On May 5, 2025 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small-molecule drugs for oncological and inflammatory diseases, reported that, it has raised $175 million in funding to date (Press release, Can-Fite BioPharma, MAY 5, 2025, View Source [SID1234652502]). These funds have enabled the advancement of its lead drug candidates, Namodenoson and Piclidenoson, into pivotal Phase III studies for liver cancer and psoriasis, respectively.

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Can-Fite is a recognized leader in the development of small-molecule therapeutics targeting the A3 adenosine receptor (A3AR), which is highly expressed in both inflammatory and cancer cells. Namodenoson, an orally bioavailable A3AR agonist, is currently enrolling patients in a pivotal Phase III study for advanced liver cancer. It has demonstrated selective targeting of liver and pancreatic tumor cells while sparing healthy tissue. Piclidenoson, also an orally administered A3AR agonist, is in a pivotal Phase III trial for patients with moderate-to-severe psoriasis.

Both candidates have shown favorable safety profiles, anti-cancer and anti-inflammatory properties, and promising efficacy in previous Phase II trials. Cumulative funding has also supported drug manufacturing, regulatory activities with the FDA and EMA, and the development of a broad patent portfolio providing extensive intellectual property protection.

In addition to its lead programs, Can-Fite is conducting a Phase IIa study of Namodenoson in pancreatic cancer, following successful preclinical studies demonstrating its ability to inhibit tumor growth by modulating the Wnt/β-catenin, NF-κB, and RAS signaling pathways. A Phase IIb trial in metabolic dysfunction-associated steatohepatitis (MASH) is also underway under an open IND with the FDA, leveraging Namodenoson’s hepatoprotective effects.

To date, Can-Fite has signed seven commercialization agreements with strategic partners for the future marketing of its drug candidates upon regulatory approval. Can-Fite continues to engage with potential partners and seeks to sign additional commercialization agreements in the future.

Motti Farbstein, CEO of Can-Fite, commented: "This funding milestone reflects the strong confidence our investors and partners have in Can-Fite’s scientific platform and clinical strategy. Progressing into pivotal Phase III trials marks a significant step toward bringing innovative, oral therapies to patients with serious unmet needs in liver cancer and psoriasis."

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]).

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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 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 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported that the Supervisory Board has appointed Ramón Zapata-Gomez to the Management Board as Chief Financial Officer ("CFO") effective July 1, 2025 (Press release, BioNTech, MAY 5, 2025, View Source [SID1234652499]). 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.

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In his new role as CFO at BioNTech, Ramón Zapata will ensure the Company’s financial direction continues to align with BioNTech’s strategy to become a multi-product company in the oncology field. In preparation for BioNTech’s oncology product launches, he will be responsible for fostering and further optimizing a strong financial infrastructure and performance in key markets. He will continue to drive sustainable organizational excellence and global execution in financial reporting, accounting, tax and treasury, and purchasing with the aim of furthering cost-effective value generation.

"Ramón Zapata is an accomplished leader with deep finance expertise who will be taking over from Jens Holstein at an exciting phase. He has gained extensive experience within the international pharmaceutical industry with a focus on North America, Europe, and Latin America, and has a deep understanding of market and business dynamics, resource optimization, and high-performing teams," said Helmut Jeggle, Chairman of the Supervisory Board. "We, the Supervisory and the Management Board, thank Jens Holstein for his financial leadership and contributions to BioNTech’s successful trajectory. He strengthened BioNTech’s global finance organization and processes and contributed to the Company’s financial stability while increasing operational efficiency. Today, the Company is uniquely positioned to continue its success story and transformation into a multi-product company."

"It has been a privilege to be part of the development of BioNTech into one of the largest global biotechnology companies," said Jens Holstein, CFO at BioNTech. "I would like to thank my colleagues on the Boards as well as my teams for their dedication and collaboration during this remarkable growth journey. For me personally, it is the right time to retire as CFO and focus on non-executive Board roles in the future."

"I am looking forward to joining BioNTech and am eager to contribute to its ambitious vision of improving patients’ lives," said Ramón Zapata, designated CFO at BioNTech. "Throughout my career, I have been driven by the goal of developing and providing access to innovative medicines for patients. As BioNTech focuses on its first oncology launches, while advancing its unique clinical pipeline with disruptive and synergistic potential, I look forward to working with the talented team at BioNTech and accelerating financial strategies that support the Company’s vision."

Ramón Zapata is a seasoned global finance executive with more than 25 years of experience in the pharmaceutical and consumer goods industries. He has held leadership roles at leading global companies including Novartis AG, Sandoz AG, and Mondelēz International. Throughout his career, Ramón Zapata has led finance functions enabling seamless execution from drug discovery through commercialization, including overseeing M&A transactions and successful integrations as well as driving digital finance transformations. Prior to joining BioNTech, Ramón Zapata served as CFO of BioMedical Research at Novartis, where he was responsible for the overall leadership of the division’s finance strategy and operations.

Ramón Zapata holds dual citizenship of the United States and Mexico. He is a Certified Public Accountant (CPA) from Universidad Panamericana in Mexico City and holds an MBA degree from IPADE Business School in Mexico City and from IESE Business School in Barcelona, Spain.

On May 5, 2025 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 5, 2025, View Source [SID1234652498]).

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"We started 2025 with another quarter of outstanding performance. ORLADEYO revenue growth was driven by moving ORLADEYO patients from free drug to paid at a much faster rate than we expected, resulting in a substantial increase to our annual guidance as we also move closer to peak sales of $1 billion. This increased financial strength accelerates our path to profitability and enables us to start paying down our debt, while continuing to invest in and advance our pipeline," said Jon Stonehouse, president and chief executive officer of BioCryst.

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the first quarter of 2025 was $134.2 million (+51 percent year-over-year (y-o-y)).

The total percentage of all ORLADEYO patients on paid drug has increased to approximately 84 percent (compared to 73.5 percent at end of 2024), generating higher than expected ORLADEYO revenue that the company expects will continue through the full year.

First quarter prescriptions were strong, above the quarterly average in 2024, and the percentage of U.S. HAE patients who describe a strong preference for an oral prophylaxis therapy increased to 70 percent, up from 50 percent in 2023, in the company’s latest market survey of HAE patients.

Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the first quarter. The number of patients treated with ORLADEYO outside the U.S. continued to grow in new and existing markets.
"By driving a dramatic increase in the rate of paid ORLADEYO patients through the prescription reauthorization period, on top of continued very strong new patient demand, our team has achieved in four months what we had expected would take three years, getting the rate of paid patients nearly to our long-term goal of 85 percent. As a result, we will capture significantly more revenue opportunity in 2025 and subsequent years and our path to peak sales of $1 billion is even more profitable than we had projected," said Charlie Gayer, chief commercial officer of BioCryst.

Rare Disease Pipeline

The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients.

The company has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) to expand the ORLADEYO label to children with HAE aged 2 to 11 using an oral granule formulation. The company also expects to submit regulatory filings in 2025 in global territories, including Europe, Japan and Canada. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.

The FDA has cleared the company’s investigational new drug application (IND) which will enable its clinical trial of BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, to enroll patients in the United States. This phase 1 trial is also open in Australia. The company expects initial data from the program in 2025.

Netherton syndrome is a serious, rare, lifelong genetic disorder causing disruption of the skin barrier with premature separation of the skin layers, chronic inflammation and vulnerability to serious infections, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have itchy, red, scaly, inflamed skin, fragile hair, and are more likely to develop severe food allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments that target the underlying cause of Netherton syndrome. BCX17725 is designed to replace missing functions of the natural KLK5 inhibitor, which could restore the normal skin barrier and result in improved skin function, including protection from severe inflammatory and infectious complications of the disease.

The first clinical trial with suprachoroidal delivery of avoralstat, the company’s investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), has been granted authorization to proceed in Australia. The company expects initial data from DME patients in 2025.

DME is an important cause of vision loss in diabetes and is due to leakage of fluid from the blood vessels in the retina. While current treatments focus on vascular endothelial growth factor (VEGF) inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space, is designed to provide long-lasting exposure to the retinal vessels, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
"Today we are reporting significant milestones for each of the next three programs in our pipeline, demonstrating our focus on advancing medicines with the potential to provide differentiated outcomes for patients of all ages. As we move closer to delivering an urgently needed oral prophylactic therapy to children with HAE, we are simultaneously progressing two clinical-stage programs in Netherton syndrome and DME towards the first patient data later this year," said Dr. Helen Thackray, chief research and development officer of BioCryst.

First Quarter 2025 Financial Results

For the three months ended March 31, 2025, total revenues were $145.5 million (+$52.7 million y-o-y), compared to $92.8 million in the first quarter of 2024 (+56.8 percent y-o-y). The increase was primarily due to $134.2 million (+$45.3 million y-o-y) in ORLADEYO net revenue in the first quarter of 2025, compared to $88.9 million in ORLADEYO net revenue in the first quarter of 2024 (+51.0 percent y-o-y).

Research and development expenses for the first quarter of 2025 decreased to $37.3 million from $46.5 million in the first quarter of 2024 (-19.8 percent y-o-y), primarily due to decreased expenses driven by the discontinuation and close-out of the Factor D programs, BCX10013 and BCX9930. These reductions were partially offset by an increase in other research, preclinical and development costs, comprised of avoralstat and other early-phase pipeline programs, and a change in general and administrative expense allocations.

Selling, general and administrative expenses for the first quarter of 2025 increased to $82.5 million, compared to $59.5 million in the first quarter of 2024 (+38.7 percent y-o-y). The increase was primarily due to increased commercial investment to support our growing ORLADEYO revenue, our newly launched regions, expanded international operations and global commercial support activities. Additionally, there was an increase to general and administrative expenses, and an offsetting reduction to research and development expenses, due to a change in the general and administrative expense allocations in 2025.

Operating income for the first quarter of 2025 was $21.2 million, compared to an operating loss of $14.5 million for the first quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense, was $42.6 million for the first quarter of 2025, compared to a non-GAAP operating loss of $0.8 million for the first quarter of 2024.

Interest expense was $23.5 million in the first quarter of 2025, compared to $24.5 million in the first quarter of 2024 (-4.1 percent y-o-y). The decrease was primarily due to a decrease in interest expense associated with the interest accrued under the Pharmakon Loan Agreement.

Net income for the first quarter of 2025 was $32 thousand, or $0.00 per share, compared to a net loss of $35.4 million, or $0.17 per share, for the first quarter of 2024.

Cash, cash equivalents, restricted cash and investments totaled $317.3 million at March 31, 2025, compared to $338.4 million at March 31, 2024. Net cash utilization for the first quarter of 2025 was $25.5 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense.

Early in the second quarter, the company paid down $75 million of the outstanding Pharmakon debt, which will result in approximately $23.5 million in interest savings over the life of the loan.

Financial Outlook for 2025
The company is increasing its outlook for full year 2025 global net ORLADEYO revenue to be between $580 million and $600 million (previously $535 million to $550 million).

The company now expects full year 2025 operating expenses will be $440 million to $450 million (previously $425 million to $435 million). This operating expense outlook does not reflect non-cash stock compensation expense.

The company is accelerating its expectation for sustainable profitability and positive cash flows by a year. The company now expects to deliver net income and positive cash flows for full year 2025 (previously expected both for full year 2026). Positive cash flow refers to the improvement in cash, cash equivalents, restricted cash and investments from year end 2024 to year end 2025, not including the impact of the $75 million Pharmakon prepayment made in April 2025.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.