Rezolute Reports Second Quarter Fiscal 2025 Financial Results and Provides Business Update

On February 12, 2025 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, reported financial results and provided a business update for the three months ended December 31, 2024 (Press release, Rezolute, FEB 12, 2025, View Source [SID1234650212]).

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"We have made significant regulatory progress with ersodetug and our focus in 2025 remains squarely on advancing both Phase 3 trials for patients with congenital HI and tumor HI," said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. "We are encouraged by our momentum and remain dedicated to providing meaningful and innovative treatments for patients with limited options."

Recent Pipeline Progress and Anticipated Milestones

Congenital HI

· The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to ersodetug for the treatment of hypoglycemia due to congenital HI.
·
sunRIZE, a Phase 3, multicenter, double-blind, randomized, controlled safety and efficacy registrational study, is ongoing.

○ Enrollment of U.S. participants is anticipated to occur in the second quarter of 2025.
○ Overall study enrollment expected to conclude in the second quarter of 2025, with topline results expected in the fourth quarter of 2025, subject to outcomes from an interim analysis.

· An independent Data Monitoring Committee (DMC) reviewed safety and pharmacokinetics of ersodetug in eight infant participants in the open-label arm of the sunRIZE study.
○ Ersodetug administered between 5-10 mg/kg during a bi-weekly loading phase and a monthly maintenance phase was generally safe and well-tolerated.
○ Observed ersodetug drug levels at peak and trough were comparable to exposures in older pediatric participants in the Phase 2b RIZE study and validate the chosen dose regimen of 5 and 10 mg/kg administered bi-weekly and monthly.
○ Subsequent infant participants may be enrolled into the double-blind, placebo-controlled study.
○ The DMC did not analyze efficacy and the Company remains blinded to the results.

· An interim analysis of the primary study endpoint (change in hypoglycemia events) is planned for this quarter, with results and study update to be announced early in the second quarter.
○ Three possible outcomes from the analysis include: (i) futility and the study should be stopped, (ii) continue the study as is or (iii) continue the study as is but increase the sample size by 33% (18 additional patients) to enhance statistical confidence in the final outcome.

Tumor HI

· During the quarter, FDA granted Orphan Drug Designation to ersodetug for the treatment of hypoglycemia due to tumor HI.
· A Phase 3 registrational study for ersodetug in patients with tumor HI is anticipated to begin in the first half of 2025.
· Topline results are expected in the second half of 2026.

Fiscal Second Quarter Financial Results

Cash, cash equivalents and investments in marketable securities were $105.3 million as of December 31, 2024, compared to $127.1 million as of June 30, 2024.

Research and development expenses were $12.6 million for the second quarter of fiscal 2025, compared with $12.0 million for the same period a year ago, with the increase primarily attributable to increased expenditures in clinical trial activities, manufacturing costs and higher personnel-related expenses, which include employee compensation.

General and administrative expenses were $4.5 million for the second quarter of fiscal 2025, compared with $3.2 million for the same period a year ago, with the increase primarily attributable to professional fees and employee-related expenses as a result of increased headcount.

Net loss was $15.7 million for the second quarter of fiscal 2025 compared with a net loss of $13.9 million for the same period a year ago.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism (HI). Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.

About sunRIZE

The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled, parallel arm study designed to evaluate the efficacy and safety of ersodetug in patients with congenital HI who are experiencing poorly controlled hypoglycemia. Participants between the ages of 3 months to 45 years old are eligible to participate. The study is enrolling up to 56 participants in more than a dozen countries around the world.

Replimune Reports Fiscal Third Quarter 2025 Financial Results and Provides Corporate Update

On February 12, 2025 Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported financial results for the fiscal third quarter ended December 31, 2024 and provided a business update (Press release, Replimune, FEB 12, 2025, View Source [SID1234650211]).

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"Over the past couple of months, we have achieved significant regulatory milestones for RP1 in anti-PD-1 failed melanoma," said Sushil Patel, Ph.D., CEO of Replimune. "With Priority Review and a PDUFA date set for July 22, 2025, by the FDA, our efforts are focused on ensuring a successful commercial launch of RP1 upon approval. Our commercial strategy is built on a deep understanding of the patient population and prescriber landscape, coupled with a launch model designed to effectively deliver intratumoral therapy. With over $500 million in cash, we are well-capitalized to execute our plans and are excited to provide further updates as we transition to a commercial-stage company."

Program Highlights & Milestones

RP1

RP1 combined with Opdivo (nivolumab) in anti-PD1 failed melanoma
In January, the FDA accepted the BLA for RP1 in combination with nivolumab for patients with advanced melanoma. The BLA was granted Priority Review by the FDA with a PDUFA action date of July 22, 2025.
The BLA is supported by the primary analysis data of the IGNYTE trial, evaluating RP1 combined with nivolumab in patients with anti-PD-1 failed melanoma.
Enrolling into the confirmatory Phase 3 trial, IGNYTE-3, with over 100 sites planned globally. This trial will assess RP1 in combination with nivolumab in patients with advanced melanoma who have progressed on anti-PD-1 and anti-CTLA-4 therapies or are ineligible for anti-CTLA-4 treatment.
RP2

RP2 in uveal melanoma
Enrolled the first patient in a registration-directed study of RP2 in metastatic uveal melanoma in patients who are immune checkpoint inhibitor-naïve. The study will enroll approximately 280 patients and evaluate RP2 in combination with nivolumab versus ipilimumab in combination with nivolumab. The primary endpoints of the study are overall survival and progression free survival and key secondary endpoints are overall response rate and disease control rate.
RP2 in hepatocellular carcinoma (HCC)
Enrolled the first patient in a Phase 2 clinical trial with RP2 combined with atezolizumab and bevacizumab in anti-PD1/PD-L1 progressed HCC. The trial is an open label trial that will enroll 30 patients and evaluate RP2 combined with the second-line therapy of atezolizumab and bevacizumab. The study is being conducted under a collaboration and supply agreement with Roche.
Financial Highlights

Financing: Completed a public offering of shares of the Company’s common stock and pre-funded warrants, raising approximately $156.0 million net of issuance costs. Proceeds from the financing will be used to fund the continued development of our RPx platform, including expanding our ongoing studies within RP1 and broadening clinical development plans for RP2, as well as for working capital and general corporate purposes.
Cash Position: As of December 31, 2024, cash, cash equivalents and short-term investments were $536.5 million, as compared to $420.7 million as of fiscal year ended March 31, 2024. The increase in cash balance was directly related to the public offering in November, somewhat offset by cash utilized in operating activities in advancing the Company’s clinical development plans.

Based on the current operating plan, the Company believes that existing cash, cash equivalents and short-term investments, as of December 31, 2024 will enable the Company to fund operations into the fourth quarter of 2026 which includes scale up for the potential commercialization of RP1 in skin cancers and for working capital and general corporate purposes and excludes any potential revenue.
R&D Expenses: Research and development expenses were $48.0 million for the fiscal third quarter ended December 31, 2024, as compared to $42.8 million for the fiscal third quarter ended December 31, 2023. This increase was primarily due to an increase in personnel-related costs, as well as consulting and facility-related costs. Research and development expenses included $4.6 million in stock-based compensation expenses for the fiscal third quarter ended December 31, 2024.
S,G&A Expenses: Selling, general and administrative expenses were $18.0 million for the fiscal third quarter ended December 31, 2024, as compared to $13.7 million for the fiscal third quarter ended December 31, 2023. Selling, general and administrative expenses included $4.1 million in stock-based compensation expenses for the fiscal third quarter ended December 31, 2024.
Net Loss: Net loss was $66.3 million for the fiscal third quarter ended December 31, 2024, as compared to a net loss of $51.1 million for the fiscal third quarter ended December 31, 2023.
About RP1

RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2

RP2 is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response. RP2 additionally expresses an anti-CTLA-4 antibody-like molecule, as well as GALV-GP R- and GM-CSF. RP2 is intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic-immune-based efficacy on tumors and limiting off-target toxicity.

Orca Bio Presents Positive Phase 1 and 1b Data on Orca-T and Orca-Q at the 2025 Tandem Meetings of ASTCT® and CIBMTR®

On February 12, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported positive data on its investigational allogeneic T-cell immunotherapies will be presented across four sessions at the 2025 Tandem Meetings of ASTCT and CIBMTR, February 12-15, 2025, in Honolulu, HI (Press release, Orca Bio, FEB 12, 2025, View Source;utm_medium=rss&utm_campaign=orca-bio-presents-positive-phase-1-and-1b-data-on-orca-t-and-orca-q-at-the-2025-tandem-meetings-of-astct-and-cibmtr [SID1234650210]).

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"We are pleased to present positive findings across four presentations at the 2025 Tandem Meetings, underscoring our commitment to engaging global experts in a collective effort to advance the field for patients and the physicians who treat them," said Scott McClellan, MD, chief medical officer at Orca Bio. "These data add to our growing body of clinical evidence which reinforces the potential of our high-precision approach to offer new and potentially transformative treatment options for patients."

Highlights include:

New research that sheds light on early T-cell activation following treatment with Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared with unmanipulated peripheral blood stem cell grafts, in addition to the identification of a novel T-cell subset which may be predictive of long-term immune activation after Orca-T administration.
Three-year follow-up data from the Phase 1b trial of Orca-T demonstrating improved overall survival in AML, ALL and high-risk MDS patients compared to a conventional allogeneic stem cell transplant (alloHSCT) with post-transplant cyclophosphamide (PTCy)-based GvHD prophylaxis. Results previously presented at the 2024 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
Encouraging results from a single-center open-label Phase 1 study of an expanded group of older patients treated with Orca Bio Orca-T and a reduced-intensity conditioning (RIC) regimen. Cohorts of fewer patients were previously presented at the 2023 ASH (Free ASH Whitepaper) Annual Meeting and the 50th Annual Meeting of the EBMT.
Results from the Phase 1 trial of Orca Bio’s second investigational allogeneic T-cell immunotherapy, Orca-Q, demonstrating promising outcomes without the use of pharmacological GvHD prophylaxis in patients with fully matched donors. Data previously presented at the 2024 ASH (Free ASH Whitepaper) Annual Meeting.
The abstracts are available at www.tandemmeetings.com. Details of the presentations follow:

Oral Session: Session L – Autoimmune Disease and Immune Reconstitution

Title: FOXP3 and Helios Expressing CD4+ T Conventional Cells Correlate with T Cell Activation after Orca-T Allogeneic T Cell Immunotherapy

Date and Time: February 15, 2025 at 10:30 AM HST

Location: Ballroom C (HCC)

Poster Session: Acute and Chronic Leukemia (AML, MDS, MPD ALL, CML) – Clinical

Title: Observational Comparison of Overall Survival between Phase 1b Orca-T and Registry-Based Post-Transplant Cyclophosphamide Patients

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

Poster Session: Graft-versus-Host and Graft-versus-Tumor – Clinical: Prevention, Treatment and Biomarkers

Title: Phase 1 Trial Results for Patients with Advanced Hematologic Malignancies Treated with Allogeneic T Cell Immunotherapy and Reduced Intensity Conditioning

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

Poster Session: Acute and Chronic Leukemia (AML, MDS, MPD ALL, CML) – Clinical

Title: Preliminary Safety and Efficacy of Myeloablative Orca-Q with No GvHD Prophylaxis for Treatment of Advanced Hematologic Malignancies

Date and Time: February 13, 2025 at 6:45 PM HST

Location: Exhibit Hall 3 (HCC)

About Orca-T
Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated in clinical trials for the treatment of multiple hematologic malignancies. Orca-T is comprised of highly purified regulatory T-cells, CD34+ stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T is currently being evaluated in Precision-T, a pivotal Phase 3 clinical trial, which has completed enrollment at leading transplant centers across the U.S. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration.

Biogen reports fourth quarter and full year 2024 results and provides full year 2025 financial guidance

On February 12, 2025 Biogen Inc. (NASDAQ: BIIB) reported fourth quarter and full year 2024 financial results (Press release, Biogen, FEB 12, 2025, View Source [SID1234650209]). Commenting on the results, President and Chief Executive Officer Christopher A. Viehbacher said:

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"We believe 2024 was an important year on our journey to deliver long-term sustainable growth. We delivered continued revenue growth from our ongoing product launches including LEQEMBI, where we believe there remains a significant long-term opportunity. We also achieved key development milestones across our late-stage pipeline, where we continue to prioritize high-conviction assets with the potential to drive growth well into the next decade. Our financial discipline has enabled a restructuring of our operating expenses with a reallocation of resources toward potential future growth drivers. We believe that continued execution against these key strategic elements, as well as a disciplined approach to business development, will allow us to generate long-term value for our shareholders by bringing innovative medicines to patients."

Financial Highlights
Q4 ’24 Q4 ’23 △
r (CC*)
FY ’24 FY ’23 △
r (CC*)
Total Revenue (in millions) $2,455 $2,386 3% 2% $9,676 $9,836 (2)% (2)%
GAAP diluted EPS $1.83 $1.71 7% N/A $11.18 $7.97 40% N/A
Non-GAAP diluted EPS $3.44 $2.95 17% N/A $16.47 $14.72 12% 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.

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, except percentages) Q4 ’24 Q4 ’23 △
r (CC#)
FY ’24 FY ’23 △ △ (CC#)
Multiple Sclerosis (MS) product revenue(1)
$1,070 $1,168 (8)% (9)% $4,350 $4,662 (7)% (7)%
Rare disease revenue(2)
$535 $472 13% 15% $1,988 $1,803 10% 11%
Biosimilars revenue $202 $188 7% 4% $793 $770 3% 2%
Other product revenue(3)
$26 $4 NMF NMF $83 $12 NMF NMF
Total product revenue $1,833 $1,832 —% —% $7,214 $7,247 —% —%
Revenue from anti-CD20 therapeutic programs $465 $436 7% 7% $1,750 $1,690 4% 4%
Alzheimer’s collaboration revenue(4)
$27 $2 NMF NMF $60 $— NMF NMF
Contract manufacturing, royalty and other revenue $130 $117 12% 9% $653 $899 (27)% (28)%
Total revenue $2,455 $2,386 3% 2% $9,676 $9,836 (2)% (2)%

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) MS includes TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI and FAMPYRA.
(2) Rare disease includes SPINRAZA, SKYCLARYS and QALSODY.
(3) Other includes ZURZUVAE, ADUHELM and FUMADERM.
(4) Includes Biogen’s 50% share of net revenue and cost of sales, including royalties, from the LEQEMBI Collaboration.
•Fourth quarter and full year 2024 ZURZUVAE revenue was approximately $23 million and approximately $72 million, respectively.
Expense Summary
(In millions, except percentages) Q4 ’24 Q4 ’23 △ FY ’24 FY ’23 △
GAAP cost of sales*
$583 $618 6% $2,310 $2,533 9%
% of Total Revenue 24% 26% 24% 26%
Non-GAAP cost of sales*
$540 $587 8% $2,137 $2,502 15%
% of Total Revenue 22% 25% 22% 25%
GAAP R&D expense $532 $571 7% $2,042 $2,462 17%
Non-GAAP R&D expense $528 $568 7% $1,930 $2,262 15%
GAAP SG&A expense $680 $609 (12)% $2,404 $2,550 6%
Non-GAAP SG&A expense $673 $588 (14)% $2,340 $2,277 (3)%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period
*Excluding amortization and impairment of acquired intangible assets

2

•The decrease in fourth quarter 2024 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by lower idle capacity charges.

•The decrease in full year 2024 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 revenue from new product launches, a decrease in contract manufacturing revenue, and lower idle capacity charges.

•The decrease in fourth quarter and full year 2024 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 increase in fourth quarter 2024 GAAP and 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.

•Full year 2024 GAAP and Non-GAAP SG&A includes higher operational spending on sales and marketing activities in support of LEQEMBI and SKYCLARYS, which was partially offset by cost-reduction measures realized in connection with the Company’s Fit for Growth program.
Other Financial Highlights

•Fourth quarter 2024 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $57 million. This includes approximately $51 million related to Biogen’s collaboration with Samsung Bioepis, and approximately $6 million related to Biogen’s collaboration with Sage Therapeutics related to the commercialization of ZURZUVAE in the U.S.

•Full year 2024 GAAP and Non-GAAP collaboration profit sharing was a net expense of approximately $254 million. This includes approximately $227 million related to Biogen’s collaboration with Samsung Bioepis, and approximately $27 million related to Biogen’s collaboration with Sage Therapeutics related to the commercialization of ZURZUVAE in the U.S.

•Fourth quarter 2024 GAAP other expense was approximately $150 million and includes approximately $78 million of net losses on strategic equity investments and approximately $42 million of net interest expense. Fourth quarter 2024 Non-GAAP other expense was approximately $72 million, primarily driven by net interest expense.

•Full year 2024 GAAP other expense was approximately $344 million and includes approximately $183 million of net interest expense and approximately $100 million of net losses on strategic equity investments. Full year 2024 Non-GAAP other expense was approximately $243 million, primarily driven by net interest expense.

•Fourth quarter 2024 GAAP and Non-GAAP effective tax rates were 8.5% and 12.2%, respectively. Fourth quarter 2023 GAAP and Non-GAAP effective tax rates were 14.7% and 17.0%, respectively.

•Full year 2024 GAAP and Non-GAAP effective tax rates were 14.4% and 14.6%, respectively. Full year 2023 GAAP and Non-GAAP effective tax rates were 10.4% and 15.2%, respectively.
Financial Position

•Fourth quarter 2024 net cash flow from operations was approximately $761 million. Capital expenditures were approximately $39 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was approximately $722 million.

•Full year 2024 net cash flow from operations was approximately $2.9 billion. Capital expenditures were approximately $154 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was approximately $2.7 billion.

3

•As of December 31, 2024, Biogen had cash and cash equivalents totaling approximately $2.4 billion with approximately $6.3 billion in total debt, resulting in net debt of approximately $3.9 billion.

•For the fourth quarter of 2024 the Company’s weighted average diluted shares were 146 million. For full year 2024 the Company’s weighted average diluted shares were 146 million.

Full Year 2025 Financial Guidance

For the full year 2025, Biogen expects a Non-GAAP diluted EPS guidance range as follows:
Full Year 2025 Guidance
Non-GAAP diluted EPS
$15.25 to $16.25

This Non-GAAP diluted EPS guidance range, which is based upon FX rates on February 7th, 2025, includes a headwind of approximately $0.35 from foreign exchange when compared to average exchange rates in 2024.

Total revenue is expected to decline by a mid-single digit percentage for 2025 compared to 2024 as further declines in multiple sclerosis product revenue are expected to be partially offset by increases in revenue from product launches.

For 2025 as compared to 2024, Biogen expects operating margin percentage to remain relatively flat. 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. Since the program was initiated in 2023, approximately $400 million of net savings have been achieved, and Biogen expects to realize the balance 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 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.

This guidance also assumes that foreign exchange rates as of February 7, 2025, will remain in effect for the remainder of the year, net of hedging activities. 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.

Key Recent Events

•As part of ongoing pipeline prioritization efforts, Biogen has decided to discontinue further development of BIIB113 in early Alzheimer’s disease, BIIB094 in early Parkinson’s disease, BIIB101 in multiple system atrophy, and BIIB143 (cemdomespib) in diabetic peripheral neuropathic pain.

4

•In February 2025, Royalty Pharma plc announced that it had entered into an agreement with Biogen to provide research and development funding of up to $250 million for litifilimab. Following potential regulatory approval, Royalty Pharma plc will be eligible for regulatory milestones and royalties of a mid-single digits percentage of the applicable net sales.

Conference Call and Webcast

The Company’s earnings conference call for the fourth quarter will be broadcast via the internet at 8:30 a.m. ET on February 12, 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.

Ascendis Pharma Reports Fourth Quarter and Full Year 2024 Financial Results

On February 12, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a business update (Press release, Ascendis Pharma, FEB 12, 2025, View Source [SID1234650208]).

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"Having achieved pivotal milestones in 2024, Ascendis is positioned to continue strong revenue growth in 2025 and beyond," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We believe YORVIPATH is well on its way to establishing itself as the new global standard for the treatment of hypoparathyroidism in adults. SKYTROFA has achieved a leading position in value in the U.S. growth hormone market. And for TransCon CNP, we have a clear path to submit our NDA and MAA as a differentiated treatment of achondroplasia in children. Together with a strong cash balance and established partnerships, I am confident in our ability to become a leading global biopharmaceutical company with multiple blockbuster products and a strong engine for future innovation."

Select 2024 Highlights & Anticipated 2025 Milestones

TransCon hGH
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA fourth quarter 2024 revenue excluding a positive impact due to reversal of €4.6 million of sales deductions related to prior years was ~€54 million (fourth quarter reported 2024 SKYTROFA revenue of €58.5 million).
SKYTROFA full year 2024 revenue excluding €4.7 million of sales deductions related to prior years was ~€202 million (full year reported 2024 SKYTROFA revenue of €197.0 million).
Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025, for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.
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 PTH
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH revenue for the fourth quarter of 2024 totaled €13.6 million and €28.7 million for the full year 2024, as previously announced.
Strong start to U.S. YORVIPATH launch, with 908 prescriptions as of Feb. 7, 2025, and 539 unique prescribing health care providers.
Expect commercial launch in at least five additional Europe Direct countries in 2025.
Eight International Markets exclusive distribution agreements signed covering 50+ countries.
TransCon CNP
(navepegritide)
Following pre-NDA meeting with FDA, on track to submit New Drug Application (NDA) for the treatment of achondroplasia in children during the first quarter of 2025, and to submit Marketing Authorisation Application (MAA) to the European Medicines Agency during the third quarter of 2025.
Presented new data demonstrating additional benefits beyond linear growth, with significant improvements in leg bowing (a common complication in achondroplasia) observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.
During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia.
TransCon hGH / TransCon CNP Combination Treatment
Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) in children with achondroplasia expected in the second quarter of 2025.
Oncology Program
Clinical development of TransCon IL-2 β/γ continues, including ongoing investigation of clinical activity in platinum-resistant ovarian cancer (PROC).
Financial Update
December 31, 2024, cash and cash equivalents totaling €559.5 million.
Subsequent to the year end, in January 2025, received $100 million related to the Exclusive License Agreement with Novo Nordisk announced last year. Including the $100 million upfront payment, cash at the end of 2024 would have totaled €655 million.
Fourth Quarter and Full Year 2024 Financial Results
Total revenue for the fourth quarter of 2024 was €173.9 million, compared to €137.7 million during the same period for 2023. The increase was primarily attributable to the upfront fee of $100 million from Novo Nordisk and the EU launch of YORVIPATH.

Total revenue for 2024 was €363.6 million compared to €266.7 million in 2023. The increase was primarily attributable to the upfront fee of $100 million from Novo Nordisk and greater commercial product revenue. Non-product revenue was €137.9 million in 2024, compared to €88.1 million in 2023.

Total Revenue
(In EUR’000s)

Three Months Ended
December 31, Twelve Months Ended
December 31,
2024
2023
2024
2023
Revenue from external customers
Commercial products 72,130 64,249 225,728 178,663
Licenses 95,853 64,304 122,343 66,077
Other 5,933 9,150 15,570 21,978
Total revenue from external customers 173,916 137,703 363,641 266,718

Commercial Product Revenue
(In EUR’000s)

Three Months Ended
December 31, Twelve Months Ended
December 31,
2024
2023
2024
2023
Revenue from commercial products
SKYTROFA 58,546 64,249 197,001 178,663
YORVIPATH 13,584 — 28,727 —
Total revenue from commercial products 72,130 64,249 225,728 178,663

Research and development (R&D) costs for the fourth quarter of 2024 were €79.3 million, compared to €90.9 million during the same period in 2023. The decline was largely due to lower external development costs for TransCon hGH and TransCon PTH, as well as the Eyconis spin-off. R&D costs for 2024 were €307.0 million compared to €413.5 million in 2023. The lower R&D costs in 2024 was driven primarily by a decrease in external program development costs as well as the Eyconis spin-off.

Selling, general, and administrative (SG&A) expenses for the fourth quarter of 2024 were €80.2 million, compared to €64.0 million during the same period in 2023. The increase was due to higher employee costs, including the impact from global commercial expansion, and higher external commercial costs. SG&A expenses for 2024 were €291.1 million compared to €264.4 million in 2023. Higher SG&A expenses were primarily due to higher employee related expenses and other general and administrative expenses attributable to organizational growth in support of launch of YORVIPATH in Europe and the U.S.

Total operating expenses for the fourth quarter of 2024 were €159.5 million compared to €154.9 million during the same period in 2023. Total operating expenses for 2024 were €598.1 million compared to €677.9 million in 2023.

Net finance expenses were €33.2 million in the fourth quarter compared to €41.6 million in the same period in 2023. Net finance expenses for 2024 were €74.4 million compared to €0.2 million in 2023. The full year net finance expense increase was driven primarily by non-cash items.

For the fourth quarter of 2024, Ascendis Pharma reported a net loss of €38.5 million, or €0.64 per share (basic and diluted) compared to a net loss of €86.9 million, or €1.54 per share (basic and diluted) for the same period in 2023. For the full year 2024, Ascendis Pharma reported a net loss of €378.1 million, or €6.53 per share (basic and diluted) compared to a net loss of €481.4 million, or €8.55 per share (basic and diluted) in 2023.

As of December 31, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €559.5 million compared to €399.4 million as of December 31, 2023. Subsequent to the year end, we received the $100 million upfront payment from Novo Nordisk which was received in January 2025. As of December 31, 2024, Ascendis Pharma had 60,689,487 ordinary shares outstanding, including 845,887 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 fourth quarter and full year 2024 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 on this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days.