On October 27, 2025 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported its participation in the upcoming investor relations events.

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Citi’s 2025 SMID Cap Biopharma Call Series
Thursday, November 6th, 2025 at 12:00 PM ET

Fireside chat with Yujiro S. Hata, President and Chief Executive Officer, hosted by Yigal D. Nochomovitz, Ph.D., Director, SMid Cap Biotech Analyst

Jefferies Global Healthcare Conference in London
Tuesday, November 18th, 2025 at 1:00 PM GMT | 8:00 AM ET

Fireside chat with Yujiro S. Hata, Chief Executive Officer, hosted by Maury Raycroft, Ph.D. Equity Research Analyst, Biotechnology
A live audio webcast of the conference events, as permitted by the conference host, will be available at the "Investors/Events" section of the IDEAYA website at View Source and/or through the conference host. A replay of available webcasts will be accessible for 30 days following the live event.

(Press release, Ideaya Biosciences, OCT 27, 2025, View Source [SID1234657046])

On October 27, 2025 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the third quarter ended September 30, 2025.

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"We are pleased with the contributions from our Enzyme Therapies and Skeletal Conditions business units to date this year driven by more than 20% revenue growth from PALYNZIQ and VOXZOGO," said Alexander Hardy, President and Chief Executive Officer of BioMarin. Our strategic investments in these focused business units are generating strong results, and we anticipate sustained financial performance from each of them. Both Enzyme Therapies and Skeletal Conditions remain central to our growth strategy, in addition to new business development opportunities and our advancing internal pipeline. As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest ROCTAVIAN and remove it from our portfolio. We continue to believe ROCTAVIAN has an important role to play in the treatment of hemophilia A and are therefore evaluating out-licensing options for this innovative gene therapy. This decision is consistent with BioMarin’s portfolio strategy and offers the most promising opportunity for ensuring continued patient access to ROCTAVIAN.

Mr. Hardy concluded, "Looking ahead, we will rely on our disciplined strategic focus and proven capabilities to develop and commercialize innovative therapies that generate sustainable value for patients, employees, and shareholders."

Third Quarter 2025 Financial Highlights
•Total Revenues for the third quarter of 2025 were $776 million, an increase of 4% compared to the same period in 2024, driven by strong revenue growth in VOXZOGO and PALYNZIQ attributable to new patients initiating therapy across all regions. These increases were partially offset by lower sales volume for ALDURAZYME due to timing of order fulfillment to Sanofi and NAGLAZYME due to timing of large government orders in Latin America.

•GAAP Net Loss increased to $31 million for the third quarter of 2025 compared to GAAP Net Income of $106 million for the same period in 2024. The increase in GAAP Net Loss was primarily due to the In-Process Research & Development (IPR&D) charge of $221 million recorded in connection with the acquisition of Inozyme Pharma, Inc. (Inozyme). This increase in GAAP Net Loss was partially offset by improved gross profit as a result of revenue growth mentioned above, lower Cost of Sales from favorable product mix during the quarter, and lower provision for income taxes.

•Non-GAAP Income for the third quarter of 2025 decreased to $22 million compared to $178 million for the same period in 2024. The decrease in Non-GAAP Income was primarily due to the factors noted above.
Third Quarter 2025 Business Highlights
Innovation
•Skeletal Conditions: At the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, investigators shared new data demonstrating improved spinal morphology – one of the factors that contributes to spinal stenosis, a leading cause of morbidity in achondroplasia – following treatment with VOXZOGO in children ages 5 and under (see PR here). VOXZOGO is the only approved therapy with data showing a positive impact on spinal morphology, and these findings add to the extensive body of evidence supporting VOXZOGO’s health benefits beyond improving growth.

•As a follow-up to the encouraging PK data announced last quarter for BMN 333, BioMarin’s long-acting C-type natriuretic peptide, the company plans to initiate dosing of children with achondroplasia in its registration-enabling Phase 2/3 study in the first half of 2026.

•VOXZOGO pivotal data for the treatment of hypochondroplasia is expected in the first half of 2026, followed by potential launch in 2027, should data be supportive. Hypochondroplasia can be associated with significant co-morbidities, including respiratory, orthopedic, mental health, and ear nose and throat complications, representing high unmet medical need for this skeletal condition with no approved therapies.

•Four new VOXZOGO indications are under development as part of BioMarin’s CANOPY clinical program, with a focus on the most severely impacted sub-set of children. These conditions include idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency, and are currently enrolling patients, with potential registration-enabling studies in 2027.

•Enzyme Therapies: Based on new data from the PALYNZIQ Phase 3 PEGASUS study in 12- to 17-year-olds demonstrating statistically significant blood phenylalanine (Phe) lowering compared to diet alone, the company is pursuing approvals in this age group in the United States and Europe, with potential approval in 2026.

•With BMN 401, a potential first-in-disease treatment for ENPP1 deficiency, initial pivotal data readout for the ENERGY 3 study in children ages 1–12 years is anticipated in the first half of 2026, with potential launch in 2027.

•Other Clinical Updates: For BMN 351, BioMarin’s next generation oligonucleotide for Duchenne muscular dystrophy, the company expects to share a clinical update for the 6 mg/kg and 9 mg/kg cohorts by year-end.

Growth

•As of the end of the quarter, children with achondroplasia in 55 countries around the world were being treated with VOXZOGO, tracking to the company’s plan to open access in more than 60 countries by 2027. Year-to-date VOXZOGO revenue increased 24% Y/Y, with Q4’25 VOXZOGO revenue expected to reach its highest level of the year. BioMarin reaffirmed full-year 2025 VOXZOGO revenue outlook of between $900 million and $935 million.

•Representing approximately 75% of total VOXZOGO revenue, markets outside of the U.S. (OUS) benefited from BioMarin’s established global footprint to drive VOXZOGO uptake across key large markets during the quarter.

•Initiatives implemented to expand treatment with VOXZOGO in the U.S. resulted in new patient starts across all ages in the third quarter, with the majority from children under 2 years of age. Due to the geographical dispersion and management across a range of specialties for older children in the U.S, the company has implemented initiatives to address slowing uptake in that demographic.

•PALYNZIQ marked its third consecutive quarter of 20%+ Y/Y growth. PALYNZIQ strength continued to be driven by greater numbers of patients titrating to daily maintenance dose and strong adherence. Total Enzyme Therapies revenue grew 8% Y/Y, year-to-date, reflecting high penetration rates and strong adherence to these treatments.

•Today, the company announced its plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. BioMarin plans to continue to make ROCTAVIAN commercially available in the U.S., Italy and Germany until next steps are finalized. The company will continue to provide support and monitoring for people treated with ROCTAVIAN.

Value Commitment

•Acquired IPR&D charges in Q3 from BioMarin’s acquisition of Inozyme resulted in Y/Y increases in GAAP and Non-GAAP R&D expenses. Q3 GAAP and Non-GAAP SG&A expenses increased Y/Y due to investment in business unit expansion initiatives. Year-to-date GAAP Diluted EPS and Non-GAAP Diluted EPS increased Y/Y, driven by underlying strong revenue performance and operational efficiencies.

•The company generated operating cash flows totaling $369 million in third quarter 2025 and generated $728 million in year-to-date operating cash flows. Total cash and investments at the end of the third quarter were approximately $2.0 billion, and increasing operating cash flow is expected to continue, supporting BioMarin’s priority of investment in innovation and future growth.

•Today, BioMarin increased total revenue guidance, at the midpoint, reflecting strong demand for its therapies through 2025. Revised Non-GAAP Operating Margin and Non-GAAP Diluted EPS guidance include the impact of Q3 acquired IPR&D expenses, partially offset by underlying strong topline performance and operational execution throughout the year.

Refer to the 2025 Full-Year Financial Guidance on page 4 of this press release.
Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)
Three Months Ended
September 30, Nine Months Ended
September 30,
2025 2024 % Change 2025 2024 % Change
Total Revenues $776 $746 4% $2,347 $2,107 11%
Net Product Revenues by Product:
VOXZOGO $218 $190 15% $654 $527 24%
Enzyme Therapies:
VIMIZIM $183 $178 3% $587 $549 7%
NAGLAZYME 122 132 (8)% 365 370 (1)%
PALYNZIQ 109 91 20% 308 255 21%
ALDURAZYME 54 71 (24)% 159 145 10%
BRINEURA 48 37 30% 137 121 13%
Total Enzyme Therapies Revenue $516 $509 1% $1,556 $1,440 8%
KUVAN $24 $28 (14)% $76 $93 (18)%
ROCTAVIAN
$3 $7 (57)% $23 $16 44%
GAAP Net Income (Loss)(1)
$(31) $106 (129)% $395 $302 31%
Non-GAAP Income (1)(2)
$22 $178 (88)% $525 $506 4%
GAAP Operating Margin % (1)(3)
(6.0)% 15.3% 19.4% 15.3%
Non-GAAP Operating Margin % (1)(2)
2.8% 27.7% 26.3% 27.7%
GAAP Diluted Earnings (Loss) per Share (EPS)(1)
$(0.16) $0.55 (129)% $2.04 $1.56 31%
Non-GAAP Diluted EPS (1)(2)
$0.12 $0.91 (87)% $2.69 $2.60 3%

September 30,
2025 December 31,
2024
Total cash, cash equivalents & investments $ 1,991 $ 1,659

(Press release, BioMarin, OCT 27, 2025, View Source [SID1234657031])

On October 27, 2025 Tolerance Bio, a biopharmaceutical company pioneering innovative approaches to extending healthspan by preserving, restoring, and manipulating the function of the thymus, the master regulator of immune tolerance, reported the publication of a scientific article titled "Patient-specific autologous thymic organoids support functional T-cell education leading to antitumor activity in humanized mice with melanoma xenografts" in Cancer Research Communications, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper). The research was led by Antonio Jimeno, M.D., Ph.D., from the Division of Medical Oncology, Department of Medicine, University of Colorado Anschutz School of Medicine, and Holger A. Russ, Ph.D., from the Diabetes Institute and the Department of Pharmacology and Therapeutics, College of Medicine at the University of Florida, Gainesville, and scientific co-founder of Tolerance Bio.

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The contributing teams utilized a novel approach, implanting patient-derived thymic cells generated via direct differentiation of induced pluripotent stem cells (iPSCs) into thymectomized and humanized immune-deficient mice, along with matched tumor tissues from a patient with metastatic melanoma. The key finding was that tumors implanted in mice with human thymic tissue grew significantly slower compared to tumors implanted in control and in humanized mice without thymic tissue. Viable melanoma content was also significantly decreased in mice bearing thymic organoids, associated with an increase in intra-tumoral activated T-cells. Whole gene sequencing identified multiple candidate neoantigens that were eliminated in cancer cells in thymus-bearing mice, suggesting a more effective identification and T-cell-driven tumor clearance.

"An experiment is only as good as the models used to conduct it. Over the last 15 years, we have generated increasingly complex laboratory and mouse models that can bear both human immune and cancer cells, which we call humanized mice, to realize the promise of immune therapy. A fundamental limitation of humanized mice, generated with donor blood stem cells and implanted with mismatched patient tumors, is that the fidelity of the interaction between the immune and tumor cells is limited by lack of the organ that facilitates immune cell education, the thymus," said Dr. Jimeno.

"Here we report the successful generation of a novel model that incorporates functional thymic organoids derived from iPSC generated using patient peripheral blood with tumors from the same patient, providing an important step towards improved personalized models," remarked Dr. Russ.

"These results represent meaningful advance in oncology, both from a therapeutic and a diagnostic point of view, as iPSC thymic cells have the potential to serve as a cell therapy for cancer, alone or along immune agents, the patient derived xenograft model can improve new drug development by more accurately identifying promising drugs and vaccines, and it will allow for personalized medicine by testing which immune therapy is more likely to work for a specific patient," added Dr. Jimeno. "We have a lot of work ahead, but I am looking forward to extending our interdisciplinary work into the future to realize this potential."

"Tolerance Bio is proud to collaborate with Dr. Jimeno and Dr. Russ, and their superb teams at U. Colorado and U. Florida. Their work lays the foundation for the use of iPSC thymic cells in immune diseases, including cancer," said Dr. Francisco Leon, M.D., Ph. D., CEO of Tolerance Bio. "The changes in the tumor growth kinetics provide critical proof of principle for the ability of iPSC thymic cells to reconstitute a T cell immune system in vivo with beneficial functional consequences. As we advance towards the clinic, we have developed a robust manufacturing process for our allogenic, off-the-shelf iPSC thymic cell product, and we plan to initiate pre-investigational new drug (IND) studies in the near future."

(Press release, Tolerance Bio, OCT 27, 2025, View Source [SID1234657047])

On October 27, 2025 Evaxion A/S (NASDAQ: EVAX) ("Evaxion"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported it has appointed Dr Helen Tayton-Martin as new Chief Executive Officer (CEO). She will take up her new position on November 24, 2025, and simultaneously step down from Evaxion’s Board of Directors (Board).

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Holding a Ph.D. in molecular immunology, and an MBA from London Business School, Helen Tayton-Martin was a co-founder of cancer-focused biotech company Adaptimmune, where she served as Chief Operating Officer and later Chief Business & Strategy Officer during her 17 years with the company. Through this time, she oversaw transatlantic growth, multiple clinical, academic and commercial collaborations and private and public financing through to its Nasdaq IPO. Dr. Tayton-Martin was responsible for all Adaptimmune’s strategic partnerships including those with Astellas, Genentech, a member of the Roche Group, GlaxoSmithKline and Galapagos NV.

Prior to Adaptimmune, Dr. Tayton Martin spent 15 years working within the pharma, biotech and consulting environments for various companies. She also previously served as a non-executive director of Trillium Therapeutics Inc. from October 2017 through to the sale of the company in November 2021 to Pfizer Inc.

"I am delighted that Helen Tayton-Martin is taking over the CEO position. She is a skilled executive with exactly the right experience in leading an organization, making partnerships and monetizing assets in biotech. Helen knows Evaxion from her time on the Board and we have seen her leadership quality and have already established a working relationship. It is with great faith that we welcome her to a new role in the company" says Marianne Søgaard, Chairman of the Board at Evaxion.

"I also want to extend my thanks to Birgitte Rønø, who has done a fantastic job as interim CEO in a crucial time for Evaxion, on top of her duties as Chief Scientific Officer. Birgitte will now again focus on this role, and we look forward to continuing to benefit from her capabilities and experience as she leads our research and development work," says Marianne Søgaard.

"I am very excited to be joining Evaxion as CEO at an incredibly exciting time for the company. I have seen first-hand from my time on the Company’s Board the strength of its AI-Immunology platform to deliver real potential products, exemplified with the recent out-licensing of EVX-B3 to MSD and the presentation of unprecedented data for EVX-01 at ESMO (Free ESMO Whitepaper), demonstrating Evaxion is in a very strong position for further future value generation. I am looking forward to working with the highly skilled and committed team of employees to build from this extraordinary scientific, medical and commercial potential of Evaxion," says Helen Tayton-Martin.

Changes to the Board of Evaxion
Helen Tayton-Martin will step down from the Board of Evaxion when taking up the CEO position. Seasoned life science executive Jens Bitsch-Norhave will join the Board of Directors as adviser and observer with the intention of seeking election as board member at the Annual General Meeting in 2026.

"We are looking forward to welcoming Jens Bitsch-Norhave to Evaxion. With more than 25 years of leadership experience in the biotech and pharmaceutical industry, specializing in corporate strategy, global expansion, and dealmaking, Jens will be a strong addition to the Board", says Marianne Søgaard.

Jens Bitsch-Norhave is currently Corporate Vice President and Global Head of Corporate Development at Hengrui Pharmaceuticals, where he is responsible for strategy, business development, licensing, M&A, and alliance management, advancing the company’s globalization and growth. He holds an MSc and Ph.D. in neuropharmacology from the University of Copenhagen and an Executive MBA in Technology & Innovation from Copenhagen Business School.

(Press release, Evaxion Biotech, OCT 27, 2025, View Source [SID1234657032])

On October 27, 2025 OncoNano Medicine, Inc. ("OncoNano") reported encouraging first-in-human results from Part 1 of its Phase 1 ON-5001 trial evaluating ONM-501, a dual-acting STING agonist, as monotherapy and in combination with Libtayo (cemiplimab), Regeneron’s PD-1 inhibitor, in patients with advanced solid tumors and lymphomas. The findings were shared during a poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, Massachusetts.

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ONM-501 is designed to activate the STING pathway and stimulate both innate and adaptive immune responses within the tumor microenvironment. By linking a cyclic dinucleotide (cGAMP) to a proprietary polymer, ONM-501 prolongs STING activation and overcomes key limitations of earlier STING agonists. In preclinical models, this approach produced durable anti-tumor responses without triggering systemic inflammation, an effect now supported by emerging clinical results.

In the dose-escalation and dose-finding study, ONM-501 was well tolerated and demonstrated early signs of clinical benefit. Among 39 patients treated (15 with ONM-501 monotherapy and 24 in combination with cemiplimab), no dose-limiting toxicities were observed, and the most common adverse events were mild fatigue and injection-site reactions. Systemic interferon-γ levels remained within normal limits across all cohorts.

In the monotherapy arm, one patient achieved an objective response and three patients had prolonged stable disease, including a case of recurrent, immunotherapy-refractory uveal melanoma with disease control lasting over one year (390 days). In the combination arm, five patients experienced objective responses, including two complete in patients with cutaneous squamous cell carcinoma (cSCC).

"These data provide the first clinical evidence that sustained STING activation through our proprietary polymer conjugate can drive meaningful immune responses in solid tumors," said Kartik Krishnan, MD, PhD, Chief Executive Officer of OncoNano Medicine. "The safety profile and emerging efficacy signals, particularly in patients with advanced cutaneous malignancies, highlight the potential of ONM-501 to expand the reach of STING-based immunotherapy."

Part 2 of the ON-5001 study (NCT06022029) is now open and enrolling patients with advanced basal cell carcinoma, cutaneous squamous cell carcinoma and melanoma.

About ONM-501
ONM-501 is a dual-activating agonist of the stimulator of interferon genes ("STING") pathway composed of cGAMP (the endogenous activator of STING) linked to a proprietary pH-activated polymer (the OMNI polymer). ONM-501 is presently being studied in a Phase 1 clinical trial (ON-5001). In preclinical models, the dual activation of STING by ONM-501 has been shown to lead to a direct anti-tumor effect, as well as leading to an anti-tumor immune response over an extended period of time. Development of ONM-501 was funded in part by the Cancer Prevention and Research Institute of Texas.

(Press release, OncoNano Medicine, OCT 27, 2025, View Source [SID1234657048])