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.

Aptose’s Frontline Triple Drug Therapy with Tuspetinib Achieves Notable Responses in Newly Diagnosed AML Patients in the Phase 1/2 TUSCANY Trial

On February 12, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company, reported promising early safety and response results from newly diagnosed acute myeloid leukemia (AML) patients dosed in Aptose’s Phase 1/2 TUSCANY trial with a 40 mg dose of tuspetinib in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) (Press release, Aptose Biosciences, FEB 12, 2025, View Source [SID1234650207]). The TUS+VEN+AZA triplet is being developed as a frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy.

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In January 2025, Aptose announced the initiation of the TUSCANY trial and dosing in the first cohort of newly-diagnosed AML patients with the lowest starting dose (40 mg) of TUS as part of the TUS+VEN+AZA triplet, and the early data reveal promising clinical safety and antileukemic activity.

To date, four newly diagnosed AML patients have received the lowest dose of TUS (40 mg) as part of the (TUS+VEN+AZA) combination.
Three patients with unmutated (wildtype) FLT3 (FLT3-WT) completed Cycle 1 of treatment with no dose-limiting toxicities (DLTs) and no dose adjustments.
Two FLT3-WT patients achieved complete remissions (CR and CRh) by the end of Cycle 1.
Notably, a patient with biallelic TP53 mutations and a complex karyotype obtained CR.
The third FLT3-WT patient experienced significant reductions in bone marrow leukemic blasts during Cycle 1 and remains on therapy in Cycle 2.
The fourth patient, harboring FLT3-ITD and NPM1 mutations, is currently dosing in Cycle 1 and is not yet eligible for response evaluation
Pharmacokinetic (PK) analyses for TUS show plasma levels unaffected by the addition of AZA, providing predictability and avoiding the need for dose alterations due to PK interactions.
"These are very promising early results from the TUSCANY trial of TUS+VEN+AZA and the first indicators of the safety and efficacy we expected to see in newly diagnosed AML patients," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "To achieve a complete remission (CR) in Cycle 1 in a subject harboring a TP53 mutation – one of the most adverse forms of AML – is particularly encouraging. With enrollment ongoing in the TUSCANY study, we look forward to reporting additional data as it becomes available."

"TUS+VEN+AZA triplet therapy has the potential to treat large AML patient populations, including those with traditionally difficult-to-treat mutations, and improve patient outcomes right from the outset of treatment," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. The ability to treat such diverse AML populations – including FLT3 wildtype patients – with a favorable safety profile and without having to alter the standard of care dosing, differentiates our drug from many AML drugs in development."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study
Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Responses to TUS were also observed in those with prior-VEN and prior-FLT3 inhibitor (FLT3i) therapies, those with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS will be administered in 28-day cycles, beginning at 40mg once daily, with dose escalations planned after a safety review of each dose level. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov.

Alkermes plc Reports Financial Results for the Fourth Quarter and Year Ended Dec. 31, 2024 and Provides Financial Expectations for 2025

On February 12, 2025 Alkermes plc (Nasdaq: ALKS) reported financial results for the quarter and year ended Dec. 31, 2024 and provided financial expectations for 2025 (Press release, Alkermes, FEB 12, 2025, View Source [SID1234650203]).

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"2024 marked the completion of a multi-year effort to transition the business into a highly profitable, pure-play neuroscience company. We enter 2025 with a diversified portfolio of proprietary commercial products generating substantial profitability and an advancing development pipeline that represents a significant value creation opportunity in one of the most exciting potential new therapeutic categories in neuroscience," said Richard Pops, Chief Executive Officer of Alkermes. "Looking ahead, we are well positioned to deliver on our financial goals and advance the development programs for our portfolio of orexin 2 receptor agonists. This year, we have clear objectives for our pipeline as we complete the phase 2 studies for ALKS 2680 in narcolepsy, with data expected in the second half of the year, and prepare to initiate the ALKS 2680 phase 2 study in idiopathic hypersomnia and advance ALKS 4510 and ALKS 7290 into planned phase 1 studies in disease areas beyond central disorders of hypersomnolence. Each of these initiatives is an important element of our strategy to unlock what we believe is a multi-billion-dollar market opportunity for this category."

"2024 was Alkermes’ strongest year of financial and operational performance to date. Financially, we generated more than $1 billion in revenue from our proprietary commercial product portfolio, delivered EBITDA from continuing operations of approximately $452 million, repurchased $200 million of the company’s ordinary shares, retired approximately $290 million of debt and ended the year debt-free with approximately $825 million of cash and investments on the balance sheet. Operationally, we completed the sale of our manufacturing business in Ireland and made significant progress advancing our neuroscience development pipeline," said Blair Jackson, Chief Operating Officer of Alkermes. "We will continue to manage the business with a sharp focus on efficiency and profitability as we invest in the programs that we believe will drive the company’s next phase of growth."

Key Financial Highlights

Revenues

(In millions)

Three Months Ended
December 31,

Twelve Months Ended
December 31,

2024

2023

2024

2023

Total Revenues

$

430.0

$

377.5

$

1,557.6

$

1,663.4*

Total Proprietary Net Sales

$

307.7

$

242.0

$

1,083.5

$

920.0

VIVITROL

$

134.1

$

102.4

$

457.3

$

400.4

ARISTADAi

$

96.6

$

83.4

$

346.2

$

327.7

LYBALVI

$

77.0

$

56.2

$

280.0

$

191.9

Profitability

(In millions)

Three Months Ended
December 31,

Twelve Months Ended
December 31,

2024

2023

2024

2023*

GAAP Net Income From Continuing Operations

$

145.7

$

160.6

$

372.1

$

519.2

GAAP Net Income (Loss) From Discontinued Operations

$

0.8

$

(47.8)

$

(5.1)

$

(163.4)

GAAP Net Income

$

146.5

$

112.8

$

367.1

$

355.8

Non-GAAP Net Income From Continuing Operations

$

173.4

$

81.8

$

494.4

$

396.5

Non-GAAP Net Income (Loss) From Discontinued Operations

$

0.8

$

(44.4)

$

(5.1)

$

(152.9)

Non-GAAP Net Income

$

174.2

$

37.4

$

489.3

$

243.7

EBITDA From Continuing Operations

$

170.0

$

72.8

$

452.4

$

486.3

EBITDA From Discontinued Operations

$

1.1

$

(40.5)

$

(5.8)

$

(162.5)

EBITDA

$

171.1

$

32.3

$

446.6

$

323.8

*As a result of the successful resolution of the arbitration with Janssen Pharmaceutica N.V., the twelve months ended December 31, 2023 included approximately $195.4 million of back royalties (and related interest) related to U.S. net sales of long-acting INVEGA products that would ordinarily have been recognized in prior periods.

Revenue Highlights

LYBALVI

Revenues for the fourth quarter were $77.0 million.
Fourth quarter revenues and total prescriptions grew 37% and 30%, respectively, compared to the fourth quarter of 2023.
During the quarter, the company recorded LYBALVI revenue of approximately $4 million related to year-end inventory fluctuations.
ARISTADAi

Revenues for the fourth quarter were $96.6 million.
Fourth quarter revenues grew 16% compared to the fourth quarter of 2023.
During the quarter, the company recorded ARISTADA revenue of approximately $9 million related to year-end inventory fluctuations and gross-to-net favorability, primarily driven by Medicaid utilization adjustments.
VIVITROL

Revenues for the fourth quarter were $134.1 million.
Fourth quarter revenues grew 31% compared to the fourth quarter of 2023.
During the quarter, the company recorded VIVITROL revenue of approximately $23 million related to year-end inventory fluctuations and gross-to-net favorability, primarily driven by Medicaid utilization adjustments.
Manufacturing & Royalty Revenues

Royalty revenues from XEPLION, INVEGA TRINZA/TREVICTA and INVEGA HAFYERA/BYANNLI for the fourth quarter were $36.5 million.
VUMERITY manufacturing and royalty revenues for the fourth quarter were $35.0 million.
FAMPYRA manufacturing and royalty revenues for the fourth quarter were $22.9 million. The company does not expect to record any FAMPYRA revenue going forward.
RISPERDAL CONSTA manufacturing revenues for the fourth quarter were $14.7 million.
Key Operating Expenses

Please see Note 1 below for details regarding discontinued operations.

(In millions)

Three Months Ended
December 31,

Twelve Months Ended
December 31,

2024

2023

2024

2023

R&D Expense – Continuing Operations

$

58.2

$

73.9

$

245.3

$

270.8

R&D Expense – Discontinued Operations

$

(1.1)

$

21.5

$

5.8

$

116.2

SG&A Expense – Continuing Operations

$

147.0

$

169.8

$

645.2

$

689.8

SG&A Expense – Discontinued Operations

$

$

19.4

$

$

48.6

Balance Sheet

At Dec. 31, 2024, the company recorded cash, cash equivalents and total investments of $824.8 million, compared to $813.4 million at Dec. 31, 2023.
In December 2024, the company prepaid and retired in full all of its outstanding long-term debt in the amount of approximately $290 million.
Financial Expectations for 2025

All line items are according to GAAP, except as otherwise noted.

In millions

2025 Expectations

Total Revenues

$1,340 – $1,430

VIVITROL Net Sales

$440 – $460

ARISTADAi Net Sales

$335 – $355

LYBALVI Net Sales

$320 – $340

Cost of Goods Sold

$185 – $205

R&D Expenses

$305 – $335

SG&A Expenses

$655 – $685

GAAP Net Income a

$175 – $205

EBITDA

$215 – $245

Adjusted EBITDA

$310 – $340

Effective Tax Rate

~17%

a Expected 2025 weighted average basic share count of approximately 165.5 million shares outstanding and a weighted average diluted share count of approximately 169.5 million shares outstanding.

Notes and Explanations

1. The company determined that upon the separation of its former oncology business, completed on Nov. 15, 2023, the oncology business met the criteria for discontinued operations in accordance with Financial Accounting Standards Board Accounting Standards Codification 205, Discontinued Operations. Accordingly, the accompanying selected financial information has been updated to present the results of the oncology business as discontinued operations for the three and twelve months ended Dec. 31, 2023.

Conference Call
Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. EST (1:00 p.m. GMT) on Wednesday, Feb. 12, 2025, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes’ website.