On May 12, 2026 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported the release of 12 Revuforj (revumenib) abstracts on the European Hematology Association (EHA) (Free EHA Whitepaper) website in advance of the EHA (Free EHA Whitepaper) 2026 Congress, taking place June 11-14, 2026, in Stockholm, Sweden.

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"The breadth of data accepted for presentation at EHA (Free EHA Whitepaper) underscores the strength of revumenib’s clinical profile, with activity observed across the acute leukemia treatment continuum in KMT2Ar, NPM1m, and NUP98r acute leukemias," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "Given the need for therapies that can reduce the risk of relapse after stem cell transplantation, we are excited for the presentation of new data from the post-transplant setting showing favorable outcomes with revumenib compared to historical data. We also look forward to sharing additional frontline and R/R combination data showing high rates of MRD negativity, transplant, and favorable tolerability, supporting physician decision making and our ongoing pivotal trials."

Dr. Botwood continued, "Collectively, these new data highlight our scientific leadership in menin inhibition and bolster our confidence that we are positioned to transform the treatment paradigm for 50% or more of patients with AML."

Key revumenib data accepted for presentation at EHA (Free EHA Whitepaper) 2026:

Findings from the ROAR study, a multicenter real-world study of revumenib in relapsed/refractory (R/R) acute leukemia.
Updated frontline data from the Phase 1 trial of revumenib in combination with intensive chemotherapy in NPM1 mutated (NPM1m), KMT2A-rearranged (KMT2Ar), or NUP98-rearranged (NUP98r) acute myeloid leukemia (AML).
Outcomes among adults and children with KMT2Ar, NPM1m, and NUP98r acute leukemia who received revumenib as maintenance following hematopoietic stem cell transplantation (HSCT).
Updated R/R data from the SAVE trial of revumenib in combination with venetoclax and decitabine/cedazuridine in NPM1m, KMT2Ar, and NUP98r acute leukemia.
Results in patients with R/R NUP98r acute leukemia treated with revumenib in AUGMENT-101 or via an expanded access program.
The accepted abstracts listed below are now available online on the EHA (Free EHA Whitepaper) conference website. Copies of the poster presentations will be made available in the ‘Publications & Meetings Presentations’ section of the Syndax website after the embargo lifts.

Full list of revumenib abstracts accepted for EHA (Free EHA Whitepaper) 2026 (all times in CEST):

Abstract Titles Presentation Details
Real-world evidence
Revumenib in the real world: interim findings from the ROAR study in relapsed/refractory acute leukemia Abstract Code: PF542
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Frontline
Revumenib + intensive chemotherapy for newly diagnosed acute myeloid leukemia harboring genetic alterations in KMT2A, NPM1, or NUP98: updated phase 1 results from SNDX-5613-0708 Abstract Code: PF489
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Post-HSCT maintenance
Revumenib as maintenance for AML following allogeneic stem cell transplantation Poster number: PS1629
Poster Session 2
Saturday, June 13, 6:45-7:45 pm
Encore of data accepted for oral presentation at ASCO (Free ASCO Whitepaper) 2026
Revumenib therapy post hematopoietic stem cell transplant for patients with relapsed/refractory KMT2Ar, NPM1m, and NUP98r acute myeloid leukemia: post hoc analysis of outcomes from AUGMENT-101 Abstract Code: PS1631
Poster Session 2
Saturday, June 13, 6:45-7:45 pm
Relapsed/refractory
Phase 1/2 study of the all-oral combination of revumenib (SNDX-5613) with decitabine/cedazuridine (ASTX727) and venetoclax (SAVE) in relapsed/refractory AML Abstract Code: PF495
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Long-term follow-up of pediatric/young adult patients with relapsed/refractory KMT2Ar acute leukemia treated with revumenib in AUGMENT-101 Abstract Code: PF508
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Efficacy of revumenib in acute myeloid leukemia harboring NPM1-mutated co-mutations: post hoc analysis of AUGMENT-101 Abstract Code: PF514
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Pharmacokinetic assessment of revumenib in patients with relapsed/refractory acute leukemias harboring a KMT2A rearrangement or NPM1 mutation: Impact of food and concomitant medications Abstract Code: PF564
Poster Session 1
Friday, June 12, 6:45-7:45 pm
Encore of data accepted for poster presentation at ASCO (Free ASCO Whitepaper) 2026
Clinical activity of revumenib in patients with relapsed/refractory NUP98-rearranged acute leukemias Abstract Code: PS1607
Poster Session 2
Saturday, June 13, 6:45-7:45 pm
Trials in progress
A phase 3 study of revumenib in combination with intensive chemotherapy in patients with newly diagnosed NPM1-mutated acute myeloid leukemia (REVEAL-ND NPM1): Trial in progress Abstract Code: PB2821
Publication only
Revumenib + venetoclax/azacitidine in adults with newly diagnosed NPM1m or KMT2Ar acute leukemia ineligible for intensive chemotherapy (EVOLVE-2/HO177/AMLSG35-24/ACT-HOV-AML-002): Trial in progress Abstract Code: PB2796
Publication only
A phase 1/2 study of the menin inhibitor revumenib with the CELMod mezigdomide in relapsed/refractory KMT2A-rearanged, NPM1-mutant, and NUP98-rearranged acute leukemias Abstract Code: PS1658
Poster Session 2
Saturday, June 13, 6:45-7:45 pm

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME, QTc PROLONGATION, and TORSADES DE POINTES

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

QTc prolongation and Torsades de Pointes have occurred in patients receiving Revuforj. Correct hypokalemia and hypomagnesemia prior to and during treatment. Do not initiate Revuforj in patients with QTcF > 450 msec. If QTc interval prolongation occurs, interrupt, reduce, or permanently discontinue Revuforj.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, rash, and/or hypotension.

In clinical trials, DS occurred in 60 (25%) of 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia. Among those with a KMT2A translocation, DS occurred in 33% of patients with acute myeloid leukemia (AML), 33% of patients with mixed-phenotype acute leukemia (MPAL), and 9% of patients with acute lymphoblastic leukemia (ALL); DS occurred in 18% of patients with NPM1m AML. DS was Grade 3 or 4 in 12% of patients and fatal in 2 patients. The median time to initial onset was 9 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours in adults or dexamethasone 0.25 mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc Interval Prolongation and Torsades de Pointes: Revuforj can cause QT (QTc) interval prolongation and Torsades de Pointes.

Of the 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia in clinical trials, QTc interval prolongation was reported as an adverse reaction in 86 (36%) patients. QTc interval prolongation was Grade 3 in 15% and Grade 4 in 2%. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 10%, and the increase from baseline QTcF was greater than 60 msec in 24%. Revuforj dose reduction was required for 7% due to QTc interval prolongation. QTc prolongation occurred in 21% of the 34 patients less than 17 years old, 35% of the 146 patients 17 years to less than 65 years old, and 46% of the 61 patients 65 years or older. One patient had a fatal outcome of cardiac arrest, and one patient had non-sustained Torsades de Pointes.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to and throughout treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia
Embryo-Fetal Toxicity: Revuforj 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 and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 9 (4%) patients who received Revuforj, including 4 with sudden death, 2 with differentiation syndrome, 2 with hemorrhage, and 1 with cardiac arrest.

Serious adverse reactions were reported in 184 (76%) patients. The most frequent serious adverse reactions (≥10%) were infection (29%), febrile neutropenia (20%), bacterial infection (15%), differentiation syndrome (13%), and hemorrhage (11%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were phosphate increased (51%), hemorrhage (48%), nausea (48%), infection without identified pathogen (46%), aspartate aminotransferase increased (44%), alanine aminotransferase increased (40%), creatinine increased (38%), musculoskeletal pain (37%), febrile neutropenia (37%), electrocardiogram QT prolonged (36%), potassium decreased (34%), parathyroid hormone intact increased (34%), alkaline phosphatase increased (33%), diarrhea (29%), bacterial infection (27%), triglycerides increased (27%), phosphate decreased (25%), differentiation syndrome (25%), fatigue (24%), edema (24%), viral infection (23%), decreased appetite (20%), and constipation (20%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec
SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: no overall differences were observed in the effectiveness of Revuforj between patients who were 65 years and older, and younger patients. Compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNINGS.

(Press release, Syndax, MAY 12, 2026, View Source [SID1234665560])

On May 12, 2026 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the first quarter ended March 31, 2026, and provided a corporate update.

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"The first quarter of 2026 marked an important period for SELLAS as we continued to execute across our clinical programs while preparing for the anticipated pivotal Phase 3 REGAL trial data readout," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "We are grateful for the ongoing commitment of the patients, caregivers, and investigators participating in the pivotal Phase 3 REGAL trial and we believe that the upcoming results, triggered by reaching the pre-specified 80th event, will be an important milestone for the Company."

"In parallel, we are generating increasingly compelling data with SLS009, including recent preclinical findings presented at AACR (Free AACR Whitepaper) demonstrating potent, mechanistically driven activity in AML through suppression of key survival pathways such as MCL-1 and induction of apoptosis, including in high-risk genetic subtypes like TP53 and ASXL1 mutations. Importantly, we have begun dosing patients in our Phase 2 study of SLS009 in newly diagnosed, first-line AML, targeting high-risk populations unlikely to benefit from standard therapies, including venetoclax. With REGAL approaching its critical readout and SLS009 advancing across clinical and translational fronts, we believe that SELLAS is entering an exciting time with multiple, high-value potential catalysts that could transform the AML treatment landscape."

Recent Corporate Highlights:

Phase 3 REGAL Trial of GPS: Ongoing Phase 3 REGAL trial evaluating GPS in AML patients who have achieved complete remission following second-line salvage therapy. Reaching the required pre-specified 80th event (death) will trigger the customary database lock, blinded data review procedures prior to statistical analysis, unblinding, and disclosure of topline results. As of May 11, 2026, 78 events have occurred and SELLAS will provide an update and announce when the 80th event has been reached.

Ongoing dosing of SLS009 in earlier-line AML: SELLAS has initiated an 80-patient Phase 2 trial in newly diagnosed AML patients, including those who become refractory early to AZA/VEN treatment identified through extensive transcriptomics, genomics, and proteomics models. The topline data are expected in Q4 2026. Additional information about the trial can be found at clinicaltrials.gov (NCT04588922).

Preclinical Data on SLS009 in AML Presented at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper): The data show that SLS009 induces apoptosis in AML cell lines, including those harboring high-risk ASXL1 and TP53 mutations. Pharmacodynamic changes were observed as early as 8 hours after treatment and became more pronounced over time, with reductions in MCL-1 and survivin levels that correlated with increased apoptosis. The poster, entitled "Tambiciclib (SLS009), a CDK9 inhibitor, promotes apoptosis and suppresses MCL-1 levels in AML cell lines," can be viewed here.

At-the-Market (ATM) Offering: SELLAS established an ATM equity offering under its S-3ASR shelf registration, providing the Company with the ability to raise up to $150 million in capital over time. The facility, to be utilized through TD Cowen, enables flexible and opportunistic access to the equity markets. The Company has not sold any shares of common stock through its ATM to date.

Financial Results for the First Quarter 2026:

Research and Development Expenses: Research and development expenses for the quarter ended March 31, 2026, were $5.1 million, compared to $3.2 million for the same period in 2025. The increase was primarily due to increases in manufacturing costs, clinical and regulatory consulting, and clinical trial expenses in preparation for a potential Biologics License Application for GPS following the final analysis of the REGAL study.

General and Administrative Expenses: General and administrative expenses for the first quarter of 2026 were $4.1 million, as compared to $2.9 million for the same period in 2025. The increase was primarily due to increases in professional fees, consulting and public company costs, and non-cash stock-based compensation.

Net Loss: The net loss was $8.4 million for the first quarter of 2026, or a basic and diluted loss per share of $0.05, as compared to a net loss of $5.8 million for the first quarter of 2025, or a basic and diluted loss per share of $0.07.
Cash Position: As of March 31, 2026, cash and cash equivalents totaled approximately $107.1 million. Subsequent to March 31, 2026, the Company received an additional $7.5 million in proceeds from the exercise of previously outstanding warrants.

(Press release, Sellas Life Sciences, MAY 12, 2026, View Source [SID1234665559])

On May 12, 2026 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported the presentation of preclinical data demonstrating that a surrogate SG293, an in vivo CAR T cell therapy, achieved cell-specific delivery, robust and dose-dependent CAR T cell generation, and deep B cell depletion in non-human primates (NHPs) without the use of lymphodepleting chemotherapy. SG293 is a CD8-targeted fusosome that delivers the genetic material to make CD19-directed CAR T cells. The data were reported in an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting.

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"SG293 represents a differentiated in vivo CAR T cell approach designed to work without lymphodepletion to deliver potent therapeutic activity and exceptional specificity to minimize off-target effects," said Dhaval Patel, MD, PhD, Executive Vice President, Chief Scientific Officer. "Data presented at ASGCT (Free ASGCT Whitepaper) highlight the fusogen platform’s potential to enable development of therapies that offer off-the-shelf treatment options for patients with blood cancers, B cell-mediated autoimmune disorders, and multiple myeloma. The data support our strategy of advancing SG293 into the clinic for the treatment of non-Hodgkin lymphoma (NHL) later this year and SG227, a BCMA-targeted in vivo CAR T cell therapy, into the clinic for the treatment of multiple myeloma as early as mid-2027."

In an NHP model, a one-time intravenous administration of the surrogate SG293 led to potent CAR T cell generation, dose-dependent CAR T cell expansion, and complete peripheral B cell depletion. At 3 weeks, B cells were undetectable or minimally detectable in lymph nodes, and as B cells returned after depletion, the vast majority exhibited a naïve phenotype indicative of a "reset" of the B cell compartment. The targeted fusogen used in SG293 demonstrates a differentiated level of protection from off-target delivery risks in vitro when compared to other targeted fusogen technologies. The specific delivery and favorable on-target safety profile were confirmed in vivo in NHPs with surrogate SG293. Post-necropsy analysis of tissues showed no evidence of delivery to non-target cells, including hepatocytes, heart, or gonadal tissue. Post-infusion symptoms were mild and managed with acetaminophen, and CAR T-associated toxicities were manageable and consistent with autologous CAR T toxicities in this NHP model. Finally, the novel transgene design used in SG293 diminishes CAR protein incorporation onto the vector during manufacturing, which mitigates anti-CAR immunogenicity in NHPs and may enable improved durability of in vivo CAR T cells. These data demonstrate that SG293 represents a differentiated approach for enabling potent and precise in vivo CAR T cell therapy for oncology and autoimmune indications. Sana intends to explore SG293 initially in NHL and expects to generate first-in-human data as early as this year. If successful, the company intends to expand clinical development with SG293 into B cell-mediated autoimmune diseases and to initiate clinical development of SG227 for patients with multiple myeloma.

About SG293
SG293, which uses Sana’s proprietary fusogen-based in vivo delivery technology, is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding potentially troublesome delivery to tissues such as the liver, heart, and gonadal tissue. Sana intends to explore SG293 in both B cell cancers and B cell-mediated autoimmune diseases.

About SG227
SG227, which uses Sana’s proprietary fusogen-based in vivo delivery technology, is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make BCMA-directed CAR T cells while avoiding potentially troublesome delivery to tissues such as the liver, heart, and gonadal tissue. Sana intends to explore SG227 as a potential therapy for the treatment of multiple myeloma.

(Press release, Sana Biotechnology, MAY 12, 2026, View Source [SID1234665558])

On May 12, 2026 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorders, reported financial results for the first quarter ended March 31, 2026 and provided a corporate update.

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"We made significant progress in the first quarter, highlighted by the robust 12-month Phase 2 data for NDV-01 in NMIBC and the successful completion of a $160 million PIPE financing, which has well-capitalized our balance sheet to fund the NDV-01 RESCUE Phase 3 program through completion," said Sergio Traversa, Chief Executive Officer of Relmada Therapeutics. "We remain on track to file the NDV-01 IND and initiate the Phase 3 RESCUE registrational program in mid-2026 – a milestone that would mark a major inflection point for Relmada and for the patients we aim to serve. We believe NDV-01 has the potential to be a best-in-class therapy for patients with NMIBC and remain focused on maximizing its potential for success. To this end, in April, we filed a provisional patent application in the U.S. directed to formulations and methods of treatment for NDV-01. This application, if issued, could form the basis for worldwide patent filings, and have a term into 2047."

"The AUA2026 Annual Meeting provides an important opportunity to introduce NDV-01 to the broader urologic community," said Raj S. Pruthi, MD, Chief Medical Officer – Urology of Relmada Therapeutics. "Our presentations will highlight the 12-month Phase 2 data generated to date for NDV-01, including observed complete responses and safety findings. We will also be sharing the design and rationale for the Phase 3 RESCUE program. NDV-01 is a sustained-release gemcitabine and docetaxel (Gem/Doce) designed to support streamlined, in-office administration in less than five minutes. We believe AUA2026 provides an important national forum to increase awareness and engagement within the investigator community as we approach the initiation of the RESCUE program in mid-2026."

Upcoming AUA2026 Presentations and NDV-01 Phase 2 Data Highlights:

Relmada will present two abstracts at AUA2026 including: (1) NDV-01 Phase 2 data (12-month follow-up), and (2) the Phase 3 RESCUE program design (Clinical Trials in Progress session). The presentations are intended to raise awareness of NDV-01 and build investigator interest in the RESCUE registrational program. Key data to be highlighted include:

95% complete response (CR) rate at any time and durable 76% CR rate at 12 months in high-risk NMIBC patients
94% CR rate at any time and durable 80% CR rate at 12 months in BCG-unresponsive NMIBC patients
No patient had progression to muscle-invasive disease, and no patient underwent a radical cystectomy
Favorable overall tolerability – no ≥ Grade 3 treatment-related adverse events and no treatment-related discontinuations or dose interruptions

NDV-01 Intellectual Property:

In April 2026, Relmada filed a provisional patent application with the United States Patent and Trademark Office (USPTO) directed to pharmaceutical formulations and methods of treatment related to NDV-01. The provisional filing has the potential to form the basis for a comprehensive world-wide patent filing program for NDV-01. If issued, patents claiming priority to the provisional filing will be expected to have a term until April 2047.

Expected Upcoming Relmada Milestones:

NDV-01 United States IND filing – Mid-2026
NDV-01 Phase 3 RESCUE Program initiation – Mid-2026
Sepranolone Phase 2 initiation in Prader-Willi syndrome – Mid-2026
Initial 3-month NDV-01 data from Phase 3 2L BCG-unresponsive study – YE 2026

Financial Results

First Quarter 2026 Financial Results

Research and development expense for the three months ended March 31, 2026, totaled $8.1 million, compared to $12.0 million for the three months ended March 31, 2025, a decrease of $3.9 million. The decrease was primarily attributable to non-recurring costs associated with the acquisition of sepranolone and the license agreement of NDV-01 recognized in 2025. This 2026 decrease was partially offset by increased costs related to the start-up of the Phase 3 NDV-01 trials and Phase 2b sepranolone study and additional R&D personnel.
General and administrative expense for the three months ended March 31, 2026, totaled $11.4 million compared to $6.3 million for the three months ended March 31, 2025, an increase of approximately $5.1 million. The increase was primarily driven by an increase in compensation costs partially offset by a decrease in stock-based compensation costs.
Net cash used in operating activities for the three months ended March 31, 2026, totaled $15.1 million compared to $18.1 million for the three months ended March 31, 2025.
The net loss for the three months ended March 31, 2026, was $19.1 million, or $0.22 per basic and diluted share, compared with a net loss of $17.6 million, or $0.58 per basic and diluted share, for the three months ended March 31, 2025.
The Company’s balance of $234.0 million in cash, cash equivalents, and short-term investments, includes net proceeds of approximately $150 million from the private placement financing announced March 9, 2026. This compares to cash, cash equivalents, and short-term investments of approximately $93.0 million at December 31, 2025.
The Company’s current cash, cash equivalents, and short-term investments as of March 31, 2026, is expected to provide sufficient resources to fund Company operations through 2029, including completion of the Phase 3 NDV-01 RESCUE program.
The Company had 104,890,223 shares outstanding, as of May 7, 2026

Conference Call and Webcast Information:
Relmada will host a conference call and webcast today at 4:30 PM ET to discuss recent business progress and financial results.

(Press release, Relmada Therapeutics, MAY 12, 2026, View Source [SID1234665557])

On May 12, 2026 The Board of Directors of Recordati S.p.A. approved the Group’s Interim Report as of 31st March 2026, representing additional voluntary financial reporting. The report was prepared using the assessment, measurement and recognition criteria prescribed by international accounting standards (IFRS). The financial statements as of 31st March 2026 will be available by 15th May 2026 at the company’s offices and on the company’s website (www.recordati.it) and can also be viewed on the authorised storage system 1Info (www.1Info.it).

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Rob Koremans, Chief Executive Officer of Recordati, commented: "Recordati has delivered a strong start to the year, driven by excellent momentum in Rare Diseases and resilient in-market growth of the Specialty & Primary Care promoted portfolio. We are pleased to report further progress in advancing our R&D pipeline. We see significant opportunity for pasireotide in PBH following positive Phase 2 results and are progressing sutimlimab into a Phase 3 study in ITP. Isturisa continues to perform very well, with ongoing expansion in the U.S. across both overt and non-overt Cushing’s syndrome populations. Overall, we remain well positioned to deliver on our strategic and financial objectives for 2026 and beyond."

Q1 2026 Financial highlights

Consolidated net revenue for the first quarter of 2026 was € 713.4 million, up 4.9% or 8.7% on a like-for-like(3) basis at CER (+7.9% excluding Türkiye) versus the first quarter of 2025. This was driven, in particular, by the strong momentum from the Rare Diseases business. The adverse FX impact for the first quarter of 2026 was € 29.1 million (-4.3%), mainly driven by the U.S. dollar and Turkish lira devaluation.

Rare Diseases revenue was € 292.4 million for the first quarter of 2026, up 14.8%, or 22.4% at CER as compared to the first quarter of 2025, driven by strong volume growth across the Endocrinology and Hemo-Oncology franchises. The Endocrinology franchise achieved net revenue of € 120.7 million, an increase of 38.1%, reflecting continued growth of Isturisa (€ 86.3 million, +56.8%), driven mostly by strong new patient uptake across geographies, particularly in the U.S., and growth of Signifor (€ 34.4 million, +6.3%). The Hema-Oncology franchise achieved net revenue of € 113.2 million, growing by 18.2%, reflecting the strong momentum of Enjaymo across geographies (€ 43.9 million, +37.6%) and Qarziba (€ 42.9 million, +13.9%), as well as growth of Sylvant (€ 22.8, million +1.8%). The Metabolic franchise achieved net revenue of € 58.5 million, a decrease of 18.3%, reflecting phasing of Carbaglu across geographies and slightly lower demand of Panhematin in the U.S. against a strong performance in the first quarter of 2025.

Specialty & Primary Care revenue was € 404.4 million for the first quarter of 2026, down 1.0% or up 0.2% on a like-for-like basis(3) at CER as compared to the first quarter of 2025(7), reflecting continued in-market growth of the promoted portfolio (+5%(8)) and some expected one-off headwinds. In particular, the Cardiovascular franchise achieved net revenue of € 113.7 million, an increase of 1.5%, and the Gastrointestinal franchise achieved net revenue of € 69.4 million, an increase of 2.1%, with continued good in-market performance of key products in both therapeutic areas. The Urology franchise achieved net revenue of € 104.4 million, a decrease of 4.3%, due to a high prior-year base following a product relaunch in Russia in 2025, and the Cough & Cold franchise achieved net revenue of € 29.6 million, a decrease of 12.8% due to a weaker season in key markets.

EBITDA(1) was € 283.6 million for the first quarter of 2026, up 5.0% compared to the first quarter of 2025, with margin of 39.7% of net revenue. Strong revenue performance and the positive mix effect at gross profit level was partially offset by a higher level of investments to support the U.S. expansion, primarily for Isturisa, the continued development of Enjaymo, ongoing geographic expansion in Rare Diseases as well as the launch of Vazkepa in Specialty & Primary Care.

Adjusted operating income(9) was €231.1 million in the first quarter of 2026, an increase of 5.4% versus the first quarter of 2025. This represents 32.4% of net revenue, compared with 32.2% in the prior year, supported by strong operating performance. Operating income was € 229.6 million in the first quarter of 2026, up 17.3% over the first quarter of 2025, reflecting gross margin-related non-cash charges of € 22.4 million in 2025, arising mostly from the unwind of the fair value step up of the acquired Enjaymo inventory. Non-recurring costs were € 1.5 million versus € 1.1 million in the first quarter of 2025.

Financial expenses were € 28.9 million, down by € 2.1 million compared to the previous year. Net exchange losses over the period were € 1.9 million, slightly higher as compared to losses of € 1.8 million in the first quarter of 2025. The impact of hyperinflation was negative € 2.0 million, the same as in the first quarter of 2025.

Adjusted net income(2) was € 188.1 million, 26.4% of net revenues, up by 7.2% compared to the same period of 2025, benefitting from higher adjusted operating income, lower financial expenses and a lower tax rate resulting from a positive country mix. Net income was € 153.1 million, 21.5% of net revenue, an increase of 22.4% versus the prior year, reflecting positive operating income, lower financial expenses and a lower income tax rate versus the first quarter of 2025.

Free cash flow(4) was € 92.1 million for the first quarter of 2026, a decrease of € 66.7 million versus the first quarter of 2025, reflecting higher EBITDA which was offset by higher working capital absorption and income taxes paid.

Net debt(5) as of March 31, 2026 was € 1,985.2 million, or leverage of just below 2.0x EBITDA, compared to net debt of € 2,037.3 million on December 31, 2025.

Shareholders’ equity was € 2,060.3 million.

Pipeline Development

The Phase 2 trial evaluating pasireotide for the treatment of post-bariatric hypoglycemia met its primary endpoint with a dose-dependent and significant increase in glucose levels during a standardized meal test (p<0.02)(10). This was associated with a lowering of level 2 and 3 hypoglycemia (not significant), particularly in patients with higher baseline hypoglycemia rates (post-hoc analysis). Recordati is scheduled to meet with the FDA to discuss potential next steps.

Immune thrombocytopenia (ITP) is a rare autoimmune disease, characterized by increased platelet destruction and decreased platelet production/release. Main symptoms include an increased risk of bleeding events, fatigue, decreased quality of life, increased risk of thrombosis (increased morbidity and mortality). Refractory ITP represents a significant unmet need, with around 20-30% failing several lines of therapy. On the basis of encouraging FDA feedback as well as early encouraging clinical evidence showing that sutimlimab, by targeting the classical complement pathway, can lead to a rapid and sustained platelet response in patients refractory to multiple lines of treatment, Recordati has decided to advance sutimlimab into a pivotal registrational Phase 3 trial for the treatment of chronic ITP.

The other lifecycle management programs are progressing in line with plans.

Corporate Development

On January 29, 2026, Recordati announced a collaboration and license agreement with Moderna to develop and commercialize worldwide mRNA-3927, an investigational product for the treatment of propionic acidemia (PA). Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, in collaboration with Recordati, and if approved, Recordati will lead global commercialization. mRNA-3927 is a post proof-of-concept, investigational product aimed to restore propionyl-CoA carboxylase (PCC) enzyme activity in patients with propionic acidemia. If approved, this could be the first disease-modifying treatment option on the market for this severe disease. mRNA-3927 is currently being evaluated in a potential registrational clinical study. The target patient enrollment has been reached, with a potential data readout expected by the end of 2026.

Business outlook

The Group confirms its financial targets for full year 2026 as follows:

Net revenue between € 2,730 and € 2,800 million with FX headwind of ~-3.5%
EBITDA(1) between € 995 and € 1,030 million; margin of +/- 36.5% with FX headwind of ~-4.0%
Adjusted net income(2) between € 655 and € 685 million; margin of +/- 24.0%
The full year 2027 targets(11) remain unchanged, with strong organic growth complemented by bolt-on business development and M&A.

Additional resolutions

Performance share plan 2023-2025: assessment of the level of achievement of the performance targets for the first cycle (2023 grant)

With reference to the incentive plan of Recordati S.p.A. ("Recordati" or the "Company") named the "2023–2025 Performance Share Plan" (the "Plan"), approved by the Shareholders’ Meeting of the Company held on 21st April 2023, and to the related first award cycle resolved upon by the Board of Directors on 27th June 2023, notice is hereby given that, on today’s date, the Board of Directors of the Company, acting upon the proposal of the Remuneration and Appointments Committee, to the extent within its remit, and in accordance with the provisions of the relevant rules, has assessed the level of achievement of the performance targets relating to the first cycle of the Plan.

On 27th June 2026, being the date on which the vesting period will be completed, the beneficiaries’ entitlement to receive up to a maximum aggregate number of 388,630 ordinary shares of Recordati, granted free of charge pursuant to the Plan, will vest. Such number may be reduced in accordance with the terms and conditions of the Plan, including in the event of termination of the relationship with the Recordati Group prior to such date. Within the above maximum aggregate number, the shares attributable to the Chief Executive Officer of Recordati amount to no. 30,485.

For further information on the Plan (12), reference should be made to the relevant Information Document, which is available on the Company’s website in the "Governance" section, under "Remunerations".

Performance share plan 2026-2028: assignment of rights provided under the first cycle (subject to certain conditions)

With reference to the "2026–2028 Performance Share Plan" (the "Plan") of Recordati approved by its shareholders’ meeting on 29th April 2026, notice is hereby given that today the Board of Directors of the Company, upon proposal of the Company’s Remuneration and Nominations Committee – within the scope of its competence and in accordance with the provisions of the relevant regulations – resolved to grant to the beneficiaries of the Plan a total of no. 528,913 rights (the "Rights") to receive ordinary shares of the Company, which fall within the first allocation cycle of the Plan. The Chief Executive Officer of Recordati has been assigned no. 24,251 Rights.

The grant of the Rights will cease to have any effect in case the potential voluntary tender offer on all ordinary shares of the Company aimed at its delisting – communicated on 25 March 2026(13) by CVC to the Company by means of a conditional non-binding indication of interest – is launched and the delisting is completed by the date of the Annual General Meeting approving the 2026 financial statements.

For further information on the Plan (12), reference should be made to the relevant Information Document, which is available on the Company’s website in the "Governance" section, under "Remunerations".

(1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS.
(2) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3, monetary net gains/losses from hyperinflation (IAS 29), net of tax effects.
(3) Proforma growth calculated excluding revenue of Vazkepa for Q1 2026 and Cardicor for Q1 2025 and Q1 2026 (Specialty & Primary Care).
(4) Total cash flow excluding financing items, milestones, dividends, purchases of treasury shares net of proceeds from exercise of stock options.
(5) Cash and cash equivalents, less bank debts and loans, which include the measurement at fair value of hedging derivatives.
(6) Italian Legislative Decree 25/2016, which implements Directive 2013/50/EU, no longer stipulates the submission of an interim management report, which was previously required in terms of paragraph 5 of Art. 154-ter of Legislative Decree 58/1998
(7) The 2025 figures have been restated to reflect the reclassification of certain brands from Other Therapeutic areas to Cardiovascular and Gastrointestinal areas in 2026. The amount of reclassification for Q1 2025 is as follows: €2.5 million from Other Therapeutic areas to Cardiovascular and €4.4 million from Other Therapeutic areas to Gastrointestinal.
(8) IQVIA Feb RQ-2026 vs Feb RQ-2025.
(9) Net income before income taxes, financial income and expenses and non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3.
(10) p=0.0106 (50 µg s.c. pasireotide vs placebo); p=0.0010 (100 µg s.c. pasireotide vs. placebo); p< 0.0001 (200 µg s.c. pasireotide vs placebo).
(11) FY 2027 targets: Net Revenue €3,000 – €3,200 million, EBITDA €1,140 – €1,225 million, Adjusted Net Income €770- €820 million, excluding potential impact from tariffs and/or most favored nation pricing policies in the U.S.
(12) Table no. 1 referred to in paragraph 4.24 of Annex 3A, Form 7, to the Issuers’ Regulation no. 11971/1999, will be published in the manner and within the timeframe set out in Article 84-bis, paragraph 5, letter a), of the same Issuers’ Regulation.
(13) Please refer to the press release issued by the Company on this regard on 26th March 2026.

Conference Call

Recordati will host a conference call on May 13th, at 2:00 p.m. CET (1:00 p.m. GMT+1) to present the results for the first quarter of 2026. Please find the pre-registration link here with all the dial-in details and a calendar invitation to follow.

Alternatively, if not pre-registered, the dial-in numbers for the conference call are:

Italy + 39 02 802 09 11, toll free 800 231 525
UK + 44 1 212818004, toll free (44) 0 800 0156371
USA +1 718 7058796, toll free (1) 1 855 2656958
France +33 1 70918704
Germany +49 6917415712

Participants are invited to dial in 10 minutes before the start of the conference call. If operator assistance is required to connect, please dial *0.

The slides that will be referenced during the call will be available at www.recordati.com under Investors/Company Presentations.

(Press release, Recordati, MAY 12, 2026, View Source [SID1234665556])