On February 26, 2026 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year ended 2025 and provided a corporate update.

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"2025 was a year of strong business execution and continued momentum in each of our potential best-in-class programs and positioned us for an exciting 2026 with important milestones and data readouts expected from each program," stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. "2026 started with derisking events for two of our key programs, ITK and ATI-052; this included compelling ATI-052 interim Phase 1a SAD/MAD results showing strong safety and tolerability profiles, robust target engagement reinforcing the potency of the compound even at very low doses, and the opportunity for extended dosing supported by dose proportional pharmacokinetic and pharmacodynamic profiles. Since then, we have initiated two Phase 1b POC trials with ATI-052, with top line results expected from both trials in the second half of this year. With a strong cash position and several meaningful catalysts across our biologics and ITK pipeline expected this year, including top line results from the Phase 2 trial of our anti-TSLP monoclonal antibody bosakitug in AD, we are looking forward to an exciting and productive year."

Fourth Quarter 2025 Highlights and Recent Updates

Pipeline:

Biologics: Antibody Franchise

Provided Positive Interim Results of Phase 1a Single (SAD) and Multiple Ascending Dose (MAD) Trial of Investigational Bispecific Anti-TSLP/IL-4Rα Antibody ATI-052; Complete Top Line Results from SAD and MAD Cohorts Expected in the Second Quarter of 2026: ATI-052 was well tolerated and demonstrated a favorable safety profile across all single and multiple ascending dose cohorts in this Phase 1a trial. Interim results included a dose proportional pharmacokinetic (PK) profile and concentration-dependent pharmacodynamics (PD) validating the potency and specificity of the compound, including robust target engagement and near complete target occupancy even at very low doses. These results support the potential for up to every three months dosing. Additional SAD and MAD results from this trial are expected in the second quarter of 2026. (press release here)
Announced Initiation of Two Phase 1b Proof-of-Concept (POC) Trials of ATI-052: Following positive interim Phase 1a SAD/MAD results, the Company has initiated Phase 1b POC studies in AD and asthma. Top line results from both trials are expected in the second half of 2026. (press releases here and here)
Planning Underway for Phase 2b Program for ATI-052: Planning is ongoing for a Phase 2b program encompassing asthma and AD as potential first indications. The Company expects to initiate this program in the second half of 2026.
Confirmed Expectation of Top Line Results in the Second Half of 2026 from Ongoing Phase 2 Trial of Investigational Anti-TSLP Monoclonal Antibody Bosakitug: This randomized, double-blind, placebo-controlled Phase 2 trial is designed to evaluate bosakitug in approximately 96 patients with moderate-to-severe AD.
Oral Inhibitors: ITK Franchise

Aclaris’ Lead ITK Inhibitor ATI-9494 Advancing Toward Expected Investigational New Drug (IND) Application in the Second Half of 2026: Aclaris’ lead ITK inhibitor candidate ATI-9494 has demonstrated potent blockade of Th1 and Th2 responses, a prolonged half-life, and high potency against ITK, potentially enabling low drug burden, dosing flexibility, and once daily (QD) administration across a broad range of disease indications. Aclaris intends to file an IND for ATI-9494 in the second half of 2026.
ATI-2138, a Potent and Selective Investigational Inhibitor of ITK and JAK3, Demonstrated Rapid and Sustained Hair Regrowth in Validated Murine Model of Severe Alopecia Areata (AA), Further Validating Best-in-Class Potential: ATI-2138 and ritlecitinib were assessed compared to control in a reversal model of murine alopecia universalis, the most severe AA phenotype. ATI-2138 demonstrated potential best-in-class results including rapid, near complete, and sustained hair regrowth compared to control and ritlecitinib including mean hair regrowth of 93% for ATI-2138 at week 6 (end of study) compared to 78% for the same dose of ritlecitinib. Mice receiving control showed no improvement in hair regrowth. The Company is completing the assessment of additional indications that are relevant to the dual pharmacology and mechanism of action, including certain alopecias and other inflammatory disorders. (press release here)
Financial Results

Liquidity and Capital Resources

As of December 31, 2025, Aclaris had cash, cash equivalents and marketable securities of $151.4 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 into the second half of 2028, without giving effect to any potential business development transactions or financing activities, or trial execution costs associated with its planned Phase 2b program for ATI-052.

Fourth Quarter and Full Year 2025

Net loss was $19.8 million for the fourth quarter of 2025 compared to $96.6 million for the fourth quarter of 2024. Net loss was $64.9 million for the year ended December 31, 2025 compared to $132.1 million for the year ended December 31, 2024.

Total revenue was $1.3 million for the fourth quarter of 2025 compared to $9.2 million for the fourth quarter of 2024. The decrease was primarily driven by the achievement of a commercial milestone under the license agreement with Eli Lilly and Company in the fourth quarter of 2024. Total revenue was $7.8 million for the year ended December 31, 2025 compared to $18.7 million for the year ended December 31, 2024.

Research and development (R&D) expenses were $16.6 million for the quarter ended December 31, 2025 compared to $9.0 million for the prior year period. The increase was primarily due to higher product candidate manufacturing costs and preclinical and clinical development expenses for bosakitug and ATI-052, and preclinical development expenses for ATI-9494. For the year ended December 31, 2025, R&D expenses were $52.6 million compared to $33.6 million for the year ended December 31, 2024.

General and administrative (G&A) expenses were $5.6 million for the quarter ended December 31, 2025 compared to $5.0 million for the prior year period. The increase was primarily due to higher compensation-related expenses and legal expenses. For the year ended December 31, 2025, G&A expenses were $22.0 million compared to $22.2 million for the year ended December 31, 2024.

Licensing expenses were $0.9 million for the quarter ended December 31, 2025 compared to $8.6 million for the prior year period. The decrease was primarily due to a milestone achieved during the fourth quarter of 2024, the entirety of which was payable to a third party. For the year ended December 31, 2025, licensing expenses were $5.2 million compared to $12.7 million for the year ended December 31, 2024.

Revaluation of contingent consideration resulted in a $0.4 million charge for the quarter ended December 31, 2025 compared to a $1.3 million gain for the prior year period. For the year ended December 31, 2025, revaluation of contingent consideration resulted in a charge of $2.3 million compared to a $2.5 million charge for the year ended December 31, 2024.

During the quarter and year ended December 31, 2024, the Company recorded $86.9 million of in-process research and development expenses, representing the fair value of consideration expensed in connection with the in-license of bosakitug (ATI-045) and ATI-052, as well as transaction costs incurred.

(Press release, Aclaris Therapeutics, FEB 26, 2026, View Source [SID1234663057])

On February 26, 2026 Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, reported financial results for the three and twelve months ended December 31, 2025, and highlighted recent corporate updates.

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"As demonstrated in today’s release, we are entering 2026 with exceptional momentum. The fourth quarter delivered the strongest results in the history of Heron’s Acute Care franchise, underscoring the success of the strategic decisions we implemented to unlock the full potential of these assets," said Craig Collard, Chief Executive Officer of Heron. "The milestones achieved in 2025, particularly for ZYNRELEF – including enhanced distributor‑partner incentives, the seamless completion of the Vial Access Needle transition, and CMS approval of a product‑specific J‑Code – are already accelerating adoption and strengthening our competitive position in a large and underpenetrated market."

"With a more powerful commercial engine, expanding demand signals, and improved reimbursement clarity, we believe Heron is well‑positioned for continued share gains and meaningful revenue expansion in 2026 and beyond."

Financial Guidance for 2026

Item
2026 Full-Year Guidance for Net Revenue and Adjusted EBITDA
(in millions)
Net Revenue $173 to $183 million
Adjusted EBITDA $10 to $20 million

Business Highlights

– Heron’s Acute Care franchise delivered revenue growth of 57.3% year-over-year in Q4 2025 and 65.1% year-over-year for 2025 compared to 2024, reflecting continued commercial acceleration.

– ZYNRELEF Updates:

The permanent, product specific J-Code (J0668) for ZYNRELEF, granted by the Centers for Medicare and Medicaid Services ("CMS"), was approved effective October 1, 2025 – streamlining reimbursement and improving billing clarity across payer types and settings of care.
Transition to the Vial Access Needle is complete, optimizing product preparation, handling, and operating field sterility with ZYNRELEF in hospitals and ambulatory surgical centers across the U.S.
Through aligned partnerships with leading distributors, we are broadening account access and elevating education around ZYNRELEF’s differentiated clinical profile, driving durable surgeon adoption and expansion of use.
Development of the proposed Prefilled Syringe market presentation is progressing and, if successful, FDA approval is anticipated in mid-to-late 2027.

– APONVIE Updates:

Inclusion of APONVIE (aprepitant) Injectable Emulsion in the Newly Released Fifth Consensus Guidelines for the Management of Postoperative Nausea and Vomiting ("PONV"), highlighting the clinical impact of aprepitant, use of multimodal PONV prophylaxis, and expanding recognition of the need for long-acting antiemetic coverage.
CMS has granted a permanent, product specific J-Code (J8502) for APONVIE.
Fully dedicated sales team, launched in Q3 2025, is gaining significant momentum in both expanding formulary access and driving successful utilization of APONVIE.

– Oncology Updates:

The Oncology franchise continues to deliver a strong revenue base, generating over $105 million in 2025 net revenue despite complex market dynamics.

– Cash, cash equivalents, and short-term investments were $46.6 million as of December 31, 2025.

Net Revenue Performance – Twelve Months Ended December 31 (in thousands)

2025 2024 Dollar Change Percentage Change

Acute Care $49,643 $30,064 $19,579 65.1%
APONVIE $11,571 $4,518 $7,053 156.1%
ZYNRELEF $38,072 $25,546 $12,526 49.0%

Oncology $105,261 $114,221 $(8,960) (7.8%)
CINVANTI $96,758 $100,079 $(3,321) (3.3%)
SUSTOL $8,503 $14,142 $(5,639) (39.9%)

Total Net Revenue $154,904 $144,285 $10,619 7.4%

Net Revenue Performance – Three Months Ended December 31 (in thousands)
(unaudited)

2025 2024 Dollar Change Percentage Change

Acute Care $16,344 $10,389 $5,955 57.3%
APONVIE $3,814 $1,932 $1,882 97.4%
ZYNRELEF $12,530 $8,457 $4,073 48.2%

Oncology $24,244 $30,392 $(6,148) (20.2%)
CINVANTI $22,917 $26,873 $(3,956) (14.7%)
SUSTOL $1,328 $3,519 $(2,191) (62.3%)

Total Net Revenue $40,588 $40,781 $(193) (0.5%)

Conference Call and Webcast

Heron will host a conference call and live webcast on Thursday, February 26, 2026, at 8:30 a.m. ET. The conference call can be accessed by phone by utilizing the following registration link which will provide participants with dial-in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. The investor presentation to be used for the conference call and webcast can be accessed from Heron’s website prior to the conference call and webcast. An archive of the teleconference, webcast, and investor presentation will also be made available on Heron’s website for sixty days following the call.

About ZYNRELEF for Postoperative Pain

ZYNRELEF is the first and only extended-release dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of nonsteroidal anti-inflammatory drug meloxicam. ZYNRELEF is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and significantly increased proportion of patients requiring no opioids through the first 72 hours following surgery compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. ZYNRELEF was initially approved by the FDA in May 2021 for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. In December 2021, the FDA approved an expansion of ZYNRELEF’s indication to include foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures. On January 23, 2024, the FDA approved ZYNRELEF for soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. Safety and efficacy have not been established in highly vascular surgeries, such as intrathoracic, large multilevel spinal, and head and neck procedures.

Please see full prescribing information, including Boxed Warning, at www.ZYNRELEF.com.

About APONVIE for Prevention of Postoperative Nausea and Vomiting ("PONV") Prevention

APONVIE is a substance P/neurokinin 1 (NK1) Receptor Antagonist (RA), indicated for the prevention of post operative nausea and vomiting (PONV) in adults. Delivered via a 30-second IV push, APONVIE 32 mg was demonstrated to be bioequivalent to oral aprepitant 40 mg with rapid achievement of therapeutic drug levels. APONVIE is the same formulation as Heron’s approved drug product CINVANTI. APONVIE is supplied in a single-dose vial that delivers the full 32 mg dose for PONV. APONVIE was approved by the FDA in September 2022 and became commercially available in the U.S. on March 6, 2023.

Please see full prescribing information at www.APONVIE.com.

About CINVANTI for Chemotherapy Induced Nausea and Vomiting (CINV) Prevention

CINVANTI, in combination with other antiemetic agents, is indicated in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC) including high-dose cisplatin as a single-dose regimen, delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC) as a single-dose regimen, and nausea and vomiting associated with initial and repeat courses of MEC as a 3-day regimen. CINVANTI is an IV formulation of aprepitant, an NK1 RA. CINVANTI is the first IV formulation to directly deliver aprepitant, the active ingredient in EMEND capsules. Aprepitant (including its prodrug, fosaprepitant) is a single-agent NK1 RA to significantly reduce nausea and vomiting in both the acute phase (0–24 hours after chemotherapy) and the delayed phase (24–120 hours after chemotherapy). The FDA-approved dosing administration included in the U.S. prescribing information for CINVANTI include 100 mg or 130 mg administered as a 30-minute IV infusion or a 2-minute IV injection.

Please see full prescribing information at www.CINVANTI.com.

About SUSTOL for CINV Prevention

SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-hydroxytryptamine type 3 RA that utilizes Heron’s Biochronomer drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical studies that evaluated SUSTOL’s efficacy and safety in more than 2,000 patients with cancer. SUSTOL’s efficacy in preventing nausea and vomiting was evaluated in both the acute phase (0–24 hours after chemotherapy) and delayed phase (24–120 hours after chemotherapy).

Please see full prescribing information at www.SUSTOL.com.

(Press release, Heron Therapeutics, FEB 26, 2026, View Source [SID1234663076])

On February 26, 2026 Johnson & Johnson (NYSE:JNJ) reported preliminary results from a Phase 1b study evaluating pasritamig (JNJ-78278343), a first-in-class bispecific T-cell engaging antibody, in combination with docetaxel in patients with metastatic castration-resistant prostate cancer. The combination demonstrated a safety profile consistent with docetaxel alone, with no new or unexpected safety signals observed. The regimen also showed clinically meaningful efficacy, including high rates of prostate-specific antigen (PSA) responses and sustained PSA reductions, supporting continued development and advancement into Phase 3 studies. The results were presented for the first time at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (Abstract #171).1

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Pasritamig is designed to engage the immune system through a novel mechanism of action, binding CD3 on T cells and human kallikrein 2 (KLK2). KLK2 is a novel, highly specific prostate cancer target with minimal expression outside prostate tissue. By both activating and directing T cells to KLK2-expressing tumor cells, pasritamig enables targeted immune engagement. This differentiated, prostate-specific approach was intentionally built to focus immune activity on prostate cancer cells, which may help limit effects on healthy tissue, and supports administration in a doctor’s office rather than hospital setting.

"These data represent an important step forward for patients with advanced prostate cancer," said Professor Shahneen Sandhu,* M.D., Ph.D., MBBS, FRACP, Associate Professor, Consultant Medical Oncologist and researcher at Peter MacCallum Cancer Centre, and study investigator. "In a disease where outcomes remain poor for many patients, seeing encouraging clinical activity alongside a favorable safety profile in combination with docetaxel reinforces the potential of this approach and supports further clinical development."

"Based on these findings, we are increasingly confident in the potential of pasritamig to meaningfully improve outcomes for people with prostate cancer," said Charles Drake, M.D., Ph.D., Vice President, Prostate Cancer and Cross Cancer Immuno-Oncology, Johnson & Johnson. "The ability to combine pasritamig with docetaxel, where prior approaches in the field have fallen short, gives us a strong foundation for Phase 3 development. What we’re seeing with this combination, including deep and durable PSA responses, underscores the promise of this combination immunotherapy approach and our commitment to advancing innovation that can make a difference for patients."

Detailed Study Results

In the study, pasritamig was evaluated in combination with docetaxel in an outpatient setting in patients with metastatic castration-resistant prostate cancer whose disease had progressed following androgen receptor pathway inhibitor therapy. Approximately half of the patients (45 percent) had received at least one prior taxane-based regimen. The primary endpoint was safety and identification of the recommended regimen for further development in Phase 2/3 studies, with secondary and exploratory endpoints assessing clinical activity, including PSA response rates.1

As of December 9, 2025, 51 patients had received pasritamig plus docetaxel, including patients who were pretreated with a median of three prior therapies (range, 1-9). Reductions of 50 percent or greater in PSA levels were achieved in 64.7 percent of patients overall and in 75.0 percent of taxane-naïve patients. Reductions of 90 percent or greater in PSA levels were achieved in 39.2 percent of patients overall and 53.6 percent of taxane-naïve patients. Among taxane-naïve patients with bone-only disease, confirmed PSA reductions of 50 percent or greater and 90 percent or greater were observed in 88.2 percent and 76.5 percent of patients, respectively. Patients were able to continue pasritamig beyond docetaxel discontinuation. Those patients received a median of six docetaxel doses every three weeks and eight pasritamig doses every six weeks, supporting the potential for sustained disease control over time.1

The safety profile of pasritamig plus docetaxel was consistent with the known safety profile of docetaxel in metastatic castration-resistant prostate cancer. The most common treatment-related adverse events (TRAEs) occurring in at least 20 percent of patients included fatigue (60.8 percent), alopecia (41.2 percent), diarrhea and nausea (31.4 percent each), peripheral edema (27.5 percent), peripheral sensory neuropathy (25.5 percent) and dysgeusia (23.5 percent). Pasritamig-related adverse events occurring in at least 10 percent of patients were fatigue (33.3 percent) and non-chronic diarrhea (11.8 percent). Grade 3 or higher TRAEs attributed to docetaxel were observed in 29.4 percent of patients, compared with only two percent attributed to pasritamig. No patients experienced cytokine release syndrome of any grade or treatment-related deaths.1

Two ongoing Phase 3 studies are evaluating pasritamig in the metastatic castration-resistant prostate cancer setting. KLK2-comPAS (NCT07164443) is evaluating pasritamig as monotherapy, and KLK2-PASenger (NCT07225946) is evaluating pasritamig in combination with docetaxel.2,3 Beyond these Phase 3 studies, pasritamig is also being evaluated in earlier-phase combination studies. Pasritamig monotherapy has received Breakthrough Therapy Designation in China and Fast Track designation from the U.S. Food and Drug Administration, supporting its continued clinical development.

About the Study

The Phase 1b study (NCT05818683) is an open-label trial evaluating the safety and clinical activity of pasritamig in combination with docetaxel in patients with metastatic castration-resistant prostate cancer (mCRPC) whose disease has progressed following treatment with an androgen receptor pathway inhibitor. The primary objective is to determine the recommended regimen for further development based on safety, with secondary and exploratory endpoints assessing clinical activity. Pasritamig was administered intravenously every six weeks, with initial step-up doses given during the first treatment cycle, in combination with docetaxel administered intravenously every three weeks. Treatment was delivered in an outpatient setting. Corticosteroids were used only as standard premedication for docetaxel, and hematopoietic growth factor support was permitted as needed.4

About Pasritamig (JNJ-78278343)

Pasritamig (JNJ-78278343) is an investigational T-cell-redirecting bispecific antibody (bsAb) targeting human kallikrein 2 (KLK2) on prostate cancer cells and CD3 receptor complexes on T cells, leveraging the body’s immune system to selectively target and eliminate cancer cells. This innovative approach is being evaluated in pretreated patients with metastatic castration-resistant prostate cancer (mCRPC), a patient population with limited treatment options.

About Metastatic Castration-Resistant Prostate Cancer (mCRPC)

Metastatic castration-resistant prostate cancer (mCRPC) is a challenging and aggressive stage of prostate cancer where the disease progresses despite androgen deprivation therapy.5 Patients often experience metastasis to bones and lymph nodes, leading to poor outcomes and limited treatment options, including chemotherapy and second-line hormone therapies.6 The median overall survival ranges from 13.5 to 31.6 months depending on the site of metastasis, with a typical range of 15 to 36 months across the broader population.7,8 Survival rates can vary significantly depending on factors such as prior treatment history, disease burden, and response to therapy. The need for more effective treatments is critical, as the disease continues to impact a large number of men globally, with metastatic castration-resistant prostate cancer (mCRPC) being responsible for a substantial number of prostate cancer-related deaths.

(Press release, Johnson & Johnson, FEB 26, 2026, View Source;johnson-show-promising-antitumor-activity-with-combination-of-pasritamig-and-docetaxel-in-advanced-prostate-cancer-302698631.html [SID1234663092])

On February 26, 2026 PMV Pharmaceuticals, Inc. ("PMV Pharma" or the "Company"; Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule therapies targeting p53, reported that results from the Phase 1, first-in-human portion of the ongoing Phase 1/2 PYNNACLE study evaluating rezatapopt in patients with advanced solid tumors harboring a TP53 Y220C mutation were published in the New England Journal of Medicine (NEJM). The publication provides a summary of the Phase 1 safety and efficacy results across 77 patients.

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The study published in NEJM entitled, "Phase 1 Study of Rezatapopt, a p53 Reactivator, in TP53 Y220C-Mutated Tumors," highlighted the antitumor activity of rezatapopt in heavily pretreated patients across multiple solid tumor-types establishing proof-of-concept for p53 reactivation. All responding patients had a TP53 Y220C mutation and were KRAS wild-type. The manuscript can be accessed here.

In the Phase 1 portion of the PYNNACLE clinical trial, 77 heavily pretreated patients with advanced solid tumors harboring a TP53 Y220C mutation received oral rezatapopt across dose-escalation cohorts to determine the maximum tolerated dose and recommended Phase 2 dose (RP2D), characterize safety, pharmacokinetics, and biomarker effects. Rezatapopt was generally well tolerated; dose-limiting toxicities were infrequent, supporting selection of the RP2D. Objective responses were observed across multiple tumor types. Clinical activity and biomarker data were consistent with selective binding to the Y220C pocket and restoration of wild-type p53 tumor suppressor function.

"Publication of the rezatapopt Phase 1 results in the New England Journal of Medicine underscores the emerging clinical impact of reactivating p53 in patients whose cancers are driven by a TP53 Y220C mutation," said Deepika Jalota, Pharm.D., Chief Development Officer of PMV Pharma. "These peer-reviewed findings further validate our scientific approach, support our registrational Phase 2 strategy and our plan to submit a New Drug Application in platinum-resistant/refractory ovarian cancer in the first quarter of 2027. We remain focused on advancing rezatapopt as a potential first-in-class therapy for ovarian cancer patients harboring a TP53 Y220C mutation, an area of high unmet medical need."

In October 2025, the Company presented updated interim clinical results from the Phase 2 pivotal portion of the PYNNACLE study, highlighting confirmed responses in patients whose tumors were TP53 Y220C mutated and KRAS wild-type across eight tumor types, including ovarian, lung, breast, endometrial, head and neck, colorectal, gallbladder cancers, and ampullary carcinoma. As of the September 4, 2025 data cutoff date, the overall response rate (ORR) across all cohorts was 34% (35/103 patients), with an ORR of 46% (22/48 patients) in ovarian cancer per investigator assessment according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, including confirmed and unconfirmed responses. Across all cohorts, the median time to response was 1.3 months, and the median duration of response was 7.6 months. In the ovarian cancer cohort, the median time to response was 1.3 months and the median duration of response was 8.0 months.

About Rezatapopt
Rezatapopt (PC14586) is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the pocket in the p53 Y220C mutant protein, restoring the wild-type tumor-suppressor function. The U.S. Food and Drug Administration granted Fast Track designation to rezatapopt for the treatment of patients with locally advanced or metastatic solid tumors with a p53 Y220C mutation.

About the PYNNACLE Clinical Trial
The ongoing Phase 1/2 PYNNACLE clinical trial is evaluating rezatapopt in patients with advanced solid tumors harboring a TP53 Y220C mutation. The primary objective of the Phase 1 portion of the clinical trial was to determine the maximum tolerated dose and recommended Phase 2 dose (RP2D) of rezatapopt when administered orally to patients. Safety, tolerability, pharmacokinetics and effects on biomarkers were also assessed. The Phase 2 portion is a registrational, single arm, expansion basket clinical trial comprising five cohorts (ovarian, lung, breast, and endometrial cancers, and other solid tumors) with the primary objective of evaluating the efficacy of rezatapopt at the RP2D in patients with TP53 Y220C and KRAS wild-type advanced solid tumors. For more information about the Phase 1/2 PYNNACLE clinical trial, refer to www.clinicaltrials.gov (NCT trial identifier NCT04585750).

(Press release, PMV Pharma, FEB 26, 2026, View Source [SID1234663108])

On February 26, 2026 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the fourth quarter and full year ended December 31, 2025 and recent business highlights related to the commercial launch of Vafseo (vadadustat) as well as its robust pipeline.

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"Vafseo commercial trends are showing marked improvement in early 2026, built on the solid foundation we created for the brand in its first year of launch," said John P. Butler, Chief Executive Officer of Akebia. "Patient access to Vafseo therapy now stands at 290,000 patients, and early data points to improved patient adherence rates, which we believe are enhanced as a result of dialysis organizations deciding to implement observed dosing protocols. These dynamics, along with the building evidence of clinical differentiation, are expected to drive significant Vafseo revenue growth in 2026 and beyond as we work toward our goal to make Vafseo standard of care for treating anemia due to CKD in patients on dialysis. Separately, we are advancing our pipeline in rare kidney disease and actively enrolling patients into the Phase 2 trial of praliciguat in FSGS. We expect to initiate an open-label rare kidney disease basket study with our tissue-targeted complement inhibitor, AKB-097, in the second half of 2026 with initial data expected in 2027. Through these efforts, we remain steadfast in working to better the lives of people impacted by kidney disease."

Vafseo Q4 2025 Commercial Updates

•Total number of prescribers increased to approximately 800 in Q4, representing an increase of 10% over the number of prescribers in Q3.
•Broadened customer base as approximately 25% of new patients in Q4 originated from dialysis organizations other than U.S. Renal Care (USRC), an increase from less than 10% in Q3.
•Patient demand was approximately $11 million. This approximately $1 million decline in demand versus Q3 was driven by fewer patient starts, which Akebia believes was a result of providers at select dialysis organizations anticipating the initiation of new in-center observed dosing protocols.
•Within centers that adopted an in-center observed dosing protocol in Q4, first refill adherence rates improved to 91% in Q4 from 75% in the first 9 months of 2025 with daily dosing.
2025 Vafseo Post Marketing Clinical Development Achievements

•In November at the American Society of Nephrology Kidney Week 2025, Dr. Glenn M. Chertow presented a post-hoc analysis of data from the INNO2VATE trials comparing dialysis patients taking vadadustat or darbepoetin alfa for CKD-related anemia. The data demonstrated statistically significant favorable outcomes in the hierarchical composite endpoint of all-cause mortality and hospitalization in patients treated with vadadustat compared to patients in the erythropoietin stimulating agent (ESA) control group.
•In July, enrolled the first patient in VOCAL, a Phase IIIb trial evaluating three times weekly (TIW) dosing of Vafseo versus ESAs, which is expected to report topline data in Q4 2026. The VOCAL trial of approximately 350 total patients also contains a sub-study of Vafseo’s impact on red blood cell characteristics.
•In June, USRC completed enrollment in VOICE, a large Phase IV trial of over 2,100 patients evaluating Vafseo TIW against standard-of-care ESAs using a hierarchical composite endpoint of all-cause mortality and all-cause hospitalization. VOICE topline results are expected in early 2027.

Rare Kidney Disease and Early-Stage Pipeline Progress

•Initiated a Phase 2 clinical trial of praliciguat, an oral, once-daily soluble guanylate cyclase (sGC) stimulator being evaluated for the treatment of biopsy-confirmed FSGS, a rare kidney disease, and dosed the first patient in December 2025. Akebia expects to enroll approximately 60 patients in this trial.
•In November 2025, Akebia acquired a humanized anti-C3d monoclonal antibody fusion protein from Q32 Bio Inc. which is designed to act as a complement inhibitor through a tissue-targeted mechanism. A Phase 2 open-label rare kidney disease basket study is expected to start in the second half of 2026 evaluating AKB-097 in IgA nephropathy, lupus nephritis and C3 glomerulopathy. The study will evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and effects on disease-relevant biomarkers, including proteinuria and measures of kidney function. Initial data is expected in 2027.

•Akebia plans to initiate a Phase 1 study of AKB-9090 in healthy volunteers. The study is expected to begin in the first half of 2026 with topline data expected by the end of the year. The initial target indication for AKB-9090 is the treatment of acute kidney injury associated with cardiac surgery.

Financial Results

•Revenues: Total revenues were $57.6 million in the fourth quarter of 2025 compared to $46.5 million in the fourth quarter of 2024, and $236.2 million for the full-year 2025 compared to $160.2 million for the full-year 2024. These increases were driven by sales of Vafseo, which was launched in the U.S. in January 2025, and an increase in Auryxia (ferric citrate) sales volumes.
◦Vafseo net product revenues were $6.2 million in the fourth quarter of 2025 and $45.8 million for the full-year 2025.

◦Auryxia net product revenues were $48.1 million in the fourth quarter of 2025 compared to $44.4 million in the fourth quarter of 2024, and $181.5 million for the full-year 2025 compared to $152.2 million for the full-year 2024. We expect generic competition for Auryxia to expand this year beyond the current authorized generic competition and therefore expect Auryxia revenues to decrease in 2026 as compared to 2025 Auryxia revenues.

◦License, collaboration and other revenues were $3.3 million in the fourth quarter of 2025 compared to $2.1 million in the fourth quarter of 2024, and $8.9 million for the full-year 2025 compared to $8.0 million for the full-year 2024.
•COGS: Cost of goods sold was $12.5 million in the fourth quarter of 2025 compared to $20.4 million in the fourth quarter of 2024, and $39.5 million for the full-year 2025 compared to $63.2 million for the full-year 2024. COGS in both periods was driven by higher Auryxia sales volumes in 2025, and was impacted by the elimination in 2025 of a quarterly $9.0 million non-cash intangible amortization charge that Akebia incurred through the fourth quarter of 2024. In addition, COGS for the full-year 2024 included a $12.3 million benefit due to our ability to sell inventory previously written-down as excess inventory. Of note, Vafseo-related COGS in both periods was derived from pre-launch inventory, which does not include the full cost of manufacturing as a portion of those inventory-related expenses were recorded as research and development expenses in the period incurred prior to Vafseo’s approval in the U.S.

•R&D Expenses: Research and development expenses were $26.6 million in the fourth quarter of 2025 compared to $11.8 million in the fourth quarter of 2024, and $62.4 million for the full-year 2025 compared to $37.7 million for the full-year 2024. The increase in expenses in both periods was driven by increased clinical trial activities related to Vafseo and our other product candidates, higher headcount related costs, as well as by a $12.8 million charge incurred during the fourth quarter of 2025 related to acquired in-process R&D costs associated with the acquisition of AKB-097 from Q32 Bio Inc.
•SG&A Expenses: Selling, general and administrative expenses were $26.1 million in the fourth quarter of 2025 compared to $27.7 million in the fourth quarter of 2024, and $107.5 million for the full-year 2025 compared to $106.5 million for the full-year 2024.
•Net Loss: Net loss was $12.2 million in the fourth quarter of 2025 compared to a net loss of $22.8 million in the fourth quarter of 2024. Net loss was $5.3 million for the full-year 2025 compared to $69.4 million for the full-year 2024. The decrease in net loss in both periods was driven by the increase in net product revenues, which was partially offset by higher expenses.

•Cash Position: Cash and cash equivalents as of December 31, 2025, were approximately $184.8 million compared to $51.9 million as of December 31, 2024. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan for at least two years.

Conference Call

Akebia will host a conference call on Thursday, February 26, 2026 at 8:00 a.m. EST to discuss fourth quarter and full year 2025 earnings. To access the call, please register by clicking on this Registration Link, and you will be provided with dial in details. To avoid delays and ensure timely connection, we encourage dialing into the conference call 15 minutes ahead of the scheduled start time.

A live webcast of the conference call will be available via the "Investors" section of Akebia’s website at: View Source/." target="_blank" title="View Source/." rel="nofollow">View Source An online archive of the webcast can be accessed via the Investors section of Akebia’s website at View Source approximately two hours after the event.

(Press release, Akebia, FEB 26, 2026, View Source [SID1234663058])