On May 5, 2025 Recursion (Nasdaq: RXRX), a leading clinical stage TechBio company decoding biology to radically improve lives, reported business updates financial results for its first quarter ending March 31, 2025 (Press release, Recursion Pharmaceuticals, MAY 5, 2025, View Source [SID1234652517]).

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Recursion will host a L(earnings) Call on May 5, 2025 at 8:00 am ET / 6:00 am MT / 1:00 pm BST from Recursion’s X (formerly Twitter), LinkedIn, and YouTube accounts giving analysts, investors, and the public the opportunity to ask questions of the company by submitting question here: View Source

"Recursion’s decade-long investment in AI is driving a decisive, data-led portfolio strategy," said Chris Gibson, Co-Founder and CEO of Recursion. "We are prioritizing high-potential programs to accelerate better treatments to patients, building on our platform’s unique ability to learn and lead this transformative shift in drug discovery. Our deep appreciation goes to the patients and investigators whose participation is invaluable on this journey."

Summary of Business Highlights

Pipeline Updates
"Our portfolio evolution reflects Recursion’s commitment to advancing medicines in areas of high unmet need where we believe we can have the greatest impact," said Najat Khan, Chief R&D Officer and Chief Commercial Officer at Recursion. "As part of our business combination with Exscientia, we are proactively streamlining our portfolio, platform, and operations, making deliberate tradeoffs to focus resources on programs with the strongest scientific rationale and the highest potential for near- and long-term impact. Powered by the integrated Recursion OS platform, we are advancing a focused set of differentiated internal and partnered programs. Our approach is grounded in rigorous science and evidenced by the consistent delivery of key milestones with leading pharmaceutical and technology collaborators."

•Preliminary Program Data:
▪REC-4881 (MEK1/2): Preliminary data presented at DDW as a late-breaking oral presentation on May 4, 2025 from the ongoing Phase 1b/2 TUPELO study of REC-4881 in familial adenomatous polyposis (FAP):
•In the Phase 2 open-label study, REC-4881 (4 mg QD) led to a preliminary median 43% reduction (n=6 patients) in polyp burden at the week 13 assessment at time of data cutoff.
•Five of six patients (83%) experienced reductions in polyp burden ranging from 31% to 82%, however, one patient showed a substantial increase from baseline.
•At Week 13, 50% of patients (3 out of 6) achieved ≥1-point improvement in Spigelman stage, a measure of upper GI disease severity.
•The early safety profile of REC-4881 was generally consistent with that of prior MEK1/2 inhibitors; among 19 patients across Phase 1b and 2, most treatment-related adverse events were Grade 1 or 2, with Grade 3 events in 16% of patients and no Grade ≥4 TRAEs reported to date.
▪REC-7735 (PI3Kα H1047R): Candidate profiling ongoing targeting PI3Kα H1047R mutant breast cancer; DC nomination expected 2H25:
•Highly selective and structurally differentiated molecule to reduce dose-limiting hyperglycemia.
•REC-7735 showed dose-dependent tumor regression in PI3Kα H1047R CDX models, with no elevation in insulin levels or hyperglycemia markers in wild-type mice, unlike standard-of-care PI3K inhibitors.
•Demonstrated dose-dependent tumor regression in preclinical models, with low-dose REC-7735 outperforming high-dose capivasertib (AKT inhibitor) in efficacy and tolerability.
▪REV102 (ENPP1): IND-enabling studies ongoing for hypophosphatasia (HPP) in development with Rallybio; Phase 1 initiation expected 2H26:
•Highly selective and orally bioavailable molecule supports QD or BID dosing.
•In vivo data in early-onset HPP model shows improved survival while treatment in late-onset HPP model improves bone defects.
•Preliminary data supports first-in-class potential for adult-onset HPP.
◦Additional Strategic Pipeline Programs
▪Focus on highest value programs in core therapeutic areas (oncology and rare disease):
•REC-617 (CDK7): Phase 1/2 ELUCIDATE study ongoing in advanced solid tumors; molecule designed to maximize therapeutic index; best-in-class potential.
•REC-1245 (RBM39): Phase 1/2 DAHLIA study with dose-escalation ongoing for biomarker-selected solid tumors and R/R lymphomas; novel mechanism of action identified to modulate DDR; first-in-class potential.
•REC-3565 (MALT1): Phase 1 EXCELERIZE study recently initiated for B cell malignancies; designed to avoid UGT1A1 on-target toxicity; best-in-class potential.
•REC-4539 (LSD1): Precision designed for reversibility and CNS penetration in solid tumors (e.g.; SCLC); strategic pause to ensure a competitive Target Product Profile.
•Continued focus on advancing additional discovery programs that meet key criteria.
▪As part of this prioritization, the Company will discontinue development and/or pursue partnering opportunities for the following clinical programs:
•REC-2282 (NF2): Totality of data supports the discontinuation of the study
◦New findings: Phase 2 passed the futility threshold primarily driven by the 40mg cohort, however the 60mg and combined dose arms did not pass the futility criteria.
◦Limited overall tumor shrinkage and clinical activity across all arms.
•REC-994 (CCM): Totality of data supports the discontinuation of study
◦Early data suggested potential promising trends in exploratory efficacy endpoints at 400mg (mean volume reduction, mRS), negative trends in efficacy at 200mg (data were not statistically significant).
◦New findings: Long-term extension results showed no promising trends in MRI or functional outcomes in the placebo-to-400mg crossover, and the 400mg-to-400mg arm did not continue prior trends and was indistinguishable from natural history.
•REC-3964 (C. difficile): The company will consider out-licensing opportunities
◦Evolved treatment options result in low recurrence rates (~5%); thus limiting unmet need.
◦Strategic decision to focus on other areas with greater unmet need.

◦Upcoming milestones:
▪REC-617 (CDK7): On track to initiate CDK7 combination studies in 1H25, additional monotherapy data expected in 2H25.
▪REC-4881 (MEK1/2): Additional data in FAP from TUPELO expected in 2H25.

▪REC-7735 (PI3Kα H1047R): preclinical studies ongoing with development candidate expected in 2H25.
▪REC-1245 (RBM39): Early Phase 1 safety and PK monotherapy data expected in 1H26.
▪REC-3565 (MALT1): Early Phase 1 safety and PK monotherapy data expected in 2H26.
▪REV102 (ENPP1): Phase 1 initiation expected in 2H26.

Partnerships Updates
Recursion and Sanofi advanced their fourth partnered program through a significant discovery milestone. This milestone involved the Recursion OS identifying differentiated, orally active small molecule leads against a high-interest immune cell target. These leads exhibit potential best-in-class properties, addressing significant liabilities seen in other candidates. As a result of this milestone, Recursion has received a $7 million milestone payment with the potential for over $300 million in additional milestone payments for this program. In total, the partnership has generated $130 million of cash inflows (including an upfront payment) to Recursion to date.

Recursion’s collaboration within Neuroscience and a GI Oncology Indication for Roche and Genentech continues to bring unbiased novel biological insights to potential programs. To date, the collaboration has built five phenomaps derived from a vast dataset of over one trillion iPSC-derived cells, one hundred billion GI Oncology relevant cells, alongside around 5,000 transcriptomes representing approximately 171 TB of data. Our approach continues to integrate high-throughput screens of genetics and small molecules with detailed cell measurements, informing our AI/ML models. Looking ahead, we are actively building additional maps and are focused on leveraging the Recursion OS and collaborating with Roche and Genentech to identify new novel programs that will fuel program advancement in both a GI Oncology indication and within Neuroscience.

Platform Updates
◦The platform is continuing to expand its ClinTech focus including high-quality, linked data assets, to industrialize clinical development, reduce costs, and accelerate the development of novel therapeutics. Updates include:
▪Leveraging Tempus data across Recursion’s oncology programs to expand therapeutic areas, which the Company believes will enrich patient population subgroups, and help increase likelihood of response for oncology clinical programs.
▪Signing an agreement with HealthVerity to integrate de-identified data for over 340 million covered lives within the US into Recursion OS, allowing for deeper insights into patient populations, enhanced trial design and feasibility assessments, as well as clinical operations workflows.
◦The collaboration with Enamine, leveraging Recursion’s massive data layer of predicted protein-small molecule interactions, resulted in the generation of enriched screening libraries to target 100 key and clinically relevant drug targets. The screening libraries are now available for purchase from Enamine.

Financial and Corporate Updates
"Recursion is stronger as a combined company, allowing us to not only deliver on operational goals more efficiently, but also be nimble in periods of uncertainty," stated Ben Taylor, CFO of

Recursion and President Recursion UK. "We have been able to significantly lower costs without cutting important platform capabilities by decreasing overall capacity. We maintain the ability to restore capacity in the future to respond to market needs. This aligns closely with our fundamental goal of using AI and automation to make drug discovery more flexible, efficient and effective."

First Quarter 2025 Financial Results

•Cash Position: Cash and cash equivalents and restricted cash were $509 million as of March 31, 2025 compared to $603 million as of December 31, 2024.
•Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $15 million for the first quarter of 2025, compared to $14 million for the first quarter of 2024 due to the timing of projects from the Company’s Sanofi, Roche and Merck KGaA, Darmstadt, Germany collaborations.
•Research and Development Expenses: Research and development expenses were $130 million for the first quarter of 2025, compared to $68 million for the first quarter of 2024. The increase was primarily driven by the Company’s agreement with Tempus as well as our business combination with Exscientia in November 2024. This includes $27 million in non-cash expenses for use of Tempus’ patient-centric multimodal oncology data for Recursion programs.
•General and Administrative Expenses: General and administrative expenses were $55 million for the first quarter of 2025, compared to $31 million for the first quarter of 2024. The increase compared to prior period was primarily due to the inclusion of G&A expenses from the business combination with Exscientia.
•Net Loss: Net loss was $202.5 million for the first quarter of 2025, compared to a net loss of $91.4 million for the first quarter of 2024.
•Net Cash: Net cash used in operating activities was $132 million for the first quarter of 2025, compared to net cash used in operating activities of $102 million for the first quarter of 2024. The difference was primarily driven by higher costs incurred for R&D and G&A due to the Company’s business combination with Exscientia, in addition to $16 million of one-time transaction related costs in the first quarter of 2025.

Integration update and guidance
•Operational teams have been functioning as consolidated groups since immediately after closing. Core integration plans are completed or on schedule across the company.
•Expected cash burn* excluding partnering or financing inflows for 2025 of equal to or less than $450 million, excluding the benefit of potential cash inflows from existing or new partnerships. In 2024, the combined cash burn excluding partnering or financing inflows was approximately $606 million, including $203 million of change in cash from Exscientia prior to the business combination and $403 million from Recursion, excluding $49 million of respective partnership inflows.
•1Q25 cash burn excluding partnering or financing inflows of approximately $118 million, excluding transaction related costs
•Projected cash runway into mid 2027 based on current business plan.
•Primary areas of combination synergies and operational savings beyond pipeline prioritization:
◦Duplicated corporate expenses
◦Reduction in capacity of drug discovery operations
◦Utilization of broader platform capabilities to reduce project costs
◦Increasing administrative efficiency
◦Rationalization of facilities and office locations
◦Greater purchasing power with vendors
◦Spin-out of Austrian operations

*Cash burn is a non-GAAP financial measure. See "Non-GAAP Financial Measures" below for additional information regarding cash burn and for a reconciliation of cash burn to net cash used in operating activities for historical periods, the most directly comparable GAAP financial measure. With respect to the expected cash burn for 2025, certain items that affect the calculation of the GAAP financial measure for net cash used by operating activities are not available on a forward-looking basis because such items cannot be reasonably calculated without unreasonable effort due to the unpredictability of the amounts and timing of events affecting the items we exclude from cash burn. Consequently, the Company is unable to provide a reconciliation of net cash used in operating activities to cash burn for the Company’s fiscal 2025 guidance.

Expanded Board:
•Namandjé Bumpus, Ph.D, and Elaine Sun have been appointed to Recursion’s Board of Directors, effective as of March 15th.
•Dr. Bumpus brings deep experience in scientific innovation and regulatory strategy, while Elaine Sun adds extensive leadership in life sciences finance and corporate strategy.

On May 5, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported financial results for the three months ended March 31, 2025 and provided an update on its corporate progress (Press release, BioNTech, MAY 5, 2025, View Source [SID1234652500]).

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"In the first quarter of 2025, we demonstrated continued execution against our strategic focus areas, highlighted by data updates for our PD-L1xVEGF-A bispecific antibody candidate BNT327 and the progress in clinical evaluation of our focus programs and combination treatment approaches," said Prof. Ugur Sahin, M.D., CEO and Co-Founder of BioNTech. "We will continue to focus on our key strategic programs as we remain steadfast in our vision to translate our science into survival for patients in need."

Financial Review for First Quarter 2025

in millions €, except per share data First Quarter 2025 First Quarter 2024
Revenues 182.8 187.6
Net loss (415.8) (315.1)
Basic and diluted loss per share (1.73) (1.31)
Revenues reported were €182.8 million for the three months ended March 31, 2025, compared to €187.6 million for the comparative prior year period. Revenues during the first quarter of 2025 were mainly driven by revenues derived from BioNTech’s COVID-19 vaccine collaboration.

Cost of sales were €83.8 million for the three months ended March 31, 2025, compared to €59.1 million for the comparative prior year period. The change was mainly due to a positive impact of an inventory revaluation in the first quarter of 2024.

Research and development ("R&D") expenses were €525.6 million for the three months ended March 31, 2025, compared to €507.5 million for the comparative prior year period. The increase was mainly driven by progressing late-stage clinical studies for candidates in BioNTech’s ADC and antibody portfolio.

Sales, general and administrative ("SG&A") expenses5, in total, amounted to €120.6 million for the three months ended March 31, 2025, compared to €132.6 million for the comparative prior year period. The decrease was primarily driven by a reduction in external services.

Income taxes were realized with an amount of €29.6 million in tax income for the three months ended March 31, 2025, compared to €16.7 million in realized tax income for the comparative prior year period.

Net loss was €415.8 million for the three months ended March 31, 2025, compared to a net loss of €315.1 million for the comparative prior year period.

Cash and cash equivalents plus security investments as of March 31, 2025, reached €15,854.4 million, comprising €10,184.9 million in cash and cash equivalents, €3,542.0 million in current security investments and €2,127.5 million in non-current security investments.

Basic and diluted loss per share was €1.73 for the three months ended March 31, 2025, compared to a basic and diluted loss per share of €1.31 for the comparative prior year period.

Shares outstanding as of March 31, 2025, were 240,392,622, excluding 8,159,578 shares held in treasury.

"Our revenues for the first quarter reflect the seasonal demand for COVID-19 vaccines and are in line with our expectations," said Jens Holstein, CFO of BioNTech. "BioNTech’s robust financial position empowers us to pursue our strategic goal of evolving into a leading biotech company with multiple oncology products by 2030."

2025 Financial Year Guidance Confirmed6

Total revenues for the 2025 financial year €1,700 million – €2,200 million
BioNTech expects its revenues for the full 2025 financial year to be in the range of €1,700 – €2,200 million and revenue phasing similar to 2024, primarily concentrated in the last three to four months, driving the full year revenue figure. The revenue guidance assumes: relatively stable vaccination rates, pricing levels and market share compared to 2024; estimated inventory write-downs and other charges by BioNTech’s collaboration partner Pfizer that negatively influence BioNTech’s revenues; anticipated revenues from a pandemic preparedness contract with the German government; and anticipated revenues from the BioNTech Group service businesses. Potential changes to the law or governmental policy, including tariffs and public health policy, and evolving public sentiment worldwide, could further negatively impact our anticipated revenues and expenses.

Planned 2025 Financial Year Expenses and Capex

R&D expenses €2,600 million – €2,800 million
SG&A expenses €650 million – €750 million
Capital expenditures for operating activities €250 million – €350 million
BioNTech expects to continuously focus investments on R&D and scaling the business for late-stage development and commercial readiness in oncology, while remaining cost-disciplined. Strategic capital allocation will continue to be a key driver of the Company’s trajectory. As part of BioNTech’s strategy, the Company may continue to evaluate appropriate corporate development opportunities with the aim of driving sustainable long-term growth and create future value.
The full interim unaudited condensed consolidated financial statements can be found in BioNTech’s Report on Form 6-K for the period ended March 31, 2025, filed today with the United States Securities and Exchange Commission ("SEC") and available at www.sec.gov.

Endnotes
1 All abbreviations are compiled in an abbreviation directory at the end of this press release.
2 All numbers in this press release have been rounded.
3 Calculated applying the average foreign exchange rate for the three months ended March 31, 2025, as published by the German Central Bank (Deutsche Bundesbank).
4 A settlement payment of $400 million related to a contractual dispute with the University of Pennsylvania is expected to be reflected in the Company’s second quarter 2025 financial results. In connection with this and another settlement with the National Institutes of Health ("NIH"), BioNTech expects to be reimbursed approximately $535 million by its collaboration partner during 2025 and 2026. Reimbursement payments have begun with the first payment received by BioNTech in the first quarter of 2025.
5 Sales, general and administrative expenses ("SG&A") include sales and marketing expenses as well as general and administrative expenses.
6 Financial guidance excludes external risks that are not yet known and/or quantifiable, including, but not limited to the effects of ongoing and/or future legal disputes and related activities, certain potential one-time effects and charges related to portfolio prioritization. It includes effects identified from licensing arrangements, collaborations or potential M&A transactions to the extent disclosed and may be subject to update. The Company does not expect to report a positive net income figure for the 2025 financial year.

Operational Review for the First Quarter 2025, Key Post Period-End Events and 2025 Outlook

Selected Oncology Pipeline Updates

Next-Generation Immunomodulators

BNT327, formerly also known as PM8002, is a bispecific antibody candidate combining PD-L1 checkpoint inhibition with VEGF-A neutralization.

In March 2025, at the European Lung Cancer Congress ("ELCC"), preliminary data from two Phase 2 clinical trials conducted in China in first-line extensive-stage small cell lung cancer ("ES-SCLC") and second-line small cell lung cancer ("SCLC") were presented.
Preliminary data from the ongoing single-arm, open-label Phase 2 clinical trial (NCT05844150) evaluating BNT327 in combination with chemotherapy as a first-line treatment in patients with ES-SCLC showed anti-tumor activity and an acceptable safety profile with no new safety signals beyond those typically described for chemotherapy agents and anti-PD-(L)1 and anti-VEGF monotherapies. These data were the first presented for BNT327 as a potential first-line treatment in ES-SCLC supporting the ongoing global randomized Phase 3 clinical trial ROSETTA Lung-01 (NCT06712355).
Preliminary data from the ongoing Phase 2 clinical trial (NCT05879068) evaluating BNT327 in combination with chemotherapy as a second-line treatment in patients with SCLC showed anti-tumor activity, which was observed regardless of prior immuno-oncology treatment, and an acceptable safety profile.
In April 2025, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2025, first data were presented for the novel combination of the PD-L1xVEGF-A bispecific antibody candidate BNT327 with various ADC candidates.
Interim data from the ongoing Phase 1/2 clinical trial (NCT05438329) evaluating BNT327 with BNT325/DB-1305, a TROP2-targeting ADC candidate being developed in collaboration with Duality Biologics (Suzhou) Co. Ltd. ("DualityBio"), in patients with advanced/metastatic solid tumors showed a manageable safety profile and early signs of anti-tumor activity in a cohort with patients with platinum-resistant ovarian cancer ("PROC"). Across the 13 efficacy evaluable patients with PROC, seven patients achieved partial response and three stable disease. Responses were also observed in patients with non-small cell lung cancer ("NSCLC") or triple-negative breast cancer ("TNBC").
First data from an ongoing Phase 2 clinical trial (NCT05918107) in first-line mesothelioma and two trial-in-progress posters for the ongoing global Phase 3 and Phase 2/3 clinical trials, ROSETTA Lung-01 (NCT06712355) and ROSETTA Lung-02 (NCT06712316) respectively, will be presented at the American Society for Clinical Oncology ("ASCO") Annual Meeting taking place from May 30 to June 3, 2025 in Chicago, Illinois.
Abstract title: First report of efficacy and safety results from a Phase 2 trial evaluating BNT327/PM8002 plus chemotherapy (chemo) as first-line treatment (1L) in unresectable malignant mesothelioma
Abstract title: A global Phase III, double-blind, randomized trial of BNT327/PM8002 plus chemotherapy (chemo) compared to atezolizumab plus chemo in patients (pts) with first-line (1L) extensive-stage small cell lung cancer (ES-SCLC)
Abstract title: A global Phase 2/3, randomized, open-label trial of BNT327/PM8002 in combination with chemotherapy (chemo) in first-line (1L) non-small cell lung cancer (NSCLC)
mRNA Cancer Immunotherapies

BNT116 is based on BioNTech’s fully owned, off-the-shelf FixVac platform, and is designed to elicit an immune response to six tumor-associated antigens that were identified to be frequently expressed in NSCLC.

In April 2025, at the AACR (Free AACR Whitepaper) Annual Meeting 2025, preliminary data from a cohort with frail patients were presented from the ongoing Phase 1 clinical trial LuCa-MERIT-1 (NCT05142189) evaluating BNT116 in combination with cemiplimab in patients with PD-L1 positive (TPS≥1%) unresectable Stage III or metastatic Stage IV NSCLC who are not eligible for chemotherapy as first-line treatment. The preliminary data showed anti-tumor activity, consistent immune response induction and a manageable safety profile.
Antibody-Drug Conjugates

BNT324/DB-1311 is an B7H3-targeted ADC candidate that is being developed in collaboration with DualityBio. The program has received Fast Track designation from the U.S. Food and Drug Administration ("FDA") for the treatment of patients with advanced castration-resistant prostate cancer ("CRPC") whose disease has progressed while undergoing or after undergoing standard systemic regimens. In addition, the program has received Orphan Drug designation from the FDA for the treatment of patients with advanced esophageal squamous cell carcinoma.

Preliminary data from the ongoing Phase 1/2 clinical trial (NCT05914116) evaluating BNT324/DB-1311 in patients with advanced solid tumors, including patients with previously treated castration-resistant prostate cancer ("CRPC"), will be presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.
Abstract title: DB‑1311/BNT324 (a novel B7H3 ADC) in patients with heavily pretreated castrate-resistant prostate cancer (CRPC)
BNT325/DB-1305 is a TROP2-targeted ADC candidate that is being developed in collaboration with DualityBio. BNT325/DB-1305 received Fast Track designation from the FDA for the treatment of patients with platinum-resistant ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.

In March 2025, at the Society of Gynecologic Oncology ("SGO") Annual Meeting, preliminary data were presented from an ongoing Phase 1/2 clinical trial (NCT05438329) evaluating BNT325/DB-1305 in signal-seeking cohorts across various cancer indications, including PROC. The data from a cohort of patients with PROC showed a manageable safety profile and early signs of anti-tumor activity.
Corporate and Commercial Update for the First Quarter 2025

Earlier today, BioNTech announced that the Supervisory Board has appointed Ramón Zapata-Gomez to the Management Board as Chief Financial Officer ("CFO") effective July 1, 2025. He will join BioNTech from Novartis AG’s global biomedical research organization where he has been serving as CFO since 2022. Ramón Zapata will succeed Jens Holstein, who, as previously planned and announced, will retire at the end of his term on June 30, 2025.
In February 2025, BioNTech completed the acquisition of Biotheus, obtaining full global rights to BNT327 and to all other candidates from Biotheus’ pipeline, as well as to its in-house antibody generation platform and bispecific ADC capability. The transaction amounted to an upfront consideration of $800 million, plus additional performance-based payments of up to $150 million.
BioNTech and Pfizer developed, manufactured and delivered their JN.1 and KP.2-adapted COVID-19 vaccines, which have received multiple regulatory approvals, including full approvals, authorizations for emergency or temporary use, or marketing authorizations, in more than 40 countries and regions. BioNTech is now focused on preparing for variant strain vaccine adaptation to be ready for commercial launch ahead of the upcoming 2025/2026 vaccination season, pending approvals.
Upcoming Investor and Analyst Events

Annual General Meeting: May 16, 2025
Innovation Series R&D Day: November 11, 2025
Conference Call and Webcast Information

BioNTech invites investors and the general public to join a conference call and webcast with investment analysts today, May 5, 2025, at 8:00 a.m. EDT (2:00 p.m. CEST) to report its financial results and provide a corporate update for the first quarter of 2025.

To access the live conference call via telephone, please register via this link. Once registered, dial-in numbers and a PIN number will be provided.

The slide presentation and audio of the webcast will be available via this link.

Participants may also access the slides and the webcast of the conference call via the "Events & Presentations" page of the Investor section of the Company’s website at www.BioNTech.com. A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

On May 5, 2025 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported first quarter 2025 financial results and corporate updates (Press release, Relay Therapeutics, MAY 5, 2025, View Source [SID1234652518]).

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"2025 is a year of execution across a range of high value clinical programs," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "The ongoing changes to our cost base are designed to enable a full funding of key initiatives including generating topline data from the ReDiscover-2 trial and clinical proof-of-concept data in vascular malformations."

Strategic Cost Reductions Implemented to Extend Runway into 2029 and to Fully Fund Key Value Drivers

•
These reductions help extend operating runway into 2029 and are aimed at funding key objectives, including:
o
Completion of ReDiscover-2 Phase 3 trial of RLY-2608 + fulvestrant in metastatic breast cancer well past topline data readout and additional breast cancer clinical trials
o
Execution of RLY-2608 Phase 1 vascular malformations trial through clinical proof-of-concept data
o
Advance Fabry and NRAS program to Investigational New Drug application (IND) readiness
o
Advance one research-stage program

•
Specific cost reductions include:
o
Over the past year, focused the research portfolio and platform on the highest value areas resulting in a reduction in the research run rate spend by approximately 80%
▪
Reduced research-stage programs from four to one
▪
Recent reduction in force by approximately 70 people
o
Executed a global out-license of RLY-4008 with Elevar Therapeutics, Inc. (Elevar) with potential for downstream economics
o
Phased the timing of entry into the clinic for Fabry and NRAS targeted programs

RLY-2608 Highlights

•
Breast Cancer
o
Initiation of Phase 3 ReDiscover-2 trial of RLY-2608 + fulvestrant in PI3Kα-mutated, CDK4/6 pre-treated, HR+/HER2- advanced breast cancer remains on track for mid-2025
o
Abstract accepted to ASCO (Free ASCO Whitepaper) for update of Phase 1b ReDiscover trial of RLY-2608 + fulvestrant
▪
Focus of the abstract is updated 600mg BID (fasted) doublet data with median follow-up now greater than 12 months
▪
Poster Title: Updated efficacy of mutant-selective PI3Kα inhibitor RLY-2608 in combination with fulvestrant in patients with PIK3CA-mutant HR+HER2- advanced breast cancer: ReDiscover trial
▪
Date/Time: Monday, June 2, 10:00-1:00 p.m. ET (9:00-12:00 p.m. CT)
o
Continued advancement of the ongoing triplet cohorts with RLY-2608 + fulvestrant + atirmociclib or ribociclib
o
Planning continues for development of next-generation endocrine therapy combinations with RLY-2608
•
Vascular Malformations
o
Initiation of Phase 1 vascular malformations clinical trial in the first quarter of 2025

First Quarter 2025 Financial Results

Cash, Cash Equivalents and Investments: As of March 31, 2025, cash, cash equivalents and investments totaled $710.3 million, as compared to $781.3 million as of December 31, 2024. The company expects its current cash, cash equivalents, and investments will be sufficient to fund its operating expenses and capital expenditure requirements into 2029.

Revenue: Revenue was $7.7 million for the first quarter of 2025, as compared to $10.0 million for the first quarter of 2024. The revenue recognized in the first quarter of 2025 was due to completion of all performance obligations under the company’s Exclusive License Agreement with Elevar. The revenue recognized in the first quarter of 2024 was due to a milestone achieved under the Collaboration and License Agreement with Genentech, Inc.

R&D Expenses: Research and development expenses were $73.8 million for the first quarter of 2025, as compared to $82.4 million for the first quarter of 2024. The decrease was primarily due to the series of strategic choices made to streamline the research organization throughout 2024.

G&A Expenses: General and administrative expenses were $18.7 million for the first quarter of 2025, as compared to $19.8 million for the first quarter of 2024. The decrease was primarily due to a decrease in stock compensation expense, partially offset by costs to obtain the agreement with Elevar, which were expensed commensurate with the timing of revenue recognized.

Net Loss: Net loss was $77.1 million for the first quarter of 2025, or a net loss per share of $0.46, as compared to a net loss of $81.4 million for the first quarter of 2024, or a net loss per share of $0.62.

On May 5, 2025 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird" or "the Company"), Carlyle (NASDAQ: CG) ("Carlyle"), SK Capital Partners, LP ("SK Capital") and Beacon Parent Holdings, L.P. ("Parent") reported that all required regulatory approvals to complete the previously announced acquisition of the Company by Carlyle and SK Capital have been received (Press release, bluebird bio, MAY 5, 2025, View Source [SID1234652501]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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No further regulatory approvals are required to complete the transaction. The parties expect to complete the merger promptly following the successful completion of the ongoing tender offer, which is scheduled to expire one minute after 11:59 p.m. New York City time on May 12, 2025, unless the tender offer is further extended or earlier terminated.

Under the terms of the merger agreement, stockholders will receive an upfront payment of $3.00 per share in cash and a contingent value right (CVR) of $6.84 per share in cash payable upon achievement of a net sales milestone, for a total potential value of $9.84 per share. The bluebird board of directors (the "Board") unanimously recommends that stockholders tender into the offer.

"The bluebird Board unanimously recommends that stockholders tender into the offer, which expires May 12, 2025. Absent a majority of stockholders tendering, bluebird is at significant risk of defaulting on its loan agreements with Hercules Capital and it is extremely unlikely that stockholders would receive any consideration for their shares in a bankruptcy or liquidation," said Mark Vachon, chairman of the bluebird bio Board of Directors. "The bluebird Board considered all reasonable alternatives during its review of strategic alternatives and concluded that the proposed transaction with Carlyle and SK Capital is the only viable solution to generate value for bluebird stockholders. We strongly urge stockholders to tender their shares before the expiration date to ensure the best possible outcome for all bluebird stakeholders and the patients that depend on its treatments."

"The receipt of all required regulatory approvals is excellent news for stockholders as well as for patients and families seeking to be treated with bluebird gene therapies," said Andrew Obenshain, chief executive officer, bluebird bio. "With this update, we have a clear path forward to close the transaction and officially begin the next chapter of bluebird’s journey to deliver potentially curative gene therapies in the commercial setting."

Stockholders who need assistance with tendering their shares of common stock of bluebird may contact the Information Agent, Innisfree M&A Incorporated, by calling toll-free at (877) 825-8793.

On May 5, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported its financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Syndax, MAY 5, 2025, View Source [SID1234652519]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"I’m very pleased to report an outstanding quarter in which Revuforj and Niktimvo generated a combined $34 million in net sales. We believe this success is a reflection of excellent commercial execution and the clinical profile of these first- and best-in-class medicines," said Michael A. Metzger, Chief Executive Officer. "We’ve also made excellent progress advancing our pipeline, including most notably the recent submission of our sNDA for R/R mNPM1 AML and the initiation of the first pivotal frontline trial of a menin inhibitor in combination with venetoclax and azacitidine for mNPM1 and KMT2Ar AML. With a solid financial position and highly skilled team, we are poised to deliver two successful product launches while aggressively advancing our development strategy designed to unlock the multi-billion-dollar opportunities for both drugs."

Recent Business Highlights and Anticipated Milestones

Revuforj (revumenib)

Achieved $20.0 million in Revuforj net revenue in the first quarter of 2025, the first full quarter of the U.S. launch. Revuforj was launched in the U.S. in late November 2024 for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older.
Completed the submission of a supplemental New Drug Application (sNDA) to the U.S. FDA in April 2025, seeking Priority Review for the approval of Revuforj for the treatment of R/R mutant NPM1 (mNPM1) AML. The sNDA was submitted under the FDA’s Real-Time Oncology Review (RTOR) program, which allows for a more efficient review and close engagement between the sponsor and FDA throughout the submission process. The sNDA is supported by the previously reported positive pivotal data from the AUGMENT-101 trial.
Submitted the pivotal R/R mNPM1 AML data from the AUGMENT-101 trial for publication. The manuscript has been accepted and the publication is expected imminently.
Opened enrollment in the EVOLVE-2 trial, a pivotal, randomized, double-blind, placebo-controlled trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2A-rearranged (KMT2Ar) AML patients who are unfit for intensive chemotherapy in the first quarter of 2025. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.
Multiple trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape are ongoing. These trials include:
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. Updated data that showed an overall response rate (ORR)1 of 100% (37/37) and a composite complete remission (CRc) rate of 95% (35/37) were reported at the Company’s investor event at the 66th ASH (Free ASH Whitepaper) Annual Meeting. The Company anticipates that an update on the trial will be available at a medical meeting in the second quarter of 2025.
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Updated data that showed an ORR of 82% (27/33) and a CR/CRh rate of 48% (16/33) were presented at the 66th ASH (Free ASH Whitepaper) Annual Meeting. The trial is now enrolling a cohort of newly diagnosed patients.
Intensive chemotherapy: A Phase 1 trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients. The Company expects to report data in the fourth quarter of 2025.
Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers.
INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial. Data that showed 54% (6/11) of patients had MRD reduction at any time, including 36% (4/11) who achieved MRD negativity, were presented at the 66th ASH (Free ASH Whitepaper) Annual Meeting.
The Company plans to initiate multiple trials of revumenib in combination with standard of care regimens in newly diagnosed acute leukemia patients who are fit to receive intensive chemotherapy, starting in the second half of 2025.
The Company is evaluating revumenib in patients with R/R metastatic microsatellite stable (MSS) colorectal cancer (CRC). The Phase 1b portion of this proof-of-concept trial is ongoing.
Niktimvo (axatilimab-csfr)

Niktimvo achieved $13.6 million in net revenue in the first quarter of 2025, the first partial quarter of the U.S. launch. Niktimvo was launched in the U.S. in late January for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Syndax and Incyte co-commercialize Niktimvo, and Syndax records 50% of the Niktimvo net profit/loss, defined as net product revenue minus the cost of sales and commercial expenses.
Presented a post-hoc analysis evaluating the effects of prior lines of therapy on clinical outcomes for patients with chronic GVHD in the AGAVE-201 trial of axatilimab. The data show that overall response rates were consistent with axatilimab regardless of the number of prior lines of therapy and that organ-specific responses were noted regardless of the last prior therapy. The data were presented at the 2025 Tandem Meetings of the American Society for Transplantation and Cellular Therapy and the Center for International Blood and Marrow Transplantation Research.
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:
A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multi-center trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD.
Enrollment is ongoing in the MAXPIRe trial, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF). The Company expects to complete enrollment in the trial in 2025 with topline data anticipated in the second half of 2026.
First Quarter 2025 Financial Results

As of March 31, 2025, Syndax had cash, cash equivalents, and short and long-term investments of $602.1 million and 86.3 million common shares and prefunded warrants outstanding.

In the first quarter of 2025, the first full quarter of the U.S. launch, Revuforj net revenue was $20.0 million. Cost of sales for the first quarter of 2025 was $0.9 million.

In the first quarter of 2025, the first partial quarter of the U.S. launch, the Company’s partner, Incyte, reported $13.6 million in Niktimvo net revenue. Syndax records 50% of the Niktimvo net commercial profit/loss, defined as net product revenue (recorded by Incyte) minus the cost of sales and commercial expenses. For the first quarter of 2025, Niktimvo posted a net commercial loss and Syndax’s share of the collaboration loss amounted to $0.2 million.

First quarter 2025 research and development expenses increased to $61.6 million from $56.5 million for the comparable prior year period. The year-over-year increase was due to an increase in axatilimab-related costs primarily driven by the IPF trial, the frontline chronic GVHD trial with ruxolitinib being conducted in partnership with Incyte, and a $10.0 million milestone payment as a result of the first patient dosed in the Phase 3 trial of axatilimab in combination with corticosteroids. The higher expenses were also driven by an increase in personnel costs and other expenses related to increased R&D support for ongoing clinical trials, sNDA activities, and medical affairs in support of commercialization. These activities were partially offset by a decrease in revumenib-related costs, primarily driven by an $8.0 million milestone expense in the 2024 period and a reduction in CMC expense due to the capitalization of inventory for commercial use.

First quarter 2025 selling, general and administrative expenses increased to $41.0 million from $23.0 million for the comparable prior year period. The year-over-year increase was primarily due to increased employee-related expenses and professional fees to support increased sales and marketing-related expenses related to the U.S. commercial launch of Revuforj.

For the three months ended March 31, 2025, Syndax reported a net loss attributable to common stockholders of $84.8 million, or $0.98 per share, compared to a net loss attributable to common stockholders of $72.4 million, or $0.85 per share, for the comparable prior year period.

Financial Guidance

For the second quarter of 2025, the Company expects research and development expenses to be $70 to $75 million and total research and development plus selling, general and administrative expenses to be $110 to $115 million. For the full year of 2025, the Company continues to expect research and development expenses to be $260 to $280 million and total research and development plus selling, general and administrative expenses to be $415 to $435 million, which includes an estimated $45 million in non-cash stock compensation expense. The Company is not providing revenue guidance at this time.

Syndax expects that its cash, cash equivalents and short- and long-term investments, combined with its anticipated product revenue and interest income, will enable the company to reach profitability.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, May 5, 2025.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: Syndax1Q25
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

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 in adult and pediatric patients one year and older.

Revumenib is in development for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial in this population with revumenib as a monotherapy were recently reported and the Company submitted a supplemental NDA for revumenib in R/R mNPM1 AML in April 2025. Additionally, multiple trials of revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia are ongoing across the treatment landscape, including in newly diagnosed patients.

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.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774). Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

About the Real-Time Oncology Review Program (RTOR)

RTOR provides a more efficient review process for oncology drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. Specifically, it allows for close engagement between the sponsor and the FDA throughout the submission process and it enables the FDA to review individual sections of modules of a drug application rather than requiring the submission of complete modules or a complete application prior to initiating review. Additional information about RTOR can be found at: View Source