On May 8, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported financial results for the first quarter ended March 31, 2025, and provided recent corporate progress (Press release, Whitehawk Therapeutics, MAY 8, 2025, View Source [SID1234652780]).

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"By uniting novel approaches to tumor targeting with next wave technologies, our goal at Whitehawk Therapeutics is to efficiently deliver improved ADC therapies for patients with difficult-to-treat cancers. In the first quarter of 2025, we made meaningful progress in advancing our portfolio toward the clinic and remain on track to bring all three assets to IND by mid-2026, with the first IND filed in Q4 this year," said Dave Lennon, President and CEO of Whitehawk Therapeutics. "Our assets target PTK7, MUC16 and SEZ6 – which we believe have the potential for broad clinical impact comparable to well-established targets like HER2 and TROP2, but with less competition and greater opportunity for differentiation. Coupled with our TOPO1-based leading next wave platform, we are confident in the strength of our strategy and remain committed to advancing with urgency and purpose."

Recent Operational Highlights:

Relaunched as Whitehawk Therapeutics marking evolution into ADC company. Launched with a three-asset portfolio consisting of clinically validated tumor targets that are upregulated in high-potential cancer indications, including lung and ovarian. These assets are engineered to produce minimal off-target toxicity, with a higher therapeutic index and greater stability than first-generation predecessors.
Completed strategic transactions. Closed $100 million PIPE financing and the divestiture of Aadi Subsidiary, Inc. ("Aadi Sub") to Kaken Pharmaceuticals ("Kaken") for $102.4 million, including specified purchase price adjustments. Kaken assumed ownership of Aadi Sub on March 25, 2025, including the Aadi name, trademark and the FYARRO business.
First Quarter 2025 Financial Results:

Cash, cash equivalents and short-term investments as of March 31, 2025, were $231.1 million as compared to $47.2 million as of December 31, 2024.
After completion of transaction-related payments, including the payment of the upfront fees under the Wuxi ADC agreement, we expect to have cash and cash equivalents of approximately $185 million, which we anticipate will fund operations into 2028 based on current plans.
Total revenue for the quarter ended March 31, 2025, was $7.1 million, resulting from sales of FYARRO through March 25, 2025, the closing date of the divestiture to Kaken.
Net income for the three months ended March 31, 2025, was $73.0 million, including a gain of $87.4 million on the sale of Aadi Sub, as compared to a net loss of $18.3 million for the three months ended March 31, 2024. Excluding the gain on the sale of Aadi Sub, net loss for the first quarter of 2025 was $14.4 million.

On May 8, 2025 Delcath Systems, Inc. (Nasdaq: DCTH) ("Delcath" or the "Company"), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, reported financial results and business highlights for the first quarter ended March 31, 2025 (Press release, Delcath Systems, MAY 8, 2025, View Source [SID1234652742]).

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First Quarter 2025 Financial Results

Total revenue of $19.8 million, compared with $3.1 million in the first quarter of 2024
HEPZATO KIT revenue of $18.0 million, compared to $2.0 million in the first quarter of 2024
CHEMOSAT revenue of $1.8 million, compared to $1.1 million in the first quarter of 2024
Gross margins of 86%, compared to 71% in the first quarter of 2024
Net income of $1.1 million, compared to a net loss of $11.1 million in the same quarter of 2024
Non-GAAP positive adjusted EBITDA in the first quarter of $7.6 million, compared to a loss of $7.3 million in the first quarter of 2024
Cash and investments of $58.9 million as of March 31, 2025
Cash provided by operations of $2.2 million in the quarter
Business Highlights and Updates

Activated three new U.S. centers in the first quarter and two more so far in the second quarter of 2025, bringing the current total to 19 active centers, with 10 additional centers accepting referrals
Received FDA clearance of an IND application for a phase 2 clinical trial of HEPZATO in liver-dominant metastatic breast cancer
Announced publication of comparative analysis from randomized portion of FOCUS Study in metastatic uveal melanoma
The exercise of 1.62 million Series F warrants resulted in $16.2 million of funding in 2025. The warrants were issued in 2020 as a component of a private placement and had an exercise price of $10.00 per share and expired on May 5, 2025
"Consistent revenue growth and the continued expansion of active treatment centers represent a strong start to 2025," said Gerard Michel, Chief Executive Officer of Delcath. "We achieved our first quarter of net income and positive operating cash flow, underscoring the accelerating clinical adoption of HEPZATO for patients with metastatic uveal melanoma. Ongoing dialogue with oncologists and interventional radiologists reinforces the importance of whole-liver treatment for patients with liver-dominant disease. These insights continue to shape our development strategy as we pursue additional indications for HEPZATO."

First Quarter 2025 Results

Total revenue for the quarter ending March 31, 2025 was $19.8 million compared to $3.1 million for the same period in the prior year. Revenue in the quarter includes sales of $18.0 million of HEPZATO in the U.S. and $1.8 million of CHEMOSAT in Europe.

Research and development expenses for the quarter ended March 31, 2025, were $5.0 million compared to $3.7 million for the same period in the prior year. The increase is primarily due to costs associated with expanding the clinical team and initiation of the Phase 2 clinical trial evaluating HEPZATO in combination with standard of care for mCRC. In 2024, these costs primarily related to medical affairs and regulatory costs associated with the approved products.

Selling, general and administrative expenses for the quarter ending March 31, 2025, were $11.3 million compared to $8.8 million for the same period in the prior year. The increase is primarily due to continued commercial expansion activities including marketing-related expenses and additional personnel in the commercial team.

Net income for the quarter ended March 31, 2025 was $1.1 million compared to net loss of $11.1 million for the same period in the prior year.

Non-GAAP adjusted EBITDA for the quarter ended March 31, 2025 was $7.6 million compared to adjusted EBITDA loss of $7.3 million for the same period in the prior year. A table reconciling non-GAAP measures is included in this press release for reference.

As of March 31, 2025, the Company had $58.9 million in cash and investments, and no debt.

Conference Call Information
To participate in this event, dial in approximately 5 to 10 minutes before the beginning of the call.

Event Date: Thursday, May 8, 2025
Time: 8:30 AM Eastern Time

Participant Numbers:
Toll Free: 1-877-407-3982
International: 1-201-493-6780
Webcast: View Source;tp_key=6de250d858
A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website View Source

On May 8, 2025 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results for the quarter ended March 31, 2025 and provided a business update (Press release, Prime Medicine, MAY 8, 2025, View Source [SID1234652758]).

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"We recently unveiled our AATD program, further demonstrating our commitment to building a liver franchise of Prime Editors designed to cure major genetic diseases," said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. "Both our Wilson’s Disease and AATD programs are advancing through preclinical development, and we look forward to initiating clinical trials in both indications in 2026. In addition, we remain on track to report initial data from our Phase 1/2 trial of PM359 in CGD this year, and continue to progress our efforts in CF."

Dr. Gottesdiener continued, "While PM359 leverages a different delivery mechanism from our programs in liver and lung diseases, we believe our forthcoming CGD data could provide important readthrough across our pipeline. If positive, these data would demonstrate the potential of Prime Editing as a powerful and differentiated technology, potentially capable of offering curative benefit to patients following a single treatment. We remain committed to executing with focus and discipline as we approach these first in-human data, which we hope will mark a key inflection point on our path to sustained growth."

Prime Medicine’s Pipeline:

Prime Medicine is advancing a set of high-value programs across its core areas of focus (hematology, immunology and oncology, liver, and lung). These include ex vivo hematopoietic stem cell (HSC) programs for the treatment of p47phox chronic granulomatous disease (CGD) and X-linked CGD; lipid nanoparticle (LNP) Prime Editors for the treatment of Wilson’s Disease and alpha-1 antitrypsin deficiency (AATD); LNP or adeno-associated virus (AAV) Prime Editors for the treatment of cystic fibrosis (CF); and ex vivo T-cell therapies, which are being developed in collaboration with Bristol Myers Squibb.

Recent Business Updates

•In March 2025, Prime Medicine unveiled its preclinical program for the treatment of AATD. Prime Medicine’s program leverages the Company’s universal liver LNP to edit the E342K (Pi*Z) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD, with the potential to treat both lung- and liver-associated disease. In initial in vivo data, Prime Medicine observed high levels of editing at the target site, with full restoration of circulating wild-type AAT protein (M-AAT) to normal human range. Additionally, based on preclinical studies using unoptimized surrogate Prime Editors across Prime Medicine’s liver franchise, the Company believes Prime Editing has the ability to correct disease-causing mutations without introducing off-target or bystander edits.
Anticipated Upcoming Milestones:

Chronic Granulomatous Disease (CGD):
•Announce initial clinical data from Cohort 1 in the Phase 1/2 trial of PM359 for p47phox CGD in 2025. The initial readout will include safety and engraftment data, as well as key outcome measures, including the reconstitution of NADPH oxidase activity as measured by the DHR assay.

Wilson’s Disease:
•Advance PM577 through investigational new drug (IND)-enabling studies for the treatment of Wilson’s Disease patients with the most prevalent Wilson’s Disease mutation in the United States.
•File investigational new drug (IND) and/or clinical trial application (CTA) for PM577 in the first half of 2026.
AATD:
•Initiate IND-enabling studies for the treatment of AATD.
•File IND and/or CTA in mid-2026.
First Quarter 2025 Financial Results
•Research and Development (R&D) Expenses: R&D expenses were $40.6 million for the three months ended March 31, 2025, as compared to $37.8 million for the three months ended March 31, 2024. The increase in R&D expenses primarily due to the expansion and build out of our laboratory space at 60 First Street and 500 Arsenal Street.
•General and Administrative (G&A) Expenses: G&A expenses were $13.3 million for the three months ended March 31, 2025, as compared to $11.2 million for the three months ended March 31, 2024. The increase in G&A expenses was driven by personnel expenses.
•Net Loss: Net loss was $51.9 million for the three months ended March 31, 2025, as compared to $45.8 million for the three months ended March 31, 2024.
•Cash Position: As of March 31, 2025, cash, cash equivalents, investments, and restricted cash were $158.3 million, as compared to $204.5 million as of December 31, 2024.
Financial Guidance
Based on its current operating plans, Prime Medicine expects that its cash, cash equivalents and investments as of March 31, 2025 will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2026.

On May 8, 2025 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, reported that the third patient in Cohort 4 of its Phase 1a clinical trial has received their initial dose of TTX-MC138 (Press release, TransCode Therapeutics, MAY 8, 2025, View Source [SID1234652781]). All cohorts have enrolled at least three patients who have been dosed with TTX-MC138 at least once. The Safety Review Committee monitoring the clinical trial unanimously approved opening the fourth cohort based on its review of available safety and pharmacokinetic (PK) data. Additionally, the Safety Review Committee approved expanded enrollment in Cohort 3 to obtain additional safety data. To date, 15 patients have received at least one dose of TTX-MC138 at 4 separate dose levels ranging from 0.8 mg/kg to 4.8 mg/kg. Three patients have been treated in the expanded enrollment.

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Ten patients remain on study for continued treatment, receiving an additional dose of TTX-MC138 during each treatment cycle every 28 days, and may remain on study absent any significant safety observations or disease progression. Two patients who have remained on study the longest have received so far seven doses of TTX-MC138 over approximately seven months and have demonstrated stable disease. The 10 patients currently on study have shown no disease progression. Further, no significant safety or dose limiting toxicities have been reported in any of the trial’s 15 patients. Analysis of PK activity from Cohorts 1, 2 and 3 is ongoing and suggests that TTX-MC138 demonstrates a PK/PD profile consistent with preclinical results and results from TransCode’s Phase 0 clinical trial. Specifically, preliminary PK and pharmacodynamic (PD) data follow a predictable dose-response relationship. Analysis of PD activity from cycle 1 treatments in Cohorts 1 and 2, treated with doses of 0.8 mg/kg and 1.6 mg/kg, respectively, demonstrates miR-10b target engagement at 24 hours post-infusion.

The observed tolerability profile and the available PK/PD results thus far support advancement of the clinical trial to further evaluate safety and potential anti-tumor activity of TTX-MC138 in the planned dose expansion (Phase 1b) portion of the trial.

About TTX-MC138

TTX-MC138 is a first-in-class therapeutic candidate designed to inhibit microRNA-10b, or miR-10b, a microRNA widely believed to be critical to the emergence and progression of many metastatic cancers. TransCode’s Phase 0 clinical trial produced evidence of delivery of a radiolabeled version of TTX-MC138 to metastatic lesions and pharmacodynamic activity, even at a microdose of the drug candidate, suggesting a broad therapeutic window for TTX-MC138.

About the Trial

TransCode’s Phase 1 clinical trial is a multicenter, open-label, dose-escalation and dose-expansion study designed to generate critical data to support evaluation of the safety and tolerability of TTX-MC138 in patients with a variety of metastatic solid cancers. While not an endpoint, the trial may provide early evidence of clinical activity of TTX-MC138. The trial comprises an initial dose-escalation stage followed by a dose-expansion stage. The primary objective of the dose-escalation stage is to evaluate the safety and tolerability of escalating dose levels of TTX-MC138. In the dose-expansion stage, the safety, tolerability and anti-tumor activity of TTX-MC138 will be further evaluated in certain tumor types and at a certain dose level selected based on preliminary results from the dose-escalation phase.

Further information is available at www.clinicaltrials.gov NCT Identifier: (NCT06260774).

On May 8, 2025 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the first quarter of 2025 and provided a corporate update (Press release, Aclaris Therapeutics, MAY 8, 2025, View Source [SID1234652717]).

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"We are entering into a potentially transformative multi-year period for Aclaris, with important milestones throughout our business that we believe will position us for future growth," stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. "Ensuring successful and timely execution of our clinical programs is our priority, and given the realities of today’s financial market environment, it’s imperative that we continue to do so in a manner that efficiently utilizes our capital. As such, we’ve announced that further global development of bosakitug – our uniquely potent anti-TSLP monoclonal antibody – in respiratory indications will be dependent on partnerships. Regarding our internal programs, we expect to initiate new clinical trials with bosakitug; ATI-2138, our highly selective oral ITK/JAK3 inhibitor; and ATI-052, our potential best-in-class bispecific anti-TSLP/IL-4R antibody, for which we recently received IND clearance from the FDA. Work is also ongoing toward next-generation small molecule kinase inhibitors and biologic antagonists of immuno-inflammatory pathways to further expand our pipeline and continue our innovative work in I&I."

"Importantly, we have the cash we believe we need to execute our plan," continued Dr. Walker. "Our expected cash runway now extends through the first half of 2028, and we will continue to practice rigorous financial stewardship with a goal of extending our runway further by exploring additional non-dilutive opportunities."

First Quarter 2025 Highlights and Recent Updates

Pipeline:

Bosakitug (ATI-045): Investigational Anti-TSLP monoclonal antibody

Aclaris Expects to Initiate Enrollment in Placebo-Controlled Two-Arm Phase 2 Trial in Atopic Dermatitis (AD) in the Second Quarter of 2025: Based on the potency of bosakitug observed in preclinical and clinical studies to date, including the compelling results of the Phase 2a single arm trial in AD, Aclaris intends to initiate a two-arm placebo-controlled Phase 2 trial of bosakitug in approximately 90 patients with moderate-to-severe AD in the second quarter of 2025. This trial is designed to evaluate the Company’s anti-TSLP therapeutic in a placebo-controlled setting in a time- and cost-efficient manner.
Results from CTTQ’s Phase 2 Trials in Respiratory Indications Support Further Development: The totality of the results received to date from Aclaris’ regional partner, Chia Tai Tianqing Pharmaceutical Group, Co., Ltd. (CTTQ) from their Phase 2 trials of bosakitug in Chinese patients with chronic rhinosinusitis with nasal polyps (CRSwNP) and severe asthma provide additional clinical evidence of the enhanced potency of bosakitug. CTTQ has announced that they are conducting Phase 3 clinical trials of bosakitug in both indications and are currently conducting a Phase 2 trial in chronic obstructive pulmonary disease (COPD). CTTQ is responsible for the disclosure and presentation of the results of their clinical trials.
Aclaris Intends to Seek Partners to Develop Bosakitug in Respiratory Indications: Aclaris’ clinical focus for bosakitug will remain on dermatological immuno-inflammatory indications. Further global (excluding China) development in respiratory indications is dependent on entering into potential partnerships.

ATI-2138: Investigational oral covalent ITK/JAK3 inhibitor

Top Line Results from Phase 2a Trial in AD Expected in June 2025: Dosing is complete in the single arm Phase 2a open-label trial of ATI-2138 designed to investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of ATI-2138 in patients with moderate-to-severe AD. The Company expects to report top line results in June 2025. Potential target indications under consideration include alopecia areata (AA) and vitiligo.

ATI-052: Investigational bispecific anti-TSLP/IL4R monoclonal antibody

Announced Clearance of ATI-052 Investigational New Drug (IND) Application and Plan to Initiate Phase 1 Trial: Aclaris announced that the U.S. Food and Drug Administration cleared its IND application for ATI-052 in April 2025. The Company expects to initiate the placebo-controlled Phase 1a/1b trial evaluating single and multiple ascending doses of ATI-052, followed by a proof-of-concept portion in an undisclosed indication, in the second quarter of 2025. (press release here)

Next Generation Kinase and Cytokine Signaling Pathway Inhibitor Development:

Confirmed Plan for Next IND Submission: Preclinical work is ongoing to develop next-generation ITK inhibitors, which the Company expects to provide the basis for new INDs starting in 2026.
Development of Novel Bispecific Antibodies Targeting Certain Cytokine Pathways Underway: Preclinical work is ongoing to develop next-generation bispecific antibodies utilizing the bosakitug anti-TSLP binding region paired with binding fragments targeting other undisclosed cytokine signaling pathways.
Corporate:

Expected Cash Runway Extends Through the First Half of 2028 and Fully Funds Preclinical and Clinical Development Plans: During the first quarter of 2025, Aclaris took certain steps to focus its pipeline investments while extending its cash runway further. In doing so, the Company continues to prioritize clinical execution and achievement of development milestones, all of which Aclaris expects to be funded by the Company’s current cash runway. Aclaris ended the first quarter of 2025 with $190.5 million in cash, cash equivalents, and marketable securities providing balance sheet strength to execute on its development plan. Aclaris’ cash management policy is focused on capital preservation and holding a portfolio of securities with short-term maturities. The investment portfolio is composed of fixed income securities, primarily high-credit quality corporate debt and U.S. government securities.
Injunction Against Sun Pharmaceuticals Lifted, Providing Aclaris with a Potential Additional Opportunity for Non-Dilutive Financing: In 2023, Aclaris granted Sun Pharma exclusive rights under certain patents for, among other things, the use of deuruxolitinib, Sun Pharma’s JAK inhibitor, to treat AA. The agreement included an upfront payment of $15.0 million, regulatory and commercial milestones, and royalties. In 2024, Incyte Corporation was granted a preliminary injunction against Sun Pharma, blocking Sun Pharma from launching its product LEQSELVI. In April 2025, a U.S. Appeals Court lifted the injunction. As Aclaris has successfully executed similar financings in the past, the Company may seek to monetize this financial asset to provide additional non-dilutive financing.
Provided Update on Senior Leadership: Jesse W. Hall, M.D. has been appointed as Chief Medical Officer.
Dr. Hall brings decades of experience in all phases of drug development, from early development through global regulatory filings and approvals, Phase IV post-marketing surveillance obligations, and commercial launch support to Aclaris. He most recently served as Chief Medical Officer for AltruBio where he was responsible for leadership of all clinical and medical functions. (press release here)

First Quarter 2025 Financial Results

As of March 31, 2025, Aclaris had cash, cash equivalents and marketable securities of $190.5 million compared to $203.9 million as of December 31, 2024. The Company believes that its cash, cash equivalents and marketable securities will be sufficient to fund its operations through the first half of 2028, without giving effect to any potential business development transactions or financing activities.

Net loss was $15.1 million for the first quarter of 2025 compared to $16.9 million for the first quarter of 2024.
Total revenue was $1.5 million for the first quarter of 2025 compared to $2.4 million for the first quarter of 2024. The decrease was primarily driven by the sale of a portion of royalty payments under the Company’s agreement with Eli Lilly and Company to OCM IP Healthcare Portfolio IP, an investment vehicle for Ontario Municipal Employees Retirement System (OMERS), in July 2024.

Research and development (R&D) expenses were $11.6 million for the quarter ended March 31, 2025 compared to $9.8 million for the prior year period. The increase was primarily driven by expenses related to the Company’s bosakitug program, specifically preclinical and clinical development expenses associated with startup activities for a Phase 2 trial in AD. The increase was partially offset by a reduction in development expenses for zunsemetinib and lepzacitinib and lower compensation-related expenses.

General and administrative (G&A) expenses were $6.1 million for the quarter ended March 31, 2025 compared to $6.8 million for the corresponding prior year period. The decrease was primarily due to a reduction in personnel expenses as a result of lower headcount and lower termination benefits.