FGFR3

Investigator-Sponsored Phase 2 Study Demonstrated Positive Results for VOXZOGO in Children with Hypochondroplasia New Analyses Highlight VOXZOGO 7-year Data and Improvement on Quality of Life in Children with Achondroplasia SAN RAFAEL, Calif., March 12, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024. Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate VOXZOGO's positive impact on quality of life. Results in Hypochondroplasia Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia. The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p<0.0001). No new safety signals were reported. "We are encouraged by preliminary results from the first study evaluating VOXZOGO in children with hypochondroplasia, suggesting the medicine has the potential to impact growth outcomes for this group of patients," said Dr. Dauber, who is the lead study author and Chief of Endocrinology at Children's National Hospital in Washington, D.C. "There are currently no approved medicines for children with hypochondroplasia, so we look forward to gathering additional data to deepen our understanding of VOXZOGO in hypochondroplasia and other growth-related conditions." In late 2023, BioMarin launched the pivotal clinical trial program studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the company plans to enter the treatment phase (Phase 3 trial) later this year. New Data Highlights VOXZOGO Long-Term Efficacy, Safety and Quality of Life in Achondroplasia 7-year Phase 2 Results in Achondroplasia Data from two studies of VOXZOGO in children ≥5 years of age with achondroplasia demonstrated a sustained improvement in AGV for each year that the participants were treated. In the Phase 2 study with more than 7 years of follow up, the mean increase in growth across each year of age up to 16 years compared with untreated participants was 1.63 cm/year for boys and 1.33 cm/year for girls. Separate cross-sectional comparative analysis assessing increase in height over a 7-year period showed an additional height gain of 11.03 cm with use of VOXZOGO compared to matched untreated children over the same period. 4-year Phase 3 Results in Achondroplasia The same analyses were produced for the Phase 3 trial in which children with achondroplasia have a mean treatment follow-up of 4 years. The mean increase in growth across each year of age up to 17 years was 1.73 cm/year for boys and 1.46 cm/year for girls. A separate cross-sectional comparative analysis assessing increase in height over a 3-year period showed an additional height gain of 5.75 cm with use of VOXZOGO compared to matched untreated children over the same period. 3-year Phase 3 Quality of Life Results in Achondroplasia Results from a separate analysis of the Phase 3 study showed that VOXZOGO improved health-related quality of life (HRQoL) among children with achondroplasia, particularly those associated with physical activities. After 3 years, the mean increase in Quality of Life in Short Stature Youth (QoLISSY) physical domain score was 6.0 as reported by caregivers and 6.3 as reported by children. These improvements were even more pronounced in children who grew more (for those with ≥ 1 SD increase in height z-score, the mean increase in physical domain score was 11.4 as reported by caregivers and 8.5 as reported by children). "The clinical results at ACMG reinforce the long-term benefit we are seeing in children treated with VOXZOGO, with a meaningful median height gain of more than 11 cm in children with achondroplasia following 7 years of treatment," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. "We are particularly encouraged by new data demonstrating the potential impact height gains from VOXZOGO can have on quality of life, an outcome of incredible importance for children and families impacted by achondroplasia." "Achondroplasia can have a negative effect on a child's quality of life, including physical, social and emotional aspects," said Melita Irving, M.D., study investigator and consultant clinical geneticist at Guy's and St. Thomas' NHS Foundation Trust in London. "Our studies showed that increasing height with long-term administration of VOXZOGO can result in meaningful improvements in quality-of-life measures for children with achondroplasia." BioMarin's full list of presentations at ACMG includes: BioMarin Poster Presentations Persistence of Growth-Promoting Effects in Children with Achondroplasia Up to 7 Years: Update from a Phase 2 Extension Study with Vosoritide Poster #: P144 Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia for Up to 4 Years: Update from Phase 3 Extension Study Poster #: P139 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia is Accompanied by Improvement in Physical Aspects of Quality of Life Poster #: P141 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Persistence of Growth-Promoting Effects in Infants and Toddlers with Achondroplasia: Results from a Phase 2 Extension Study with Vosoritide Poster #: P131 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Exploring the Landscape of Phenylketonuria Education and Learning Needs of Genetics Trainees Poster #: P049 Thursday, March 14, 2024, 10:30 a.m. – 12 p.m. ET Reduction of Blood Phenylalanine in Participants Enrolled in OPAL, an Observational Study, Mirror Findings from the U.S.-based PRISM Population Poster #: P034 Friday, March 15, 2024, 10:30 a.m. – 12 p.m. ET Investigator-Sponsored Oral Presentation Vosoritide Increases Growth Velocity in Hypochondroplasia: Phase 2 Trial Results Oral Session: O03 Thursday, March 14, 2024, 1:30 – 3 p.m. ET About VOXZOGO In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. VOXZOGO is approved in the U.S. and indicated to increase linear growth in children with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history. Patient Support Accessing VOXZOGO To reach a BioMarin RareConnections® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail VOXZOGOSupport@biomarin-rareconnections.com. For more information about VOXZOGO, please visit . For additional information regarding this product, please contact BioMarin Medical Information at medinfo@bmrn.com. About Achondroplasia Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth. More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category. VOXZOGO U.S. Important Safety Information What is VOXZOGO used for? VOXZOGO is a prescription medicine used to increase linear growth in children with achondroplasia and open growth plates (epiphyses). VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. What is the most important safety information about VOXZOGO? VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should eat a meal and drink 8 to 10 ounces of fluid within 1 hour before receiving VOXZOGO. What are the most common side effects of VOXZOGO? The most common side effects of VOXZOGO include injection site reactions (including redness, itching, swelling, bruising, rash, hives, and injection site pain), high levels of blood alkaline phosphatase shown in blood tests, vomiting, joint pain, decreased blood pressure, and stomachache. These are not all the possible side effects of VOXZOGO. Ask your healthcare provider for medical advice about side effects, and about any side effects that bother the patient or that do not go away. How is VOXZOGO taken? VOXZOGO is taken daily as an injection given under the skin, administered by a caregiver after a healthcare provider determines the caregiver is able to administer VOXZOGO. Do not try to inject VOXZOGO until you have been shown the right way by your healthcare provider. VOXZOGO is supplied with Instructions for Use that describe the steps for preparing, injecting, and disposing VOXZOGO. Caregivers should review the Instructions for Use for guidance and any time they receive a refill of VOXZOGO in case any changes have been made. Inject VOXZOGO 1 time every day, at about the same time each day. If a dose of VOXZOGO is missed, it can be given within 12 hours from the missed dose. After 12 hours, skip the missed dose and administer the next daily dose as usual. The dose of VOXZOGO is based on body weight. Your healthcare provider will adjust the dose based on changes in weight following regular check-ups. Your healthcare provider will monitor the patient's growth and tell you when to stop taking VOXZOGO if they determine the patient is no longer able to grow. Stop administering VOXZOGO if instructed by your healthcare provider. What should you tell the doctor before or during taking VOXZOGO? Tell your doctor about all of the patient's medical conditions including If the patient has heart disease (cardiac or vascular disease), or if the patient is on blood pressure medicine (anti-hypertensive medicine). If the patient has kidney problems or renal impairment. If the patient is pregnant or plans to become pregnant. It is not known if VOXZOGO will harm the unborn baby. If the patient is breastfeeding or plans to breastfeed. It is not known if VOXZOGO passes into breast milk. Tell your doctor about all of the medicines the patient takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088. Please see additional safety information in the full Prescribing Information and Patient Information. About BioMarin Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of the genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit . Forward-Looking Statements This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: data to be presented at the 2024 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting, including the six poster presentations and the investor-sponsored oral presentation; the development of BioMarin's VOXZOGO program generally; the potential benefits of VOXZOGO for children with achondroplasia, including the duration of such benefits, potential improvement in annualized growth velocity (AGV), and potential improvement in health-related quality of life; the potential efficacy of VOXZOGO for children with hypochondroplasia, including potential to impact growth outcomes; and the continued clinical development of VOXZOGO, including BioMarin's plans for launching the treatment phase of the Phase 3 trial studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency, the European Commission and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the year ended December 31, 2023 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc. Company Codes: NASDAQ-NMS:BMRN

Johnson & Johnson (J&J) has announced that a reduced dosing frequency of Tecvayli (teclistamab-cqyv) has been approved by the US Food and Drug Administration (FDA) for certain patients with relapsed or refractory multiple myeloma (RRMM). The regulator’s decision means that RRMM patients who have achieved a complete response or better for at least six months can receive a 1.5mg/kg dose of the BCMA-targeting bispecific antibody every two weeks. More than 35,000 new cases of multiple myeloma, a currently incurable blood cancer that affects a type of white blood cell called plasma cells, are expected to be diagnosed in the US this year. Tecvayli, which is administered subcutaneously, was granted accelerated approval by the FDA in October 2022 to treat adults with RRMM who received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug and an anti-CD38 monoclonal antibody. The FDA’s latest approval on the drug was supported by positive results from the phase 1/2 MajesTEC-1 study, in which patients were initially treated with the recommended dose of 1.5mg/kg Tecvayli weekly. Those who achieved a confirmed complete response or better for six months or longer were eligible to receive the reduced dosing frequency of 1.5mg/kg every two weeks until disease progression or unacceptable toxicity. Rachel Kobos, vice president, oncology research and development, J&J Innovative Medicine, said: “Tecvayli is the only BCMA-targeted immune-based therapy with weight-based dosing. “[The] approval of bi-weekly dosing for eligible patients will further enable clinicians to meet the individual needs of patients who may want flexibility in their dosing schedules.” The reduced dosing frequency of Tecvayli was approved by the European Commission in August for the same patient population. The approval comes just one month after the FDA granted full approval to J&J's bladder cancer drug, Balversa (erdafitinib). The oral FGFR kinase inhibitor, which originally received accelerated approval by the FDA in 2019, is now fully authorised for use in adults with locally advanced or metastatic urothelial carcinoma with susceptible FGFR3 genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy.

Upon closing, XOMA anticipates adding approximately $9.5 million in cash to its balance sheet and several early-stage programs to potentially add to its royalty portfolioEMERYVILLE, Calif., Feb. 16, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA) announced today it has entered an agreement to acquire Kinnate Biopharma Inc. (NASDAQ: KNTE) for (i) a base cash price of $2.3352 per share (the Base Price) and (ii) an additional cash amount of not more than $0.2527 per share (together with the Base Price, the Cash Amount) at the closing of the merger plus a non-transferable contingent value right (CVR), representing the right to receive 85% of the net proceeds from any out license or sale of the Kinnate programs effected within one year of closing of the merger or 100% of the net proceeds from any out license or sale executed prior to the closing. “This acquisition will further add to our sizable cash balance that resulted from the VABYSMO transaction, as well as potentially add several programs to our royalty portfolio,” stated Owen Hughes, Chief Executive Officer of XOMA. “Given our successful history of out licensing assets and technologies, we will look to monetize Kinnate’s precision oncology programs to the benefit of Kinnate and XOMA shareholders alike.” Following a thorough review process conducted with the assistance of its legal and financial advisors, Kinnate's Board of Directors has determined that the acquisition by XOMA is in the best interests of all Kinnate stockholders and has unanimously approved the Merger Agreement. TermsPursuant and subject to the terms of the Merger Agreement, a wholly owned subsidiary of XOMA will commence a tender offer (the "Offer") by March 4, 2024, to acquire all outstanding shares of Kinnate common stock. Closing of the Offer is subject to certain conditions, including the tender of Kinnate common stock representing at least a majority of the total number of outstanding shares; the availability of at least $120.0 million of cash, net of transaction costs, wind-down costs, and other liabilities, at closing, and other customary closing conditions. Immediately following the closing of the tender offer, Kinnate will merge with a subsidiary of XOMA, and all remaining shares not tendered in the offer, other than appraisal shares, will be converted into the right to receive the same cash and CVR consideration per share as is provided in the tender offer. Kinnate shareholders holding approximately 46% of Kinnate common stock have signed support agreements under which such shareholders agreed to tender their shares in the Offer and support the merger. The acquisition is expected to close in April 2024. AdvisorsXOMA was represented by Gibson, Dunn & Crutcher LLP. About XOMA CorporationXOMA is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of milestone and royalty assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com. About Kinnate Biopharma Inc.Kinnate Biopharma Inc. is a clinical-stage precision oncology company founded with a mission to inspire hope in those battling cancer by expanding on the promise of targeted therapies. Kinnate concentrates its efforts on addressing known oncogenic drivers for which there are currently no approved targeted therapies and to overcome the limitations associated with existing cancer therapies, such as non-responsiveness or the development of acquired and intrinsic resistance. Kinnate’s lead product candidates are investigational pan-RAF inhibitor, exarafenib, which targets cancers with BRAF and NRAS-driven alterations, and investigational FGFR inhibitor, KIN-3248, which is designed for cancers with FGFR2 and FGFR3 alterations. Kinnate also has early stage programs, including a c-MET inhibitor that targets resistant variants and a brain penetrant CDK4 selective program. For more information, visit Kinnate.com and on LinkedIn to learn about its most recent initiatives. Forward-Looking Statements/Explanatory Notes Certain statements contained in this press release are forward-looking statements, including statements regarding the expected timing and ability to satisfy the conditions required to close the tender offer, the merger and transactions related to the Merger Agreement, the ability of XOMA to monetize Kinnate’s programs for the benefit of XOMA and Kinnate shareholders, and the ability to achieve any dispositions within the disposition period under the CVR. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “approximately,” “look to,” “plan,” “expect,” “may,” “will,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks including the risk that XOMA does not achieve anticipated net cash after winding down Kinnate’s operations and concluding remaining clinical trial activities, the risk that XOMA is unable to develop or otherwise enter into dispositions related to the Kinnate programs, and risks that the conditions to the closing the merger in the Merger Agreement are not satisfied. Other potential risks to XOMA meeting these expectations are described in more detail in XOMA's most recent filing on Form 10-K and in other filings with the Securities and Exchange Commission. Any forward-looking statement in this press release represents XOMA's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. Additional Information and Where to Find It The tender offer for the outstanding shares of Kinnate referenced in this press release has not yet commenced. This press release is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell shares, nor is it a substitute for the tender offer materials that XOMA and its subsidiary will file with the SEC. At the time the tender offer is commenced, XOMA and its subsidiary will file tender offer materials on Schedule TO, and, thereafter, Kinnate will file a Solicitation/Recommendation Statement on Schedule 14D-9 with the SEC with respect to the tender offer. The Offer to Purchase, related Letter of Transmittal and certain other tender offer documents, including Kinnate’s Solicitation/Recommendation Statement, will contain important information. Holders of Kinnate common stock are urged to read these documents carefully when they become available as they will contain important information. These materials will be made available to Kinnate common stock holders at no expense upon request and will be made available for free at the SEC’s website at www.sec.gov or by accessing the Investor Relations section of the Company’s website at https://www.investors.xoma.com. XOMA Investor ContactJuliane SnowdenXOMA Corporation+1 646-438-9754juliane.snowden@xoma.comXOMA Media ContactKathy VincentKV Consulting & Management+1 310-403-8951kathy@kathyvincent.com