Core Insights - SpringWorks Therapeutics has received FDA approval for GOMEKLI™ (mirdametinib), the first and only treatment for both adults and children with neurofibromatosis type 1 (NF1) associated plexiform neurofibromas (PN) [3][5][17] - The approval is based on positive results from the Phase 2b ReNeu trial, demonstrating significant efficacy and a manageable safety profile [6][8][9] - The company has been granted a rare pediatric disease priority review voucher by the FDA, highlighting the importance of this treatment for the NF1-PN patient community [3][6] Company Overview - SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer [27] - The company has developed GOMEKLI as the first FDA-approved medicine for NF1-PN, and it also markets OGSIVEO (nirogacestat) for adults with desmoid tumors [27] - SpringWorks is advancing a diverse portfolio of targeted therapy candidates for solid tumors and hematological cancers [27] Product Details - GOMEKLI is an oral MEK inhibitor approved for patients aged 2 years and older with symptomatic plexiform neurofibromas not amenable to complete resection [17][12] - The product is available in 1 mg and 2 mg capsules, as well as a 1 mg tablet for oral suspension [12] - GOMEKLI is expected to be available through specialty pharmacies and distributors in the U.S. within two weeks of approval [12] Clinical Trial Insights - The Phase 2b ReNeu trial enrolled 114 patients (58 adults and 56 pediatric patients) and met its primary endpoint with a confirmed objective response rate (ORR) of 41% in adults and 52% in children [7][8] - Tumor volume reductions were significant, with a median best percentage change of -41% in adults and -42% in children [8] - The trial also reported improvements in pain and quality of life for patients, indicating the treatment's potential to enhance overall well-being [8] Market Context - NF1 is a genetic disorder affecting approximately 100,000 individuals in the U.S., with a 30-50% lifetime risk of developing plexiform neurofibromas [5][14] - Prior to GOMEKLI, there were no approved treatments for NF1-PN, making this approval a significant advancement for the patient community [5][6] - The approval of GOMEKLI represents a critical new option for NF patients and their families, addressing a substantial unmet medical need [10][5]

– GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN – – Approval based on positive data from Phase 2b ReNeu trial, which showed GOMEKLI treatment resulted in robust ORR, deep and durable reductions in tumor volume, and a manageable safety profile – – SpringWorks granted rare pediatric disease priority review voucher by the FDA – Photos accompanying this announcement are available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/8eb8effc-49a5-4b24-911f-d3e6dfa9c8 ...

STAMFORD, Conn., Jan. 30, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, today announced that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, February 20, 2025 to report its fourth quarter and full year financial results and discuss recent business updates. To join the live webcast and view the corresponding slides, please click here. To participate in the live confe ...

SpringWorks Therapeutics (SWTX) announced preliminary net product revenues for fourth-quarter and full-year 2024 for its sole marketed drug, Ogsiveo (nirogacestat). The drug is approved for treating adult patients with progressing desmoid tumors who require systemic treatment.The company also outlined some expected milestones related to its commercial portfolio and other pipeline candidates for 2025.Shares of SWTX were down 13.9% on Jan. 13 following the news announcement.In the past year, shares of SpringW ...

– Achieved $61.5 million and $172.0 million in preliminary fourth quarter and full year 2024 OGSIVEO® (nirogacestat) U.S. net product revenues, respectively – – Ended 2024 with approximately $462 million in cash, cash equivalents, and marketable securities – – Additional updates across Company's commercial portfolio and investigational pipeline to be provided during J.P. Morgan Healthcare conference presentation today at 11:15 a.m. PT – STAMFORD, Conn., Jan. 13, 2025 (GLOBE NEWSWIRE) -- SpringWorks Therapeu ...

SpringWorks Therapeutics (SWTX) shares ended the last trading session 6% higher at $41.20. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 7.2% loss over the past four weeks.The company’s sole marketed drug, Ogsiveo, which is approved for treating adult patients with progressing desmoid tumors, has been witnessing a strong initial uptake. The growing optimism surrounding Ogsiveo might have driven the recent share p ...

Company Overview - SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer [3] - The company applies a precision medicine approach to develop and deliver life-changing medicines [3] - OGSIVEO® (nirogacestat) is the company's first FDA-approved therapy for adult patients with progressing desmoid tumors requiring systemic treatment [3] - SpringWorks has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with programs from preclinical development to advanced clinical trials [3] - The company has entered into multiple collaborations with industry and academia to unlock the full potential of its portfolio [3] Upcoming Event - SpringWorks Therapeutics CEO Saqib Islam will present at the 43rd Annual J P Morgan Healthcare Conference on January 13, 2025 at 11:15 a m PT [1] - The live webcast can be accessed on the company's website, with a replay available for a limited time [2] Additional Information - More information about SpringWorks Therapeutics is available on their website and social media channels [4]

Core Insights - Jonathan Faison highlights Tarsus Pharmaceuticals and SpringWorks Therapeutics as key investment opportunities in the biotech sector, with Tarsus's XDEMVY showing strong performance and SpringWorks's Ogsiveo gaining traction in the market [1][2] Tarsus Pharmaceuticals - Tarsus Pharmaceuticals has a market cap of $1.8 billion and an enterprise value of approximately $1.5 billion, with a valuation of 1x to 1.5x peak sales [2] - The lead drug, XDEMVY, targets Demodex blepharitis and has seen a price increase from $15 to the mid-$40s, indicating strong market performance [1][2] SpringWorks Therapeutics - SpringWorks Therapeutics is currently the number one holding in the portfolio, representing around 13% of it, with Ogsiveo launched for desmoid tumors [2] - Ogsiveo has achieved a 70% market share for new prescriptions in its category and is projected to have an annualized run rate of $200 million to $250 million, with only 10% market penetration so far [2] - The company anticipates three launches next year, including the European Union launch for Ogsiveo and the U.S. launch for Mirdametinib, which has a priority review with a PDUFA date in February [2] Market Dynamics - Over 90% of doctors have indicated they will use Ogsiveo again, with 90% stating they will use it as a frontline treatment [2] - The addressable market for Ogsiveo is larger than previously anticipated, strengthening the investment thesis for SpringWorks Therapeutics [2] - The competitive landscape includes AstraZeneca's Koselugo, which is currently approved only for pediatric indications, presenting an opportunity for SpringWorks to capture a larger market share [2]

It has been about a month since the last earnings report for SpringWorks Therapeutics (SWTX) . Shares have added about 14.5% in that time frame, outperforming the S&P 500.Will the recent positive trend continue leading up to its next earnings release, or is SpringWorks Therapeutics due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important catalysts. SpringWorks' Q3 Lo ...

SpringWorks Therapeutics (SWTX) closed the last trading session at $34.71, gaining 15.2% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $66.88 indicates a 92.7% upside potential.The mean estimate comprises eight short-term price targets with a standard deviation of $9.69. While the lowest estimate of $52 indicates a 49.8% increase from the current price level, the most optimistic ...