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Business Wire
13 hours ago
- Business
- Business Wire
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). 'The receipt of Rare Pediatric Disease Designation highlights the significant unmet need that necessitates new therapeutic options for boys living with DMD,' said Cindy Atwell, Chief Development and Business Officer at Precision BioSciences. 'Our first-in-class PBGENE-DMD program, which leverages the differentiated capabilities of our ARCUS gene editing platform to excise exons 45-55 of the dystrophin gene, holds the potential to drive meaningful improvement and durable functional benefit over time for up to 60% of patients with DMD. Our approach is designed to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein known to be functional in humans. We look forward to completing the final IND-enabling toxicology studies currently underway and generating initial clinical data which is expected in 2026.' The FDA grants Rare Pediatric Disease Designation to therapies targeting serious and life-threatening diseases that affect fewer than 200,000 people in the U.S. and primarily affect individuals under the age of 18. Approximately 15,000 people in the U.S. alone are living with Duchenne muscular dystrophy. With this designation, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. The Priority Review Voucher program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a Priority Review Voucher can be redeemed by the original sponsor to receive FDA priority review for a different product. In addition, the Priority Review Voucher can also be sold to a different sponsor, serving as a source of non-dilutive capital to the original sponsor. About PBGENE-DMD PBGENE-DMD is Precision's development program for the treatment of DMD. The approach uses two complementary ARCUS nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene with the aim of restoring near-full length dystrophin protein within the body to improve functional outcomes. PBGENE-DMD is intended to address more than 60% of the DMD patient population. In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored the body's ability to produce a near full length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement. About Precision BioSciences, Inc. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV such as in the DMD program). Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the clinical development and expected safety, efficacy and benefit of our and our partners' and licensees' product candidates and gene editing approaches; the potential of PBGENE-DMD to drive meaningful improvement in functional and durable benefit over time for up to 60% of patients with DMD; the design on PBGENE-DMD to permanently edit a patient's own DNA sequence, resulting in naturally-produced, near full-length dystrophin protein proven known to be functional in humans; the potential value of a Priority Review Voucher(if awarded) including priority review for a different product or sale to another sponsor; the recent sale of a Priority Review Voucher for more than $150 million; the approach of using a single AAV to deliver PBGENE-DMD to excise exons 45-55 of the dystrophin gene with the aim of restoring a near-full length dystrophin protein within the body to improve functional outcomes; the expected timing of regulatory processes and clinical operations (including IND and/or CTA filings, studies, enrollment and clinical data for PBGENE-DMD; and anticipated timing of clinical data. In some cases, you can identify forward-looking statements by terms such as 'aim,' 'anticipate,' 'appear,' 'approach,' 'believe,' , 'confidence', 'contemplate,' 'could,' 'design' 'designed,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look,' 'may,' 'mission,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'pursue,' 'should,' 'strive,' 'suggest,' 'target,' 'will,' 'would,' or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim 'top-line' and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption 'Risk Factors' in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Yahoo
14-05-2025
- Business
- Yahoo
Zevra Therapeutics Inc (ZVRA) Q1 2025 Earnings Call Highlights: Navigating Growth and Challenges
Net Revenue: $20.4 million for Q1 2025. MIPLYFFA Revenue: $17.1 million. OLPRUVA Revenue: $0.1 million. Operating Expense: $22.8 million for Q1 2025. R&D Expenses: $3.3 million, a decrease from the previous year. SG&A Expenses: $19.5 million, an increase of $9.6 million from the previous year. Net Loss: $3.1 million or $0.06 per share for Q1 2025. Cash, Cash Equivalents, and Investments: $68.7 million as of March 31, 2025. Total Debt: Approximately $60 million. Prescription Enrollment Forms for MIPLYFFA: 122 total, with 13 in Q1 2025. Prescription Enrollment Forms for OLPRUVA: 28 total, with 5 in Q1 2025. Covered Lives for MIPLYFFA: 38% coverage achieved by end of Q1 2025. Covered Lives for OLPRUVA: 78% coverage achieved. Warning! GuruFocus has detected 7 Warning Signs with ZVRA. Release Date: May 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Zevra Therapeutics Inc (NASDAQ:ZVRA) exceeded internal expectations for the MIPLYFFA launch, enrolling over one-third of diagnosed Niemann-Pick disease type C (NPC) patients in the US. The company added $148.3 million of non-dilutive capital to its balance sheet through the monetization of a pediatric rare disease Priority Review Voucher, enhancing financial strength. Zevra Therapeutics Inc (NASDAQ:ZVRA) was recognized on Fast Company's Top 10 Most Innovative Companies list in the medicines, therapeutics, and pharmaceuticals category. The Phase 3 DISCOVER trial for Celiprolol, targeting Vascular Ehlers-Danlos syndrome (VEDS), is progressing with patient enrollments, supported by Orphan Drug and Breakthrough Therapy designations. The company is actively expanding MIPLYFFA's availability outside the US, with plans for a Marketing Authorization Application in Europe, targeting approximately 1,100 people living with NPC. Despite the progress, Zevra Therapeutics Inc (NASDAQ:ZVRA) faces challenges with payer reimbursement, with only 38% of covered lives achieved for MIPLYFFA. The launch of OLPRUVA has been slower than expected, with only 28 prescription enrollment forms received since its availability in July 2023. The company reported a net loss of $3.1 million for the first quarter of 2025, although this was an improvement from the previous year. Zevra Therapeutics Inc (NASDAQ:ZVRA) is facing challenges in enrolling patients for the Phase 3 DISCOVER trial for Celiprolol, with only 32 patients enrolled out of a target of 150. The company has moderated expectations for OLPRUVA's launch pace due to unique dynamics in the UCD commercial landscape, indicating potential hurdles in market penetration. Q: Could you provide details on the number of patients currently on MIPLYFFA and the spread of reimbursed patients? A: We have received 122 enrollment forms since the launch, and while we don't report the exact number of patients actively on the drug, the majority are on MIPLYFFA. Those not yet on the drug are undergoing benefits investigation or receiving Quick Start, which is free drug while benefits are being verified. Q: What are the main reasons for reimbursement denials for MIPLYFFA? A: Initial denials are common with rare disease products due to the need for prior authorization. However, we've been successful in securing coverage through direct formulary status or medical exception processes, thanks to MIPLYFFA's demonstrated differentiation and clinical data. Q: How do you track the source of new MIPLYFFA patients, and what is the expected cadence for bringing them on board? A: We track patients from three cohorts: diagnosed, undiagnosed, and those from our Expanded Access Program (EAP). We continue to see enrollments from diagnosed and undiagnosed cohorts due to our awareness initiatives. Inventory levels are maintained at target levels to support patient needs. Q: Can you provide insights into refill rates, patient retention, and average net price for MIPLYFFA? A: Most patients who have started on MIPLYFFA continue to receive refills, indicating strong retention. The distribution of doses aligns with our EAP experience, with most patients receiving higher doses, which informs the average net price. Q: What does the 38% coverage of lives mean for MIPLYFFA, and how does it impact patient access? A: The 38% coverage indicates that plans covering a significant number of lives have reviewed and made MIPLYFFA available. This does not limit patient access, as we successfully secure reimbursement through medical exception pathways, and we expect coverage to increase as more plans evaluate the drug. Q: Can you update us on the enrollment progress for the celiprolol trial? A: We have enrolled 32 patients in the event-driven trial, which targets 150 patients. We are implementing strategies to accelerate enrollment, including targeting COL3A1 patient centers and using genetic testing to identify eligible patients. Q: How does the suspension of a competitor's trial impact your celiprolol trial timeline? A: The suspension of a competitor's trial highlights the unmet need in the space. While we can't provide a specific timeline for enrollment completion, our strategies are starting to pay off, and we are committed to advancing the trial. Q: Are all EAP patients now receiving MIPLYFFA through the commercial channel? A: The process of transitioning EAP patients to commercial drug involves benefits investigation and can vary in duration. Our team is working diligently to ensure patients receive the drug as soon as possible, with some transitions occurring within 24 hours and others taking longer. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. 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Yahoo
31-03-2025
- Business
- Yahoo
OS Therapies Provides Regulatory Update on Rare Pediatric Cancer Immunotherapy Candidate OST-HER2 for Human Osteosarcoma
Dialogue open with FDA in preparation for End of Phase 2 Meeting request New canine elite responder biomarkers added to human biomarker strategy NEW YORK, March 31, 2025--(BUSINESS WIRE)--OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, today provided a regulatory update for its OST-HER2 listeria monocytogenes (Lm) immunotherapeutic cancer biologic drug candidate in the prevention or delay of fully-resected, recurrent, lung metastatic osteosarcoma. "We are making rapid progress in putting together an appropriate data package to achieve accelerated approval for OST-HER2 given the feedback we've received to date from the FDA," said Paul Romness, MHP, Chairman & CEO of OS Therapies. "We know one of the US government's stated priorities is to treat deadly childhood cancers, and we believe that OST-HER2 for osteosarcoma aligns well with that mission." Regulatory Timelines The Company initiated regulatory correspondence with the US Food & Drug Administration (FDA) and the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) in the first quarter of 2025. The Company intends to initiate regulatory interaction with the European Medicines Agency (EMA) and EMA National Competent Authorities in the second quarter of 2025. FDA Q1/25: Regulatory communication regarding endpoints for accelerated approval Q2/25: End of Phase 2 Meeting Q3/25: Initiation of rolling BLA submission Q4/25: Conditional BLA via Accelerated Approval Program MHRA Q1/25: Scientific Advice Meeting requested and granted Q3/25: Scientific Advice Meeting with MHRA and ILAP application submission Q4/25: Application for joint Scientific Advice Meeting with MHRA and National Institute for Health and Care Excellence (NICE) Q4 25: MHRA Conditional Marketing Authorisation application. EMA Q2/25: EMA National Competent Authority Scientific Advice Meeting Request (Medicines Evaluation Board, Netherlands) Q3/25: EMA PRIME and EMA Orphan Designation applications Q4/25: EMA-FDA Parallel Scientific Advice application Q1 26: EMA Conditional Marketing Authorisation application. The Company has recently buttressed its regulatory and clinical strategy & operations infrastructure with the addition of key consulting agencies with significant track records in rare pediatric cancer, Priority Review Voucher (PRV) program approvals, and worldwide Conditional Market Access Applications including in the EU and UK. The Company reiterates its commitment to seeking to obtain FDA approval for OST-HER2 in the rare pediatric cancer osteosarcoma in 2025. The Company is eligible to receive a Priority Review Voucher (PRV) if OST-HER2 is approved under its Rare Pediatric Disease Designation (RPDD) by September 30, 2026. The Company has sufficient cash on hand to operate into 2026. Potential Treatment Response Biomarkers from Canine Osteosarcoma Program The Company's canine osteosarcoma program recently made significant progress in understanding which biomarkers likely drive anti-tumor activity in prevention of recurrence of metastases, metastasis treatment (lung resection is not standard of care in canines) and primary tumor treatment to achieve limb sparing in dogs. The data generated creates the potential for the eventual expansion of the uses of OST-HER2 in osteosarcoma into treatment of unresectable lung metastases and limb sparing prior to surgical resection. The Company intends to evaluate these biomarkers as potentially predictive of treatment response in humans in preparation for regulatory discussions with FDA, MHRA and EMA. Canine equivalent biomarkers have previously been used as surrogate endpoints for rare diseases in Comparative Oncology, which is the study of diseases that occur in humans and animals and have significant genetic homology. Human and Canine osteosarcoma have 96% genetic homology. The Company is preparing to initiate regulatory correspondence with the United States Department of Agriculture (USDA) with the Company's updated manufacturing process which incorporates key improvements covered under a patent that was recently granted a notice of allowance by the United States Patent & Trademark Office (USPTO). Shelter Me is a film that was produced to highlight the important role of human and canine Comparative Oncology disease research. The movie will premiere on April 3, 2025 at AMC Century City in Los Angeles and will later be available to stream through the PBS app and on About Shelter Me Shelter Me: The Cancer Pioneers also features leading scientists at the National Cancer Institute/National Institutes of Health, the University of Pennsylvania, the University of Illinois, the University of Wisconsin, and Colorado State University. It is part of an Emmy Award-winning PBS series, Shelter Me, that celebrates the life-changing relationships between people and animals. A trailer of the movie can be viewed here. About OS Therapies OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other solid tumors. OST-HER2, the Company's lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the US Food & Drug Administration and Fast-Track and Orphan Drug designations from the US FDA and European Medicines Agency. The Company positive data in its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma demonstrating statistically significant benefit in the 12-month event free survival (EFS) primary endpoint of the study. The Company anticipates submitting a Biologics Licensing Application (BLA) to the US FDA for OST-HER2 in osteosarcoma in 2025 and, if approved, would become eligible to receive a Priority Review Voucher that it could then sell. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the U.S. Department of Agriculture for the treatment of canines with osteosarcoma. In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) and Drug Conjugates (DC), known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone Si-Linker and Conditionally Active Payload (CAP) technology, enabling the delivery of multiple payloads per linker. For more information, please visit Forward-Looking Statements Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of the federal securities laws. These forward-looking statements and terms such as "anticipate," "expect," "intend," "may," "will," "should" or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. OS Therapies cautions readers that forward-looking statements are based on management's expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially, including, but not limited to the approval of OST-HER2 by the US FDA and grant of a priority review voucher and other risks and uncertainties described in "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" in the Company's registration statement on Form S-1 filed with the Securities and Exchange Commission (the "SEC") on November 12, 2024, as amended on November 27, 2024, and other subsequent documents we file with the SEC, including but not limited to our Quarterly Reports on Form 10-Q. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. View source version on Contacts OS Therapies Contact Information: Jack Doll571.243.9455Irpr@
Associated Press
05-02-2025
- Business
- Associated Press
Biotech Investing: How PRV's Can Unlock Major Opportunities (OSTX, DAWN, SWTX, PTCT)
In the world of biotechnology investing, companies often struggle to bring new drugs to market. Developing treatments for rare diseases can be incredibly expensive, and because these conditions affect relatively few patients, the financial payoff isn't always clear. To help incentivize drugmakers, the U.S. government created the Priority Review Voucher (PRV) program in 2007. PRVs act as a powerful tool that can significantly speed up the FDA approval process, making them a highly valuable asset—especially to major pharmaceutical companies. A PRV is awarded when a company gains approval for a drug that treats a rare pediatric disease (RPD), a tropical disease, or serves as a medical countermeasure for serious public health threats. Normally, FDA drug approvals take around 10 months, but using a PRV shortens that timeline to just six months. This four-month head start can be a game-changer, allowing companies to get a competitive edge or generate extra sales from blockbuster drugs. Since these vouchers don't expire and can be freely bought and sold, they often end up in the hands of big pharmaceutical companies willing to pay top dollar for the advantage. PRV prices have historically hovered around $100 million, but with uncertainty surrounding the renewal of the rare pediatric disease PRV program, recent sales have surged past $150 million. If the program isn't renewed, PRVs could become even scarcer, potentially driving prices even higher. Big pharma companies are fiercely competing for these vouchers, particularly in lucrative fields like obesity treatments, where shaving months off a drug's approval timeline could mean capturing billions in market share. For small biotech companies, receiving a PRV can be a major financial boost. Take Day One Biopharmaceuticals (NASDAQ: DAWN) as an example. The company was awarded a PRV after its pediatric cancer drug, OJEMDA (tovorafenib), received FDA approval. Rather than using the voucher itself, Day One sold it for $108 million, securing much-needed funding without diluting shareholder value. This kind of transaction highlights why PRVs can be a game-changer for biotech firms—providing an immediate cash infusion that can support further drug development. With PRVs becoming an increasingly hot commodity, investors should keep an eye on biotech companies with potential voucher-earning drugs in development. With PRVs becoming increasingly valuable, several biotech companies could be next in line to benefit. Let's take a look at three stocks with potential PRVs on the horizon. OS Therapies: OS Therapies (NYSE-A: OSTX) is emerging as a strong contender for a Priority Review Voucher (PRV), a potential game-changer for the company. The company is developing OST-HER2, an immunotherapy designed to prevent the spread of osteosarcoma (OS), a rare and aggressive bone cancer that primarily affects children and young adults. Because OS Therapies holds a Rare Pediatric Disease Designation (RPDD) from the FDA, an approval for OST-HER2 would make it eligible for a PRV—potentially bringing in around $150 million in non-dilutive funding. For a small biotech company, this is a game-changing opportunity. 'The data we generated in our Phase 2b trial with OST-HER2 provides the first glimmer of hope in over 40 years that a paradigm shift could radically change the course of this deadly disease,' said CEO Paul Romness. If OS Therapies secures FDA approval, not only would it mark a major breakthrough for osteosarcoma treatment, but the PRV could also provide the financial runway needed for future expansion. OS Therapies has already hit a key milestone, achieving the primary endpoint in its Phase 2b trial for OST-HER2. The treatment demonstrated a 33% Event-Free Survival (EFS) rate at 12 months, compared to just 20% in historical controls. Even more compelling, 91% of patients treated with OST-HER2 were alive at the one-year mark, versus 80% in the control group. These results indicate a significant improvement over existing treatment options, which have remained largely unchanged for decades. With these promising results in hand, OS Therapies plans to file for FDA approval (Biologics Licensing Application, or BLA) in late 2025, with potential approval by mid-2026. If successful, the company would receive a PRV just before the program sunsets, making it one of the last biotechs to benefit from this lucrative incentive. Financial Stability and Growth Potential Despite being a small-cap biotech, OS Therapies has positioned itself well financially. Over the past six months, the company raised $13.1 million through an IPO and a subsequent private placement. These funds are earmarked for final clinical trial payments, commercial manufacturing, and regulatory expenses—all crucial steps toward bringing OST-HER2 to market. Moreover, OS Therapies recently acquired key clinical assets from Ayala, including two additional Listeria-based immunotherapy candidates for lung and prostate cancer. This acquisition not only expands the company's pipeline but also significantly reduces future cash obligations, improving long-term financial prospects. The Bigger Picture Beyond osteosarcoma, OST-HER2 has potential applications in other HER2-positive cancers, including breast cancer and canine osteosarcoma, where it has already received conditional approval from the U.S. Department of Agriculture. Additionally, the company is advancing its tunable Antibody Drug Conjugate (tADC) platform, a next-generation cancer therapy designed to improve targeted drug delivery. With multiple clinical programs, a solid financial strategy, and a real chance at securing a PRV, OS Therapies stands out as a compelling investment opportunity in biotech. If OST-HER2 gains FDA approval, the PRV could provide a significant financial boost—offering the company valuable resources for further development and creating substantial upside potential for investors. SpringWorks Therapeutics: SpringWorks Therapeutics (NASDAQ: SWTX) is a biotech company focused on developing treatments for severe rare diseases and cancer. Its first FDA-approved drug, OGSIVEO® (nirogacestat), treats desmoid tumors, but the company's next major opportunity lies with mirdametinib—a drug currently in development for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN), a rare condition that causes tumors to form along nerves. In the ReNeu trial, mirdametinib showed impressive results in both adults and children, reducing tumor size and improving pain and quality of life. Because of these promising outcomes, the FDA has granted Priority Review status to the drug, with a decision expected by February 28, 2025. If mirdametinib is approved, SpringWorks could earn a Priority Review Voucher (PRV), which would be a highly valuable asset for the company. A PRV allows for a faster approval process, providing a significant advantage for drugmakers. The company could choose to sell the PRV for a substantial financial boost, potentially funding future drug development or pipeline expansion without diluting shareholder value. With SpringWorks poised to potentially earn a PRV through mirdametinib, 2025 could be a pivotal year for the company, positioning it as a leader in rare disease treatments. Investors should keep a close eye on SpringWorks as it works toward FDA approval for mirdametinib and the possible PRV windfall. PTC Therapeutics: PTC Therapeutics, Inc. (NASDAQ: PTCT) is a biopharmaceutical company dedicated to developing innovative treatments for rare diseases. The company has two important drugs that could earn PRVs in the near future, each representing a valuable opportunity for investors. First, vatiquinone, a drug for Friedreich ataxia (FA)—a rare, progressive disease that affects the nervous system—has been submitted for FDA approval. The company believes this drug could fill a big gap in treatment for both children and adults with FA, as there are very few options available right now. Vatiquinone has shown strong results in clinical trials, with evidence of slowing disease progression and improving quality of life. If approved, it could earn PTC a PRV, which could then be sold for a significant financial boost. The second drug, Sepiapterin, is being developed for phenylketonuria (PKU), a rare metabolic disorder that can cause serious developmental issues. PTC recently submitted Sepiapterin for FDA approval, based on promising trial results showing it can help patients manage the disease and even reduce their reliance on strict diets. If this drug gets FDA approval, it could also bring in a PRV for PTC, providing an additional source of funding. With two drugs in the pipeline that have the potential to earn PRVs, PTC Therapeutics is an exciting company to watch. These PRVs could provide significant financial support, helping the company continue its important work in rare disease treatments. Disclaimers: RazorPitch Inc. 'RazorPitch' is not operated by a licensed broker, a dealer, or a registered investment adviser. This content is for informational purposes only and is not intended to be investment advice. The Private Securities Litigation Reform Act of 1995 provides investors a safe harbor in regard to forward-looking statements. Any statements that express or involve discussions with respect to predictions, expectations, beliefs, plans, projections, objectives, goals, assumptions, or future events or performances are not statements of historical fact and may be forward-looking statements. Forward-looking statements are based on expectations, estimates, and projections at the time the statements are made that involve a number of risks and uncertainties that could cause actual results or events to differ materially from those presently anticipated. Forward-looking statements in this action may be identified through the use of words such as projects, foresee, expects, will, anticipates, estimates, believes, understands, or that by statements indicating certain actions & quote; may, could, or might occur. Understand there is no guarantee past performance will be indicative of future results. Investing in micro-cap and growth securities is highly speculative and carries an extremely high degree of risk. It is possible that an investor's investment may be lost or impaired due to the speculative nature of the companies profiled. RazorPitch has been retained and compensated by O S Therapies Inc to assist in the production and distribution of content related to OSTX. RazorPitch is responsible for the production and distribution of this content. It should be expressly understood that under no circumstances does any information published herein represent a recommendation to buy or sell a security. This content is for informational purposes only; you should not construe any such information or other material as legal, tax, investment, financial, or other advice. Nothing contained in this article constitutes a solicitation, recommendation, endorsement, or offer by RazorPitch or any third-party service provider to buy or sell any securities or other financial instruments. All content in this article is information of a general nature and does not address the circumstances of any particular individual or entity. Nothing in this article constitutes professional and/or financial advice, nor does any information in the article constitute a comprehensive or complete statement of the matters discussed or the law relating thereto. RazorPitch is not a fiduciary by virtue of any persons use of or access to this content. Media Contact Email: Send Email City: NAPLES State: Florida Country: United States
