On March 11, 2026 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical-stage biotechnology company focused on the discovery and development of integrin-based therapeutics, reported a corporate update and announced fourth quarter 2025 financial results.

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"We ended 2025 with encouraging data from our lead oncology program in ICI-refractory patients, an area of unmet medical need, that informed the initiation of our accelerated development plan for PLN-101095," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant. "In addition to oncology and the early-stage pipeline programs emerging from Pliant’s proprietary integrin platform, we continue to assess opportunities to expand our clinical-stage pipeline that leverage our areas of expertise and align with creating shareholder value."

Fourth Quarter and Recent Developments

Oncology Program
PLN-101095 is an oral, small molecule, dual selective inhibitor of αvβ8 and αvβ1 integrins designed to overcome checkpoint resistance by blocking TGF-β activation in the tumor microenvironment. Pliant is currently conducting a Phase 1a/1b open-label, dose-escalation and indication expansion trial to evaluate the safety, tolerability, pharmacokinetics, and preliminary evidence of antitumor activity of PLN-101095, as monotherapy and in combination with pembrolizumab, in patients with immune checkpoint inhibitor (ICI)-refractory advanced or metastatic solid tumors.
•Data from the Phase 1 trial of PLN-101095 showed deep and durable ongoing responses. In December, the Company announced positive data showing that, in a heavily pretreated patient population, PLN-101095 demonstrated anti-tumor activity in combination with pembrolizumab, an FDA-approved ICI. Four responders were observed consisting of one confirmed complete response and three partial responses (two confirmed, one unconfirmed) out of the 10 secondary ICI refractory patients. These clinical responses were observed in patients with cholangiocarcinoma, melanoma, head and neck squamous cell carcinoma and non-small cell lung cancer (NSCLC). Notably, all responding patients showed large increases in plasma interferon gamma (IFN-γ) after 14 days of monotherapy with PLN-101095 prior to the addition of pembrolizumab. No non-responders showed meaningful increases in plasma IFN- γ. PLN-101095 was generally well tolerated across all doses tested. IFN-γ is known to play a multifaceted role in modulating anti-tumor immunity, with increased tumor expression levels having previously been linked with better outcomes from immune checkpoint blockade.

•Accelerated development plan of PLN-101095 underway with initiation of Phase 1b indication expansion trial.
Based on the encouraging response data and supportive IFN- γ biomarker data from the Phase 1 trial, the Company is advancing an accelerated clinical development plan of PLN-101095 and has initiated a Phase 1b indication expansion trial. The Phase 1b open-label, single dose trial will enroll three cohorts of patients including NSCLC, clear cell renal cell carcinoma and tumors with high tumor mutational burden. Tumor selection was based on data from the Phase 1 trial, as well as strong mechanistic rationale for integrin inhibition. Patients will be treated for 14 days with PLN-101095 dosed at 1,000 mg twice daily as monotherapy, after which pembrolizumab will be added as combination therapy. Study start activities for this trial are underway with first patient enrollment anticipated in second quarter. Interim data is expected in 2027.

•PLN-101095 Phase 1 data accepted for presentation at AACR (Free AACR Whitepaper) Annual Meeting 2026. Data from the Phase 1 trial of PLN-101095 will be the subject of a poster presentation and an oral presentation as part of the Clinical Trials Minisymposium at the upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) conference to be held April 17-22, 2026, in San Diego, California.

Integrin-Targeted Delivery Platform

•Utilizing cell-specific integrin receptors, Pliant has developed a platform to deliver drug payloads, including siRNAs, to selective tissue types. Current programs are focused on delivering siRNAs to skeletal muscle cells and other tissues. The Company believes this integrin-targeting delivery platform has the potential for broad applicability across multiple disease areas utilizing a variety of drug payloads.

Corporate Highlights

•Appointment of Minnie Kuo as Chief Operating Officer. Ms. Kuo joined Pliant in September 2023 as Chief Development Officer, bringing more than 20 years of multinational clinical development experience across various therapeutic areas. In this expanded role, Ms. Kuo bridges Pliant’s science and operations with oversight of clinical operations, early development, program management, regulatory affairs and compliance.
•INTEGRIS-PSC results published in the Journal of Hepatology. The manuscript, "Phase II INTEGRIS-PSC trial of bexotegrast, an αvβ6 and αvβ1 integrin inhibitor, in primary sclerosing cholangitis", appears in the January 2026 issue of the Journal of Hepatology.
Fourth Quarter 2025 Financial Results
•Research and development expenses were $15.6 million as compared to $38.8 million for the prior-year quarter. The decrease was primarily driven by the discontinuation of BEACON-IPF.
•General and administrative expenses were $8.0 million as compared to $14.5 million for the prior-year quarter. The decrease was primarily due to lower personnel-related costs resulting from the strategic restructuring of our workforce.
•Net loss was $23.6 million as compared to $49.7 million for the prior-year quarter. The decrease was primarily attributable to the discontinuation of BEACON-IPF coupled with the decrease in personnel-related costs resulting from the strategic restructuring of our workforce.
•As of December 31, 2025, the Company had cash, cash equivalents and short-term investments of $192.4 million which the Company expects to be sufficient to fund operations into the second half of 2028.

(Press release, Pliant Therapeutics, MAR 11, 2026, View Source [SID1234663458])

On March 11, 2026 Tempest Therapeutics, Inc. (Nasdaq: TPST) ("Tempest"), a clinical-stage biotechnology company developing a pipeline of advanced CAR-T cell therapy product candidates to treat cancer, reported the selection of Cincinnati Children’s Applied Gene and Cell Therapy Center ("AGCTC") as the lead contract development and manufacturing partner to conduct the formal technology transfer of TPST-2003, Tempest’s dual-targeting CD19/BCMA CAR-T therapy under development for the treatment of relapsed/refractory multiple myeloma ("rrMM").

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Tempest recently announced that, as of a January 31, 2026 data cutoff, a total of 36 patients with rrMM had received one infusion of TPST-2003, including 24 patients in a prior Phase 1/2 investigator-initiated trial (IIT) and 12 patients in the ongoing REDEEM-1 trial, representing one of the largest datasets evaluating a CD19/BCMA dual-targeting CAR-T therapy. As of the data cutoff, all six efficacy evaluable patients enrolled in the REDEEM-1 trial had achieved a complete response (CR) according to the International Myeloma Working Group (IMWG) uniform response criteria. Among 25 evaluable patients with measurable disease at baseline across both studies, the overall response rate ("ORR") was 100% (25/25). Tempest plans to present the results of the REDEEM-1 trial and updated results from the IIT at a scientific meeting later this year.

"The recent positive results from the ongoing REDEEM-1 trial position TPST-2003 as a potential class-leading therapy for rrMM, which, subject to FDA approval, could offer a meaningful option for patients with rrMM," said Dr. Matt Angel, President and Chief Executive Officer of Tempest. "The potential to treat patients with extramedullary disease (EMD) and patients who have previously received a BCMA-targeting CAR-T is particularly exciting. We are very pleased to have selected the AGCTC as our partner to execute the technology transfer of TPST-2003 as we prepare for our planned U.S. clinical development."

The AGCTC is a research, development, and manufacturing hub advancing future cell and gene therapy (CGT) treatments for patients with unmet needs. Established in 2001, the center has evolved into a nationally recognized leader in CTG CDMO services with a proven track record that reflects Cincinnati Children’s commitment to solving unmet medical needs through translational science. AGCTC is part of the Cincinnati Children’s Cancer and Blood Diseases Institute, which is ranked #1 in the nation by U.S. News & World Report for pediatric cancer care.

"We are proud to have been selected to support the development of this exciting product candidate," said Dr. Chaozhong Zou, Executive Director and General Manager of AGCTC, Cancer and Blood Diseases Institute, Cincinnati Children’s Hospital Medical Center. "The parallel-structure dual-targeting CAR architecture of TPST-2003 is truly innovative, and fits perfectly within our experience supporting technology transfer of engineered cell therapy products, including our extensive experience making novel CAR-T programs IND-ready. We look forward to generating the information needed to advance TPST-2003 to the next stage of development."

About TPST-2003

TPST-2003 is an autologous CD19/BCMA dual-targeting CAR-T therapy designed to improve response depth and durability in patients with relapsed/refractory multiple myeloma (rrMM) through a parallel dual-targeting CAR structure designed to address tumor heterogeneity and antigen escape. TPST-2003 is being developed in China by Tempest’s partner, Novatim Immune Therapeutics ("Novatim"). Under its agreement with Novatim, Tempest has the exclusive right to develop TPST-2003 outside of China, India, Turkey, and Russia.

About REDEEM-1

REDEEM-1 (Study nos. CTR20233309/NCT06223646) is a Phase 1/2a clinical trial evaluating TPST-2003 in patients with relapsed/refractory multiple myeloma, including patients with high-risk cytogenetics and patients with extramedullary disease. The REDEEM-1 trial has a targeted full enrollment of 29 patients. The REDEEM-1 trial is sponsored and being conducted by Tempest’s partner, Novatim Immune Therapeutics, with a total of eight clinical sites registered in China: Peking Union Medical College Hospital (Dr. Jian Li; lead site), The First Affiliated Hospital of Nanchang University (Dr. Fei Li), Peking University First Hospital (Dr. Yujin Dong), Henan Cancer Hospital (Dr. Baijun Fang), Shanxi Provincial Cancer Hospital (Dr. Liping Su), The Second Xiangya Hospital of Central South University (Dr. Hongling Peng), The First Affiliated Hospital of China Medical University (Dr. Xiaojing Yan), and The Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College (Dr. Dehui Zou).

Additional clinical trials evaluating TPST-2003

A Phase 1/2 IIT (Study no. NCT04714827) is evaluating TPST-2003 in patients with relapsed/refractory multiple myeloma, including patients with high-risk cytogenetics and patients with extramedullary disease. The IIT is sponsored and being conducted by Tempest’s partner, Novatim Immune Therapeutics, with a total of two clinical sites registered in China: Shanghai Fourth People’s Hospital (Dr. Weijun Fu; lead site) and Shanxi Provincial Cancer Hospital (Dr. Liping Su).

A Phase 1 trial (Study nos. CTR20242409/NCT06518876) is evaluating TPST-2003 in patients with POEMS, a rare blood disorder caused by abnormal plasma cells. The Phase 1 trial is sponsored and being conducted by Tempest’s partner, Novatim Immune Therapeutics, with a total of three clinical sites registered in China: Peking Union Medical College Hospital (Dr. Jian Li; lead site), Xuanwu Hospital Capital Medical University (Dr. Wanling Sun), and West China Hospital, Sichuan University (Dr. Yu Wu).

(Press release, Tempest Therapeutics, MAR 11, 2026, View Source [SID1234663459])

On March 11, 2026 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a therapeutics company developing and commercializing medications for the treatment of dermatological diseases, including skin cancers, reported financial results for the fourth quarter and full year ended December 31, 2025.

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"In 2025, Verrica successfully implemented a series of transformational changes that we believe have fundamentally improved the future growth and strategic value of our entire business," said Jayson Rieger, PhD, MBA, President and Chief Executive Officer of Verrica. "Our focused and efficient commercial strategy allowed us to nearly double dispensed applicator units of YCANTH from the prior year while cutting selling, general and administrative expenses by over 40% over that same period. This February, we dispensed more applicators of YCANTH per selling day than in any month in our history, reflecting strong and increasing demand for YCANTH. We are poised to advance our late-stage clinical pipeline in common warts and basal cell carcinoma, which collectively could represent a multiple billion-dollar opportunity. Finally, in 2025 we significantly improved our financial position, repaying our outstanding debt while extending our cash runway into the first quarter of 2027.

"Looking ahead, in addition to growing YCANTH sales in the United States we have multiple potential avenues to continue building value by entering new markets and expanding our product portfolio. Our first international partnership for YCANTH, with Torii Pharmaceutical, has now launched in Japan. After recently gaining alignment with regulators for YCANTH’s approval pathway in the European Union, we are now able to more meaningfully engage in discussions with additional potential commercialization partners, which could provide a significant source of non-dilutive funding and future revenue for the Company," Dr. Rieger continued.

"We are also advancing toward pivotal Phase 3 studies of VP-315 for the treatment of basal cell carcinoma. VP-315 represents a unique opportunity to introduce a novel immunotherapy with potential abscopal activity that could become a primary or neoadjuvant, non-surgical treatment option for this large patient population. With the opportunity to grow YCANTH in the United States, enter new markets and develop transformative medicines, we are tremendously excited about what 2026 has in store for Verrica and our patients," Dr. Rieger concluded.

Conference Call and Webcast Information

The Company will host a conference call on Wednesday, March 11, 2026, at 8:30 am, to discuss its fourth quarter and full year 2025 financial results and provide a business update. To participate in the conference call, please utilize the following information:

Domestic Dial-In Number: Toll-Free: 1-800-343-4136

International Dial-In Number: 1-203-518-9843

Conference ID: VERRICA

Participants can use Guest dial-in #s above and be answered by an operator.

Webcast:

View Source;tp_key=9018bdf1ab

The call will be broadcast live over the Web and can also be accessed on Verrica Pharmaceuticals’ website: www.verrica.com.

The conference call will also be available for replay for one month on the Company’s website in the Events Calendar of the Investors section.

Business Highlights and Recent Developments

YCANTH (VP-102)

•
During the fourth quarter of 2025, YCANTH dispensed applicator units totaled 13,654, representing a year-over-year increase of 58% from the fourth quarter of 2024. On a sequential basis, YCANTH dispensed applicator units decreased approximately 3% from the prior quarter.

•
In the first quarter of 2026, while January was likely impacted somewhat by significant winter weather across the East Coast, dispensed applicator units per selling day in February rebounded, reaching a record monthly high since launch.

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In September 2025, Verrica announced that its partner, Torii Pharmaceutical Co. Ltd. ("Torii"), a wholly-owned subsidiary of Shionogi & Co., Ltd., received approval from the Japanese Ministry of Health, Labour and Welfare for YCANTH for the treatment of molluscum. On February 9, 2026, the Company announced the commercial launch of YCANTH in Japan by Torii for the treatment of molluscum contagiosum.

•
On January 7, 2026, the Company announced that the first patient was dosed in December 2025 in the global Phase 3 program evaluating YCANTH (VP-102) for the treatment of common warts. The Phase 3 program was initiated based upon the clinically meaningful activity observed for the primary endpoint of complete clearance in the Phase 2 COVE-1 study. These results, if replicated in the Phase 3 program, support the potential for YCANTH to become the first therapy approved in either the United States or Japan for the treatment of common warts, a condition that impacts over 22 million people in the United States alone. The Company has retained full commercial rights for all potential YCANTH indications outside of Japan and believes that YCANTH for common warts could represent a substantial commercial and licensing opportunity.

•
The Company launched YcanthRx in the fourth quarter of 2025, a new non-dispensing pharmacy option. YcanthRx is designed to provide prescribers a central hub option for YCANTH prescriptions to assist with benefits investigations and prior authorizations. Prescriptions written to YcanthRx are then routed to a dispensing pharmacy in the Company’s pharmacy network that is contracted with the patient’s insurance plan, where available.

•
On October 20, 2025, the Company announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency provided positive feedback that supports the filing of a Marketing Authorization Application for YCANTH as a treatment for molluscum contagiosum. Based on Verrica’s comprehensive efficacy and safety data from the well-controlled Phase 3 studies successfully conducted in both the U.S. and Japan, the CHMP concluded that no further Phase 3 clinical studies are needed to progress toward a filing for approval.

VP-315

•
On November 4, 2025, the Company presented new data on VP-315 from its Phase 2 trial in basal cell carcinoma (BCC) at the 40th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. The presentation revealed supportive immunologic mechanistic data that helps explain why VP-315 shrinks treated basal cell carcinomas in many patients (as evidenced by a 97% objective response rate and an 86% reduction in overall tumor size).

•
The Company is now providing additional data on the abscopal response in 14 observed but not treated lesions from the Phase 2 study, which suggests immune system engagement. Specifically, 3 out of the 14 lesions had complete histologic clearance and there was a 67% overall reduction in tumor size across all 14 lesions. Additional details on this observation are being planned for discussion at a scientific conference later this year.

•
On November 14, 2025, the Company announced that the FDA confirmed alignment with the Company’s plan for the Phase 3 program for BCC to encompass two placebo-controlled Phase 3 studies with approximately 100 subjects each and a primary endpoint of complete clearance as assessed at week 14. Based on the discussion with the FDA, the Company expects these studies will be adequate to support a New Drug Application filing, with long-term follow-up studies to be conducted as post-approval commitments.

CORPORATE

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On February 12, 2026, the Company announced the appointment of Chris Chapman as its Chief Commercial Officer. Mr. Chapman brings over 25 years of commercial experience in the pharmaceutical industry to Verrica, and most recently served as Chief Commercial Officer at Dermavant Sciences through its acquisition by Organon, where he played an instrumental role in launching VTAMA (tapinarof) cream, 1%, approved for adult plaque psoriasis in June 2022 and atopic dermatitis in December 2024.

•
On November 24, 2025, the Company announced a private placement of $50 million anchored by Caligan Partners LP and PBM Capital, along with other new and existing investors. The Company used $35.0 million of the net proceeds to fully repay its outstanding obligations and terminate all outstanding commitments under its Credit Agreement, and the remainder for working capital and general corporate purposes. With these proceeds and its existing cash and cash equivalents, the Company expects its cash runway to fund operations into the first quarter of 2027.

Financial Results

Fourth Quarter 2025 Financial Results

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Product revenue, net was $3.7 million for the three months ended December 31, 2025, compared to net product revenue of $0.3 million for the three months ended December 31, 2024. The increase in product revenue was based on higher demand for YCANTH. Product revenue, net, relates to the delivery of YCANTH to Verrica’s distribution partners.

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License and collaboration revenue was $1.4 million for the three months ended December 31, 2025, consisting primarily of commercial supply for Torii’s YCANTH launch in Japan. License and collaboration revenue was not material for the three months ended December 31, 2024.

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Costs of product revenue were $0.7 million for the quarter ended December 31, 2025, compared to $0.6 million for the quarter ended December 31, 2024.

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Selling, general and administrative expenses were $8.1 million for the quarter ended December 31, 2025, compared to $9.9 million for the same period in 2024. Excluding the impact of stock-based compensation, the decrease of $1.8 million was primarily due to lower expenses related to commercial activities for YCANTH.

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Research and development expenses were $2.5 million for the quarter ended December 31, 2025, compared to $1.2 million for the same period in 2024. Excluding the impact of stock-based compensation, the increase was primarily attributable to costs associated with the Phase 3 program for common warts and compensation.

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Interest income was $0.2 million for the quarter ended December 31, 2025, which was unchanged from the quarter ended December 31, 2024.

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Interest expense was $1.3 million for the quarter ended December 31, 2025, compared to $2.3 million for the same period in 2024. Interest expense was related to borrowings under the Company’s now-repaid Credit Agreement. The decrease of $1.0 million was related to a lower principal balance and the termination of the Credit Agreement in November 2025.

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For the quarter ended December 31, 2025, net loss was $8.1 million, or $0.57 per basic and diluted share, compared to a net loss of $16.2 million, or $2.41 per share, for the same period in 2024.

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For the quarter ended December 31, 2025, non-GAAP net loss was $7.2 million, or $0.51 per basic and diluted share, compared to a non-GAAP net loss of $12.2 million, or $1.81 per share, for the same period in 2024.

Full Year 2025 Financial Results

•
Product revenue, net was $15.3 million for the twelve months ended December 31, 2025, compared to $6.6 million for the twelve months ended December 31, 2024. For the twelve months ended December 31, 2025, product revenue, net was primarily related to increased demand for YCANTH.

•
License and collaboration revenue was $20.3 million for the twelve months ended December 31, 2025, compared to $1.0 million for the twelve months ended December 31, 2024. License and collaboration revenue for the twelve months ended December 31, 2025, consisted primarily of $18.0 million in milestone payments and $2.3 million of revenue related to commercial supplies and development activity from the Collaboration and License Agreement with Torii.

•
Costs of product revenue were $2.2 million for the twelve months ended December 31, 2025, compared to $1.9 million for the twelve months ended December 31, 2024. The increase was due to increased sales of YCANTH partially offset by higher obsolete inventory reserves in the prior period.

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Selling, general and administrative expenses were $35.2 million during the twelve months ended December 31, 2025, compared to $58.8 million for the same period in 2024. Excluding the impact of stock-based compensation, the decrease of $20.6 million was primarily due to lower expenses related to commercial activities for YCANTH, including decreases in compensation, benefits and travel due to reduced sales force of $6.9 million, decreased commercial costs of $6.6 million, decreased compensation of $2.7 million related to termination of non-sales employees, decreased travel and fleet costs of $2.0 million and decreased legal and administrative costs of $2.3 million.

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Research and development expenses were $8.9 million for the twelve months ended December 31, 2025, compared to $11.8 million for the same period in 2024. Excluding stock-based compensation, the decrease of $2.1 million was primarily attributable to decreased clinical costs for VP-315.

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Interest income was $0.9 million for the twelve months ended December 31, 2025, compared to $1.4 million for the same period in 2024. The decrease of $0.5 million was primarily due to a lower cash balance.

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Interest expense was $7.7 million for the twelve months ended December 31, 2025, and $9.4 million for the same period in 2024. Interest expense is related to borrowings under the Credit Agreement, which was terminated in November 2025. The decrease of $1.7 million was primarily related to a lower principal balance and the termination of the Credit Agreement.

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For the twelve months ended December 31, 2025, net loss was $17.9 million, or $1.68 per share, compared to a net loss of $76.6 million, or $14.78 per share, for the same period in 2024.

•
For the twelve months ended December 31, 2025, non-GAAP net loss was $13.2 million, or $1.24 per share, compared to a non-GAAP net loss of $64.6 million, or $12.47 per share, for the same period in 2024.

(Press release, Verrica Pharmaceuticals, MAR 11, 2026, View Source [SID1234663460])

On March 11, 2026 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, reported the publication of a new study in JCO Precision Oncology highlighting how advanced features of comprehensive genomic profiling (CGP) expand treatment options for cancer patients in community oncology settings. The study, conducted in collaboration with The Oncology Institute (TOI), reveals that features such as tumor-normal matched sequencing, RNA sequencing, and liquid biopsy reflex identify actionable findings that are missed by more limited standard in-network testing.

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While organizations like ASCO (Free ASCO Whitepaper) and the NCCN strongly advocate for CGP to guide precision therapies, current guidelines often lack specificity regarding the exact composition or essential features required within these panels. This research demonstrates that CGP value extends far beyond simple panel size, highlighting the utility of advanced testing features to more fully capture clinically relevant findings.

In the study, 12% (approximately 1 in 8) of patients across the pilot and expanded cohorts had potentially actionable findings associated with an approved therapy identified solely through advanced Tempus features—such as tumor-normal matching, RNA sequencing, and liquid biopsy reflex testing—that would otherwise have been missed by less comprehensive tests.

"This study demonstrates a clear clinical mandate: to truly provide precision medicine, we must utilize the most comprehensive tools available," said Ezra Cohen, MD, Chief Medical Officer, Oncology at Tempus and a coauthor of the study. "Advanced testing capabilities expand access to targeted treatments and clinical trial opportunities, while providing clinicians with a more comprehensive view of clinically relevant findings. This underscores the critical role Tempus plays in helping clinicians ensure that no stone is left unturned for their patients."

(Press release, Tempus, MAR 11, 2026, View Source [SID1234663461])

On March 11, 2026 Parabilis Medicines, a clinical-stage biopharmaceutical company committed to creating extraordinary medicines for people living with cancer using its Helicon TM peptide platform to drug historically undruggable targets, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to zolucatetide (previously known as FOG-001), the company’s lead investigational HeliconTM peptide and the first and only direct inhibitor of the elusive β-catenin:TCF interaction, for the treatment of desmoid tumors. Zolucatetide has also received FDA Fast Track Designation in desmoid tumors.

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"Patients living with desmoid tumors have lacked therapies that directly address the root biological cause of their disease because that biology, the β-catenin:TCF interaction, has long been considered ‘undruggable,’" said Mathai Mammen, M.D., Ph.D., Chairman, CEO and President of Parabilis Medicines. "With Orphan Drug Designation and Fast Track Designation from the FDA, along with compelling early clinical data showing encouraging evidence of clinical activity, we are building momentum behind zolucatetide as a potential first-in-class therapy that we believe could help redefine the standard of care for patients with desmoid tumors and other tumors driven by the same elusive biology. We are committed to advancing this program with rigor and speed to deliver meaningful impact for patients."

Desmoid tumors are considered a rare condition involving locally invasive soft-tissue tumors that form in the connective tissues. While they do not metastasize, desmoid tumors can be aggressive and recurrent, often causing chronic and significant pain, limited mobility, disfigurement and organ dysfunction, and no existing therapies target the underlying biology of the disease.

Zolucatetide is an investigational first-in-class inhibitor of the Wnt/β-catenin pathway, which is the fundamental driver of desmoid tumor growth. Orphan Drug Designation follows early data, first released at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and Connective Tissue Oncology Society (CTOS) 2025 Annual Meeting, demonstrating that zolucatetide has shown evidence of clinically meaningful antitumor activity in desmoid tumors, including tumor reductions and 100% disease-control rate (DCR) in all 10 patients with at least one post-baseline scan and an 80% objective response rate (ORR) in the five patients with more than one post-baseline scan per RECIST 1.1.

The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits towards the cost of clinical trials upon approval and prescription drug user fee waivers. Products with Orphan Drug Designation status are also entitled to a potential seven years of regulatory exclusivity following regulatory approval.

Beyond desmoid tumors, zolucatetide is being evaluated across a broad range of rare and common Wnt/β-catenin-driven tumor types. Clinical data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC 2025 meeting showed zolucatetide had single-agent activity in five low-complexity tumor types where Wnt/β-catenin mutations are the primary drivers of disease – including desmoid, adamantinomatous craniopharyngioma (ACP), ameloblastoma, salivary gland cancer, and solid pseudopapillary neoplasm (SPN) – with strong scientific rational for combination therapy in more complex cancers such as microsatellite-stable colorectal cancer (MSS CRC). Parabilis also shared early data indicating the potential of zolucatetide in hepatocellular carcinoma (HCC) and familial adenomatous polyposis (FAP) at the JP Morgan Healthcare conference earlier this year.

The company plans to share additional data readouts in 2026 from its ongoing Phase 1/2 trial of zolucatetide, in which more than 150 patients have been dosed to date.

About Zolucatetide (Previously FOG-001)

Zolucatetide is an investigational first-in-class competitive inhibitor of β-catenin interactions with the T-cell factor (TCF) family of transcription factors and is currently in clinical development. By directly targeting the β-catenin:TCF protein-protein interaction, zolucatetide is intended to block the Wnt signaling pathway irrespective of the various APC and β-catenin mutations that typically drive disease.

Zolucatetide combines key features that distinguish it from previously reported Wnt/β-catenin pathway modulators: zolucatetide acts inside the cell where it binds directly to the key oncogenic driver β-catenin; and zolucatetide blocks the Wnt pathway at the key downstream node, disrupting the interaction between β-catenin and the TCF transcription factors, thereby abrogating the signal transmission by which Wnt pathway mutations are believed to drive oncogenesis.

Zolucatetide is currently being evaluated in a Phase 1/2 clinical trial in patients with locally advanced or metastatic solid tumors. Zolucatetide has received Fast Track Designation and Orphan Drug Designation for the treatment of desmoid tumors from the U.S. Food and Drug Administration (FDA).

About the Phase 1/2 trial of Zolucatetide

Zolucatetide is being evaluated in a Phase 1/2 multicenter, open-label study (NCT05919264) assessing its safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity. The trial includes dose-escalation and dose-expansion phases and is testing zolucatetide both as a monotherapy and in combination with other anticancer agents in patients with advanced or metastatic solid tumors likely or known to harbor a Wnt pathway–activating mutation (WPAM).

(Press release, Parabilis Medicines, MAR 11, 2026, View Source;cateninTCF-Interaction-Receives-FDA-Orphan-Drug-Designation-for-the-Treatment-of-Desmoid-Tumors [SID1234663462])