On February 26, 2026 OPKO Health, Inc. (NASDAQ: OPK) (OPKO) reported business highlights and financial results for the three and 12 months ended December 31, 2025, and introduces financial guidance for the first quarter and full year 2026.

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Highlights from the fourth quarter of 2025 and recent weeks include the following:

Entered into a research collaboration with Regeneron Pharmaceuticals to develop multispecific antibodies. This new partnership leverages ModeX’s MSTAR technology platform with Regeneron’s proprietary binders to develop single molecule candidates that target multiple distinct biological pathways in several clinical indications. ModeX received an upfront payment and is entitled to potential milestone payments exceeding $200 million for each program. The overall potential value of the collaboration exceeds $1 billion if multiple products from the collaboration are successful. In addition, ModeX is eligible to receive tiered royalties on global net sales, reaching low double digits at the highest tier. Regeneron is responsible for funding all preclinical and clinical development, as well as all commercialization activities.

Merck completed the Phase 1 Epstein-Barr virus vaccine trial (NCT06655324). This investigational vaccine candidate is being developed in collaboration with Merck. The Phase 1 trial evaluating safety and tolerability in over 200 healthy adults was completed in the fourth quarter of 2025. Additional studies are in progress to inform dose and adjuvant selection for potential Phase 2 studies.

MDX2003, a first-in-class trispecific T-cell engager-expander for the treatment of leukemia and lymphoma, was approved for Phase 1 studies in Australia, which are expected to begin in the first half of 2026. Also, an abstract was presented at 2025 ASH (Free ASH Whitepaper) Annual Meeting. In November, an abstract titled "MDX2003, a novel tetraspecific T cell engager-expander targeting CD19xCD20xCD3xCD28, demonstrates potent preclinical activity against B cell malignancies" was presented at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s 67th ASH (Free ASH Whitepaper) Annual Meeting and Exposition. In an animal model, MDX2003 prevented further tumor growth at low doses and was well-tolerated with low levels of cytokines in the blood and no observable toxicity.

Abstract for MDX2004, a first-in-class trispecific antibody-fusion protein immune rejuvenator, was presented at SITC (Free SITC Whitepaper) 2025. In November, an abstract titled "MDX2004, a novel immune rejuvenator targeting CD3, CD28, and 4–1BB, augments tumor immunity in preclinical animal models" was presented at SITC (Free SITC Whitepaper) 2025, the annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper). The ongoing Phase 1/2a study (NCT07110584) is designed to evaluate the safety, tolerability and biologic activity of MDX2004 as an immunotherapy for advanced cancers.

Abstract for MDX2001 cMet-Trop2/CD3-CD28, a first-in-class tetraspecific T-cell engager, was presented at ESMO (Free ESMO Whitepaper) 2025. In October, an abstract titled "A phase I/IIa, multicenter, first-in-human, open-label clinical trial evaluating MDX2001, a tetraspecific T cell engager-expander in patients with advanced solid tumors" was presented at ESMO (Free ESMO Whitepaper) Congress 2025, the annual meeting of the European Society for Medical Oncology. The MDX2001 cMet-Trop2/CD3-CD28 tetraspecific antibody has advanced to the fifth dose level in its Phase 1 clinical trial, with Phase 1b studies in select solid tumors expected to begin in the first half of 2026.

FDA permission to proceed to Phase 1 granted to MDX2301, a tetravalent bispecific antibody that neutralizes all known strains of SARS-CoV2, for the prevention and treatment of COVID-19 infection. Supported by non-dilutive funding from BARDA, the clinical trial in healthy volunteers is scheduled to begin in the second quarter of 2026, with pharmacokinetic and immunogenicity data expected later this year.

Expanded partnership with Entera Bio to advance first-in-class oral long-acting PTH tablet for patients with hypoparathyroidism. This third program under the collaboration combines OPKO’s proprietary long-acting PTH variants with Entera’s proprietary N-Tab technology. Following favorable pharmacodynamic and pharmacokinetic data reported in December 2025, the companies have jointly decided to accelerate development and expect to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) in late 2026. OPKO and Entera Bio each hold a 50% pro-rata ownership interest in the long-acting PTH hypoparathyroidism program and each is responsible for 50% of the program’s development costs.

Fourth Quarter Financial Results

Consolidated: Consolidated total revenues for the fourth quarter of 2025 were $148.5 million compared with $183.6 million for the 2024 period, with the decrease principally resulting from the sale of certain BioReference assets in 2025. Operating loss for the fourth quarter of 2025 was $38.3 million compared with operating loss of $33.1 million for the 2024 quarter. Net loss for the fourth quarter of 2025 was $31.3 million, or $0.04 per share, compared with net income of $14.0 million, or $0.01 per diluted share, for the 2024 quarter. Net income for the fourth quarter of 2024 included a realized gain of $54.1 million from the sale of shares of GeneDx, as well as non-cash other income of $21.4 million.
Pharmaceuticals: Revenue from products in the fourth quarter of 2025 was $43.7 million compared with $37.4 million in the fourth quarter of 2024, driven by a positive net foreign exchange impact of $4.0 million and by higher sales volumes in our international operations. Revenue from Rayaldee was $8.8 million compared with $9.1 million in the comparable prior-year quarter. Revenue from the transfer of intellectual property and other was $33.7 million in the fourth quarter of 2025 compared with $43.1 million in the 2024 period. Gross profit share payments for NGENLA, which totaled $12.5 million in the 2025 period compared with $9.6 million in the 2024 period, reflect the global commercial progress by Pfizer. In addition, the fourth quarter of 2025 included $4.3 million in royalty revenue from Eli Lilly following the commercial launch of mazdutide in China by their partner Innovent Biologics and a $7.0 million upfront payment from Regeneron. The comparable period of 2024 included a $12.5 million milestone payment from Merck, as well as a $7.0 million decrease in commercial milestones at our Eirgen business and a $4.1 million decrease in revenue from our contract with BARDA. Total costs and expenses increased to $88.0 million in the fourth quarter of 2025 from $82.6 million in the prior-year period, primarily due to higher cost of revenue related to higher sales volume, higher research and development expenses driven by advancements in early-stage programs and employee-related expenses reflecting an increase in headcount to support ongoing clinical activities. Operating loss was $10.7 million in the fourth quarter of 2025, which included $18.3 million in depreciation and amortization expense, compared with operating loss of $2.1 million in the fourth quarter of 2024, which included $18.1 million of depreciation and amortization expense.

Diagnostics: Revenue from services in the fourth quarter of 2025 was $71.1 million compared with $103.1 million in the prior-year period. The decrease was principally a result of the sale of certain BioReference assets in 2025 and the resulting decline in clinical test volume, and lower clinical test reimbursement rates, partially offset by increased demand and higher average reimbursement for the 4Kscore test, resulting in a 16% increase in revenue to $7.0 million in the 2025 quarter compared with $6.0 million in revenue in the 2024 quarter. Total costs and expenses were $89.4 million in the fourth quarter of 2025 compared with $124.8 million in the fourth quarter of 2024. The decrease was primarily attributable to the assets sold and to continued cost-reduction initiatives at BioReference. Operating loss was $18.3 million in the fourth quarter of 2025, which included $4.1 million of depreciation and amortization expense, compared with operating loss of $21.7 million in the 2024 period, which included $6.0 million of depreciation and amortization expense. The fourth quarter of 2025 was impacted by non-recurring transition-related adjustments of $5.8 million, primarily from severance, asset write-offs, third-party revenue adjustments, and capital tax expense. The fourth quarter of 2024 included revenue of $26.3 million and costs and expenses of $32.9 million from the oncology assets that were sold to Labcorp on September 15, 2025.

Cash, cash equivalents, marketable securities and restricted cash: Cash, cash equivalents and restricted cash were $369.1 million as of December 31, 2025. As of December 31, 2025, approximately $87.3 million of OPKO’s common stock had been repurchased, including $13.5 million in the fourth quarter of 2025, under the program since its authorization in July 2024. Approximately $112.7 million remained authorized and available for future repurchases.

2026 Financial Guidance: The table below reflects financial guidance for the first quarter and full year 2026 (in millions):

For the three months ended March 31, 2026 For the year ended December 31, 2026
Low High Low High
Revenue
Services revenue $ 71 $ 75 $ 300 $ 312
Product revenue 38 45 160 170
IP and other revenue 15 20 70 80
Total revenues 125 140 530 560

Included in revenue
Pfizer profit share 5 6 34 37
BARDA contract 7 9 18 22

Total costs and expenses 170 180 725 750

R&D included in costs and expenses 30 32 125 135

Conference Call and Webcast Information

OPKO’s senior management will provide a business update, discuss fourth quarter financial results, provide financial guidance and answer questions during a conference call and live audio webcast today beginning at 4:30 p.m. Eastern time. Participants are encouraged to pre-register for the conference call here. Callers who pre-register will receive a unique PIN to gain immediate access to the call and bypass the live operator. Participants may register at any time, including up to and after the call start time. Those unable to pre-register may participate by dialing 833-630-0584 (U.S.) or 412-317-1815 (International). A webcast of the call can also be accessed at OPKO’s Investor Relations page and here.

A telephone replay will be available until March 5, 2026, by dialing 855-669-9658 (U.S.) or 412-317-0088 (International) and providing the passcode 2367034. A webcast replay will be available beginning approximately one hour after the completion of the live conference call here.

(Press release, Opko Health, FEB 26, 2026, View Source [SID1234663084])

On February 26, 2026 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported pipeline progress, reiterated key anticipated milestones, and announced fourth quarter and full year 2025 financial results.

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"As we advance toward the culmination of our OnTarget 2026 operating plan with a first potential FDA approval targeted for later this year, our focus remains on applying the disciplined, patient-centric approach that has enabled rapid progress in discovery and development across our pipeline towards building the capabilities needed to effectively deliver new medicines to patients," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "We are executing against our initial registration paths for zidesamtinib and neladalkib in TKI pre-treated patients and are well underway with launch readiness efforts to ensure we have the commercial infrastructure in place to deliver these medicines to patients, if approved. In parallel, we continue to pursue label expansion opportunities for TKI-naïve patients towards our goal of bringing new therapies to all patients with ROS1-positive or ALK-positive NSCLC, and advance our earlier-stage and discovery programs. With a steady cadence of anticipated milestones across our pipeline in 2026 and a strong balance sheet, we believe we are positioned to become an enduring leader in precision oncology across the full continuum of discovery, development, and delivery, built to serve patients for years to come."

Recent Pipeline Achievements and Anticipated 2026 Milestones

ROS1 Program

•
The U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for zidesamtinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who received at least 1 prior ROS1 tyrosine kinase inhibitor (TKI), and assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 18, 2026. Pending FDA review, Nuvalent anticipates U.S. commercial launch of zidesamtinib in 2026.
•
Nuvalent plans to submit data to the FDA to support a potential label expansion of zidesamtinib in TKI-naïve patients with advanced ROS1-positive NSCLC in the second half of 2026.

ALK Program

•
Nuvalent completed its pre-NDA meeting with the FDA and aligned on a submission strategy for neladalkib in TKI pre-treated ALK-positive NSCLC. The company plans to move forward with an NDA submission of the data for TKI pre-treated patients with advanced ALK-positive NSCLC from the ALKOVE-1 study of neladalkib in the first half of 2026.
•
Enrollment is ongoing in ALKAZAR, the company’s global Phase 3 randomized, controlled trial designed to evaluate neladalkib for the treatment of TKI-naïve patients with advanced ALK-positive NSCLC. Patients are randomized 1:1 to receive neladalkib or alectinib, a front-line standard of care, reflecting input from collaborating physician-scientists and alignment with global regulatory agencies. The company expects to continue to progress the ALKAZAR trial throughout 2026.

HER2 Program

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Enrollment is ongoing in the HEROEX-1 Phase 1a/1b clinical trial evaluating the overall safety and tolerability of NVL-330 for pre-treated patients with HER2-altered NSCLC. Additional objectives include determination of the recommended Phase 2 dose, characterization of NVL-330’s pharmacokinetic profile, and preliminary evaluation of anti-tumor activity. The company expects to continue to progress the HEROEX-1 trial throughout 2026.

Discovery Research Programs

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Nuvalent continues to advance its discovery research programs and expects to disclose a new development candidate by year-end 2026.

Business Highlights

•
Completed Successful Public Offering of Common Stock Raising $500.0 Million in Gross Proceeds: On November 20, 2025, Nuvalent closed an underwritten public offering of 4,950,496 shares of Class A common stock at a price to the public of $101.00 per share. The gross proceeds to Nuvalent from the offering were approximately $500.0 million, before deducting underwriting discounts and commissions and other offering expenses.
•
Ron Squarer Appointed to Board of Directors: Nuvalent appointed Ron Squarer to its board of directors in December 2025. Mr. Squarer brings more than 30 years of proven leadership in oncology drug development and commercialization to the Nuvalent Board.

Upcoming Events

•
TD Cowen 46thAnnual Health Care Conference in Boston: Management will be participating in a fireside chat on Wednesday, March 4, 2026, at 9:45 a.m. ET.
•
Leerink Global Healthcare Conference 2026 in Miami: Management will be participating in a fireside chat on Monday, March 9, 2026, at 2:20 p.m. ET.
Live webcasts of the fireside chats will be available in the Investors section of Nuvalent’s website at www.nuvalent.com, and will be archived for 30 days following each conference.

Fourth Quarter and Full Year 2025 Financial Results

•
Cash Position: Cash, cash equivalents and marketable securities were $1.4 billion as of December 31, 2025. Nuvalent continues to believe that its existing cash, cash equivalents and marketable securities will be sufficient to fund its operations into 2029.
•
R&D Expenses: Research and development (R&D) expenses were $67.8 million for the fourth quarter of 2025 and $307.0 million for the year ended December 31, 2025.
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G&A Expenses: General and administrative (G&A) expenses were $34.4 million for the fourth quarter of 2025 and $107.3 million for the year ended December 31, 2025.
•
Net Loss: Net loss was $118.7 million for the fourth quarter of 2025 and $425.4 million for the year ended December 31, 2025.

(Press release, Nuvalent, FEB 26, 2026, View Source [SID1234663083])

On February 26, 2026 Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) reported financial results for the three and twelve months ended December 31, 2025, and provided an operating forecast and business update. Ligand management will host a conference call and webcast today beginning at 8:30 a.m. Eastern time to discuss this announcement and answer questions.

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"We delivered strong fourth quarter results, exceeding our initial full-year adjusted EPS guidance by approximately 30%. This growth was driven by better-than-expected performance across several products in our royalty portfolio, including the successful out-licensing and partner launch of Zelsuvmi. These results highlight the strength of our team and their ability to invest in high-value assets that address significant unmet clinical needs. As we enter 2026, we have a strong balance sheet and are well positioned to execute on our broad pipeline of investment opportunities that we believe position us to continue driving growth and creating long-term shareholder value," said Todd Davis, CEO of Ligand.

Fourth Quarter 2025 Financial Results

Total revenues and income for the fourth quarter of 2025 were $59.7 million, compared with $42.8 million for the same period in 2024, with the 39% increase driven primarily by royalty revenue. Royalties for the fourth quarter of 2025 were $50.5 million, compared with $34.8 million for the same period in 2024, with the 45% increase primarily attributable to increases in sales of Travere Therapeutics’ Filspari, and Merck’s Capvaxive and Ohtuvayre. Captisol sales were $7.8 million for the fourth quarter of 2025, compared with $7.9 million for the same period in 2024. Contract revenue and income was $1.3 million for the fourth quarter of 2025, compared with $0.1 million for the same period in 2024.
Cost of Captisol was $3.0 million for the fourth quarter of 2025, compared with $2.8 million for the same period in 2024. Amortization of intangibles was $8.1 million for the fourth quarter of 2025, compared with $8.3 million for the same quarter in 2024. Research and development expenses were $3.5 million for the fourth quarter of 2025, compared with $4.4 million for the same period in 2024. General and administrative expenses were $25.0 million for the fourth quarter of 2025, compared with $25.6 million for the same period in 2024. Financial royalty asset impairment was $6.2 million for the fourth quarter of 2025 related to Agenus returned partner programs, compared to $4.1 million for the same period in 2024 related to the discontinuation of Takeda’s soticlestat program. There was no fair value adjustment to partner program derivatives for the fourth quarter of 2025. Fair value adjustment to partner program derivatives was $7.2 million for the fourth quarter of 2024 primarily due to the discontinued development of certain Agenus partnered programs.
GAAP net income was $44.8 million, or $2.12 per diluted share for the fourth quarter of 2025, compared with a net loss of $31.1 million, or $1.64 per share, for the same period in 2024. GAAP net income for the fourth quarter of 2025 included a gain of $22.1 million from our short-term investments. Adjusted net income for the fourth quarter
1 A reconciliation of forward‑looking non‑GAAP core adjusted earnings per diluted share to the most directly comparable GAAP measures was provided in the Company’s Investor Day presentation on December 9, 2025, which is available on the Company’s investor relations website. The Company is reiterating that guidance in this release and has not updated the underlying assumptions reflected in that reconciliation

of 2025 was $42.7 million, or $2.02 per diluted share, compared with $25.2 million, or $1.27 per diluted share, for the same period in 2024. The increase in adjusted net income was driven primarily by the 45% increase in royalty revenue. See the table below for a reconciliation of net income (loss) to adjusted net income.
As of December 31, 2025, Ligand had cash, cash equivalents and short-term investments of $733.5 million.
Full Year 2025 Financial Results
Total revenues and income for the full year 2025 were $268.1 million, compared with $167.1 million for the full year 2024. Royalties for the full year 2025 were $161.0 million, compared with $108.8 million for the full year 2024, with the increase primarily attributable to increases in sales of Travere Therapeutics’ Filspari, Recordati’s Qarziba, and Merck’s Capvaxive and Ohtuvayre. Captisol sales were $40.2 million for full year 2025, compared with $30.9 million for the full year 2024, with the increase due to the timing of customer orders. Contract revenue and income was $66.9 million for the full year 2025, compared with $27.5 million for the full year 2024, with income from the Pelthos Therapeutics spin-out transaction being the main driver of the increase, partially offset by 2024 Ohtuvayre approval and commercial launch milestone payments.
Cost of Captisol was $14.5 million for the full year 2025, compared with $11.1 million for the full year 2024, with the increase due to higher Captisol sales. Amortization of intangibles was $32.7 million for the full year 2025, compared with $33.0 million for the full year 2024. Research and development expenses were $81.2 million for the full year 2025, compared with $21.4 million for the full year 2024, with the increase primarily attributable to a $44.3 million one-time charge in connection with the royalty financing agreement with Castle Creek Biosciences to fund the Phase 3 clinical study of D-Fi (FCX-007) and a $17.8 million one-time charge in connection with the royalty financing agreement with Orchestra BioMed to fund its late-stage partnered cardiology programs, which were accounted for as research and development funding arrangements under ASC 730-20. General and administrative expenses were $92.4 million for the full year 2025, compared with $78.7 million for the full year 2024. This increase was primarily driven by higher stock-based compensation expenses associated with the vesting of performance stock unit awards, investments made in scaling the Company’s business development function and Pelthos transaction costs. Financial royalty asset impairment was $6.2 million for the full year 2025 related to the Agenus returned partnered programs, compared to $30.6 million for the full year 2024, primarily due to the impairment loss related to the discontinuation of Takeda’s soticlestat program. There was no fair value adjustment to partner program derivatives for the full year 2025. Fair value adjustment to partner program derivatives was $15.1 million for the full year 2024 primarily due to the discontinued development of certain Agenus partnered programs.
GAAP net income was $124.5 million, or $6.13 per diluted share, for the full year 2025, compared to a net loss of $4.0 million, or $0.22 per share, for the full year 2024. Core adjusted net income for the full year 2025 was $165.1 million, or $8.13 per diluted share, compared with core adjusted net income of $108.5 million, or $5.74 per diluted share, for the full year 2024. The increase in core adjusted net income in 2025 was driven primarily by increases in royalty revenue and Zelsuvmi out-license income. Core adjusted net income represents a non-GAAP financial measure. See the table below for a reconciliation of net income (loss) to core adjusted net income.
2026 Financial Guidance
Ligand is reaffirming its 2026 financial guidance introduced at the Company’s Investor Day on December 9, 2025. The Company continues to expect adjusted earnings per diluted share1 of approximately $8.00 to $9.00. Ligand also expects 2026 royalty revenue to be in the range of $200 million to $225 million, revenue from sales of Captisol to be in the range of $35 million to $40 million and contract revenue to be in the range of $10 million to $20 million, resulting in total revenue of $245 million to $285 million.
Fourth Quarter 2025 and Recent Business Highlights
Ohtuvayre
•On January 27, 2026, Nuance Pharma announced that the National Medical Products Administration (NMPA) of China has officially accepted for review the New Drug Application (NDA) for Ohtuvayre (ensifentrine) for the maintenance treatment of chronic obstructive pulmonary disease.
Filspari

•On November 26, 2025, Renalys Pharma, Inc., now Chugai Pharmaceuticals, announced positive topline results from its Phase 3 clinical study of sparsentan, an orally administered dual endothelin and angiotensin II receptor antagonist, in Japanese patients with IgA nephropathy (IgAN). Based on these results, Chugai plans to submit a NDA in Japan in 2026.
•On January 13, 2026, Travere announced the U.S. Food and Drug Administration (FDA) extended the review timeline of its supplemental New Drug Application (sNDA) for Filspari in focal segmental glomerulosclerosis (FSGS). The new Prescription Drug User Fee Act (PDUFA) target action date is April 13, 2026.
•On February 19, 2026, Travere announced total U.S. Filspari net product sales for the fourth quarter of 2025 to be $103 million, representing 108% growth compared to the prior year period. U.S. Filspari new patient start forms reached an all time high of 908 in the fourth quarter of 2025.
Qtorin Rapamycin
•On December 15, 2025, Palvella announced positive topline results from its Phase 2 TOIVA study of Qtorin 3.9% rapamycin anhydrous gel for the treatment of cutaneous venous malformations (cutaneous VMs). The trial achieved statistical significance on multiple pre-specified clinician-reported and patient-reported efficacy endpoints, including dynamic change endpoints and static severity endpoints and was well-tolerated, with no drug-related serious adverse events reported.
•On December 16, 2025, Palvella announced that the FDA granted Fast Track Designation to Qtorin rapamycin for the treatment of angiokeratomas. Angiokeratomas are characterized by lymphatic-derived skin lesions that can persistently bleed and significantly impact quality of life. There are currently no FDA-approved therapies in existence for the estimated 50,000 diagnosed U.S. patients. With Fast Track designation, Qtorin rapamycin for angiokeratomas may be eligible for Accelerated Approval and Priority Review in the future, if applicable criteria are met. Palvella plans to initiate a Phase 2 trial evaluating Qtorin rapamycin for clinically significant angiokeratomas in the second half of 2026.
•On January 9, 2026, Palvella provided a corporate update and 2026 outlook providing the following Qtorin rapamycin updates:
◦Palvella is accelerating U.S. launch readiness for Qtorin rapamycin for microcystic LMs which has the potential to become the first FDA-approved therapy and a first-line, standard-of-care treatment for this serious, lifelong disease affecting an estimated more than 30,000 diagnosed patients in the U.S.
◦Following positive Phase 2 results for Qtorin rapamycin for the treatment of cutaneous venous malformations announced in December 2025, requested a Preliminary Breakthrough Therapy Designation Advice meeting with the FDA, anticipated in the first quarter of 2026
•On February 24, 2026, Palvella announced positive topline results from its Phase 3 SELVA study of Qtorin rapamycin for the treatment of microcystic LMs. The Phase 3 trial met its primary endpoint with statistically significant improvement on the Microcystic LM Investigator Global Assessment and achieved statistical significance on its pre-specified key secondary endpoint and all four secondary efficacy endpoints. Qtorin rapamycin was well tolerated, with no drug-related serious adverse events reported and systemic rapamycin levels below 2 ng/mL at all timepoints for all participants. 98% of participants who completed the efficacy evaluation period elected to continue to receive Qtorin rapamycin in the ongoing treatment extension period. An NDA submission is planned for the second half of 2026.
Tzield/Teizeild
•On October 20, 2025, Sanofi announced the FDA accepted for expedited review the sBLA for Tzield to delay the progression of stage 3 type 1 diabetes (T1D) in adults and pediatric patients eight years of age and older recently diagnosed with stage 3 T1D. The FDA nominated Tzield for the Commissioner’s National Priority Voucher (CNPV) pilot program based on its potential to address a large unmet medical need. Sanofi expects a regulatory decision from the FDA in the first half of 2026.

•On January 5, 2026, Sanofi announced the FDA accepted for priority review the sBLA for Tzield to expand the current age indication from eight years and above, to as young as one year old and above to delay the onset of stage 3 T1D in patients diagnosed with stage 2 T1D. The sBLA is supported by the positive interim one-year data from the ongoing PETITE-T1D phase 4 study. The target action date for the FDA decision is April 29, 2026.
•On January 12, 2026, Sanofi announced the European Commission has approved Teizeild (teplizumab) to delay the onset of stage 3 type 1 diabetes (T1D) in adult and pediatric patients eight years of age and older with stage 2 T1D. The approval is based on positive results from the TN-10 phase 2 study demonstrating that Teizeild delayed the onset of stage 3 T1D by a median of two years compared to placebo.
Other Program Updates
•On November 6, 2025, UroGen announced it made the strategic decision to discontinue development of UGN-301 (zalifrelimab) following completion of its Phase 1 dose escalation study and provided notice of termination to Agenus. While the study confirmed proof of concept, UGN-301’s overall clinical profile did not meet UroGen’s internal benchmarks for advancement to Phase 2.
•On November 7, 2025, Pelthos Therapeutics announced it acquired U.S. commercialization rights to Xepi (ozenoxacin) Cream, 1%. Xepi is a non-fluorinated quinolone antimicrobial indicated for the topical treatment of impetigo due to Staphylococcus aureus or Streptococcus pyogenes in adult and pediatric patients two months of age and older. Ligand is entitled to a low single-digit royalty on net sales of Xepi.
•On December 3, 2025, Gilead announced that it provided 1,200 vials of its antiviral therapy, remdesivir, to the Ministry of Health of Ethiopia to help combat the country’s first outbreak of Marburg Virus Disease (MVD). Marburg Virus Disease is a rare but severe hemorrhagic fever with high mortality rates, requiring swift intervention to prevent further spread. Gilead is working closely with the Ministry of Health of Ethiopia to provide remdesivir for emergency use. Remdesivir is Captisol enabled and Ligand is entitled to revenue from material sales of Captisol for the use of remdesivir.
•On December 18, 2025, Athira, now Leona Bio, announced that it acquired the development and commercialization rights to lasofoxifene, a promising clinical asset in a potentially registrational Phase 3 trial for the treatment of metastatic breast cancer. The ongoing Phase 3 ELAINE-3 clinical trial is greater than 50% enrolled with data expected in mid-2027. Ligand is entitled to a tiered 6-10% royalty on future net sales of lasofoxifene.
•On January 15, 2026, Agenus announced the closing of its $141 million strategic collaboration with Zydus. The agreement is designed to accelerate global development and potential commercialization of Agenus’ botensilimab and balstilimab (BOT/BAL) immunotherapy combination program. The collaboration provides Agenus with strategic capital and committed, long-term biologics manufacturing capacity in the United States to support BOT/BAL clinical development, authorized early access pathways, and commercial supply preparation. Agenus is initiating a global Phase 3 registrational trial evaluating BOT/BAL in patients with refractory, unresectable microsatellite stable (MSS)/mismatch repair proficient (pMMR) colorectal cancer. The trial will enroll approximately 800 patients across more than 100 sites in Canada, France, Australia, and New Zealand.
Adjusted Financial Measures
Ligand reports adjusted net income from continuing operations, adjusted net income per diluted share and adjusted earnings per diluted share in addition to, and not as a substitute for, financial measures calculated in accordance with GAAP, and does not consider such measures superior to GAAP results. The Company also reports "core" versions of these measures, which exclude any realized gains from the sale of Viking Therapeutics common stock.
Adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to evaluate management performance and determine certain elements of management compensation. GAAP results include items such as share‑based compensation expense, amortization of acquisition‑related and intangible assets, changes in contingent liabilities, mark‑to‑market adjustments on investments in public companies, transaction‑related costs and related tax effects, which are excluded from adjusted results and are detailed in the reconciliations included at the end of this press release.

A reconciliation of forward‑looking non‑GAAP adjusted earnings per diluted share to the most directly comparable GAAP measures was provided in the Company’s Investor Day presentation on December 9, 2025, which is available on the Company’s investor relations website. The Company is reiterating that guidance in this release and has not updated the underlying assumptions reflected in that reconciliation.

Conference Call
Ligand management will host a conference call and webcast today beginning at 8:30 a.m. Eastern time (5:30 a.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 715-9871 (North America toll-free number) using the conference ID 3661098. International participants outside of Canada may use the toll number (646) 307-1963 and use the same conference ID. To participate via live or replay webcast, a link is available at www.ligand.com.

(Press release, Ligand, FEB 26, 2026, View Source [SID1234663082])

On February 26, 2026 Kairos Pharma, Ltd. (NYSE American: KAPA), a clinical-stage biopharmaceutical company focused on innovative cancer therapeutics, reported the signing of a term sheet for a strategic asset acquisition with Celyn Therapeutics, Inc., a privately held biotechnology company backed by OrbiMed and Torrey Pines Investment. Under the proposed terms of the agreement, Kairos Pharma will acquire worldwide rights to two highly differentiated, clinical-stage oncology assets targeting non-small cell lung cancer (NSCLC): CL-273, a pre-IND, reversible, wild-type-sparing pan-EGFR inhibitor, and CL-741, a Phase 1-ready, orally available type IIb c-MET kinase inhibitor.

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John Yu, M.D., Kairos Pharma Chief Executive Officer, commented: "We anticipate this acquisition will significantly expand our oncology pipeline with late-preclinical and Phase 1-ready assets in a multi-billion dollar market with substantial unmet medical needs. With this acquisition, if completed, we will strengthen our armamentarium to reverse oncology drug resistance – by implementing therapeutics that specifically target resistance mutations that arise from targeting the EGFR receptor. Importantly, our established clinical consortia on the West Coast, anchored at Cedars-Sinai Medical Center in Los Angeles, provides us with the clinical infrastructure and expertise to rapidly initiate and execute Phase 1 and Phase 2 studies for both compounds."

Kairos Pharma believes the scientific rationale for combining a pan-EGFR inhibitor with a c-MET inhibitor in non-small cell lung cancer as demonstrated with these two assets is compelling and well-validated clinically. MET amplification represents one of the most important resistance mechanisms in EGFR-mutant NSCLC, and the ability to address both pathways with highly selective, brain-penetrant molecules represents a significant therapeutic advance. The Company anticipates that CL-273’s wild-type-sparing profile and broad coverage of EGFR mutations, combined with CL-741’s potent and selective c-MET inhibition, upon acquisition, will position it to develop best-in-class monotherapies as well as a differentiated combination regimen. Mechanistically, dual inhibition of EGFR and MET pathways can overcome compensatory signaling that drives resistance, deepens tumor responses, and extends progression-free survival in this difficult-to-treat patient population.

Kinase inhibitors for cancer treatment were estimated to be valued at $60.7B in 2025. Of these, EGFR inhibitors represented 32.5% of the market in 2025 (Future Market Insights).

CL-273, developed using a proprietary AI-driven drug discovery platform, targets the EGFR mutated lung cancer treatment, a market valued at $16.2B in 2026 (Future Market Insights). EGFR mutations are present in approximately 10-15% of NSCLC cases in Western populations and up to 50% in Asian populations (CoherentMI), creating a substantial addressable patient population for targeted therapies.

CL-741 addresses the cMet inhibitor market which is experiencing rapid growth, valued at more than $2B and projected to reach over $10B by 2030 with a CAGR in excess of 17% (Biospace). The c-MET metastatic NSCLC market represents a high-value niche with significant unmet medical needs, with c-MET amplification being a critical resistance mechanism for EGFR-targeted therapies. C-MET alterations, including MET exon 14 skipping mutations and MET amplification, is a driver of multiple cancer types inclusive of gastric, liver, and renal cancer.

"Our proprietary AI-driven drug design platform has enabled the discovery of a highly efficacious, wild-type-sparing, pan-mutant EGFR inhibitor. This molecule offers a 4-to-5-fold broader safety margin than current competitive inhibitors," stated Nikolay Savchuk, Ph.D., CEO of Celyn Therapeutics. "By partnering with Kairos Pharma and leveraging their clinical consortia at Cedars-Sinai Medical Center, we are positioned to rapidly advance CL-273 and CL-741. This collaboration combines Kairos’s operational expertise with our innovative pipeline to create an optimal pathway for patients fighting EGFR-mutant and c-MET-driven lung cancers."

Clinical studies have demonstrated that combination treatment with EGFR and MET inhibitors for EGFR-mutant, MET-amplified NSCLC patients is able to achieve progression-free survival of approximately 7 months, representing a significant advance over single-agent therapy (SAVANNAH trial).

CL-273 is an investigational, reversible, wild-type-sparing pan-EGFR small-molecule inhibitor specifically engineered for EGFR-mutant non-small cell lung cancer (NSCLC). Preclinical data demonstrate that CL-273 maintains broad-spectrum activity against classical EGFR mutations including Exon 19 and 21 deletions and Exon 20 insertions, atypical mutations, and resistance-associated variants that bypass currently approved tyrosine kinase inhibitors (TKIs).

A defining feature of CL-273 is its exceptional selectivity index. By sparing wild-type EGFR, studies to date have shown CL-273 offers a 4–5 fold wider therapeutic window, suggesting significantly improved safety and tolerability over existing therapies. Designed for high brain and lung permeability to address metastatic disease, CL-273 possesses favorable ADME properties and has successfully completed GLP toxicology studies. The program is currently pre-IND, with first-in-human clinical trials projected to commence in 2026.

CL-741 is an orally available, small-molecule, type IIb c-MET kinase inhibitor designed to be highly selective for c-MET with broad coverage of activating and acquired resistance mutations in solid tumors. The compound was discovered as a drug-like lead with potent activity across multiple c-MET resistance mutants and is being developed for c-MET-driven advanced solid tumors, with a primary focus on non-small cell lung cancers harboring MET exon 14 skipping alterations and MET amplification.

The acquisition of both CL-273 and CL-741, if the acquisition is successfully completed, are anticipated to enable Kairos Pharma to pursue a differentiated dual-target strategy addressing both primary EGFR mutations and MET-mediated resistance mechanisms. MET amplification is one of the most common mechanisms of acquired resistance to EGFR TKIs.

Developing CL-273 and CL-741 together provides a rational combination therapy approach for EGFR-mutant NSCLC patients who either harbor baseline MET amplification/overexpression or acquire MET-driven resistance on EGFR-TKI therapy. Combined EGFR and MET inhibition has already demonstrated meaningful clinical response rates and survival benefit with other agents in this setting. Pairing CL-273 with CL-741 could deepen and prolong responses, reduce outgrowth of MET-mediated escape clones, and potentially expand the addressable population of MET-dependent, EGFR-mutant tumors.

(Press release, Kairos Pharma, FEB 26, 2026, View Source [SID1234663081])

On February 26, 2026 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies, reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a business update.

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"The past year marked a period of significant execution and progress for Janux as we continued to translate the promise of our tumor-activated platform into meaningful clinical and strategic advances. Recent JANX007 clinical results demonstrate clinical activity and a consistent and predictable safety profile in mCRPC and support continued advancement in taxane-naïve patients as well as other expansion cohorts," said David Campbell, Ph.D., President and CEO of Janux.

"In early 2026, we announced a collaboration with Bristol Myers Squibb that provides near-term capital and further validates the potential of our platform. We also continue to strengthen our team to support the next phase of clinical and pipeline growth. With additional product candidates entering the clinic in 2026, we believe Janux is well positioned to execute on our clinical strategy and continue to build long-term value for patients and shareholders."

BUSINESS HIGHLIGHTS AND RECENT DEVELOPMENTS:

Clinical & Pipeline Progress

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Janux presented updated interim Phase 1 data for JANX007 (PSMA-TRACTr) in December 2025. As of the October 15, 2025 data cutoff, 109 patients had been treated across Phase 1 cohorts:
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Durable clinical activity was observed across both once-weekly (QW) and every-two-week (Q2W) cohorts, with median radiographic progression-free survival (rPFS) ranging from 7.9 to 8.9 months. The rPFS in the Q2W cohort compared favorably to the QW expansion group.
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Data demonstrated high prostate-specific antigen (PSA) response rates including deep PSA declines. Anti-tumor activity was observed, with confirmed and unconfirmed partial responses in 30% (8/27) of RECIST-evaluable patients.

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Ongoing dose optimization of JANX007 monotherapy is focused on taxane-naïve mCRPC.
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The Phase 1 study also includes expansion cohorts evaluating JANX007 in combination with darolutamide, an androgen receptor pathway inhibitor, to further characterize safety and clinical activity in taxane-naïve mCRPC.
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Additional expansion cohorts are evaluating JANX007 monotherapy in patients with PARP inhibitor-refractory mCRPC, supporting assessment of activity in a high-unmet-need population.
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JANX008 (EGFR-TRACTr) continues to enroll in its Phase 1 clinical trial in a defined group of solid tumors, with expansion cohorts underway to further characterize safety and clinical activity.
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JANX011 (CD19-ARM) is actively enrolling its Phase 1 clinical trial in healthy volunteers.

Strategic Collaboration

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In January 2026, Janux announced a collaboration and exclusive worldwide license agreement with Bristol Myers Squibb to develop a novel tumor-activated therapeutic targeting a validated solid tumor antigen.
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Under the terms of the agreement, Janux is eligible to receive $50 million in upfront and near-term milestone payments, with the potential for additional development, regulatory and commercial milestones totaling approximately $800 million, as well as tiered royalties on global product sales, if successful.
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Janux will lead preclinical development through IND submission, with Bristol Myers Squibb assuming responsibility for subsequent clinical development and global commercialization.

Corporate & Leadership

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Janux recently announced the appointment of William Go, M.D., Ph.D. as Chief Medical Officer, strengthening the Company’s clinical leadership as additional programs enter the clinic in 2026 and progress toward later-stage development.

Upcoming Milestones

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The Company expects to provide additional clinical data for JANX007 in 2026 or present these data at a future medical meeting.
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The Company expects to provide additional clinical update for JANX008 in 2026.
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The Company expects to announce initial clinical update from the Phase 1 study of JANX011 in healthy volunteers by year-end 2026.

FOURTH QUARTER AND FULL YEAR 2025 FINANCIAL RESULTS:

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Cash and cash equivalents and short-term investments: As of December 31, 2025, Janux reported cash and cash equivalents and short-term investments of $966.6 million, compared to $1.03 billion on December 31, 2024.
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Research and development expenses: Research and development expenses were $31.5 million for the quarter and $125.9 million for the year ended December 31, 2025, compared to $20.8 million and $68.4 million for the same quarter and year in 2024.

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General and administrative expenses: General and administrative expenses were $10.9 million for the quarter and $41.8 million for the year ended December 31, 2025, compared to $8.2 million and $41.0 million for the same quarter and year in 2024.
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Net loss: Net loss was $31.9 million for the quarter and $113.6 million for the year ended December 31, 2025, compared to $20.2 million and $69.0 million for the same quarter and year in 2024.

(Press release, Janux Therapeutics, FEB 26, 2026, View Source [SID1234663080])