BRAF V600 mutation-positive Neoplasms

Initial Phase 2 data of BDTX-1535 in 2L/3L EGFRm NSCLC patients on track for Q3 2024Enrollment ongoing in Phase 2 trial of BDTX-1535 in 1L patients with non-classical EGFRm NSCLC; initial data expected Q1 2025Presented real world evidence on the evolving EGFR mutation landscape in NSCLC, and the potential of BDTX-1535 across multiple lines of therapy at the 2024 AACR annual meetingPoster presentations upcoming June 1st at 2024 ASCO Annual Meeting on BDTX-1535 in GBM patients: Phase 1 dose-escalation data and initial “window of opportunity” trial resultsCash, cash equivalents, and investments of $115.2 million as of March 31, 2024, expected to be sufficient to fund operations into Q3 of 2025 CAMBRIDGE, MA, May 09, 2024 (GLOBE NEWSWIRE) -- Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, today reported financial results for the first quarter ended March 31, 2024, and provided a corporate update. “We reached an important inflection in BDTX-1535 development this past quarter, as we initiated dosing of patients with non-classical EGFR mutant NSCLC in the first-line setting, and are on track to release initial results from the ongoing Phase 2 second/third-line cohorts in the third quarter of this year ”, said Mark Velleca, M.D., Ph.D., Chief Executive Officer of Black Diamond Therapeutics. “Our recent AACR presentation on the evolving EGFR mutation landscape highlights the major unmet need in EGFR mutant NSCLC, and the differentiated BDTX-1535 profile as the potential first- and best-in-class fourth-generation oral TKI in clinical development addressing the full spectrum of classical, non-classical, and C797S resistance mutations in NSCLC.” Recent Developments & Upcoming Milestones BDTX-1535 Following receipt of End of Phase 1 feedback from the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2023, Black Diamond initiated dosing of a Phase 2 first-line cohort in patients with non-classical epidermal growth factor receptor mutation positive (EGFRm) non-small cell lung cancer (NSCLC) during the first quarter of 2024. (NCT05256290)In April 2024, Black Diamond described real world evidence of the evolving EGFR mutation landscape in patients with NSCLC and the potential of BDTX-1535 to address a broader range of mutations compared to existing therapies at the 2024 American Association for Cancer Research (AACR) annual meeting. The oral presentation titled “BDTX-1535 – A MasterKey EGFR Inhibitor Targeting Classical, Non-Classical and the C797S Resistance Mutation to Address the Evolved Landscape of EGFR Mutant NSCLC,” evaluated more than 235,000 sequenced cases of NSCLC sourced from Guardant Health (GuardantINFORM™) and Foundation Medicine (FoundationInsights™). The analyses revealed a broad spectrum of non-classical mutations, as well as an increased prevalence of the acquired resistance mutation, C797S. These non-classical EGFR mutations were present in 20-30% of newly diagnosed EGFRm NSCLC patients.Black Diamond anticipates the following upcoming key milestones for BDTX-1535: Poster presentations at the American Society of Clinical Oncology (ASCO) Annual Meeting on June 1, 2024, of Phase 1 dose escalation data in patients with relapsed/recurrent GBM, and initial intratumoral pharmacokinetic results from a “window of opportunity” (also known as a Phase 0/1 “Trigger”) trial sponsored by the Ivy Brain Tumor Center, in patients with recurrent high-grade glioma (HGG).Disclosure of initial Phase 2 data in 2L/3L EGFRm NSCLC patients with non-classical mutations or the acquired resistance C797S mutation remains on track for Q3 2024. BDTX-4933 Enrollment of patients with BRAF and select RAS/MAPK mutation-positive cancers, with an emphasis on patients with KRAS mutant NSCLC is ongoing in a Phase 1 dose escalation trial of BDTX-4933, a brain-penetrant oral inhibitor of oncogenic alterations in KRAS, NRAS and BRAF (NCT05786924). An update from this trial is anticipated in Q4 2024. Corporate In April 2024, the Company announced changes to its Board of Directors. Industry veterans Shannon Campbell and Prakash Raman, Ph.D., have been appointed to the Company’s Board of Directors. In addition, Wendy Dixon, Ph.D., and Alex Mayweg, Ph.D., have stepped down as members of the Board of Directors. Financial Highlights Cash Position: Black Diamond ended the first quarter of 2024 with approximately $115.2 million in cash, cash equivalents, and investments compared to $131.4 million as of December 31, 2023. Net cash used in operations was $21.2 million for the first quarter of 2024 compared to $20.0 million for the first quarter of 2023.Research and Development Expenses: Research and development (R&D) expenses were $13.5 million for the first quarter of 2024, compared to $14.8 million for the same period in 2023. The decrease in R&D expenses was primarily due to reduced spending on early discovery projects.General and Administrative Expenses: General and administrative (G&A) expenses were $6.7 million for the first quarter of 2024, compared to $6.8 million for the same period in 2023. The decrease in G&A expenses was primarily due to a decrease in personnel related costs and legal and other professional fees.Net Loss: Net loss for the first quarter of 2024 was $18.2 million, as compared to $20.9 million for the same period in 2023. Financial Guidance Black Diamond ended the first quarter of 2024 with approximately $115.2 million in cash, cash equivalents and investments which the Company believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the third quarter of 2025. About Black Diamond Therapeutics Black Diamond Therapeutics is a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer. The Company’s MasterKey therapies are designed to address a broad spectrum of genetically defined tumors, overcome resistance, minimize wild-type mediated toxicities, and be brain penetrant to treat CNS disease. The Company is advancing two clinical-stage programs: BDTX-1535, a brain-penetrant fourth-generation EGFR MasterKey inhibitor targeting EGFR mutant NSCLC and GBM, and BDTX-4933, a brain-penetrant RAF MasterKey inhibitor targeting KRAS, NRAS and BRAF alterations in solid tumors. For more information, please visit www.blackdiamondtherapeutics.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: the potential of BDTX-1535 to address the full spectrum of EGFR mutations in NSCLC, the continued development and advancement of BDTX-1535 and BDTX-4933, including the ongoing clinical trials and the timing of clinical updates for BDTX-1535 in patients with NSCLC and in patients with recurrent GBM, and for Phase 1 clinical trial results for BDTX-4933, the expected timing for additional updates on data from the “window of opportunity” clinical trial of BDTX-1535 in patients with recurrent HGG, the potential of BDTX-1535 to benefit patients with NSCLC across multiple lines of therapy, potential future development plans for BDTX-1535 in NSCLC and GBM, including in treatment naïve and recurrent settings, and the Company’s expected cash runway. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include those risks and uncertainties set forth in its Annual Report on Form 10-K for the year ended December 31, 2023, filed with the United States Securities and Exchange Commission and in its subsequent filings filed with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Black Diamond Therapeutics, Inc.Condensed Consolidated Balance Sheet Data (Unaudited)(in thousands) March 31, 2024 December 31, 2023 (in thousands)Cash, cash equivalents, and investments$115,199 $131,400 Total assets$142,521 $158,567 Accumulated deficit$(435,656) $(417,431)Total stockholders’ equity$104,306 $116,736 Black Diamond Therapeutics, Inc.Consolidated Statements of Operations (Unaudited)(in thousands, except per share data) Three Months EndedMarch 31, 2024 2023 Operating expenses: Research and development$13,545 $14,753 General and administrative 6,701 6,808 Total operating expenses 20,246 21,561 Loss from operations (20,246) (21,561)Other income (expense): Interest income 637 622 Other income (expense) 1,384 64 Total other income (expense), net 2,021 686 Net loss$(18,225) $(20,875)Net loss per share, basic and diluted$(0.35) $(0.57)Weighted average common shares outstanding, basic and diluted 51,808,849 36,483,878 Contact For Investors:Mario Corso, Head of Investor Relations, Black Diamond Therapeuticsmcorso@bdtx.com For Media:media@bdtx.com

PHILADELPHIA--(BUSINESS WIRE)-- FORE Biotherapeutics today announced the acceptance of two abstracts on new clinical data related to FORE8394, the company's novel, investigational, small-molecule, next-generation, orally available selective inhibitor of BRAF alterations at the American Society of Clinical Oncology (ASCO) Annual Meeting, taking place June 3-7, 2023, in Chicago and virtually. The datasets from the phase 1/2a clinical trial, evaluating the safety, tolerability, and efficacy of FORE8394 as a monotherapy will be presented. Details of the presentations are as follows: Oral Presentation Abstract number: 3006 Title: Safety and efficacy of the novel BRAF inhibitor FORE8394 in patients with advanced solid and CNS tumors: Results from a phase 1/2a study. Presenter: Macarena de la Fuente, MD, University of Miami Sylvester Comprehensive Cancer Center Presentation Session Date/Time: The oral presentation will take place on Monday, June 5, 2023, from 8:00 – 11:00 a.m. CDT, during the session titled "Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology." Poster Presentation Abstract number: 3106 Title: Dose optimization of novel BRAF inhibitor FORE8394 based on PK and efficacy results. Presenter: Eric Sherman, MD, Memorial Sloan-Kettering Cancer Center Presentation Session Date/Time: The poster will be presented on Friday, June 3, 2023, from 8:00 – 11:00 a.m. CDT, during the session titled "Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology.” About FORE8394 FORE8394 is an investigational, novel, small-molecule, next-generation, orally available selective inhibitor of mutated BRAF. It was designed to target a wide range of BRAF mutations while sparing wild-type forms of RAF. Preclinical studies and clinical trials have shown that its unique mechanism of action effectively inhibits not only the constitutively active BRAFV600 monomers targeted by first-generation RAF inhibitors but also disrupts constitutively active dimeric BRAF class 2 mutants, fusions, splice variants and others. Unlike first-generation RAF inhibitors, FORE8394 does not induce paradoxical activation of the RAF/MEK/ERK pathway. As a “paradox breaker,” FORE8394 could therefore treat acquired resistance to current RAF inhibitors and, more generally, yield improved safety and more durable efficacy than first-generation RAF inhibitors. The company previously announced interim data from the ongoing Phase 1/2a clinical trial evaluating FORE8394 in advanced solid and CNS tumors with activating BRAF alterations, providing evidence of durable anti-tumor activity in patients with BRAF-mutated (V600+) cancers. The data were presented at the European Society of Medical Oncology Congress (ESMO) in September 2022. About Fore Biotherapeutics Fore Bio is a precision oncology company dedicated to developing innovative treatments that provide a better outcome for cancer patients. Its lead asset FORE8394 is a Class 1/V600 and 2 BRAF inhibitor with demonstrated clinical safety and early efficacy signals in an ongoing Phase 1/2a clinical trial. Leveraging a proprietary functional genomics platform that can screen a wide range of known mutations for cancer-driving genes, the Fore R&D team is optimizing drug development by identifying existing compounds with known clinical profiles and a clear path through clinical development to advance new medicines for patients without treatment options. For more information, please visit or follow us on Twitter and LinkedIn.

BASEL, Switzerland & BEIJING & CAMBRIDGE, Mass. & SAN MATEO, Calif. & STAMFORD, Conn.--(BUSINESS WIRE) -- MapKure, LLC, BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), and SpringWorks Therapeutics, Inc. (NASDAQ: SWTX), today announced that they will present updated clinical data from the Phase 1a/1b study of BGB-3245, an investigational, selective RAF dimer inhibitor, in adult patients with advanced or refractory solid tumors harboring MAPK pathway aberrations. The data are being presented today in an oral presentation at the American Association for Cancer Research (AACR) Annual Meeting 2023, taking place in Orlando, Florida. This press release features multimedia. View the full release here: “The promising data we are sharing at AACR demonstrate the value of our next-generation RAF dimer inhibitor, BGB-3245, as a monotherapy in patients with MAPK pathway-altered cancers, especially its potential to address key primary and resistance gene alterations that are currently unaddressed by approved therapies,” said Lusong Luo, Ph.D., Acting CEO of MapKure and Senior Vice President, External Innovation at BeiGene. “We believe that our early clinical data supported the advancement into our selected expansion cohorts which are now underway.” “SpringWorks is committed to developing genomically-targeted therapies for patients with cancer and our MAPK directed portfolio is a cornerstone of our efforts. In particular, we are encouraged by the promising activity BGB-3245 is showing in patients with MAPK pathway aberrations,” said Saqib Islam, CEO of SpringWorks. “We and our partners are excited by the advancement of this program and are eager to continue making strides to benefit patients in significant need of new therapeutic options.” Oral Presentation at the 2023 AACR Annual Meeting: A first-in-human, phase 1a/1b, open-label, dose-escalation and expansion study to investigate the safety, pharmacokinetics, and antitumor activity of the RAF dimer inhibitor BGB-3245 in patients with advanced or refractory tumors Presenter: Alison M. Schram, M.D., Memorial Sloan Kettering Cancer Center in New York Session Title: Mini-symposium Session CTMS02 - Targeting the KRAS Pathway in the Clinic Abstract #: CT031 Session Date and Time: Monday, April 17, 2023, from 3:20-3:30 PM ET This ongoing Phase 1a/1b trial (NCT04249843) is an open-label, dose escalation and expansion study of BGB-3245 in adult patients with advanced or refractory solid tumors harboring MAPK pathway alterations. Results from the Phase 1a dose-escalation and dose-finding study are being presented at AACR. This portion of the study was designed to evaluate the safety, pharmacokinetics, preliminary antitumor activity of BGB-3245, and to determine its maximum tolerated dose and/or recommended Phase 2 dose to be used in select expansion cohorts. As of the data cut-off of September 1, 2022, 42 patients were treated across six dose levels (5-60 mg daily). Patients were heavily pre-treated, having received a median three prior lines of therapy (range 1-9), including standard of care immunotherapy and targeted therapy regimens. Results demonstrated that BGB-3245 had a manageable safety profile, with adverse event findings consistent with other MAPK pathway inhibitors. The most common treatment-related adverse events (>15%) were rash acneiform (33%), rash maculopapular (24%), and fever (17%). The 40 mg once daily dose was determined to be the maximum tolerated dose of BGB-3245. In addition, encouraging anti-tumor activity was observed in heavily pretreated patients, with an objective response rate of 18% (6 confirmed responses including one complete response in 33 efficacy evaluable patients). The disease control rate was 79% and clinical benefit rate was 42%. Objective responders include patients with tumors harboring BRAF V600E that had progressed on prior BRAF/MEK inhibitors with or without checkpoint inhibitor treatment, BRAF Class II mutation, BRAF fusion, NRAS and KRAS mutations. Median time on treatment was approximately 5 months (range: 1.9-23.6 months) and 9 patients remain on treatment. These data supported the advancement of BGB-3245 into the Phase 1b dose expansion portion of the study, which has been enrolling patients since October 2022 in defined cohorts. “These data support the ongoing investigation of BGB-3245 in defined cohorts, including BRAF V600 tumors that have progressed after prior BRAF and/or MEK inhibitor treatment, solid tumors with BRAF class II mutations and BRAF fusions, and NRAS mutant melanoma,” said Alison M. Schram, M.D., Assistant Attending Physician at Memorial Sloan Kettering Cancer Center in New York. “These patients have very limited treatment options and I look forward to the further development of BGB-3245 as it continues to advance through the dose expansion cohorts.” About BGB-3245 BGB-3245 is an investigational, oral, selective small molecule inhibitor of RAF monomer and dimer forms. Preclinical data demonstrate that BGB-3245 has activity in tumor models that have BRAF/MEK inhibitor-resistance mutations, suggesting BGB-3245 could provide a therapeutic option for patients who have progressed on prior BRAF and/or MEK inhibitions. In addition, BGB-3245 has shown activity in preclinical models that have BRAF class II/III mutations, fusions, and splice isoforms, for which approved BRAF inhibitors are ineffective. These mutations and fusions have been identified in several solid tumors to be drivers of cancer growth, including in melanoma, non-small cell lung cancer, colorectal cancer, thyroid cancer, and other cancers. In addition to its development as a monotherapy in several genetically defined solid tumor types, a Phase 1/2a combination study of BGB-3245 and mirdametinib is ongoing (NCT05580770) and BGB-3245 also has the potential to be used in rational combination therapies in the future. About MapKure MapKure is a clinical-stage company created in 2019 to develop precision medicines for patients with life-threatening diseases, with an initial focus on cancer. By focusing on genetically defined disease drivers, MapKure is positioned to advance the development of transformative medicines to patients whose unmet medical needs are largely unaddressed. MapKure is jointly owned by BeiGene and SpringWorks and is currently developing BGB-3245 under an exclusive license from BeiGene in solid tumor patients harboring specific B-RAF driver mutations and RAF fusions, as well as in patients who have developed resistance to first-generation B-RAF inhibitors. About BeiGene BeiGene is a global biotechnology company that is developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 9,000 colleagues spans five continents, with administrative offices in Basel, Beijing and Cambridge, Mass. To learn more about BeiGene, please visit and follow us on Twitter at @BeiGeneGlobal. About SpringWorks Therapeutics SpringWorks is a clinical-stage biopharmaceutical company applying a precision medicine approach to acquiring, developing and commercializing life-changing medicines for patients living with severe rare diseases and cancer. SpringWorks has a differentiated targeted oncology pipeline spanning solid tumors and hematological cancers, including two late-stage clinical trials in rare tumor types as well as several programs addressing highly prevalent, genetically defined cancers. SpringWorks’ strategic approach and operational excellence in clinical development have enabled it to rapidly advance its two lead product candidates into late-stage clinical trials while simultaneously entering into multiple shared-value partnerships with innovators in industry and academia to unlock the full potential for its portfolio and create more solutions for patients with cancer. For more information, please visit and follow @SpringWorksTx on Twitter and LinkedIn. BeiGene Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the potential for BGB-3245 to address key primary and resistance gene alterations that are currently unaddressed by approved therapies; the future development and regulatory filing and approval of BGB-3245; and BeiGene’s plans, commitments, aspirations, and goals under the heading “About BeiGene.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products and its ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and the impact of the COVID-19 pandemic on BeiGene’s clinical development, regulatory, commercial, manufacturing, and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law. SpringWorks Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, relating to our business, operations, and financial conditions, including, but not limited to, current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our development plans, our preclinical and clinical results, as well as relating to other future conditions. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “would,” “should” and “could,” and similar expressions or words, identify forward-looking statements. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks relating to: (i) the success and timing of our product development activities, including the initiation and completion of SpringWorks’ clinical trials, (ii) the fact that topline or interim data from a clinical study may not be predictive of the final or more detailed results of such study, or the results of other ongoing or future studies, (iii) the success and timing of our collaboration partners’ ongoing and planned clinical trials, (iv) the timing of our planned regulatory submissions and interactions, including the timing and outcome of decisions made by the U.S. Food and Drug Administration (FDA) and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; (v) whether FDA or other regulatory authorities will require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, (vii) our ability to obtain and maintain regulatory approval of any of our product candidates, (viii) our plans to research, discover and develop additional product candidates, (ix) our ability to maintain adequate patent protection and successfully enforce patent claims against third parties, (x) our ability to enter into collaborations for the development of new product candidates, (xi) our ability to establish manufacturing capabilities, and our and our collaboration partners’ abilities to manufacture our product candidates and scale production, (xii) our ability to meet any specific milestones set forth herein, and (xiii) uncertainties and assumptions regarding the impact of the COVID-19 pandemic on SpringWorks’ business, operations, clinical trials, supply chain, strategy, goals and anticipated timelines. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. Accordingly, readers are cautioned not to place undue reliance on these forward-looking statements. For further information regarding the risks, uncertainties and other factors that may cause differences between SpringWorks’ expectations and actual results, you should review the “Risk Factors” in Item 1A of Part I of SpringWorks’ Annual Report on Form 10-K for the year ended December 31, 2022, as well as discussions of potential risks, uncertainties and other important factors in SpringWorks’ subsequent filings. View source version on businesswire.com: Contacts BeiGene Contacts: Investors Kevin Mannix +1 857-302-5189 ir@beigene.com Media Kathleen Cuca +1 551 222 6790 media@beigene.com SpringWorks Contacts: Kim Diamond Vice President, Communications and Investor Relations Phone: +1 203-561-1646 Email: kdiamond@springworkstx.com Samantha Hilson Sandler Senior Director, Investor Relations Phone: +1 203-461-5501 Email: samantha.sandler@springworkstx.com Source: BeiGene View this news release online at: