Full Year and Q4 2024 results

On February 6, 2025 AstraZeneca reported full year and fourth quarter 2024 financial results (Press release, AstraZeneca, FEB 6, 2025, View Source [SID1234650236]).

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Neurocrine Biosciences Reports Fourth Quarter and Fiscal 2024 Financial Results and Provides Financial Expectations for 2025

On February 6, 2025 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the fourth quarter and full year ended December 31, 2024 and provided financial guidance for 2025 (Press release, Neurocrine Biosciences, FEB 6, 2025, View Source [SID1234650085]).

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"I’m proud of the tremendous progress we made last year with the continued growth of INGREZZA for patients living with tardive dyskinesia or Huntington disease chorea. With the approval and launch of CRENESSITY, we look forward to delivering the first new treatment for the congenital adrenal hyperplasia community in over 70 years, transforming the standard of care for patients," said Kyle W. Gano, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With a rapidly advancing and growing pipeline and a strong financial profile, we are well positioned to build a leading neuroscience company."

Financial Highlights

Three Months Ended

December 31,

Twelve Months Ended

December 31,

(unaudited, in millions, except per share data)

2024

2023

2024

2023

Revenues:

Net Product Sales

$ 621.2

$ 507.2

$ 2,330.6

$ 1,860.6

Collaboration Revenue

6.5

8.0

24.7

26.5

Total Revenues

$ 627.7

$ 515.2

$ 2,355.3

$ 1,887.1

GAAP Research and Development (R&D)

$ 185.6

$ 137.5

$ 731.1

$ 565.0

Non-GAAP R&D

$ 164.4

$ 124.3

$ 662.3

$ 497.0

GAAP Selling, General, and Administrative (SG&A)

$ 287.8

$ 218.9

$ 1,007.2

$ 887.6

Non-GAAP SG&A

$ 241.6

$ 194.0

$ 862.5

$ 757.4

GAAP Net Income

$ 103.1

$ 147.7

$ 341.3

$ 249.7

GAAP Earnings Per Share – Diluted

$ 1.00

$ 1.44

$ 3.29

$ 2.47

Non-GAAP Net Income

$ 173.4

$ 157.7

$ 656.3

$ 390.0

Non-GAAP Earnings Per Share – Diluted

$ 1.69

$ 1.54

$ 6.33

$ 3.86

(unaudited, in millions)

December 31,

2024

December 31,

2023

Total Cash, Cash Equivalents, and Marketable Securities

$ 1,815.6

$ 1,719.1

Net Product Sales Highlights

INGREZZA fourth quarter and fiscal 2024 net product sales were $615 million and $2.3 billion, respectively
INGREZZA fourth quarter net product sales grew 23% compared to fourth quarter 2023, driven by strong underlying patient demand and improvement in gross-to-net dynamics
CRENESSITY fourth quarter and fiscal 2024 net product sales were $2 million reflecting initial pharmacy orders following approval by the U.S. Food and Drug Administration (FDA) in December 2024
Other Key Financial Highlights

Differences in fourth quarter 2024 GAAP and Non-GAAP operating expenses compared with fourth quarter 2023 were driven by:
Increased R&D expense in support of an expanded and advancing portfolio including investments in osavampator in major depressive disorder, our muscarinic franchise and preclinical research and discovery activities.
Increased SG&A expense includes incremental investment in CRENESSITY-related headcount, CRENESSITY-related pre-launch activities, and continued investment in INGREZZA, including the recent expansion of our psychiatry and long-term care sales teams in September 2024.
Increased stock-based compensation expense (GAAP) of $28 million primarily related to performance-based awards achievement associated with CRENESSITY FDA approval and a charge associated with the retirement of our CEO in October 2024.
Fourth quarter 2024 GAAP net income and earnings per share were $103 million and $1.00, respectively, compared with $148 million and $1.44, respectively, for fourth quarter 2023
Fourth quarter 2024 Non-GAAP net income and earnings per share were $173 million and $1.69, respectively, compared with $158 million and $1.54, respectively, for fourth quarter 2023
Differences in fourth quarter 2024 GAAP and Non-GAAP net income compared with fourth quarter 2023 driven by:
Higher INGREZZA net sales
Increased operating expenses to support expanding and advancing R&D portfolio, incremental investments for CRENESSITY pre-launch activities and INGREZZA recent sales force expansion
Fourth quarter 2024 includes $66 million of stock-based compensation expense compared with $38 million for fourth quarter 2023 (Non-GAAP adjustment)
Fourth quarter 2024 includes a $2 million loss from changes in fair values of equity investments compared with a $29 million gain for fourth quarter 2023 (Non-GAAP adjustment)
As of December 31, 2024, repurchased and retired approximately 2.0 million shares of the Company’s common stock valued at approximately $240.5 million pursuant to a previously announced $300 million accelerated share repurchase (ASR) program. The $300 million program was completed in early February 2025 repurchasing and retiring approximately 2.3 million shares.
At December 31, 2024, the Company had cash, cash equivalents and marketable securities totaling approximately $1.8 billion
A reconciliation of GAAP to Non-GAAP financial results can be found in Table 3 and Table 4 at the end of this news release.
Recent Developments

CRENESSITY was approved in December 2024 by the FDA as an adjunctive treatment to glucocorticoid replacement to control androgens in adult and pediatric patients four years of age and older with classic congenital adrenal hyperplasia (CAH).
Announced the initiation of the Phase 3 program for osavampator (formerly NBI-1065845 / TAK-653), a potential first-in-class AMPA positive allosteric modulator in development for patients with inadequate response to treatment of major depressive disorder (MDD).
Announced amendment to strategic collaboration with Takeda to develop and commercialize osavampator. Under the amended agreement, Neurocrine will obtain exclusive rights for all indications to develop and commercialize osavampator in all territories worldwide except Japan, where Takeda will acquire exclusive rights. Under the terms of the updated agreement, each company is responsible for development costs in their respective region, and both companies are eligible to receive royalty payments.
Received Centers for Medicare and Medicaid Services (CMS) notification in January that INGREZZA qualifies for the small biotech exemption under the Medicare Drug Price Negotiation Program.
Announced the initiation of its Phase 1 clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of investigational compound NBI-921355 in healthy adult participants. NBI-921355 is an investigational, selective inhibitor of voltage-gated sodium channels Nav1.2 and Nav1.6 and in development for the potential treatment of certain types of epilepsy.
Presented subgroup analyses and data from the KINECT-HD study showing the impact of INGREZZA capsules on emotional health and psychiatric stability in patients with chorea associated with Huntington’s disease. The subgroup analysis showed consistent efficacy in reducing chorea compared to placebo across all identified subgroups, categorized by demographics and baseline assessment scores. A separate data analysis showed improvements in some aspects of emotional health with no worsening of psychiatric symptoms.
Presented data from more than 300 patients diagnosed with tardive dyskinesia and treated with INGREZZA capsules. These data showed significant improvements in functional, social, emotional and health-related quality of life measures in Phase 3 and 4 studies and improvements in functional, social, independence, emotional and physical aspects of patients’ lives and antipsychotic adherence in real-world practice.
Full Year 2025 Financial Guidance

Range

(in millions)

Low

High

INGREZZA Net Product Sales 1

$ 2,500

$ 2,600

GAAP R&D Expense 2

$ 960

$ 1,010

Non-GAAP R&D Expense 2, 3

$ 890

$ 940

GAAP SG&A Expense 4

$ 1,110

$ 1,130

Non-GAAP SG&A Expense 3, 4

$ 955

$ 975

1.

INGREZZA sales guidance reflects expected net product sales of INGREZZA in tardive dyskinesia and chorea associated with Huntington’s disease.

2.

R&D guidance reflects the continued advancement of our pre-clinical and clinical portfolio including the initiation of our Phase 3 programs for osavampator in MDD and NBI-568 in schizophrenia. R&D guidance includes $60 million of expense for development milestones primarily in connection with our collaborations with Takeda and Nxera achieved or deemed probable to achieve. Acquired in-process research and development expense is included in guidance once significant collaboration and licensing arrangements have been completed.

3.

Non-GAAP guidance adjusted to exclude estimated non-cash stock-based compensation expense of $70 million in R&D and $130 million in SG&A and vacated legacy campus facility costs.

4.

SG&A guidance range reflects expense for ongoing commercial initiatives supporting INGREZZA growth and the launch of CRENESSITY.

Conference Call and Webcast Today at 4:30 PM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). Participants can access the live conference call by dialing 800-225-9448 (US) or 203-518-9708 (International) using the conference ID: NBIX. The webcast and accompanying slides can also be accessed at approximately 4:30 p.m. Eastern Time on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

Full Year and Q4 2024 Results

On February 6, 2025 AstraZeneca reported full Year and fourth quarter 2024 Results (Presentation, AstraZeneca, FEB 6, 2025, View Source [SID1234650276]).

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Rakovina Therapeutics Announces Additional Synthesized Compounds in its AI-Driven Cancer Drug Discovery Program

On February 6, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO), a biopharmaceutical company advancing cancer therapies based on DNA-damage response technologies, reported continued advancement of its AI-driven drug discovery program (Press release, Rakovina Therapeutics, FEB 6, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-announces-additional-synthesized-compounds-in-its-ai-driven-cancer-drug-discovery-program [SID1234650087]). Over the next four months, additional small-molecule drug candidates, designed in collaboration with Pharma Inventor Inc., will be synthesized and delivered for further evaluation.

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The expanded program builds on the success of the first batch of AI-designed PARP1-selective inhibitors announced earlier this year. These new compounds are being tailored to optimize efficacy, safety, and pharmacokinetic profiles, targeting critical unmet needs in oncology, including central nervous system (CNS) malignancies.

Separately, Rakovina Therapeutics expects to receive the first synthesized drug candidates from its second AI-driven drug discovery program targeting ATR.

AI-derived drug candidates are first validated in silico before being synthesized and evaluated for in vitro and in vivo activity against a specific target product profile in the Company’s laboratories at the University of British Columbia. Rakovina Therapeutics plans to advance the most promising lead candidates into human clinical trials in collaboration with pharmaceutical industry partners.

Leveraging AI for Revolutionary Advances in Cancer Treatment
The potential of artificial intelligence to accelerate medical breakthroughs has never been more apparent. Recent comments from leaders in AI and healthcare highlight the transformative role this technology will play in curing diseases at an unprecedented pace. Sam Altman, CEO of OpenAI, recently stated, "We will see diseases get cured at an unprecedented rate. We will be amazed at how quickly we’re curing this cancer and that one — and heart disease. AI will cure the diseases at a rapid, rapid rate."

Similarly, Oracle CEO Larry Ellison emphasized AI’s ability to revolutionize early cancer detection and personalized treatments, stating, "If you can do it using AI, you can do early cancer detection with a blood test… Once we gene sequence that cancer tumor, you can then vaccinate the person — design a vaccine for every individual person… in about 48 hours."

These bold predictions align with Rakovina’s approach to combine cutting-edge AI with world-class scientific expertise. By leveraging AI platforms like Deep Docking and Enki,

Rakovina Therapeutics is poised to contribute to this new era of rapid medical advancements, accelerating the development of innovative cancer therapies.

Advancing Innovation and Expanding Potential
Each set of compounds is being developed to address specific therapeutic gaps, including the treatment of cancers that involve the brain, such as BRCA-mutated metastatic breast cancer and glioblastoma. Leveraging its collaborations for utilizing the Deep Docking and Enki systems, Rakovina continues to employ cutting-edge artificial intelligence to accelerate drug discovery and bring meaningful therapies closer to patients in need.

"This expansion demonstrates the scalability and impact of our AI platform partnership," said Prof. Mads Daugaard, Chief Scientific Officer. "With additional compounds in the pipeline, we are dramatically increasing the potential for identifying novel therapeutics to tackle some of the most challenging cancers. This ongoing development exemplifies our commitment to innovation and efficiency in cancer research."

Jeffrey Bacha, Executive Chairman, added, "The addition of these AI-designed compounds significantly broadens our pipeline potential and will accelerate progress toward delivering life-changing treatments. By rapidly scaling our efforts, we are addressing the growing demand for innovative cancer therapies with unprecedented precision and speed."

Allarity Therapeutics Announces Expansion of Phase 2 Clinical Trial to Accelerate Development of Stenoparib in Advanced Ovarian Cancer

On February 6, 2025 Allarity Therapeutics, Inc. ("Allarity" or the "Company") (NASDAQ: ALLR), a Phase 2 clinical-stage pharmaceutical company dedicated to developing stenoparib—a differentiated dual PARP/Wnt pathway inhibitor—as a personalized cancer treatment using its proprietary, drug-specific patient selection technology—the Drug Response Predictor (DRP)—reported plans for the next step in advancing the clinical development of stenoparib toward FDA approval in advanced ovarian cancer (Press release, Allarity Therapeutics, FEB 6, 2025, View Source [SID1234650088]). With this new phase 2 protocol, Allarity will capitalize and expand on the ongoing phase 2 clinical trial data to optimize the dose of stenoparib and to refine the DRP patient selection criteria in order to maximize the clinical benefit from stenoparib.

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An in-depth review of Allarity’s maturing clinical data, in collaboration with leading clinical investigators, prompted Allarity to design this new Phase 2 protocol to enable a more aggressive push of stenoparib toward regulatory approval. The protocol will enable new enrollment expressly focusing on patients with advanced, recurrent, platinum-resistant disease, a group that has shown durable clinical benefit from stenoparib in the current phase 2 trial and who have only limited treatment options following standard chemotherapy. In addition to expanding and deepening the clinical data set necessary for regulatory approval, this trial is also designed to enhance the understanding of stenoparib’s unique therapeutic mechanism of action. Of particular interest will be the impact of stenoparib treatment on the Wnt pathway, a key cellular pathway repeatedly shown to be involved in advanced ovarian cancers as well as many other advanced cancer types, most notably colon cancers. A richer understanding of stenoparib’s potential for controlling the Wnt pathway—a key aspect of the newly designed trial—could further distinguish stenoparib as a unique investigational cancer treatment.

The protocol will also further refine Allarity’s understanding of the stenoparib-DRP for identifying patients most likely to benefit from stenoparib treatment. As part of the trial, a DRP score will be assessed for all enrolled patients to generate the deeper, more robust data set necessary to refine the DRP cut-off that would ring-fence patients most likely to benefit from stenoparib. These data will be fundamental for supporting any regulatory approval for the stenoparib-DRP as a companion diagnostic (CDx) specific to stenoparib.

The Company has previously announced long-lasting clinical benefit for advanced, recurrent ovarian cancer patients, with some of these patients continuing on therapy for more than 14 months. Based upon these encouraging clinical benefit data, Allarity intends to pursue an advantaged regulatory path toward approval to further enable stenoparib’s clinical development.

Patient enrollment is expected to begin in the first half of 2025, subject to final protocol review by regulatory authorities. Recruitment will commence at leading U.S. trial sites, with the potential inclusion of additional sites in the U.K. as warranted.

"Until now, patients receiving stenoparib have had diverse and often extensive treatment histories, with many being very heavily pretreated. Based on data we have gathered to date from our clinical trials, and through in-depth analysis and consultation with leading gynecologic oncologists, we have developed a new clinical trial protocol focused on a well-defined, commercially significant patient population in desperate need of newer, safer treatment options beyond more chemotherapy, which comes with well-documented side effects," said Thomas Jensen, CEO of Allarity Therapeutics. "We are therefore pleased to have submitted this trial design and are eager to begin patient enrollment as soon as we receive the final green light from the regulatory authorities, including the FDA."

About Stenoparib
Stenoparib is an orally available, small-molecule dual-targeted inhibitor of PARP1/2 and tankyrase 1/2. At present, tankyrases are attracting significant attention as emerging therapeutic targets for cancer, principally due to their role in regulating the Wnt signaling pathway. Aberrant Wnt/β-catenin signaling has been implicated in the development and progression of numerous cancers. By inhibiting PARP and blocking Wnt pathway activation, stenoparib’s unique therapeutic action shows potential as a promising therapeutic for many cancer types, including ovarian cancer. Allarity has secured exclusive global rights for the development and commercialization of stenoparib, which was originally developed by Eisai Co. Ltd. and was formerly known under the names E7449 and 2X-121.

About the Drug Response Predictor – DRP Companion Diagnostic
Allarity uses its drug-specific DRP to select those patients who, by the gene expression signature of their cancer, are found to have a high likelihood of benefiting from a specific drug. By screening patients before treatment, and only treating those patients with a sufficiently high, drug-specific DRP score, the therapeutic benefit rate may be significantly increased. The DRP method builds on the comparison of sensitive vs. resistant human cancer cell lines, including transcriptomic information from cell lines, combined with clinical tumor biology filters and prior clinical trial outcomes. DRP is based on messenger RNA expression profiles from patient biopsies. The DRP platform has shown an ability to provide a statistically significant prediction of the clinical outcome from drug treatment in cancer patients in dozens of clinical studies (both retrospective and prospective). The DRP platform, which may be useful in all cancer types and is patented for more than 70 anti-cancer drugs, has been extensively published in the peer-reviewed literature.