On March 31, 2026 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer and Simcere Zaiming Pharmaceutical Co., Ltd., (Simcere) an oncology-focused biopharmaceutical company, reported that an abstract for SIM0505 has been accepted for the American Society for Clinical Oncology (ASCO 2026) meeting being held May 29 – June 2 in Chicago, Illinois. SIM0505 is an investigational antibody drug conjugate (ADC) targeting Cadherin-6 (CDH6) with a proprietary topoisomerase 1 inhibitor (TOPOi) payload.

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The ongoing open-label, Phase 1 study (NCT06792552) is evaluating SIM0505 in advanced solid tumors, including ovarian cancer, with an emphasis on platinum resistant ovarian cancer.

About SIM0505

SIM0505 is a novel antibody drug conjugate (ADC) directed to cadherin-6 (CDH6 ADC), featuring a proprietary topoisomerase 1 inhibitor (TOPOi) payload. The ADC is designed for broad anti-tumor activity, fast systemic clearance and an improved potential therapeutic window. SIM0505 is being evaluated in an open-label, Phase 1 study (NCT06792552) for the potential treatment of advanced solid tumors, including ovarian cancer, with an emphasis on platinum resistant ovarian cancer. NextCure holds exclusive global rights for SIM0505, excluding China, Hong Kong, Macau, and Taiwan which are retained by Simcere Zaiming Pharmaceutical Co., Ltd.

(Press release, NextCure, MAR 31, 2026, View Source [SID1234664100])

On March 31, 2026 Biogen Inc. (Nasdaq: BIIB) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) reported the companies have entered into a definitive agreement under which Biogen has agreed to acquire all outstanding shares of Apellis for $41.00 per share in cash at closing, or approximately $5.6 billion. Apellis stockholders will also receive a nontransferable CVR for each Apellis share held, entitling the holder to receive two payments of $2 per share each, contingent on certain annual global net sales thresholds being met for SYFOVRE.

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The addition of Apellis, a leader in advancing treatments for serious, complement-driven diseases, is expected to enhance Biogen’s short- and long-term revenue growth profile by adding two commercialized differentiated immunology and rare disease medicines to its growth portfolio. EMPAVELI and SYFOVRE add immediate sales revenue to Biogen, having achieved combined net sales of $689 million in 2025, which is expected to grow at a rate in the mid-to-high teens at least through 2028.

EMPAVELI is FDA-approved in rare immune-mediated kidney diseases (C3G and primary IC-MPGN) and in PNH and SYFOVRE is FDA-approved in geographic atrophy secondary to age related macular degeneration, an immune-mediated retinal disease. Apellis brings an established U.S. sales infrastructure and capabilities, including in nephrology, that Biogen believes will accelerate and strengthen Biogen’s commercial readiness for felzartamab, which is currently in Phase 3 studies for three kidney diseases with the first trial readout expected in the first half of 2027. Upon closing, Biogen expects a significant proportion of Apellis employees to join the company. Biogen will continue to work with Sobi, which retains commercial rights to EMPAVELI (Aspaveli in the EU) outside the U.S.

"Consistent with our strategy, this acquisition immediately advances Biogen’s ongoing transformation. The addition of Apellis expands our growth portfolio in immunology and rare disease with two approved, best-in-class medicines that complement our existing portfolio and bolsters our near-and long-term growth potential," said Christopher A. Viehbacher, Biogen’s President and Chief Executive Officer. "We look forward to welcoming Apellis employees to Biogen. We believe our combined capabilities and experience will allow us to maximize the potential of SYFOVRE and EMPAVELI, while Apellis’ talent, expertise and field capabilities will further strengthen Biogen, deepening the foundation for our growing nephrology franchise with felzartamab and serving many more patients with immune-mediated retinal disease."

"I am incredibly proud of the Apellis team and what we have achieved, including bringing two transformational medicines – SYFOVRE and EMPAVELI – to patients and building an innovative pipeline leveraging our deep expertise in complement science," said Cedric Francois, M.D., Ph.D., co-founder and Chief Executive Officer of Apellis. "With Biogen’s extensive experience with immunology and rare disease, we believe this transaction will accelerate our impact and enable us to reach more patients. This transaction represents a compelling outcome for our shareholders and a strong validation of our strategy, scientific innovation, and execution."

Two differentiated, complement therapies to enhance Biogen’s growth portfolio and expand Biogen’s nephrology capabilities

EMPAVELI (pegcetacoplan) is a targeted complement component 3 (C3) therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system. EMPAVELI has been approved with a broad label for adults and adolescents with C3 glomerulopathy (C3G) or primary IC-MPGN (primary immune-complex membranoproliferative glomerulonephritis) that also includes data on patients with post-transplant C3G disease recurrence. In the Phase 3 VALIANT study, EMPAVELI demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits (as measured by C3 staining by biopsy), compared to placebo.

Positive results were seen in adolescent and adult patients with C3G and primary IC-MPGN and in C3G patients with post-transplant disease recurrence.
EMPAVELI is the only FDA-approved treatment for pediatric patients (12+ years) with C3G and the only FDA-approved treatment for patients with post-transplant C3G disease recurrence.
EMPAVELI is also the first and only FDA-approved treatment for both adult and pediatric (12+ years) populations in primary IC-MPGN.
EMPAVELI/Aspaveli is also approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in the United States, European Union, and other countries globally.

SYFOVRE (pegcetacoplan injection) is an approved therapy for geographic atrophy (GA) secondary to age-related macular degeneration. By targeting C3, SYFOVRE has been shown to provide comprehensive control of the complement cascade, part of the body’s immune system. SYFOVRE was approved based on positive results from the Phase 3 OAKS and DERBY studies at 24 months, which included a broad and representative patient population.

In these studies, SYFOVRE reduced the rate of geographic atrophy (GA) lesion growth compared to sham treatment.
The treatment demonstrated increasing effects over time, with the greatest benefit — up to 36% reduction in lesion growth with monthly treatment in the DERBY study — occurring between months 18 and 24.
In November 2025, Apellis presented a post-hoc analysis of its five-year long-term efficacy data from the GALE open-label extension study, showing SYFOVRE delayed GA lesion progression by approximately 1.5 years in patients with nonsubfoveal GA when compared to sham/projected sham.
SYFOVRE is approved in the United States and Australia.
Apellis has completed a clinical trial for a SYFOVRE prefilled syringe (PFS) and plans to submit its application for FDA approval in the first half of 2026.

Transaction brings significant opportunity for value creation
Upon closing of the transaction, expected in the second quarter of 2026, the addition of Apellis is expected to add commercial products to Biogen that together recorded $689 million in revenue in 2025, and which are expected to grow at a rate in the mid-to-high teens at least through 2028.

This transaction is expected to strengthen Biogen’s revenue and EPS growth potential and is expected to be increasingly accretive to Biogen’s Non-GAAP diluted EPS starting in 2027. Importantly, the transaction is expected to meaningfully increase Biogen’s non-GAAP EPS compounded annual growth rate (CAGR) through the end of the decade. Biogen expects to finance the acquisition with a combination of cash and borrowings and believes it can fully de-lever by the end of 2027, allowing it to maintain financial flexibility for future investments.

Biogen plans to update full year 2026 guidance when it reports earnings for the first quarter of 2026.

Transaction and financial details
Under the terms of the agreement, Biogen will commence a tender offer to acquire all of the outstanding shares of Apellis common stock for $41 per share in cash at closing (representing an 86% premium to the 90-day volume-weighted average stock price and a 35% premium to the 52-week high stock price) and a non-transferable CVR per share. Each CVR represents the right to receive a cash payment of $2 per share if SYFOVRE achieves $1.5 billion in annual global net sales in any calendar year between 2027 and 2030, a right to receive an additional cash payment of $2 per share if SYFOVRE achieves $2 billion in annual global net sales in any of these calendar years, and, if these thresholds are not met and no CVR payment is due for any of these years, but if SYFOVRE achieves $2 billion in annual global net sales in the 2031 calendar year, a cash payment of $4 per share. Upon the successful completion of the tender offer, Biogen will acquire all remaining Apellis shares that are not tendered into the tender offer through a second-step merger at the same consideration.

The transaction, which was approved by the boards of directors of both companies, is subject to successful completion of the tender offer, customary closing conditions, and the receipt of necessary regulatory approvals.

Conference Call Details
Biogen will host an investor call on March 31, at 8:30 a.m. ET. The conference call will be accessible through the Investors section of Biogen’s website, www.biogen.com. Supplemental information in the form of a slide presentation will also be accessible at the same location on the internet and will be subsequently available on the website for at least 90 days.

Advisors
Lazard acted as sole financial advisor and Cravath, Swaine & Moore LLP and Arnold & Porter acted as legal advisors to Biogen. Evercore acted as sole financial advisor and Wachtell, Lipton, Rosen & Katz and WilmerHale acted as legal advisors to Apellis.

(Press release, Apellis Pharmaceuticals, MAR 31, 2026, View Source [SID1234668984])

On March 31, 2026 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported a rinzimetostat (ORIC-944) program update and potential best-in-disease efficacy and safety data from the Phase 1b trial of once daily rinzimetostat in combination with darolutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) who were previously treated with abiraterone acetate (abiraterone).

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"The combination dose optimization data announced today provide confirmatory evidence that support rinzimetostat’s potential best-in-disease clinical profile, reinforcing its path towards becoming a practice-changing therapy for patients with prostate cancer," said Jacob M. Chacko, M.D., president and chief executive officer. "The durability observed to date is consistent with the competitor PRC2 inhibitor currently in Phase 3 in mCRPC and appears meaningfully improved relative to other approved standard of care therapies. Notably, rinzimetostat demonstrated this impressive durability with a markedly cleaner safety profile than competitor regimens."

"These data provide compelling validation for advancing rinzimetostat in combination with darolutamide into Phase 3 registrational trials in patients with prostate cancer," said Pratik S. Multani, M.D., chief medical officer. "We expect our first Phase 3 trial, Himalayas-1, in patients with mCRPC previously treated with abiraterone, to initiate in the first half of 2026 while we continue to evaluate rinzimetostat in additional indications in prostate cancer and beyond."

Rinzimetostat Phase 1b Dose Optimization Data in Combination with Darolutamide
Patients were previously treated with a median of two prior lines of therapy, including abiraterone, up to one prior line of chemotherapy, and a variety of other approved and investigational treatment regimens. This median excludes background androgen deprivation therapy or first-generation androgen receptor (AR) inhibitors that the patients may have received. 18 patients were treated with 400 mg of rinzimetostat once daily and 15 patients were treated with 600 mg of rinzimetostat once daily, both in combination with the standard dose of darolutamide at 600 mg twice daily.

Rationale for Selection of the Recommended Phase 3 Dose (RP3D)
At the 400 mg dose, rinzimetostat in combination with darolutamide continues to be well tolerated and demonstrates a safety profile supportive of long-term administration and sustained patient adherence. As of the January 16, 2026 data cutoff, the vast majority of treatment-related adverse events (TRAEs) were Grade 1 in severity and consistent with PRC2 and AR inhibition. The most common TRAEs were fatigue (39%; 17% Grade 1 and 22% Grade 2), diarrhea (22%; 17% Grade 1 and 6% Grade 2) and nausea (22%; all Grade 1). A single Grade 3 TRAE was observed, and no Grade 4 or 5 AEs were attributed to rinzimetostat or darolutamide. Dose modifications were rare (one interruption and one discontinuation), with no dose reductions required. The 600 mg dose of rinzimetostat in combination with darolutamide was associated with a modestly higher rate of adverse events and dose modifications. In a broader dataset of post-ARPI (androgen receptor pathway inhibitor) patients (n=72, inclusive of the post-abiraterone patients), the safety and tolerability profile was consistent with the post-abiraterone cohort.

To support the selection of the recommended Phase 3 dose in accordance with the FDA’s Project Optimus initiative, the company conducted a comprehensive exposure-response (E-R) analysis in over 100 patients across both the single-agent and combination data evaluating the relationship between rinzimetostat drug exposure versus efficacy and safety outcomes. The analysis demonstrated that 400 mg and 600 mg once daily provided comparable efficacy. In contrast, the E-R analysis identified statistically significant relationships between higher drug exposure and toxicities as well as increased rates of treatment modifications, clearly favoring the 400 mg dose on the basis of safety. Based on these findings 400 mg once daily in combination with darolutamide has been selected as the RP3D for rinzimetostat.

Preliminary Efficacy Analysis at RP3D
As of March 6, 2026, radiographic progression-free survival (rPFS) was assessed in 18 efficacy-evaluable patients. PSA responses were assessed in a subset of 15 patients with at least one post-baseline assessment as of the January 16, 2026 clinical cutoff. ctDNA molecular response was evaluated in 14 patients with detectable ctDNA at baseline.

Early efficacy data, including landmark rPFS rates, PSA reductions and ctDNA responses, are suggestive of meaningful and durable clinical benefit for rinzimetostat in combination with darolutamide compared to competitor data and historical outcomes with approved therapies. Rinzimetostat 400 mg once daily in combination with darolutamide demonstrated compelling efficacy across multiple endpoints:

With a median follow up of 4.9 months, landmark rPFS rates of 93%, 84%, and 84% at 3, 4, and 5 months, respectively, are consistent with the competitor PRC2 inhibitor currently in Phase 3 in post-abiraterone mCRPC patients and superior to available standard-of-care therapies, including Xtandi, Jevtana, Taxotere, and Pluvicto. For reference, the 5-month landmark rPFS for these approved therapies ranges from approximately 60% to 75%.
47% of patients (7/15) achieved a PSA50 response, with 33% (5/15) confirmed.
Impressive ctDNA reductions observed across a range of AR mutations, with 71% of patients (10/14) achieving >50% ctDNA reduction.
Himalayas-1 Phase 3 Trial
The company expects to initiate the Himalayas-1 global Phase 3 registrational trial in mCRPC patients previously treated with abiraterone in the first half of 2026. This proposed trial will enroll approximately 600 patients from over 250 sites in over 20 countries, randomized 1:1 to receive the RP3D of 400 mg QD rinzimetostat in combination with darolutamide versus physician’s choice of an AR inhibitor or chemotherapy. The primary endpoint is rPFS, the key secondary endpoint is overall survival, and additional secondary endpoints include PSA response rate, objective response rate and patient reported outcomes.

In the US, the annual incidence of mCRPC patients previously treated with abiraterone is approximately 17,000, with an estimated addressable market of greater than $3.5 billion and total global addressable market of $7 billion. This market lacks oral, well-tolerated therapies with meaningful efficacy.

Next Steps
Rinzimetostat 400 mg QD plus darolutamide is currently being evaluated in a food effect cohort. Thus far, there does not appear to be a significant food effect, consistent with previous single agent studies. Furthermore, as of March 6, 2026, early efficacy and safety are consistent with fasted results, with two confirmed PSA50 responses and one confirmed PSA90 response in the first five patients dosed, and all TRAEs reported being Grade 1 with a single Grade 2 TRAE.

The company is also evaluating additional prostate cancer populations for future potential Phase 3 trials in mCRPC and mCSPC. Early data from rinzimetostat in 19 patients with mCRPC previously treated with AR inhibitors demonstrated compelling landmark rPFS rates of 93%, 85%, and 85% at 3, 4, and 5 months, respectively, with a median follow-up of 4.8 months.

Rinzimetostat Phase 1b Trial Design
Rinzimetostat is being evaluated in a Phase 1b dose optimization trial in combination with ERLEADA (apalutamide), Johnson & Johnson’s AR inhibitor, and NUBEQA (darolutamide), Bayer’s AR inhibitor, in patients with mCRPC. Patients are eligible if they have received prior treatment with an ARPI and up to one prior chemotherapy. The primary objective of the trial is to determine the RP3D, and additional objectives include safety, tolerability, pharmacokinetics, and preliminary clinical activity.

Conference Call and Webcast Details
ORIC will host a conference call and webcast today at 4:30 p.m. ET. To join the conference call via phone and participate in the live Q&A session, please pre-register online here to receive a telephone number and unique passcode required to enter the call. A live webcast and audio archive of the conference call will be available through the investor section of the company’s website at www.oricpharma.com. The webcast will be available for replay for 90 days following the presentation.

(Press release, ORIC Pharmaceuticals, MAR 31, 2026, View Source [SID1234664083])

On March 31, 2026 MAIA Biotechnology, Inc. (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer, reported highlights from a poster presented on March 27, 2026, at the European Lung Cancer Congress 2026 (ELCC), a premier thoracic oncology forum held March 25-28, 2026, in Copenhagen, Denmark.

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MAIA reports overall survival (OS) beyond two years for eight patients treated with ateganosine sequenced with cemiplimab in Parts A and B of its ongoing Phase 2 THIO-101 clinical trial in non-small cell lung cancer (NSCLC). The patients did not receive subsequent lines of therapy.

The eight patients featured in the poster include:

1 patient in third-line (3L) therapy with survival of 33 months. Expected survival in this heavily pre-treated population is 5.8 months.2
4 patients in 2L therapy with survival over 30 months. Documented OS for standard of care treatment (chemotherapy or checkpoint inhibitors alone) in second-line (2L) therapy is 10.5 months.3
All patients have failed previous treatment (prior to THIO-101) with a checkpoint inhibitor (CPI) alone.
All patients completed 29-34 cycles of therapy, except for 1 patient who completed 2 cycles of therapy with survival follow-up of 725 off therapy.
5 of the 8 patients have survival follow-up ongoing.

"It’s very encouraging to see such outstanding survival from these patients extending beyond our 24-month trial protocol and without any subsequent treatment. OS surpassing two-years bodes well as we continue to monitor patients in our ongoing Phase 3 pivotal trial and in THIO-101 Part C," said Vlad Vitoc, M.D., Founder and Chief Executive Officer of MAIA. "These results illuminate ateganosine’s valuable role in targeting telomeres to eliminate NSCLC tumor cells and support this treatment—ateganosine sequenced by a CPI—as a potential breakthrough therapeutic option for NSCLC."

THIO-101 treated 79 patients in Parts A and B of the trial. The Part C expansion is currently enrolling up to 48 participants in Asia and Europe. Treatment with ateganosine followed by cemiplimab (Libtayo) has shown an acceptable safety profile to date in a heavily pre-treated population.

MAIA’s ELCC poster is available on MAIA’s website at maiabiotech.com/publications.

About Ateganosine

Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in non-small cell lung cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment of ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101 Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate ateganosine’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of ateganosine administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of ateganosine administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of ateganosine using Overall Response Rate (ORR) as the primary clinical endpoint. The expansion of the study will assess overall response rates (ORR) in advanced NSCLC patients receiving third line (3L) therapy who were resistant to previous checkpoint inhibitor treatments (CPI) and chemotherapy. Treatment with ateganosine followed by cemiplimab (Libtayo) has shown an acceptable safety profile to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

(Press release, MAIA Biotechnology, MAR 31, 2026, View Source [SID1234664101])

On march 31, 2026 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported publication of the final data from the Phase 1/2 ARROW study evaluating pralsetinib for the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) in the Journal of Clinical Oncology. The final data includes an additional 42 months of follow-up from previously published data. Pralsetinib is the only once daily, oral RET-inhibitor therapy that is designed to selectively target RET in metastatic NSCLC and advanced or metastatic thyroid carcinoma.

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"The final data from the ARROW study shows robust and durable responses with a manageable safety profile in patients with RET fusion-positive NSCLC, emphasizing the importance of early biomarker testing and suggesting that pralsetinib may be a valuable tool in the treatment armamentarium," said Justin F. Gainor, M.D., Phase 1/2 trial investigator and Director of the Center for Thoracic Cancers at Mass General Brigham Cancer Institute. "In addition, the responses observed in the subset of patients with measurable CNS metastases at baseline further expand the potential clinical value of pralsetinib in everyday practice."

"The results published in the Journal of Clinical Oncology demonstrate the positive impact pralsetinib can have for patients with RET fusion-positive NSCLC," said Lisa Rojkjaer, M.D, Rigel’s chief medical officer. "These longer-term data further support pralsetinib’s role as a first-line treatment option for RET fusion-positive NSCLC patients."

Additional key points from the paper include:

Consistent with previous reports from the ARROW study NSCLC cohort, pralsetinib was generally well tolerated with a manageable toxicity profile. Three treatment-related deaths occurred in treatment-naive patients in Asia (pneumonia, n=2; interstitial lung disease and rhabdomyolysis, n=1 each), no new safety signals were observed and no hypersensitivity reactions were reported in patients receiving prior immunotherapies.
Among patients with measurable disease (n=259), the overall response rate (ORR) was 70%, including 7% complete responses and 63% partial responses.
ORR was 78% among treatment-naïve patients and 63% among patients receiving prior platinum-based chemotherapy.
At final data lock, median treatment duration was 15.0 months.
Median overall survival (OS) was 44.3 months in the overall measurable disease patient population, 50.1 months in treatment-naïve patients, and 39.7 months in prior-platinum patients, with median follow-ups of 47.6, 43.7, and 49.7 months, respectively. Longer median OS in the overall measurable disease patient population was seen in patients treated in the United States (62.4 months) vs. Asia (44.5 months) or Europe (29.6 months).
Median overall progression-free survival (PFS) was 13.1 months in the overall measurable disease patient population, but was longer in patients in the United States (25.9 months) vs. Asia (12.6 months) or Europe (12.8 months).
ORR remained high in subgroups bearing the RET fusion partners KI5FB and CCDC6 among both treatment-naive and prior platinum-based chemotherapy patients. Among all patients, median duration of response (DOR) was longer in patients with CCDC6 (47.9 months) vs. KIF5B (13.1 months).
Fifteen patients had measurable central nervous system (CNS) metastases at baseline. The intracranial response rate (CNS ORR) among these patients was 53%. In the 11 response-evaluable patients with CNS metastases, CNS ORR was 73%.
The publication, titled "Final Efficacy and Safety Data From the Phase 1/2 ARROW Study of Pralsetinib in Patients With Advanced RET Fusion-Positive Non-Small Cell Lung Cancer (NSCLC)," was published online and can be found here.

About NSCLC
It is estimated that over 229,000 adults in the U.S. will be diagnosed with lung cancer in 2026. Lung cancer is the leading cause of cancer death in the U.S., with non-small cell lung cancer (NSCLC) being the most common type accounting for 77% of all lung cancer diagnoses.1 RET fusions are implicated in approximately 1-2% of patients with NSCLC.2

About GAVRETO (pralsetinib)

INDICATIONS

GAVRETO (pralsetinib) is indicated for the treatment of:

Adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test
Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)*
*This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

WARNING: SERIOUS INFECTIONS, INCLUDING OPPPORTUNISTIC INFECTIONS

GAVRETO may increase the risk for serious infections, including bacterial, fungal, viral and opportunistic infections, which can lead to hospitalization or death. Withhold, reduce the dose or permanently discontinue GAVRETO based on severity.

WARNINGS AND PRECAUTIONS

Serious Infections, Including Opportunistic Infections: GAVRETO may increase the risk for serious infections, including fatal and opportunistic infections. In the AcceleRET-Lung trial, infections occurred in 72% of patients who received GAVRETO, including 18% with Grade 3 and 3.7% with Grade 4 and 7% with fatal outcomes. Among the patients who received chemotherapy/immunotherapy, infections occurred in 52%, including 10% with Grade 3. Infections in the GAVRETO arm included pneumonia, urinary tract infection, opportunistic infections (such as pneumocystis jirovecii pneumonia and fungal infections) and others. Monitor patients for signs and symptoms of infection and treat appropriately. Withhold, reduce the dose, or permanently discontinue GAVRETO based on severity.
Interstitial Lung Disease (ILD)/Pneumonitis: Severe, life-threatening, and fatal ILD/pneumonitis can occur in patients treated with GAVRETO. Pneumonitis occurred in 12% of patients who received GAVRETO, including 3.3% with Grade 3-4, and 0.2% with fatal reactions. Monitor for pulmonary symptoms indicative of ILD/pneumonitis. Withhold GAVRETO and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue GAVRETO based on severity of confirmed ILD.
Hypertension: Occurred in 35% of patients, including Grade 3 hypertension in 18% of patients. Overall, 8% had their dose interrupted and 4.8% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate GAVRETO in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating GAVRETO. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue GAVRETO based on the severity.
Hepatotoxicity: Serious hepatic adverse reactions occurred in 1.5% of patients treated with GAVRETO. Increased aspartate aminotransferase (AST) occurred in 49% of patients, including Grade 3 or 4 in 7% and increased alanine aminotransferase (ALT) occurred in 37% of patients, including Grade 3 or 4 in 4.8%. The median time to first onset for increased AST was 15 days (range: 5 days to 2.5 years) and increased ALT was 24 days (range: 7 days to 3.7 years). Monitor AST and ALT prior to initiating GAVRETO, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue GAVRETO based on severity.
Hemorrhagic Events: Serious, including fatal, hemorrhagic events can occur with GAVRETO. Grade ≥3 events occurred in 4.1% of patients treated with GAVRETO including one patient with a fatal hemorrhagic event. Permanently discontinue GAVRETO in patients with severe or life-threatening hemorrhage.
Tumor Lysis Syndrome (TLS): Cases of TLS have been reported in patients with medullary thyroid carcinoma receiving GAVRETO. Patients may be at risk of TLS if they have rapidly growing tumors, a high tumor burden, renal dysfunction, or dehydration. Closely monitor patients at risk, consider appropriate prophylaxis including hydration, and treat as clinically indicated.
Risk of Impaired Wound Healing: Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, GAVRETO has the potential to adversely affect wound healing. Withhold GAVRETO for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of GAVRETO after resolution of wound healing complications has not been established.
Embryo-Fetal Toxicity: Based on findings from animal studies and its mechanism of action, GAVRETO can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with GAVRETO and for 2 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with GAVRETO and for 1 week after the last dose.
ADVERSE REACTIONS

Common adverse reactions (≥25%) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough. Common Grade 3/4 laboratory abnormalities (≥2%) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased phosphate, decreased leukocytes, decreased sodium, increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased calcium (corrected), decreased platelets, increased alkaline phosphatase, increased potassium, decreased potassium, and increased bilirubin.
DRUG INTERACTIONS

Avoid coadministration of GAVRETO with strong or moderate CYP3A inhibitors, P-gp inhibitors, or combined P-gp and strong or moderate CYP3A inhibitors. If coadministration cannot be avoided, reduce the GAVRETO dose. Avoid coadministration of GAVRETO with strong or moderate CYP3A inducers. If coadministration cannot be avoided, increase the GAVRETO dose.
Lactation: Advise women not to breastfeed during treatment with GAVRETO and for 1 week after the last dose.

Pediatric Use: Monitor open growth plates in adolescent patients. Consider interrupting or discontinuing GAVRETO if abnormalities occur.

Click here for Important Safety Information and Full Prescribing Information, including Boxed WARNING.

To report side effects of prescription drugs to the FDA, visit www.fda.gov/medwatch or call 1-800-FDA-1088 (800-332-1088).

GAVRETO is a registered trademark of Rigel Pharmaceuticals, Inc.

(Press release, Rigel, MAR 31, 2026, View Source [SID1234664084])