million as of March 31, 2025, includes $19.5 million attributable to Fortress and the private subsidiaries, $3.5 million attributable to Avenue, $33.0 million attributable to Checkpoint, $14.2 million attributable to Mustang Bio and $21.1 million attributable to Journey Medical.
o Fortress’ consolidated cash and cash equivalents totaled $57.3 million as of December 31, 2024, and included $20.9 million attributable to Fortress and private subsidiaries, $2.6 million attributable to Avenue, $6.6 million attributable to Checkpoint, $6.8 million attributable to Mustang and $20.3 million attributable to Journey Medical.
● Fortress’ consolidated net product revenue totaled $13.1 million for the first quarter ended March 31, 2025, which is generated from Journey Medical’s marketed dermatology products. This compares to consolidated revenue totaling $13.0 million for the first quarter of 2024.
● Consolidated research and development expenses totaled $3.9 million for the first quarter ended March 31, 2025, compared to $24.8 million for the first quarter ended March 31, 2024.
● Consolidated selling, general and administrative costs were $25.7 million for the first quarter ended March 31, 2025, compared to $17.9 million for the first quarter ended March 31, 2024.
● Consolidated net loss attributable to common stockholders was $(12.7) million, or $(0.48) per share, for the first quarter ended March 31, 2025, compared to net loss attributable to common stockholders of $(17.9) million, or $(1.04) per share for the first quarter ended March 31, 2024.

On May 15, 2025 Elevation Oncology, Inc. (Nasdaq: ELEV), an innovative oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors with significant unmet medical needs, reported financial results for the quarter ended March 31, 2025, and provided recent business updates (Press release, Elevation Oncology, MAY 15, 2025, View Source;utm_medium=rss&utm_campaign=elevation-oncology-reports-first-quarter-2025-financial-results-and-provides-business-updates [SID1234653166]).

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"We recently presented preclinical proof-of-concept data for EO-1022, reaffirming its potential as a differentiated HER3 ADC, and supporting our goal of providing a safer and more effective option for patients with HER3-expressing solid tumors," said Joseph Ferra, President and Chief Executive Officer of Elevation Oncology. "In parallel, we are engaged in efforts to explore a range of strategic alternatives, with the objective of identifying and capitalizing on the opportunity that is in the best interest of our shareholders. We look forward to providing an update at the appropriate time."

Recent Business Updates

Pipeline

Elevation Oncology is developing EO-1022, a HER3 antibody-drug conjugate (ADC) for the treatment of patients with HER3-expressing solid tumors, including breast cancer and non-small cell lung cancer. The Company expects to file an Investigational New Drug (IND) application for EO-1022 in 2026.

In April 2025, Elevation Oncology presented new preclinical proof-of-concept data supporting the development of EO-1022 in a late-breaking poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The in vitro and in vivo data indicate EO-1022 may offer reduced payload-associated toxicity and an improved safety profile, as well as improved anti-tumor activity, for patients living with solid tumors that express HER3. Specifically, data show:
EO-1022 is highly stable in human serum, with a homogenous drug-to-antibody ratio (DAR) of 4 and minimal free payload compared to seribantumab-vcMMAE and patritumab-DXd, two benchmark HER3 ADCs, both of which use stochastic conjugation. These findings illustrate that a key feature of EO-1022 is minimal systemic exposure to free payload, potentially resulting in reduced payload-associated toxicity in patients and an improved safety profile.
EO-1022 exhibits potent in vitro cytotoxicity that is dependent on HER3 expression levels.
EO-1022 elicits anti-tumor activity in in vivo models of low, medium and high HER3 expression levels, including in a patient derived xenograft (PDX) model of low HER3-expressing EGFR-mutant lung cancer.
Corporate

In March 2025, Elevation Oncology elected to discontinue development of EO-3021. In parallel, the Company implemented a workforce reduction of approximately 70%. Elevation Oncology is in the process of evaluating strategic options with a commitment to maximizing shareholder value. There is currently no timetable set for completion of the strategic alternatives review process.
Financial Outlook

Elevation Oncology ended the first quarter of 2025 with $80.7 million in cash, cash equivalents and marketable securities. Subsequent to the first quarter, on May 2, 2025, Elevation Oncology voluntarily prepaid the $32.3 million aggregate principal, interest, fees and expenses due under its loan agreement with K2 HealthVentures LLC. Elevation Oncology expects that a significant majority of expenses incurred in relation to its workforce reduction and EO-3021 program closure will be paid in the second quarter of 2025.

Elevation Oncology estimates that it will have cash, cash equivalents and marketable securities in a range of approximately $30 million to $35 million as of June 30, 2025, which is expected to fund its current operations into the second half of 2026.

First Quarter 2025 Financial Results

Research and development expenses for the first quarter of 2025 were $6.9 million, compared to $6.0 million for the first quarter of 2024. The increase of $0.9 million was primarily due to $1.3 million of increased costs associated with the preclinical development of EO-1022 and a $0.6 million increase in clinical trial expenses for EO-3021, partially offset by a $1.0 million decrease in clinical trial expenses for seribantumab.

General and administrative expenses for the first quarter of 2025 were $4.0 million, compared to $3.9 million for the first quarter of 2024. The increase of $0.1 million was mainly due to increased personnel costs, including stock-based compensation.

Restructuring charges were $3.4 million for the first quarter of 2025 and consisted primarily of charges related to the workforce reduction in connection with the discontinuation of development of EO-3021. No such charges were incurred during the first quarter of 2024.

Net loss for the first quarter of 2025 was $14.2 million, compared to $10.7 million for the first quarter of 2024.

About EO-1022

Elevation Oncology is developing EO-1022, a potentially differentiated HER3 ADC for the treatment of HER3-expressing solid tumors, including breast cancer and non-small cell lung cancer. EO-1022 consists of seribantumab, a fully human IgG2 anti-HER3 antibody, site-specifically conjugated at glycan to the MMAE payload with a DAR of 4. It leverages seribantumab’s desirable internalization properties and advanced site-specific ADC technology which makes possible the use of the potent cytotoxic MMAE payload. Elevation Oncology expects to file an IND application in 2026.

On May 15, 2025 Pulmatrix ("Pulmatrix" or the "Company") (Nasdaq: PULM), a biopharmaceutical company that has focused on the development of novel inhaled therapeutic products intended to prevent and treat migraine and respiratory diseases with important unmet medical needs using its patented iSPERSE technology, reported first quarter financial results for 2025 and provided a corporate update (Press release, Cullgen, MAY 15, 2025, View Source [SID1234653162]).

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Peter Ludlum, Interim Chief Executive Officer of Pulmatrix, commented, "Our focus in the first quarter has been to advance steps to complete the proposed merger with Cullgen, a privately held, clinical-stage biopharmaceutical company applying its proprietary targeted protein degradation uSMITE platform to discover and advance therapeutics for the treatment of cancer and other diseases. If successful, the proposed merger would create a Nasdaq-listed company focusing on targeted protein degradation technology with three degrader programs in Phase 1 clinical trials – two for the treatment of cancer and one for the treatment of acute and chronic pain. As part of the proposed merger, Pulmatrix is currently in a process to divest its clinical assets, including our Phase 2-ready acute migraine product candidate PUR3100, along with our iSPERSE technology."

Proposed Merger with Cullgen

As previously reported, on November 13, 2024, the Company entered into an agreement and plan of merger with Cullgen Inc. ("Cullgen"), as amended by Amendment No. 1 thereto on April 7, 2025 (the "Merger Agreement" and such transaction, the "Merger"). The proposed Merger is anticipated to close in June 2025, subject to the satisfaction of certain closing conditions, among others, however the exact timing of the consummation of the proposed Merger cannot be predicted.

Additional information about the Merger Agreement and proposed Merger was previously disclosed in a registration statement on Form S-4 (File No. 333-284993) initially filed with the Securities and Exchange Commission (the "SEC") on February 14, 2025, as amended on April 17, 2025, and May 7, 2025.

Pulmatrix Currently Seeking Divestment of Clinical Assets and Proprietary iSPERSE Technology

PUR3100

PUR3100 is an orally inhaled dihydroergotamine ("DHE") engineered with Pulmatrix’s iSPERSE dry powder inhalation technology for the treatment of acute migraine.
In 2023, Pulmatrix announced the Food and Drug Administration’s acceptance of an Investigational New Drug ("IND") application for PUR3100 and receipt of a "study may proceed" letter to proceed with a Phase 2 study, positioning PUR3100 as a Phase 2-ready asset. The IND includes a Phase 2 clinical protocol where safety and preliminary efficacy of PUR3100 will be investigated in patients with acute migraine.
The Phase 2 IND builds on the Phase 1 trial results of PUR3100, which were published in 2024 in a peer-reviewed publication, Headache: The Journal of Head and Face Pain.
The study showed that PUR3100 achieved peak exposures in the targeted therapeutic range and time to maximum concentration occurred at five minutes after dosing at all dosing levels. The PUR3100 dose groups also showed a lower incidence of nausea and no vomiting compared to observations of nausea and vomiting in the intravenously ("IV") administered DHE dose group.
PUR1800

PUR1800 is a Narrow Spectrum Kinase Inhibitor ("NSKI"), engineered with our iSPERSE technology, for the treatment of acute exacerbations in chronic obstructive pulmonary disease ("AECOPD"). In 2023, Pulmatrix presented complete results from a Phase 1b study of PUR1800 for AECOPD, indicating PUR1800 was safe and well tolerated with no observed safety signals. The topline data, along with the results from chronic toxicology studies, support the continued development of PUR1800 for the treatment of AECOPD and other inflammatory respiratory diseases.
PUR1900

PUR1900 is the Company’s inhaled iSPERSE formulation of the antifungal drug itraconazole for indications where an orally inhaled antifungal may provide a therapeutic benefit or fulfill an unmet medical need.
The Company completed all Phase 2b wind down activities in the third quarter of 2024. With the study wind down complete, Pulmatrix bears no further financial responsibility for the development of PUR1900.
The Company’s partner Cipla has continued clinical development outside the United States and has advised us that they have completed their Phase 2 study in India and have been approved by India’s Central Drug Standard Control Organization to proceed with a Phase 3 clinical trial.
Should Cipla successfully market PUR1900 outside the United States, Pulmatrix will receive 2% royalties on any potential future net sales by Cipla outside the United States. Within the United States, the Company and Cipla will seek to monetize PUR1900 for indications where an orally inhaled antifungal may provide a therapeutic benefit or fulfill an unmet medical need.
iSPERSE Technology

iSPERSE particles are engineered with a small, dense and dispersible profile to exceed the performance of traditional dry powder particles as the iSPERSE particles have the dispersibility advantages of porous engineered particles. Pulmatrix believes this results in superior drug delivery compared to traditional oral and injectable forms of treatment for certain diseases.
As of March 31, 2025, Pulmatrix’s patent portfolio related to iSPERSE included approximately 146 granted patents, 18 of which are granted U.S. patents, with expiration dates from 2026 to 2037, and approximately 48 additional pending patent applications in the U.S. and other jurisdictions.
First Quarter 2025 Financial Results

Revenues decreased approximately $5.9 million to $0 for the three months ended March 31, 2025, compared to $5.9 million for the three months ended March 31, 2024. The decrease is primarily related to completion of the wind down of the PUR1900 Phase 2b clinical trial during the year ended December 31, 2024.

Research and development expenses decreased approximately $3.5 million to less than $0.1 million for the three months ended March 31, 2025, compared to $3.5 million for the three months ended March 31, 2024. The decrease was primarily due to winding down the PUR1900 Phase 2b clinical trial, disposal of the Company’s lab and facilities lease and employee terminations.

General and administrative expenses increased approximately $0.2 million to $1.8 million for the three months ended March 31, 2025, compared to $1.6 million for the three months ended March 31, 2024. The increase was primarily due to incurred costs related to the proposed Merger, partially offset by decreased employment and other operating costs.

The Company’s total cash and cash equivalents balance as of March 31, 2025, was $7.7 million. The Company anticipates that its cash position, based on operational efficiencies and prioritization of spending, is sufficient to fund its operations at least through the anticipated closing of the proposed Merger with Cullgen.

On May 15, 2025 Century Therapeutics, Inc. (‘Century’, NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in autoimmune disease and cancer, reported financial results and business highlights for the first quarter ended March 31, 2025 (Press release, Century Therapeutics, MAY 15, 2025, View Source [SID1234653161]).

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"We have made important progress focusing on pipeline programs with transformational potential in high-value commercial markets. In a recently held company webinar and at ASGCT (Free ASGCT Whitepaper), we showcased developmental progress with our preclinical pipeline, iPSC platform, and manufacturing capabilities, highlighting the potential for a pathway to developing iPSC-derived cell therapies at antibody-like scale. We are on track to initiate IND-enabling studies for our lead preclinical program, CNTY-308, in mid-2025," said Brent Pfeiffenberger, Pharm.D., Chief Executive Officer of Century Therapeutics. "For our clinical-stage pipeline, patient dosing is now underway in the U.S. for the CALiPSO-1 trial and, with expansion into Europe, we remain on track to deliver clinical data for CNTY-101 by the end of 2025. We look forward to building on this momentum and pursuing a disciplined approach across our pipeline to bring investigational cell therapies closer to patients and generate value for all our stakeholders."

First Quarter 2025 and Recent Highlights

CNTY-101 in Autoimmune Disease

· Patient dosing underway in CALiPSO-1 trial in the U.S.: In March 2025, the first patient was dosed in the Phase 1 CALiPSO-1 trial which is currently evaluating CNTY-101 as a potential treatment for patients with B-cell-mediated autoimmune diseases.

· Focus on CALiPSO-1 clinical trial expansion and patient recruitment: Five clinical trial sites in the U.S. are active with additional U.S. clinical trial sites expected to open in 2025. Site activation activities are underway in select European countries, with enrollment at these sites expected to initiate in the second half of 2025. The company’s Clinical Trial Application (CTA) has been authorized in Germany, France, and Italy, providing important external validation of Century’s approach to clinical studies with allogeneic iPSC-derived therapies.

· CARAMEL IIT CTA authorized and study on track to commence in mid-2025: The CARAMEL investigator-initiated trial (IIT), a Phase 1/2 trial of CNTY-101 in patients with B-cell-mediated autoimmune diseases led by Professors Georg Schett and Andreas Mackensen and sponsored by the Friedrich-Alexander University Erlangen-Nürnberg, is expected to commence in mid-2025. The CTA has been authorized in Germany

· Clinical data by end of 2025: The company remains on track to report clinical data for CNTY-101 by the end of 2025.

CNTY-308 and Other Preclinical Programs

· ASGCT presentations highlighting preclinical pipeline: At the ongoing American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, the company is presenting data on the company’s emerging preclinical cell therapy pipeline for autoimmune diseases and cancer. The data from the poster presentation suggest certain engineering modifications to CAR-iT and CAR-iNK cells can improve the anti-tumor activity of iPSC-derived immunotherapies to withstand the immunosuppressive nature of the solid tumor microenvironment. The data from the oral presentation characterize the generation of allogeneic iPSC-derived targeted CD4+/CD8+ ab CAR-T cells with comparable function, in vivo proliferation, and tumor control to primary T cells, supporting their use for the development of scalable, accessible CAR-T cell therapies.

· CNTY-308 on track to enter into IND-enabling studies in mid-2025: The company continues to expect to initiate Investigational New Drug (IND)-enabling studies with CNTY-308, a CD19-targeted CD4+/CD8+ ab CAR-iT cell therapy engineered with Allo-Evasion 5.0 as a potential treatment for B-cell-mediated autoimmune diseases and malignancies, in mid-2025.

· Progressing early-stage development activities for iPSC-derived ab CAR-T cells in oncology and opportunities with non-immune effector cells in high-impact diseases: The company is advancing CNTY-341, a CD19/CD22 dual-targeted CAR-iT investigational cell therapy intended for B-cell malignancies, and the company’s first solid tumor CAR-iT investigational program exploiting nectin-4 CAR and other validated targets. Each of these programs is anchored by advanced iPSC-derived ’tunable’ CD4+/CD8+ ab T cells and is engineered with the company’s proprietary immune evasion technology, Allo-Evasion 5.0, which is designed to enable holistic evasion of T cell, NK cell, and humoral immunity. In addition, the company is leveraging its deep expertise in selective iPSC differentiation to non-immune effector cells with opportunities to potentially accelerate in high-impact therapeutic areas where the company believes its technology and capabilities provide meaningful differentiation.

First Quarter 2025 Financial Results

· Cash Position: Cash, cash equivalents, and marketable securities were $185.8 million as of March 31, 2025, as compared to $220.1 million as of December 31, 2024. The company estimates its cash, cash equivalents, and investments will support operations into the fourth quarter of 2026.

· Collaboration Revenue: Collaboration revenue generated through the company’s collaboration, option, and license agreement with Bristol-Myers Squibb (the Collaboration Agreement) was $109.2 million for the three months ended March 31, 2025, compared to $0.9 million for the same period in 2024. The Collaboration Agreement was terminated effective March 12, 2025. As such, the company recognized $109.2 million of collaboration revenue for the quarter ended March 31, 2025. There will be no future collaboration revenues recognized under the Collaboration Agreement.

· Research and Development (R&D) Expenses: R&D expenses were $26.6 million for the three months ended March 31, 2025, compared to $23.4 million for the same period in 2024. The increase in R&D expenses is most notably due to increased clinical trial costs and advancing of preclinical programs.

· General and Administrative (G&A) Expenses: G&A expenses were $8.4 million for the three months ended March 31, 2025, compared to $8.7 million for the same period in 2024. The decrease was primarily due to a decrease in salary and benefit expense.

· Net Income (Loss): Net income was $76.6 million for the three months ended March 31, 2025, compared to net (loss) of $28.1 million for the same period in 2024.

On May 15, 2025 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, reported financial results for the first quarter ended March 31, 2025, and provided recent business highlights (Press release, Aura Biosciences, MAY 15, 2025, View Source [SID1234653160]).

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"Aura has started 2025 with strong momentum, making meaningful strides across both our ocular and urologic oncology programs," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "Our global Phase 3 CoMpass trial in early-stage choroidal melanoma continues to advance, and we enrolled the first patient in our multi-dose Phase 1b/2 trial in NMIBC. At Aura, we remain deeply focused on transforming the treatment landscape in ocular and urologic cancers—two areas where patients urgently need innovative therapies."

Recent Pipeline Developments

Early-Stage Choroidal Melanoma

Update on Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling study in early-stage choroidal melanoma. The study is a global, Phase 3, randomized trial evaluating bel-sar treatment against a sham control arm and includes an enrichment strategy to enroll approximately 100 patients with documented tumor growth.

The CoMpass trial is actively enrolling globally. To identify appropriate patients to meet the enrichment strategy of documented growth, the Company has enabled a pre-screening ‘run in’ period. Globally, since June 2024, investigators have registered over 220 patients in a pre-screening tool as having met initial enrollment criteria for the study, highlighting the global need for a frontline vision-preserving therapy. Given the momentum in the study globally, the Company believes study enrollment may be completed as early as the end of 2025.

The Company previously received Orphan Drug Designation from the FDA and the European Medicines Agency and Fast Track designation from the FDA for the treatment of early-stage choroidal melanoma. The CoMpass trial is under a Special Protocol Assessment agreement with the FDA.

Additional Ocular Oncology Indications

In addition to early-stage choroidal melanoma, bel-sar is being explored for metastases to the choroid and cancers of the ocular surface. These three ocular oncology indications have a collective incidence of greater than 60,000 patients annually in the United States and Europe.

Metastases to the Choroid

Metastases to the choroid is an indication with high unmet medical need and no approved therapies. Bel-sar has the potential to treat a wide variety of tumor types that metastasize from several primary tumors. The Company has initiated a Phase 2 clinical trial in metastases to the choroid from breast and lung cancer and have activated sites with patients in prescreening in the United States. The Company is currently implementing a protocol amendment for the Phase 2 trial to broaden the inclusion criteria beyond breast and lung cancer to include all metastases from different solid tumors as a basket study approach. The Company believes that this approach, in addition to advancing bel-sar in metastases to the choroid, can provide clinical insights into multiple tumor types that could be impacted by bel-sar. The Company expects initial data from this trial in 2025.

Metastases to the choroid represents the second potential ocular oncology indication for bel-sar, affecting approximately 20,000 patients annually in the United States and Europe. The Company previously received FDA Fast Track designation for bel-sar in this indication.

Cancers of the Ocular Surface

The Company’s third potential ocular oncology indication is cancers of the ocular surface, which affects approximately 35,000 patients in the United States and Europe annually and has no approved therapies. We continue to advance pre-clinical activities in cancers of the ocular surface, and we plan to initiate a Phase 1 trial in 2025.

Bladder Cancer

Patent Application Filed for New Formulation of Bel-sar for Use in Bladder Cancer: The Company has filed a patent application for a new formulation of bel-sar for use in urologic oncology. This new formulation is designed to enable convenient in-office urologist procedures with enhanced storage and handling at refrigerator temperatures, as well as an adjusted volume and concentration.

Positive Data from Completed Phase 1 Window-of-Opportunity Trial: In the completed Phase 1 window-of-opportunity trial for NMIBC, the administration of a single, low dose of the ocular formulation of bel-sar resulted in multiple clinical complete responses among patients with intermediate and high-risk NMIBC. These histopathologic outcomes highlight robust cell-mediated immunity and a urothelial field effect. Additionally, the study demonstrated a favorable safety profile, with only grade 1 drug-related adverse events occurring in less than 10% of patients. Detailed data can be accessed here: link. Based on these findings, the Company believes bel-sar has the potential to transform treatment of patients with intermediate and high-risk NMIBC with its immune-ablative, front-line approach.

Ongoing Phase 1b/2 Trial: Based on the positive data from the Phase 1 window of opportunity trial, the Company is advancing the development of bel-sar in NMIBC. The ongoing Phase 1b/2 trial will evaluate additional doses and cycles of bel-sar in approximately 26 intermediate and high-risk patients. The trial will evaluate two approaches: an immune ablative design and a multimodal neoadjuvant design. In the immune ablative approach, bel-sar will be administered in two cycles without the need for a transurethral resection of the bladder tumor (TURBT). In the multimodal neoadjuvant cohorts, bel-sar will be administered in two cycles ahead of TURBT. For both approaches, patients will be monitored for response assessments and recurrence at 3, 6, 9, and 12 months.

Endpoints of this trial include multiple efficacy assessments, such as complete response rate at 3 months and durability of response up to 12 months in the immune ablative cohorts and recurrence-free survival in the neoadjuvant cohorts. Patients will also be monitored for safety. The Company expects initial efficacy data at 3 months by year-end 2025.

The Company has filed a patent application with the U.S. Patent and Trademark Office covering the new formulation, which if issued, would provide patent coverage for this formulation into 2046.

Corporate Updates

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The Company strengthened the leadership team with the appointment of Tony Gibney as Chief Finance and Business Officer. Mr. Gibney is an experienced biotechnology leader and former investment banker who brings over 30 years of experience dedicated to leading and advising biotechnology companies across their businesses, including corporate strategy, business development, finance and investor relations, among others. Following his investment banking career, he has worked as Chief Business Officer at Achillion Pharmaceuticals, Inc. and Iveric Bio, Inc. and as Chief Business and Financial Officer at Fog Pharmaceuticals, Inc.

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The Company hosted a virtual urologic oncology investor event on March 24, 2025. A replay of the webcast is available on the "Investors & Media" page under the "Events & Presentations" section of Aura’s website at View Source

First Quarter 2025 Financial Results

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As of March 31, 2025, Aura had cash and cash equivalents and marketable securities totaling $128.0 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the second half of 2026.

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Research and development expenses increased to $23.3 million for the three months ended March 31, 2025 from $17.1 million for the three months ended March 31, 2024, primarily due to ongoing clinical and contract research organization costs associated with the progression of the Company’s Phase 3 trial of bel-sar in early-stage choroidal melanoma and manufacturing and development costs for bel-sar.

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General and administrative expenses increased to $5.7 million for the three months ended March 31, 2025 from $5.3 million for the three months ended March 31, 2024. General and administrative expenses include $1.6 million and $1.4 million of stock-based compensation for the three months ended March 31, 2025 and 2024, respectively. The increase was primarily driven by higher personnel expenses related to the growth of the Company.

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Net loss for the three months ended March 31, 2025 was $27.5 million compared to $19.7 million for the three months ended March 31, 2024.