RegulationPriority Review (United States), Accelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (Australia), Priority Review (Australia)

Structure/Sequence

Sequence Code 12981416H

Source: *****

Sequence Code 13030670L

Source: *****

Clinical Trials associated with Retifanlimab

NCT07472322

/ Not yet recruitingPhase 1/2IIT

UW26001 Phase I/II Study on the Safety, Tolerability, and Preliminary Efficacy of ASTX727 (Decitabine/Cedazuridine) and Retifanlimab-dlwr in Patients With Advanced Merkel Cell Carcinoma Who Have Progressed on Anti-PD-(L)1 Inhibitor

The goal of this clinical trial is to learn if ASTX727 can be combined with retifanlimab to treat Merkel cell cancer. It will also learn about the safety of combining these drugs. The main questions it aims to answer are:
* Can the combination shrink cancer and lower the chance of the cancer growing or spreading?
* Is the combination better than standard of care for Merkel cell cancer?
Participants will:
* Take oral ASTX727 and retifanlimab through a vein in the arm for about 2 years.
* Visit the clinic once every 2 weeks for checkups and tests

Start Date01 May 2026

Sponsor / Collaborator

University of Wisconsin-Madison

NCT07468136

/ Not yet recruitingPhase 1/2IIT

Phase I/IIa Trial of Retifanlimab and Difluoromethylornithine (DFMO) in Patients With Progressive High-Grade Glioma

This phase I/II trial tests the safety, side effects best dose and effect of retifanlimab with or without difluoromethylornithine (DFMO) for the treatment of high grade gliomas that are growing, spreading, or getting worse (progressive). Immunotherapy with monoclonal antibodies, such as retifanlimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. DFMO is in a class of medications called ornithine decarboxylase (ODC) inhibitors. It works by blocking the action of a substance that signals tumor cells to multiply. This helps stop or slow the spread of tumor cells. Giving retifanlimab with or without DFMO mat be safe, tolerable and/or effective in treating patients with progressive high grade glioma.

Start Date24 Apr 2026

Sponsor / Collaborator

Mayo Clinic

NCT07425054

/ Not yet recruitingPhase 2IIT

ctHPVDNA Adapted ChemoRadiation +/- Retifanlimab Treatment in Locally-Advanced Anal Cancer (CHART-AC)

This study is for people who have anal cancer and have not yet had treatment. The regular treatment for people who have anal cancer is chemoradiation therapy (CRT). CRT is when chemotherapy and radiation therapy are given at the same time. Studies show that CRT works well to treat anal cancer and prevents many people from needing surgery which may require a colostomy bag. Doctors know that CRT is an effective way to treat anal cancer. But, they are doing studies to find out how much dose of radiation and chemotherapy should be given during the CRT. Higher doses of chemotherapy and radiation could increase the risk of side effects, but lowering the dose of chemoradiation has the risk of not being as effective to treat the cancer. One way to predict whether participants need higher or lower doses of radiation therapy is to do a blood test called ctDNA (circulating tumor DNA) to test for the presence of human papillomavirus (HPV). This test is done at certain times while participants are getting CRT. This has been shown to be a marker for the presence of anal cancer.
In this study, doctors will tailor lower versus higher doses of CRT based on the tumor response that is measured by ctDNA. The purpose of this study is to see if customizing the dose of chemoradiation based on the amount of ctDNA will increase survival in participants with anal cancer and/or decrease the risk of side effects. Some participants in this study whose cancer does not respond as well to the CRT may have the opportunity to receive a drug called Retifanlimab that stimulates the body's immune system. Retifanlimab is approved by the Federal Drug Administration (FDA) for treating anal cancer that is recurrent or metastatic since there is proven benefit in these situations.

Start Date01 Mar 2026

Sponsor / Collaborator

The Case Comprehensive Cancer Center

[+1]

100 Clinical Results associated with Retifanlimab

100 Translational Medicine associated with Retifanlimab

100 Patents (Medical) associated with Retifanlimab

Literatures (Medical) associated with Retifanlimab

16 Mar 2026·Future Oncology

Avelumab real-world use in advanced Merkel cell carcinoma: a systematic review and non-comparative meta-analysis

Review

Author: Moradian, Hoora ; Freitag, Andreas ; Rutherford, Megan ; Kearney, Mairead ; Kwon, Christina ; Lan, Zhiyi

AIM:

Programmed death (ligand) 1 inhibitors (e.g., avelumab, pembrolizumab, and retifanlimab) are first-line treatment options for patients with locally advanced or metastatic Merkel cell carcinoma (MCC). In the absence of comparative and randomized trials, we aimed to systematically identify and synthesize real-world evidence (RWE) on the effectiveness and safety of immunotherapies in patients with advanced MCC.

METHODS & MATERIALS:

MEDLINE and Embase searches were conducted for observational RWE studies in locally advanced or metastatic MCC from January 2017 to December 2023. Avelumab was the only immunotherapy with sufficient data for analysis. Landmark 12-month overall survival (OS) and progression-free survival (PFS) were assessed by meta-analysis.

RESULTS:

Screening of 1731 records identified 37 publications eligible for inclusion (16 unique studies), of which eight were included in the meta-analysis. In line with results from the JAVELIN Merkel 200 trial, pooled 12-month OS rates of 77.7% in stage III and 63.0% in stage IV MCC and 12-month PFS rates of 53.3% and 39.3%, respectively, were estimated. Although safety data were insufficient for meta-analysis, two retrospective studies reported lower adverse event rates than JAVELIN Merkel 200.

CONCLUSIONS:

The results of this study support broader use of avelumab in advanced MCC.

01 Mar 2026·International Journal of Clinical Oncology

PMDA regulatory update on approval and revision of the precautions for use of anticancer drugs in Japan; imlunestrant for breast cancer, tafasitamab for follicular lymphoma, atezolizumab for thymic carcinoma, retifanlimab for anal canal squamous cell carcinoma, and tagraxofusp for blastic plasmacytoid dendritic cell neoplasm

Author: Matsumura, Noriomi

01 Feb 2026·Cancer research communications

Tumor and Immune Dynamics Following Sequential CDK4/6 and PD-1 Inhibition: Results from a Phase 2 Study in Dedifferentiated Liposarcoma

Article

Author: Reed, Damon R. ; Adamow, Mathew ; D’Angelo, Sandra P. ; Tap, William D. ; Movva, Sujana ; Wong, Phillip ; Avutu, Viswatej ; Desir, Rhoena ; Biniakewitz, Matthew ; Keohan, Mary Louise ; Lefkowitz, Robert A. ; Singer, Samuel ; Chi, Ping ; Koff, Andrew ; Erinjeri, Joseph P. ; Maki, Robert G. ; Kelly, Ciara M. ; Antonescu, Cristina R. ; Seffar, Evan ; Bradic, Martina ; Lee, Jasme ; Shen, Ronglai ; Chan, Jason E. ; Gularte-Mérida, Rodrigo ; Rosenbaum, Evan ; Dickson, Mark A. ; Qin, Li-Xuan ; Banks, Lauren B. ; Gounder, Mrinal M. ; Panageas, Katherine S.

Abstract:

Purpose::

The CDK4/6 inhibitor palbociclib delays progression in patients with advanced dedifferentiated liposarcoma (DDLPS). In carcinoma models, CDK4/6 and PD-1 inhibition induce intratumoral inflammation and synergistic activity. Comprehensive assessment of tumor and host dynamics is needed to understand response and resistance to this combination.

Patients and Methods::

We performed a phase 2 study of palbociclib and the PD-1 inhibitor retifanlimab in advanced DDLPS. Palbociclib was administered 2 weeks prior to retifanlimab. Tumor biopsies were analyzed by single-cell RNA sequencing (scRNA-seq), and peripheral blood by high-parameter flow cytometry. Analyses focused on cell cycle, senescence, and immune-related changes.

Results::

Twelve patients were treated before the study was halted because of a 42% immune-related toxicity rate. The overall response rate was 8.3%, and the disease control rate was 75%. Median progression-free and overall survival rates were 7.1 and 26.8 months, respectively. Nine patients had at least one tumor biopsy analyzed; three had paired. Ten patients had paired blood samples analyzed. Following treatment, tumor cells demonstrated decreased cycling and increased cell-cycle checkpoint activity. Transcriptional scores for senescence increased in cancer cells, as did the proportion of intratumoral T and B cells. A cluster of cells emerged with upregulated cell-cycle genes and downregulated HLA class I, suggesting innate or acquired resistance to treatment. In blood, a subset of CD4+ T cells decreased, whereas expression of LAG-3, ICOS, and CD38 increased in select subsets.

Conclusions::

A palbociclib lead-in prior to retifanlimab had a high rate of immune-related toxicities. Correlative analyses identified changes in tumor and immune cells attributable to treatment. A study of concurrent dosing of the combination is ongoing.

Significance::

In this phase 2 study of palbociclib and retifanlimab, treatment led to immune-related adverse events, halting enrollment. scRNA-seq and flow cytometry revealed cell-cycle suppression, senescence, and T-cell changes. These findings highlight the rationale for this combination and the need for safer dosing strategies in larger cohorts.

147

News (Medical) associated with Retifanlimab

13 May 2026

·ir.macrogenics.com

MacroGenics Reports First Quarter 2026 Financial Results and Highlights Business Transformation

Manufacturing operations divestiture sharpens focus on core capabilities in novel drug discovery and development Manufacturing divestiture and expanded monetization of ZYNYZ royalty anticipated to provide up to $202.5 million in combined proceeds ADC pipeline remains on track for multiple data disclosures and program milestones Cash runway guidance extended through 2028, based on anticipated closing of manufacturing divestiture ROCKVILLE, Md., May 13, 2026 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on developing innovative antibody-based therapeutics for the treatment of cancer, today reported financial results for the quarter ended March 31, 2026, and highlighted its recent corporate progress. “We are very pleased to report a strong start to the year, building on the momentum generated in 2025. These results reflect our team’s disciplined execution of a strategy designed to sharpen our focus, maximize the value of our pipeline, and strengthen our financial position. As part of this effort, we recently announced the sale of our GMP manufacturing operations to Bora Pharmaceuticals and the monetization of additional ZYNYZ royalties with Sagard Healthcare Partners. Subject to the closing of the manufacturing operations divestiture, these transactions are expected to provide significant non-dilutive capital to support growth opportunities in 2026 and beyond,” said Eric Risser, President and CEO of MacroGenics. “We look forward to providing multiple updates during the remainder of the year, including key programmatic milestones for MGC026, MGC028, and MGC030. We believe our increased focus on discovering and developing breakthrough medicines has the potential to enhance patients’ lives while creating meaningful value for our shareholders.” Focus and Realignment Across Our Business MacroGenics recently took a series of significant steps designed to focus resources on the Company’s innovative oncology programs. These steps include: Divestiture of Manufacturing Operations. As announced earlier this week, MacroGenics entered into a definitive agreement with Bora Pharmaceuticals Co., Ltd. and Bora Biologics USA, LLC (collectively, Bora) to sell its manufacturing operations, inclusive of drug substance manufacturing, development and quality services. Subject to customary closing conditions, MacroGenics is expected to receive an upfront payment of $122.5 million, before transaction fees and expenses. As part of this transaction, MacroGenics’ headquarters and warehouse sites in Maryland will transfer to Bora. The transaction is expected to close in the third quarter of this year. At closing, MacroGenics will enter into a supply agreement with Bora to support development and production of clinical drug substance for current and future pipeline programs. The Company has historically leveraged both internal manufacturing capabilities as well as those of external contract manufacturing partners. Through this transaction, the Company will transition to a fully outsourced model, which is expected to provide increased flexibility and cost advantages. Expanded ZYNYZ Royalty Monetization. Earlier this month, MacroGenics announced that it had entered into an amended royalty purchase agreement with Sagard Healthcare Partners (Sagard) in exchange for a revised capped royalty interest in future global net sales of ZYNYZ. MacroGenics received a $60.0 million cash payment from Sagard with the potential to receive an additional milestone, based on 2026 ZYNYZ sales performance, of up to $20.0 million. Corporate Restructuring. As part of the manufacturing divestiture transaction, approximately 140 employees are expected to transfer to Bora. With some additional reductions across the company, MacroGenics is expected to have approximately 135 employees at closing, enabling a more agile organization that is focused on the research and clinical development of novel therapeutics. Advancement of Innovative Pipeline MacroGenics is developing potential best-in-class or first-in-class antibody-drug conjugates (ADCs) and T-cell engagers (TCEs). MacroGenics' two clinical-stage ADC programs, MGC026 and MGC028, continue to demonstrate acceptable safety profiles to date, with no observations of interstitial lung disease, as well as evidence of anti-tumor activity by Response Evaluation Criteria in Solid Tumors (RECIST). MGC026 is a novel ADC that targets B7-H3, which is overexpressed in multiple solid tumors. The Company completed enrollment of the dose escalation portion of a Phase 1 study in late 2025 and is currently enrolling patients in the dose expansion portion of the study in selected solid tumor indications. The Company anticipates reporting initial MGC026 clinical data in mid-2026. MGC028 is a first-in-class ADC that targets ADAM9, which is overexpressed in multiple solid tumors. MGC028 is currently being evaluated in the dose escalation portion of a Phase 1 study in patients with advanced solid tumors. The Company anticipates reporting initial MGC028 clinical data in the second half of 2026. MGC030 is a first-in-class preclinical ADC that targets an undisclosed antigen expressed across several solid tumors. An Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MGC030 is planned for the third quarter of 2026. Future Pipeline. MacroGenics is advancing additional preclinical programs that incorporate proprietary platforms for next-generation TCEs and ADCs with novel payloads. The company expects to nominate two additional product candidates by the end of 2026. Lorigerlimab Update MacroGenics continues its LINNET Phase 2 monotherapy study of lorigerlimab, a PD-1 × CTLA-4 bispecific DART® molecule, in patients with gynecological cancers. As of a data cut-off on May 7, 2026, 17 patients with clear cell gynecologic cancer (CCGC) were treated at 6 mg/kg every three weeks (Q3W). Of the 16 evaluable patients with CCGC, 4 (25%) had objective responses, including 4 with confirmed partial responses (PR), of which 1 patient subsequently had an unconfirmed complete response (CR). Of the 17 CCGC patients evaluable for safety, Grade ≥3 treatment-related adverse events (TRAEs) occurred in 8 patients (47%) and 2 patients (12%) discontinued treatment due to AEs. No treatment-related fatalities were reported in these patients. Going forward, MacroGenics intends to enroll an additional 20 CCGC patients at a lower dose of 3 mg/kg Q3W, which was selected based on pharmacokinetic and pharmacodynamic modeling, with the goal of improving safety while maintaining clinical benefit. The Company anticipates completing enrollment of these 20 patients by year-end 2026 and reporting updated study results in the first half of 2027. Based on an assessment of results from the high-grade serous and platinum-resistant ovarian cancer (PROC) cohort of the LINNET study, the predetermined response rate was not achieved, and the Company no longer intends to pursue development in this indication. Current Partnerships MacroGenics maintains partnerships with Incyte Corporation, Sanofi, and Gilead Sciences, which span multiple commercial, clinical and preclinical programs. These include ZYNYZ® (retifanlimab-dlwr), TZIELD® (teplizumab-mzwv), and MGD024, a clinical-stage CD123 × CD3 bispecific DART molecule. Across these collaborations, the Company remains eligible to receive up to approximately $2.5 billion in aggregate future milestones, in addition to royalties on partnered products. First Quarter 2026 Financial Results Cash Position: Cash, cash equivalents and marketable securities balance as of March 31, 2026, was $154.2 million, compared to $189.9 million as of December 31, 2025. The balance as of March 31, 2026, did not include the $60.0 million received from Sagard earlier this month. In addition, the Company is expected to receive $122.5 million proceeds from Bora, less related transaction fees and expenses, in connection with the manufacturing operations divestiture, which is expected to close in the third quarter of this year, subject to customary closing conditions. Revenue: Total revenue was $20.8 million for the quarter ended March 31, 2026, compared to $13.2 million for the quarter ended March 31, 2025. The increase was due to higher contract manufacturing revenue from higher production volume for external clients and royalty revenue recognized from higher sales of ZYNYZ, offset by decreased collaborative revenue. Cost of Manufacturing Services: Cost of manufacturing services was $9.5 million for the quarter ended March 31, 2026, compared to $5.4 million for the quarter ended March 31, 2025. The increase was due to increased production for external clients. R&D Expenses: Research and development expenses were $35.0 million for the quarter ended March 31, 2026, compared to $39.7 million for the quarter ended March 31, 2025. The decrease is primarily due to the discontinuation of further development of vobramitamab duocarmazine. This was partially offset by increased development costs related to MGC028 and the preclinical TCE programs. G&A Expenses: General and administrative expenses were $9.7 million for the quarter ended March 31, 2026, compared to $10.7 million for the quarter ended March 31, 2025. Net Loss: Net loss was $36.8 million for the quarter ended March 31, 2026, compared to $41.0 million for the quarter ended March 31, 2025. Shares Outstanding: Shares of common stock outstanding as of March 31, 2026, were 63,560,068. Cash Runway Guidance: MacroGenics anticipates that its cash, cash equivalents and marketable securities balance of $154.2 million as of March 31, 2026, in addition to projected and anticipated future payments from partners, including $60.0 million received from Sagard earlier this month plus anticipated sale proceeds of $122.5 million, less related transaction fees and expenses, from Bora related to divestiture of the Company’s manufacturing operations, is expected to support its cash runway through 2028. About MacroGenics, Inc. MacroGenics (the Company) is a biopharmaceutical company focused on developing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at www.macrogenics.com. MacroGenics, the MacroGenics logo, DART and TRIDENT are trademarks or registered trademarks of MacroGenics, Inc. Cautionary Note on Forward-Looking Statements Any statements in this press release about future expectations, plans and prospects for MacroGenics (“Company”), including statements about the Company’s strategy, future operations, clinical development of and regulatory plans for the Company’s therapeutic candidates, expected timing of the release of clinical updates and safety and efficacy data for the Company’s ongoing clinical trials, anticipated cash runway and other statements containing the words “subject to”, "believe", “anticipate”, “plan”, “expect”, “intend”, “estimate”, “potential,” “project”, “may”, “will”, “should”, “would”, “could”, “can”, the negatives thereof, variations thereon and similar expressions, or by discussions of strategy, including our ability to execute on our key strategic priorities for 2026, constitute forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: risks that TZIELD, lorigerlimab, ZYNYZ, or any other product candidate’s revenue, expenses and costs may not be as expected, risks relating to TZIELD, lorigerlimab, ZYNYZ, or any other product candidate’s market acceptance, competition, reimbursement and regulatory actions; future data updates, including timing and results of efficacy and safety data with respect to product candidates in ongoing clinical trials; our ability to provide manufacturing services to our customers; the uncertainties inherent in the initiation and enrollment of future clinical trials; the availability of financing to fund the internal development of our product candidates; expectations of expanding ongoing clinical trials; expectations for the timing and steps required in the regulatory review process; expectations for regulatory approvals; expectations of future milestone payments; the impact of competitive products; our ability to enter into agreements with strategic partners and other matters that could affect the availability or commercial potential of the Company's product candidates; business, economic or political disruptions due to catastrophes or other events, including natural disasters, terrorist attacks, civil unrest and actual or threatened armed conflict, or public health crises; costs of litigation and the failure to successfully defend lawsuits and other claims against us; the risk that one or more of the closing conditions to the sale of our CDMO operations (the "Transaction") may not be satisfied or waived, on a timely basis or at all, including the risk that any required landlord consents or other third-party consents are not obtained; the risk that the Transaction may not be completed on the timeline currently expected, or at all, or on the terms currently contemplated; the occurrence of any event, change, or other circumstance that could give rise to the termination of the purchase agreement related to the Transaction; the effect of the announcement, pendency, or consummation of the Transaction on the Company's business, operating results, employees, customers, suppliers, and other business relationships, including the Company's CDMO operations; risks related to the transition of the CDMO operations to the purchaser in the Transaction, including the diversion of management's attention from the Company's ongoing business operations; risks related to the Company's post-closing manufacturing arrangements with the purchaser in the Transaction including under the manufacturing and supply agreement and the transition services agreement; the possibility that the anticipated benefits of the Transaction, including that the additional post-closing cash payments may not be earned or received, in whole or in part; the costs and expenses associated with the Transaction; potential litigation relating to the Transaction; and other risks described in the Company's filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views only as of the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so, except as may be required by law. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. CONTACTS Jim Karrels, Senior Vice President, CFO 1-301-251-5172 info@macrogenics.com Argot Partners 1-212-600-1902 macrogenics@argotpartners.com Source: MacroGenics, Inc.

Phase 1Phase 2Financial StatementADCAcquisition

13 May 2026

·www.biospace.com

Bora inks $122.5M takeover of MacroGenics facility to further US expansion

inkoly /Getty Images MacroGenics is selling the manufacturing plant to Bora, a Taiwan-based CDMO, to raise cash to support the progress of its drug development pipeline. Bora Pharmaceuticals has struck a deal to buy MacroGenics’ production plant for $122.5 million upfront, securing ownership of a facility that manufactures materials for companies including Incyte. MacroGenics, a developer of antibody-based therapeutics, uses the Rockville, Maryland, site to produce drug substances for most of its clinical trials. The company also makes the drug substance for Margenza and Zynyz, cancer drugs that originated from its pipeline but are sold by TerSera and Incyte, respectively. MacroGenics planned to use the facility to make its own commercial products in the event of notching a regulatory approval. Now, Bora—a Taiwan-based contract development and manufacturing organization (CDMO)—is acquiring the facility and related warehouse operations for the upfront fee and up to $5 million in milestones. The $5 million is tied to the progress of the CDMO operation and professional development program services in 2027 and 2028. The biotech expects about 140 of its employees to transfer to Bora as part of the deal. The employees will operate the 11,000 liters of capacity at the facility to serve the site’s existing customers and any new clients Bora onboards. MacroGenics plans to strike a manufacturing and supply deal with Bora to support process development and drug substance production for its internal pipeline. The agreement will allow MacroGenics to retain access to development and manufacturing capabilities while generating nondilutive capital to lengthen its cash runway, which extended into 2027 before factoring in the sale. Earnings Daiichi takes $850M charge, axes facility investment as ADC demand forecast falls While Daiichi Sankyo brought in $13.4 billion in 2025, setbacks forced the company to update its antibody-drug conjugate forecast, pushing demand below the minimum supply agreed upon with CMOs and prompting the cancellation of an in-house investment. May 11, 2026 · 2 min read · Nick Paul Taylor Read more The deal comes 21 years after MacroGenics established in-house manufacturing capabilities. While some biotechs rely on contract manufacturing organizations even after bringing drugs to market, MacroGenics’ management opened a manufacturing facility five years after founding the business. The choice to revert to third-party reliance marks a sharp shift in MacroGenics’ strategy. MacroGenics’ CDMO plant is an “embedded part of the business” and “benefiting from all the onshoring interest,” CEO Eric Risser said at a TD Cowen event in March. The company’s CDMO sales grew 300% last year, jumping to $52.6 million, and Risser said that by the third quarter the unit was generating $19 million to $20 million of quarterly revenue. For Bora, buying the facility extends its long history of expanding through acquisitions. Since 2013, Bora has bought facilities from Eisai , Impax Laboratories , GSK , Eden Biologics and Upsher-Smith Laboratories . The deals have expanded Bora’s capabilities and geographic footprint, moving the CDMO into biologics and giving it a presence in the U.S. GSK signed a $250 million, five-year deal with Bora in February.

AcquisitionADC

04 May 2026

·endpoints.news

Celcuity's breast cancer win; Odyssey plans $205M IPO

Celcuity’s breast cancer drug wins again: The company said two regimens with its experimental drug gedatolisib succeeded in PIK3CA mutant patients as part of its Phase 3 trial. Last year, Celcuity reported that the drug staved off cancer progression in patients without PIK3CA mutations, and the company is awaiting a July FDA approval in that setting. The company is studying its drug as a second-line treatment for patients with HR-positive, HER2-negative breast cancer. The Minneapolis-based company said Friday that gedatolisib plus two other drugs —fulvestrant and Pfizer’s Ibrance — beat a combination of Novartis’ Piqray and fulvestrant, meeting the study’s primary endpoint. Another secondary analysis also showed that gedatolisib plus fulvestrant — without Ibrance — also beat Piqray plus fulvestrant. Celcuity is now headed to the FDA to ask for approval of gedatolisib in PIK3CA mutant patients as well. Unlike Piqray or Roche’s Itovebi, which are only approved for patients with a PIK3CA mutation, gedatolisib is meant to hit multiple parts of the key cancer pathway. As a result, Celcuity is aiming for a broad approval encompassing both wildtype and mutant forms of the cancer. — Lei Lei Wu 🚀 Odyssey charts $205M IPO: The biotech plans to raise that amount in net proceeds from its proposed Nasdaq listing as $ODTX . The autoimmune and inflammatory disease startup previously attempted an IPO in 2025 but pulled back and went with a Series D instead. Now, it seeks to sell 13.24 million shares at $16 to $18 apiece after Kailera Therapeutics, Avalyn Pharma, Seaport Therapeutics and Hemab Therapeutics opened the IPO window heading into the summer. — Kyle LaHucik 💸 Gene therapy biotech gets $43M: Philadelphia startup Latus Bio has extended its 2024 Series A to a total of $97 million as it looks to begin two clinical trials this year. The company will test AAV-based gene therapies for Huntington’s disease and CLN2 disease. Its backers include 8VC, DCVC Bio and others. — Kyle LaHucik MacroGenics expands royalty deal: The biotech will get $60 million upfront and up to $20 million more based on a 2026 sales milestone for Zynyz. Sagard Healthcare Partners and MacroGenics formed the initial deal around the Incyte-commercialized PD-1 inhibitor last year. — Kyle LaHucik Mirum scores Phase 2b win for liver drug: The biotech said volixibat hit its primary endpoint in the trial, which enrolled patients with a rare liver condition called primary sclerosing cholangitis. Individuals with the chronic disease have inflammation in the bile ducts, leading to liver damage, according to the Mayo Clinic. Volixibat is designed to treat a common itching symptom (cholestatic pruritus) that comes with the disease. Mirum plans an FDA submission in the second half of 2026. — Max Gelman Indivior begins $175M share repurchase: The move comes after the company said last week that it would punt on Phase 3 studies for a program called INDV-6001, and that a Phase 2 trial for INDV-2000 in opioid use disorder had failed. — Max Gelman

Phase 2Drug ApprovalPhase 3

100 Deals associated with Retifanlimab

External Link

KEGG	Wiki	ATC	Drug Bank
D11827	-	-	DB15766

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Merkel Cell Carcinoma	United States	Incyte Corp.	30 Mar 2026
Anal canal squamous cell carcinoma	United States	Incyte Corp.	15 May 2025
Metastatic Merkel Cell Carcinoma	United States	Incyte Corp.	22 Mar 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
squamous cell carcinoma of the anus	NDA/BLA	European Union	Incyte Biosciences Distribution BV	29 Jan 2026
Non-Small Cell Lung Cancer	NDA/BLA	United States	Incyte Corp.	-
Endometrial Carcinoma	Phase 3	China	Zai Lab (Shanghai) Co., Ltd.	20 Sep 2021
metastatic non-small cell lung cancer	Phase 3	China	Zai Lab (Shanghai) Co., Ltd.	18 Sep 2020
metastatic non-small cell lung cancer	Phase 3	China	Incyte Corp.	18 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	United States	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	China	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Brazil	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Bulgaria	Incyte Corp.	11 Sep 2020
Metastatic Non-Squamous Non-Small Cell Lung Carcinoma	Phase 3	Czechia	Incyte Corp.	11 Sep 2020

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05287113 (CTgov)	Phase 2	Squamous Cell Carcinoma of Head and Neck \| PD-L1 positive Solid Tumors	176	Retifanlimab (Retifanlimab)	xdeyxxsjel(mbhfztzzuw) = mdwqzckcve mxynglvhlj (nmvwwmimjb, uzhfsygwgj - xfcptfrqtk) View more	-	27 Mar 2026
				Retifanlimab (Retifanlimab Plus INCAGN02385)	xdeyxxsjel(mbhfztzzuw) = umpugsczup mxynglvhlj (nmvwwmimjb, eloxoivkgo - lngpgwoevp) View more
NCT04472429 (POD1UM-303, ESMO_IO2025)	Phase 3	squamous cell carcinoma of the anus First line	308	Retifanlimab + carboplatin-paclitaxel	xzluntomdr(kgcposbfkv) = jxlbxbrrjf ksaxegsqxe (xxcleylnaa ) View more	Positive	12 Nov 2025
				Placebo + carboplatin-paclitaxel	xzluntomdr(kgcposbfkv) = mlwxxzmtrm ksaxegsqxe (xxcleylnaa ) View more
EUCTR2021-005775-39-BE (ESMO2025)	Phase 2	PD-L1+	176	Retifanlimab (RETI)	kocmqcyurg(bfdecyrqvt) = srpebgsazx ldxbnkqyym (wbirbkjwbs, 3.5 - 9.7) View more	Negative	17 Oct 2025
				Retifanlimab + Tuparstobart (doublet tx)	kocmqcyurg(bfdecyrqvt) = rzubuzytav ldxbnkqyym (wbirbkjwbs, 3.7 - 7.6) View more
NCT04205812 (POD1UM-304, Pubmed) Manual	Phase 3	Non-Small Cell Lung Cancer First line	583	Retifanlimab + chemotherapy	oxmfprpgjk(rhktkatjug) = skzgqmtuef qonntcosgy (wnjufyysae, 16.2 - 21.0) View more	Positive	19 Sep 2025
				Placebo + chemotherapy	oxmfprpgjk(rhktkatjug) = afazbosgxq qonntcosgy (wnjufyysae, 11.0 - 16.7) View more
NCT03532295 (CTIM-23, CTgov)	Phase 2	Glioblastoma Multiforme \| Glioblastoma \| Recurrent Glioblastoma	51	Radiation therapy+Bevacizumab+Retifanlimab (Regimen A: Retifanlimab+RT+bevacizumab)	xenqyodwrv = pwvgiefykh vbzqudnrgs (xyipbmkikj, bisdqzbcrp - ifbhattcmu) View more	-	07 Aug 2025
				Radiation therapy+Bevacizumab+Epacadostat+Retifanlimab (Regimen B: Retifanlimab+RT+bevacizumab+epacadostat)	xenqyodwrv = rvwbyciqaa vbzqudnrgs (xyipbmkikj, hglejisgnr - mkajsusweo) View more
NCT04116073 (CTgov)	Phase 2	Pancreatic carcinoma non-resectable	25	PD-1 antibody+INCMGA00012	sltaexciaj = emozpmieac zicbccfigd (icfhhpzxnx, oimmnxmkbp - hmqjdbdljh) View more	-	29 Jul 2025
NCT05239182 (RiLEY, CTgov)	Phase 2	Pancreatic adenocarcinoma \| Pancreatic Ductal Adenocarcinoma \| Advanced Pancreatic Adenocarcinoma	7	9-ING-41+Abraxane+Gemcitabine+Retifanlimab	rfzfbjawey = xyqtyivikj wpmgghqjzm (poweiphdhi, heijsdhfsa - omvrandqid) View more	-	17 Jul 2025
NCT04472429 (POD1UM-303/InterAACT 2, BusinessWire) Manual	Phase 3	Anal canal squamous cell carcinoma	308	Retifanlimab + Carboplatin + Paclitaxel	lcktjexfpg(xhmuirwlkw) = jtvvngntrc oqfgvzkoii (erywussurz, 7.5 - 11.3) View more	Positive	14 Jun 2025
				Placebo + Carboplatin + Paclitaxel	lcktjexfpg(xhmuirwlkw) = rtwkwdfsba oqfgvzkoii (erywussurz, 7.1 - 7.7) View more
NCT04472429 (POD1UM-303/InterAACT 2, ASCO2025) Manual	Phase 3	Anal canal squamous cell carcinoma	308	Retifanlimab + chemotherapy (PD-L1 ≥ 1%)	lxranhujlt(fdfszwlrfp) = nvnqoaelnw poetmrkxkv (cdfjhmdhnu ) View more	Positive	30 May 2025
				Retifanlimab + chemotherapy (PD-L1 < 1%)	lxranhujlt(fdfszwlrfp) = kunhafzrgj poetmrkxkv (cdfjhmdhnu )
NCT03599713 (POD1UM-201, ASCO2025)	Phase 2	Metastatic Merkel Cell Carcinoma	101	Retifanlimab 500 mg	yhxruyubqz(bsyftolbka) = xhjjqparff acoegopsay (vykdcncihs ) View more	Positive	30 May 2025

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