On May 1, 2025 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the first quarter ended March 31, 2025 (Press release, BioMarin, MAY 1, 2025, View Source [SID1234652427]).

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"During the first quarter, we saw continued high demand for our innovative medicines resulting in strong revenue growth and profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Products in our pipeline also advanced according to plan. In April, we shared positive top-line results from the Phase 3 PALYNZIQ study for the treatment of adolescents with phenylketonuria between the ages of 12 and 17. Also in April, we were pleased to conclude enrollment in the pivotal study in hypochondroplasia with VOXZOGO, keeping us on track to launch in 2027, should data be supportive."

Mr. Hardy continued, "For the remainder of 2025, we look forward to continued momentum in our global expansion of VOXZOGO for achondroplasia. Across our Enzyme Therapies, we plan to build upon strong PALYNZIQ performance in the quarter, as well as initiatives to drive uptake of our other therapies to reach an even greater number of patients around the world. In addition to our strong financial outlook, we expect to advance multiple new indications with VOXZOGO in our CANOPY clinical program, share early clinical results from both BMN 351 for Duchenne Muscular dystrophy and BMN 333, our long-acting C-type natriuretic peptide, as well as execute on our business development strategy. We are delivering strong growth and profitability while we continue to implement BioMarin’s new strategy and operating model. We look forward to seeing the benefits of this transformation flow through our results in the coming quarters and beyond."

First Quarter 2025 Financial Highlights
•Total Revenues for the first quarter of 2025 were $745 million, an increase of 15% compared to the same period in 2024, driven by strong 40% year-over-year VOXZOGO revenue growth from new patients initiating therapy across all regions. In the quarter, revenues from BioMarin’s Enzyme Therapies (ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM) increased 8% compared to the first quarter of 2024, driven by a combination of increased patient demand and the timing of large government orders in all regions. The increase was partially offset by lower KUVAN product revenues attributed to continued generic competition as a result of the loss of market exclusivity.

•GAAP Net Income increased by $97 million to $186 million in the first quarter of 2025 compared to the same period in 2024, an increase of 109%, primarily attributed to higher gross profit driven by the factors noted above. The increase was also attributed to lower operating expenses following the termination of certain early stage development programs following the company’s 2024 strategic portfolio review and focused ROCTAVIAN strategy announced in the second half of 2024. These increases were partially offset by higher tax provision primarily due to increase in taxable income.

•Non-GAAP Income increased by $81 million to $221 million in the first quarter of 2025 compared to the same period in 2024, representing 58% growth. The increase in Non-GAAP Income was primarily due to the factors noted above.

First Quarter 2025 Business Highlights

Innovation
•Skeletal Conditions: In March 2025, BioMarin presented new data demonstrating favorable safety and strong adherence in real-world clinical practice with VOXZOGO in children with achondroplasia under the age of 3 years old at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Meeting. No treatment-related adverse events nor any dose interruptions were reported among 63 children followed for up to 23.7 months. These real-world findings further validate VOXZOGO’s established safety profile and reinforce the therapeutic benefit seen in clinical studies. The study’s safety results, including in infants as young as 1 month old, add to the growing body of evidence supporting early treatment initiation with VOXZOGO, consistent with new international treatment guidelines published in the journal Nature Reviews Endocrinology earlier this year.

•In April 2025, BioMarin completed enrollment in its pivotal Phase 3 study with VOXZOGO in hypochondroplasia and the company is on track to share topline data in 2026, with a potential launch in 2027. BioMarin plans to leverage its multiyear track record treating children with achondroplasia, a related condition, to raise awareness and treat children with hypochondroplasia across the globe. The CANOPY clinical program is continuing to advance VOXZOGO in additional new indications, including idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.

•With BMN 333, BioMarin’s long-acting C-type natriuretic peptide (CNP), the company enrolled multiple cohorts of healthy volunteers in its first-in-human study, with initial pharmacokinetic (PK) data expected by year-end. Detailed data from this study is expected to be presented at a scientific forum in the first half of 2026. Pre-clinical data with BMN 333 demonstrated sustained 100 pM concentrations for free CNP, representing an approximate 2-3 fold increase versus published data in an analogous pre-clinical model for other long-acting CNP analogs.

•Additionally, BioMarin recently met with FDA and reached agreement on an overall clinical development plan for BMN 333 in achondroplasia. Assuming the Phase 1 data are supportive, the company plans to initiate a registration-enabling study in 2026, supporting a previously disclosed target for 2030 approval. BioMarin plans to seek similar agreements with additional global regulators in the coming months.

•The company announced in April that its pivotal study with PALYNZIQ for the treatment of adolescents between the ages of 12 and 17 met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels. These data will support the planned submission of applications in the second half of 2025 to expand PALYNZIQ age eligibility in the United States and Europe.

•Other Clinical Pipeline Programs: BMN 351, BioMarin’s next generation oligonucleotide for Duchenne Muscular Dystrophy, and BMN 349, an oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, continue to advance. Initial data for BMN 351 is anticipated to be presented at a scientific congress in the second half of 2025 (including muscle dystrophin levels from the 6 mg/kg cohort after 25 weeks of dosing).

•During a recent strategic portfolio assessment of R&D programs, BioMarin determined that the evolving profile for BMN 370, a pre-clinical candidate for the treatment of von Willebrand disease, did not meet its threshold for further development and commercialization. The program has been discontinued and impacted employees have been redeployed within BioMarin.

Growth

•Total VOXZOGO revenue in the first quarter increased 40% compared to the same period in 2024, representing continued strong global demand since its commercial launch in 2021. As of the end of the quarter, children with achondroplasia in 49 countries around the world were being treated with VOXZOGO.

•In the U.S., BioMarin is investing in focused initiatives to drive continued expansion. These efforts include increasing field personnel to broaden the prescriber base and adding awareness platforms to drive adoption of VOXZOGO treatment. This is expected to begin increasing the rate of U.S. expansion in the second half of the year. Outside of the U.S. (OUS), from where the majority of VOXZOGO revenue is generated, uneven ordering patterns, consistent with BioMarin’s other brands, were observed. This OUS dynamic is expected to result in VOXZOGO full-year revenues being more weighted towards the second half of 2025.

•Enzyme Therapies revenues grew 8% in the first quarter Y/Y, driven by strong continued demand for PALYNZIQ. Strong PALYNZIQ performance as well as solid growth from BioMarin’s other enzyme treatments are expected to continue throughout 2025.

Value Commitment

•In the first quarter of 2025, BioMarin delivered strong results across the business. Total revenues for the first quarter grew 15% Y/Y. First quarter GAAP Operating Margin of 30.0% expanded 16.4 percentage points Y/Y while GAAP Diluted EPS of $0.95 increased 107% Y/Y. First quarter Non-GAAP Operating Margin of 35.7% expanded 11.9 percentage points Y/Y while Non-GAAP Diluted EPS of $1.13 increased 59% Y/Y. These measures of profitability increased at rates faster than revenue growth, representing the company’s focus on operational efficiency.

•During the quarter, BioMarin continued to realize the benefits of cost transformation initiatives implemented in 2024, resulting in a decrease in GAAP and Non-GAAP R&D and SG&A expenses Y/Y. Throughout the remainder of 2025, BioMarin expects to increase investments in VOXZOGO indication expansion, clinical pipeline development, and commercialization initiatives supporting the company’s Skeletal Conditions and Enzyme Therapies business units.

•The company generated operating cash flows totaling $174 million in first quarter 2025, an increase of 271% compared to first quarter 2024. Total cash and investments at the end of the first quarter were approximately $1.8 billion, and with anticipated increasing profitability, BioMarin is positioned to generate increasing operating cash flow into the future.

•Today, the company reaffirmed its previously communicated 2025 full-year financial guidance, which reflects the impact of tariffs that have already been enacted but does not reflect the impact of potential future pharmaceutical tariffs. BioMarin has immaterial exposure to U.S. tariffs for China, Mexico and Canada across its global supply chain operations and product sales.

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
March 31,
2025 2024 % Change
Total Revenues $745 $649 15%
Net Product Revenues by Product:
VOXZOGO $214 $153 40%
Enzyme Therapies:
VIMIZIM $188 $193 (3)%
NAGLAZYME 114 106 8%
PALYNZIQ 93 76 22%
ALDURAZYME 49 35 40%
BRINEURA 40 39 3%
Total Enzyme Therapies Revenue $484 $449 8%
KUVAN $25 $36 (31)%
ROCTAVIAN
$11 $1 1,000%
GAAP Net Income $186 $89 109%
Non-GAAP Income (1)
$221 $140 58%
GAAP Operating Margin % (2)
30.0% 13.6%
Non-GAAP Operating Margin % (1)
35.7% 23.8%
GAAP Diluted Earnings per Share (EPS) $0.95 $0.46 107%
Non-GAAP Diluted EPS (1)
$1.13 $0.71 59%

March 31,
2025 December 31,
2024
Total cash, cash equivalents & investments $ 1,779 $ 1,659

On May 1, 2025 Biogen Inc. (NASDAQ: BIIB) reported first quarter 2025 financial results (Press release, Biogen, MAY 1, 2025, View Source [SID1234652426]).

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"Biogen delivered strong first-quarter results, supporting our strategy for long-term growth. We are encouraged by the transformation in our commercial product portfolio, with approximately 45% of total product revenue in the first quarter derived from important medicines outside of our MS business," said Christopher A. Viehbacher, President and Chief Executive Officer. "In an environment of potential tariffs and medical supply security concerns, Biogen operates a significant manufacturing presence in the U.S. Roughly 75% of our 2024 U.S. product revenues were generated by products which have manufacturing operations in the U.S. In addition, Biogen pays substantial taxes in the U.S. since our U.S. market revenues are almost entirely taxable in the U.S. at Federal and state tax rates."

Financial Highlights
Q1 ’25 Q1 ’24 △
r (CC*)
Total Revenue (in millions) $2,431 $2,290 6% 8%
GAAP diluted EPS $1.64 $2.70 (39)% N/A
Non-GAAP diluted EPS $3.02 $3.67 (18)% N/A

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period.
N/A = not applicable.
* Percentage changes in revenue growth at constant currency (CC) are presented excluding the impact of changes in foreign currency exchange rates and hedging gains or losses. Foreign currency revenue values are converted into U.S. Dollars using the exchange rates from the end of the previous calendar year.

First quarter 2025 GAAP and Non-GAAP diluted EPS reflects the ~($0.95) impact from a $165 million upfront transaction payment to Stoke related to the collaboration agreement for zorevunersen in Dravet syndrome.

A reconciliation of GAAP to Non-GAAP financial measures can be found in Table 4 at the end of this news release.
Revenue Summary
(in millions) Q1 ’25 Q1 ’24 △
r (CC*)
Multiple sclerosis (MS) product revenue(1)
$953 $1,076 (11)% (10)%
Rare disease revenue(2)
$563 $424 33% 36%
Biosimilars revenue $181 $197 (8)% (5)%
Other product revenue(3)
$29 $15 93% 92%
Total product revenue $1,727 $1,712 1% 3%
Revenue from anti-CD20 therapeutic programs $378 $394 (4)% (4)%
Alzheimer’s collaboration revenue(4)
$33 $3 NMF NMF
Contract manufacturing, royalty and other revenue $293 $182 61% 63%
Total revenue $2,431 $2,290 6% 8%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period. Numbers may not foot or recalculate due to rounding.
NMF = no meaningful figure.
(1) Multiple sclerosis includes TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI and FAMPYRA. Effective
January 1, 2025, our collaboration and license agreement for FAMPYRA global commercialization rights was terminated.
(2) Rare disease includes SPINRAZA, SKYCLARYS and QALSODY.
(3) Other includes ADUHELM, FUMADERM and ZURZUVAE – First quarter 2025 ZURZUVAE revenue was approximately $28 million.
(4) Includes Biogen’s 50% share of net revenue and cost of sales, including royalties, from the LEQEMBI Collaboration.
Expense Summary
(in millions) Q1 ’25 Q1 ’24 △
GAAP cost of sales*
$629 $542 (16)%
% of Total Revenue 26% 24%
Non-GAAP cost of sales*
$580 $500 (16)%
% of Total Revenue 24% 22%
GAAP R&D expense $434 $445 3%
Non-GAAP R&D expense $427 $439 3%
GAAP SG&A expense $573 $582 2%
Non-GAAP SG&A expense $572 $569 (1)%
GAAP acquired in-process R&D (IPR&D), upfront and milestone expense $201 $8 NMF
Non-GAAP acquired IPR&D, upfront and milestone expense $201 $8 NMF

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period
NMF = no meaningful figure.
* Excluding amortization and impairment of acquired intangible assets

•The increase in first quarter 2025 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by product mix, particularly the year-over-year increase in contract manufacturing revenue, partially offset by an increase in launch product revenue.

•The decrease in first quarter 2025 GAAP and Non-GAAP R&D expense was driven primarily by savings from the Company’s R&D prioritization and Fit for Growth initiatives.

•The decrease in first quarter 2025 GAAP SG&A was driven primarily by the Company’s Fit for Growth initiative, partially offset by sales and marketing spend to support product launches.

•The increase in first quarter 2025 Non-GAAP SG&A was driven primarily by sales and marketing spend to support product launches, partially offset by savings from the Company’s Fit for Growth initiative.

•First quarter 2025 GAAP and Non-GAAP acquired IPR&D, upfront and milestone expense was approximately $201 million and includes a $165 million upfront transaction payment to Stoke related to the collaboration agreement for zorevunersen, and a $35 million milestone to MorphoSys AG as part of the initiation of the Phase 3 trial of felzartamab in antibody-mediated rejection.
Other Financial Highlights

•First quarter 2025 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $58 million, which includes approximately $48 million related to Biogen’s collaboration with Samsung Bioepis, and approximately $10 million related to Biogen’s collaboration with Sage Therapeutics, Inc. and the commercialization of ZURZUVAE in the U.S.

•First quarter 2025 GAAP other expense was approximately $68 million, primarily driven by net interest expense and net losses on strategic equity investments. First quarter 2025 Non-GAAP other expense was approximately $33 million, primarily driven by net interest expense.

•First quarter 2025 GAAP and Non-GAAP effective tax rates were 22.7% and 19.4%, respectively. First quarter 2024 GAAP and Non-GAAP effective tax rates were 15.4% and 15.9%, respectively.
Financial Position

•First quarter 2025 net cash flow from operations was approximately $259 million. Capital expenditures were approximately $37 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was approximately $222 million.

•As of March 31, 2025, Biogen had cash and cash equivalents totaling approximately $2.6 billion and approximately $6.3 billion in total debt, resulting in net debt of approximately $3.7 billion.

•For the first quarter of 2025 the Company’s weighted average diluted shares were approximately 147 million.
3

Full Year 2025 Financial Guidance

For the full year 2025, Biogen’s expected underlying business outlook remains unchanged and Biogen is updating its expected Non-GAAP diluted EPS guidance range to reflect the $165 million upfront transaction payment to Stoke and more favorable foreign exchange as follows:
Full Year 2025 Non-GAAP Diluted EPS
Prior Guidance (February 2025) $15.25 to $16.25
Approx. impact from $165 million Stoke upfront
($0.95)
Benefit mainly from foreign exchange +$0.20
Updated Guidance $14.50 to $15.50

This updated Non-GAAP diluted EPS guidance range reflects the ~($0.95) impact from a $165 million upfront transaction payment to Stoke related to the collaboration agreement for zorevunersen in Dravet syndrome, partially offset by a $0.20 benefit mainly from foreign exchange.
For 2025 as compared to 2024, Biogen expects total revenue to decline by a mid-single digit percentage as further declines in multiple sclerosis product revenue are expected to be partially offset by increases in revenue from product launches.
The Fit for Growth program is expected to generate approximately $1 billion of gross savings and $800 million net of reinvestment by the end of 2025. Biogen expects combined Non-GAAP R&D expense and Non-GAAP SG&A expense to total approximately $3.9 billion in 2025.
This financial guidance incorporates the Company’s view that Biogen’s 2025 financial outlook is not currently expected to be materially impacted by potential tariffs as previously announced by the U.S. Administration on April 2, 2025, even if the exemption for pharmaceuticals were to be removed. This is based on both a significant proportion of U.S. revenue being derived from products which have manufacturing operations in the United States, and the Company’s current global inventory positions. The U.S. and international tariff landscape remains uncertain, and this guidance does not include contemplation of any new tariffs.
This financial guidance also assumes that foreign exchange rates as of April 25, 2025, will remain in effect for the remainder of the year, net of hedging activities.
This financial guidance does not include any impact from potential acquisitions or business development transactions or pending and future litigation or any impact of potential tax or healthcare reform, as all are hard to predict. Other modeling considerations will be provided on the conference call and webcast.
Biogen may incur charges, realize gains or losses, or experience other events or circumstances in 2025 that could cause any of these assumptions to change and/or actual results to vary from this financial guidance.
Biogen does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the Company is unable to predict with reasonable certainty the financial impact of items such as the transaction, integration, and certain other costs related to acquisitions or large business development transactions; unusual gains and losses; potential future asset impairments; gains and losses from equity security investments; and the ultimate outcome of pending or future significant litigation without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the Company is unable to address the significance of the unavailable information, which could be material to future results.

Other Key Recent Events

•Today Biogen announced that BIIB122, a LRRK2 inhibitor for Parkinson’s disease developed in collaboration with Denali Therapeutics, Phase 2b LUMA study has fully enrolled with a readout expected in 2026.

•Today Biogen announced that it plans to host a series of investor events to highlight the development pipeline. Biogen plans to hold the first virtual event on June 11th at 10 a.m. ET with a focus on felzartamab and rare disease. Biogen does not intend to disclose new clinical data on the call.

Conference Call and Webcast

The Company’s earnings conference call for the first quarter will be broadcast via the internet at 8:30 a.m. ET on May 1, 2025 and will be accessible through the Investors section of Biogen’s website, www.biogen.com. Supplemental information in the form of a slide presentation is also accessible at the same location on the internet and will be subsequently available on the website for at least 90 days.

On May 1, 2025 Bicycle Therapeutics plc (NASDAQ: BCYC), a pharmaceutical company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported recent business progress and financial results for the first quarter ended March 31, 2025 (Press release, Bicycle Therapeutics, MAY 1, 2025, View Source [SID1234652425]).

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"In the first quarter, we continued to advance our business priorities and our pipeline of oncology therapeutics. We were pleased to share additional human imaging data that continue to validate the potential of MT1-MMP as a novel cancer target and demonstrate the positive properties of our Bicycle Radioconjugate molecules for radiopharmaceutical use," said Bicycle Therapeutics CEO Kevin Lee, Ph.D. "Our work to develop zelenectide pevedotin for various Nectin-4 associated cancers continues to progress, as we recently initiated our Phase 1/2 Duravelo-3 trial for NECTIN4 gene-amplified breast cancer and remain on track for dose selection in our Phase 2/3 Duravelo-2 trial for metastatic urothelial cancer in the second half of this year. Additionally, with new members on our leadership team, Board of Directors and Clinical Advisory Board, and expected financial runaway extending to the second half of 2027, we remain focused on continuing to execute our strategy and make meaningful advances for patients."

First Quarter 2025 and Recent Events

Additional human imaging data for an early Bicycle Radioconjugate (BRC) molecule targeting MT1-MMP presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025. A poster presentation included new data from a second patient who underwent MT1-MMP-PET/CT imaging that build on previously announced data. Altogether, the data continue to validate the potential of MT1-MMP as a novel cancer target and demonstrate the positive properties of BRC molecules for radiopharmaceutical use. Importantly, the imaging data from these two patients are representative of the data generated to date in 12 out of 14 patients with various solid tumors. Bicycle Therapeutics continues to advance its emerging BRC pipeline, with initial EphA2 human imaging data expected in 2H 2025 and company-sponsored clinical trials planned for 2026.
Two abstracts accepted for poster presentation at the 2025 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Bicycle Therapeutics will present two abstracts highlighting the development of zelenectide pevedotin for metastatic urothelial cancer (mUC). The first abstract outlines previously disclosed topline combination data for zelenectide pevedotin plus pembrolizumab in first-line mUC from the Phase 1 Duravelo-1 trial, while the second abstract provides an overview of the ongoing Phase 2/3 Duravelo-2 registrational trial for zelenectide pevedotin in mUC. Dose selection in Duravelo-2 remains on track for the second half of the year.
Phase 1/2 Duravelo-3 trial for zelenectide pevedotin in NECTIN4-amplified breast cancer open and actively recruiting patients. Bicycle Therapeutics previously announced a development strategy leveraging NECTIN4 amplification for zelenectide pevedotin in breast cancer, lung cancer and multiple tumor types. The strategy is based on the company’s discovery that the NECTIN4 gene sits on a commonly amplified chromosomal site in cancer, creating more copies of the gene and often translating to more protein expression. Data from post-hoc analyses of late-line breast cancer and lung cancer patients enrolled in Duravelo-1 showed enhanced anti-tumor activity of zelenectide pevedotin in patients with NECTIN4 amplification and/or polysomy. Based on these data, the U.S. Food and Drug Administration granted Fast Track designation to zelenectide pevedotin for the treatment of adult patients with previously treated, NECTIN4-amplified, advanced or metastatic triple-negative breast cancer and non-small cell lung cancer. The Duravelo-3 breast cancer trial is the first of several planned trials to expand the development of zelenectide pevedotin for additional solid tumors.
Announced new Board of Directors and key clinical leadership appointments. Felix J. Baker, Ph.D., will succeed Pierre Legault, MBA, CPA, as chairman of the Bicycle Therapeutics Board of Directors as Mr. Legault and Richard Kender, MBA, will retire from the Board following the company’s Annual General Meeting on June 17, 2025. In addition, world-renowned oncology experts Alessandro Riva, M.D., and Fabrice André, M.D., Ph.D., have joined the company’s Board of Directors and Clinical Advisory Board, respectively. Furthermore, Eric Westin, M.D., has been promoted to chief medical officer and Jim MacDonald-Clink has been promoted to senior vice president, head of business development, following the transitions of Santiago Arroyo, M.D., Ph.D., chief development officer, and Nigel Crockett, Ph.D., chief business officer, to advisor roles as distinguished fellows.
Participation in Upcoming Investor Conferences

Bicycle Therapeutics management will participate in a fireside chat at the 2025 RBC Capital Markets Global Healthcare Conference on Tuesday, May 20, at 2:05 p.m. ET. A live webcast of the fireside chat will be accessible from the Investor section of the company’s website at www.bicycletherapeutics.com. A replay of the webcast will be archived and available following the event.

First Quarter 2025 Financial Results

Cash and cash equivalents were $793.0 million as of March 31, 2025, compared to $879.5 million as of December 31, 2024. The decrease in cash and cash equivalents is primarily due to cash used in operations, including increased cash payments for clinical program activities.
Research and development (R&D) expenses were $59.1 million for the three months ended March 31, 2025, compared to $34.9 million for the three months ended March 31, 2024. The increase in expense of $24.2 million was primarily due to increased clinical program expenses for zelenectide pevedotin development, increased personnel-related expenses and lower U.K. R&D tax credits period over period.
General and administrative expenses were $21.1 million for the three months ended March 31, 2025, compared to $16.4 million for the three months ended March 31, 2024. The increase in expense of $4.7 million was primarily due to increased professional and consulting fees as well as increased personnel-related costs, including incremental share-based compensation expense of $0.5 million for the three months ended March 31, 2025.
Net loss was $60.8 million, or $(0.88) basic and diluted net loss per share, for the three months ended March 31, 2025, compared to net loss of $26.6 million or $(0.62) basic and diluted net loss per share, for three months ended March 31, 2024.

On May 1, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the first quarter ended March 31, 2025, and provided a business update (Press release, Ascendis Pharma, MAY 1, 2025, View Source [SID1234652424]).

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"The strong global launch of YORVIPATH positions 2025 to be an inflection point for Ascendis with growing revenue and a path to cashflow breakeven in the near term," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We look forward to potential approvals of our third product, TransCon CNP, as a monotherapy in children with achondroplasia, and we believe the upcoming topline COACH combination trial data may demonstrate improved outcomes, including growth, compared to TransCon CNP alone. With our diversified pipeline, robust supply chain, and strong global commercial capabilities we are well-positioned to deliver on our commitment to provide patients with highly differentiated medicines."

Select Highlights & Anticipated 2025 Milestones

TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH revenue for the first quarter of 2025 totaled €44.7 million.
Continued strong start to U.S. YORVIPATH launch, with more than 1,750 prescriptions as of March 31, 2025, and more than 1,000 unique prescribing health care providers.
On track for commercial launch in at least five additional Europe Direct countries in 2025.
International Markets exclusive distribution agreements covering 75+ countries.
TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the first quarter of 2025 totaled €51.3 million.
Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025, for Food & Drug Administration (FDA) review of supplemental Biologics License Application (BLA) for the treatment of adults with growth hormone deficiency.
During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications.
TransCon CNP
(navepegritide)
Submitted New Drug Application (NDA) to the FDA for the treatment of children with achondroplasia in the first quarter of 2025; expect to submit Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during the third quarter of 2025.
During the fourth quarter of 2025, plan to submit an IND or similar to investigate TransCon CNP alone or in combination with TransCon hGH for the treatment of hypochondroplasia.
TransCon CNP + TransCon hGH Combination Therapy
(navepegritide plus lonapegsomatropin, marketed as SKYTROFA)
Topline Week 26 data from COACH, the combination TransCon CNP and TransCon hGH trial of children with achondroplasia (ages 2-11 years) expected in the second quarter of 2025.
Oncology Programs
Clinical development of TransCon IL-2 β/γ continues, including ongoing investigation of clinical activity in platinum-resistant ovarian cancer.
Financial Update
As of March 31, 2025, Ascendis Pharma had cash and cash equivalents totaling €518 million which includes the completion of previously announced share repurchase program and the net settlement of certain Restricted Stock Units for €29 million, compared to €560 million as of December 31, 2024.
On March 21, 2025, VISEN Pharmaceuticals ("VISEN") closed its initial public offering on the Hong Kong Stock Exchange and began trading under the stock code 2561.HK. Ascendis Pharma holds 41,136,364 shares in VISEN. As of March 31, 2025, the total market value of our equity position in VISEN was approximately €260 million.
First Quarter 2025 Financial Results
Total revenue for the first quarter of 2025 was €101.0 million, compared to €95.9 million during the same period in 2024. The year-over-year increase in revenue was primarily attributable to an increase in product revenue which reflected a contribution of €44.7 million from YORVIPATH, following its commercial launch. Non-product revenue decreased to €4.9 million in the first quarter of 2025, compared to €29.4 million for the same period for 2024.

Total Revenue
(In EUR’000s) Three Months Ended
March 31,
2025 2024
Revenue
Commercial products 96,028 66,499
Rendering of services and clinical supply 3,524 4,625
Licenses 1,402 24,770
Total revenue 100,954 95,894

Commercial Product Revenue
(In EUR’000s) Three Months Ended
March 31,
2025 2024
Revenue from commercial products
SKYTROFA 51,340 65,005
YORVIPATH 44,688 1,494
Total revenue from commercial products 96,028 66,499

Research and development costs for the first quarter of 2025 were €86.6 million, compared to €70.7 million during the same period in 2024. The first quarter of 2024 was positively impacted by a reversal of prior period write-downs of pre-launch inventories related to TransCon PTH. The first quarter of 2025 was negatively impacted by an impairment charge related to property, plant and equipment due to change in activities at one of our sites in the U.S.

Selling, general, and administrative expenses for the first quarter of 2025 were €101.0 million, compared to €66.8 million during the same period in 2024. The increase was primarily due to the impact from commercial expansion including global launch activities for YORVIPATH, as well as an impairment charge related to property, plant and equipment due to change in activities at one of our sites in the U.S.

Total operating expenses for the first quarter of 2025 were €187.6 million compared to €137.5 million during the same period in 2024.

Net finance expenses for the first quarter of 2025 was €15.9 million compared to €73.6 million during the same period in 2024. The decrease was primarily driven by non-cash items.

For the first quarter of 2025, Ascendis Pharma reported a net loss of €94.6 million, or €1.58 per share (basic and diluted) compared to a net loss of €131.0 million, or €2.30 per share (basic and diluted) for the same period in 2024.

As of March 31, 2025, Ascendis Pharma had cash and cash equivalents totaling €518 million compared to €560 million as of December 31, 2024. As of March 31, 2025, Ascendis Pharma had 60,970,565 ordinary shares outstanding, including 597,055 ordinary shares represented by ADSs held by the company.

Conference Call and Webcast Information
Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its first quarter 2025 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available in this section of the Ascendis Pharma website shortly after the conclusion of the event for 30 days.

On May 1, 2025 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company working to develop a new class of drugs based on targeted protein degradation, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Arvinas, MAY 1, 2025, View Source [SID1234652423]).

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"The positive readout from our first Phase 3 trial, the first ever for a PROTAC, is a tremendous accomplishment and one which we are very proud of," said John Houston, Ph.D., Chairperson, Chief Executive Officer and President at Arvinas. "Our conviction is high that vepdegestrant can be highly competitive as a monotherapy treatment option for metastatic breast cancer in the second-line, ESR1 mutant setting. We are on track to submit a regulatory filing with health authorities, which we believe could result in the first ever approval of a PROTAC and an exciting opportunity to bring a novel treatment to patients with ESR1 mutant advanced metastatic breast cancer."

"Beyond the second-line monotherapy opportunity, we and our partners at Pfizer have removed plans for a Phase 3 first-line combination trial with atirmociclib, as well as the planned Phase 3 second-line combination trial with a CDK4/6 inhibitor, from our joint development plan," continued Dr. Houston. "This decision was made following a review of the totality of emerging information, including external data results, the evolving treatment landscape in metastatic breast cancer, and long-term capital allocation. We and Pfizer are working to evaluate future combination plans with the potential to maximize patient benefit and shareholder value."

1Q 2025 Business Highlights and Recent Developments

Vepdegestrant: Oral PROTAC ER degrader: As part of Arvinas global collaboration with Pfizer, the companies:

Reported positive topline data for VERITAC-2, the Phase 3 pivotal 2L+ trial of monotherapy vepdegestrant in patients with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) advanced or metastatic breast cancer, in the estrogen receptor 1-mutant population.
VERITAC-2 data accepted for oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (May 30 – June 3, 2025).
Announced that Pfizer will add a vepdegestrant/KAT6 cohort to its ongoing Phase 1 trial evaluating its investigational KAT6 inhibitor (PF-07248144) in combination with endocrine therapies following CDK4/6 inhibitor treatment.
Removed the first-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib, from the agreed-upon joint development plan.
Removed the second-line Phase 3 combination trial with a CDK4/6 inhibitor from the agreed-upon joint development plan.
ARV-102: Oral PROTAC LRRK2 degrader

Presented single ascending dose (SAD) and multiple ascending dose (MAD) data from the ongoing Phase 1 clinical trial in healthy volunteers in an oral session at the Alzheimer’s Disease/Parkinson’s Disease (AD/PD) conference in Vienna, Austria demonstrating blood-brain barrier penetration, and central and peripheral LRRK2 degradation:
At a single oral dose of at least 60 mg, and once daily repeated oral doses of at least 20 mg, ARV-102 achieved greater than 50% LRRK2 reduction in the cerebral spinal fluid (CSF) and greater than 90% LRRK2 reduction in the peripheral blood mononuclear cells (PBMCs), indicating substantial central and peripheral LRRK2 protein degradation.
Inhibition of Rab10 phosphorylation in PBMCs and reduction of bis(monoacylglycerol)phosphate (BMP) in urine following single doses of ARV-102, signifying downstream LRRK2 pathway engagement.
Bioavailable and brain penetrant with dose dependent exposure in the CSF.
ARV-102 was generally safe and well tolerated with no serious adverse events reported after single or multiple doses.
ARV-393: Oral PROTAC BCL6 degrader

Continued recruiting patients in the first-in-human Phase 1 clinical trial in patients with non-Hodgkin lymphoma (NHL) (ClinicalTrials.gov Identifier: NCT06393738).
Presented new preclinical data of ARV-393 in combination with standard of care (SOC) biologic agents and small molecule inhibitors (SMI) in high-grade B-cell lymphoma (HGBCL) and aggressive diffuse large B-cell lymphoma (DLBCL) models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating that ARV-393 had broad and significant combinability with:
SOC chemotherapy:
ARV-393 in combination with R-CHOP, induced significantly greater tumor growth inhibition (TGI) compared with rituximab, CHOP, R-CHOP, or ARV-393 alone, with complete tumor regressions enabled by the combination.
SOC biologics:
Similarly, the combination of ARV-393 and SOC biologics targeting CD19 (tafasitamab), CD79b (polatuzumab vedotin), or CD20 (rituximab) resulted in tumor regressions and demonstrated significantly stronger TGI compared with either agent alone.
Investigational small molecule inhibitors targeting clinically validated oncogenic drivers of lymphoma:
Combination of ARV-393 with a BTK inhibitor (acalabrutinib), a BCL2 inhibitor (venetoclax), or an EZH2 inhibitor (tazemetostat) induced tumor regressions in the majority of mice.
Overall, current preclinical data suggest that ARV-393 has the potential to be an attractive combination partner for development of novel therapies for lymphoma, including chemo-free combination regimens and/or "all oral" treatment options.
ARV-806: Novel PROTAC KRAS G12D degrader

Filed an Investigational New Drug (IND) application and received a safe-to-proceed letter from the U.S. Food and Drug Administration.
Corporate updates:
As part of a Company-wide cost reduction effort:

Announced the removal of two Phase 3 trials from the vepdegestrant development plan
Announced a reduction in workforce of approximately one-third across the Company to streamline operations across the organization and enable the efficient progression of the Company’s portfolio. The reduction is planned to be completed in the second quarter of 2025.
Announced a reprioritization of the Company’s preclinical portfolio to focus on assets that have the greatest potential to deliver value for patients, physicians and shareholders.
Updated guidance for its cash runway into the second half of 2028.
"We continuously evaluate and refine our long-term strategy, and recent challenges in the capital markets have required us to expeditiously evaluate our business priorities and capital needs," continued Dr. Houston. "Although difficult, the workforce reduction is a prudent decision that we believe will right-size the Company for future success. I want to thank all the talented employees who were directly impacted by this decision. I’m proud of the progress we have made together, and want to acknowledge their contributions, and commitment, to discovering and developing new treatment options for patients with serious diseases."

Anticipated Upcoming Milestones and Expectations

Vepdegestrant: Oral PROTAC ER degrader
As part of Arvinas’ global collaboration with Pfizer, the companies plan to:

Present detailed results from the VERITAC-2 Phase 3 clinical trial in a late-breaker oral presentation at ASCO (Free ASCO Whitepaper) (2Q 2025).
VERITAC-2 abstract will be featured in the ASCO (Free ASCO Whitepaper) press program and has been selected to be included in the "2025 Best of ASCO (Free ASCO Whitepaper) Program."
Share data with global regulatory authorities to potentially support regulatory filings (2Q 2025) and submit new drug application to the U.S. Food and Drug Administration for potential approval (2H 2025).
Add a combination cohort of vepdegestrant plus Pfizer’s KAT6 inhibitor (PF-07248144) to Pfizer’s ongoing Phase 1 trial (NCT04606446).
The trial is currently evaluating Pfizer’s KAT6 inhibitor in combination with endocrine therapies following CDK4/6 inhibitor treatments; the trial is being operationalized and funded by Pfizer.
ARV-102: Oral PROTAC LRRK2 degrader

Present final data from the SAD/MAD cohorts of the Phase 1 clinical trial in healthy volunteers (2H 2025).
Continue enrollment and present initial data from the SAD cohort of the ongoing Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025).
Initiate the MAD cohort of the Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025).
ARV-393: Oral PROTAC BCL6 degrader

Present new preclinical data demonstrating single agent activity of ARV-393 in patient derived xenograft models of transformed Follicular Lymphoma and a patient-derived xenograft model of nTFHL-AI (angioimmunoblastic type of nodal T-follicular helper cell lymphoma), a rare and aggressive non-Hodgkin lymphoma with high unmet need and limited treatment options, at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 conference in Milan, Italy (June 12-15, 2025).
Share preclinical data in combination with an emerging SOC option in 2L DLBCL (2H 2025).
Share preliminary clinical data from the ongoing Phase 1 clinical trial in patients with NHL (NCT06393738) (2H 2025).
ARV-806: Novel PROTAC KRAS G12D degrader

Initiate a first-in-human Phase 1 trial in patients with solid tumors harboring KRAS G12D mutations (2H 2025).
Financial Guidance
Based on its current operating plan, Arvinas believes its cash, cash equivalents, and marketable securities as of March 31, 2025, is sufficient to fund planned operating expenses and capital expenditure requirements into the second half of 2028.

First Quarter Financial Results
Cash, Cash Equivalents, and Marketable Securities Position: As of March 31, 2025, cash, cash equivalents and marketable securities were $954.3 million as compared with cash, cash equivalents and marketable securities of $1,039.4 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities of $85.1 million for the three months ended March 31, 2025 was primarily related to cash used in operations of $85.1 million, the purchase of lab equipment and leasehold improvements of $0.4 million and $0.1 million of long term debt repayments, partially offset by unrealized gains on marketable securities of $0.5 million.

Research and Development Expenses: Generally Accepted Accounting Principles (GAAP) Research and development (R&D) expenses were $90.8 million for the quarter ended March 31, 2025, as compared with $84.3 million for the quarter ended March 31, 2024. The increase in research and development expenses of $6.5 million for the quarter was primarily due to an increase in external expenses of $7.8 million, partially offset by a decrease in compensation and related personnel expenses of $1.4 million, which are not allocated by program. External expenses include (i) program-specific expenses, which increased by $10.0 million, primarily driven by increases in our ARV-102, vepdegestrant, ARV-393 and other programs of $5.2 million, $5.0 million, $1.4 million and $2.5 million, respectively, offset by decreases in our luxdegalutamide (ARV-766) and bavdegalutamide (ARV-110) programs of $3.6 million and $0.5 million, respectively, and (ii) our non-program specific expenses, which decreased by $2.2 million.

Non-GAAP R&D expenses were $79.3 million for the quarter ended March 31, 2025, as compared with $71.9 million for the quarter ended March 31, 2024, excluding $11.5 million and $12.4 million of non-cash stock-based compensation expense for the quarters ended March 31, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

General and Administrative Expenses: GAAP General and administrative (G&A) expenses were $26.6 million for the quarter ended March 31, 2025, as compared with $24.3 million for the quarter ended March 31, 2024. The increase in general and administrative expenses of $2.3 million for the quarter was primarily due an increase in professional fees of $2.4 million, inclusive of an increase in the amortization of costs to obtain a contract of $2.6 million related to changes in total Vepdegestrant (ARV-471) Collaboration Agreement program cost estimates resulting from the removal of two Phase 3 combination trials from the development plan and an increase in costs related to developing our commercial operations of $2.3 million, partially offset by a decrease in personnel and infrastructure related costs of $2.4 million.

Non-GAAP G&A expenses were $23.1 million for the quarter ended March 31, 2025, as compared with $18.0 million for the quarter ended March 31, 2024, excluding $3.5 million and $6.3 million of non-cash stock-based compensation expense for the quarters ended March 31, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

Revenue: Revenue was $188.8 million for the quarter ended March 31, 2025 as compared with $25.3 million for the quarter ended March 31, 2024. Revenue for the quarter is related to the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer and the collaboration and license agreement with Pfizer. The increase in revenue of $163.5 million was primarily due to an increase in revenue from the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer totaling $167.8 million related to changes in total program cost estimates resulting from the removal of the first-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib, and the removal of the second-line Phase 3 combination trial with a CDK4/6 inhibitor from the development plan, offset by a decrease in revenue from the Pfizer Research Collaboration Agreement of $2.7 million due to changes in estimates of the performance period duration under the agreement resulting from updated research timelines and a decrease in revenue from the Bayer Collaboration Agreement of $1.6 million related to the termination of the Bayer Collaboration Agreement in August 2024.

Investor Call & Webcast Details
Arvinas will host a conference call and webcast today, May 1, 2025, at 8:00 a.m. ET to review its first quarter 2025 financial results and discuss recent corporate updates. Participants are invited to listen by going to the Events and Presentation section under the Investors page on the Arvinas website at www.arvinas.com. A replay of the webcast will be available on the Arvinas website following the completion of the event and will be archived for up to 30 days.