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Business Wire
08-05-2025
- Business
- Business Wire
PepGen Reports First Quarter 2025 Financial Results and Recent Corporate Highlights
BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results and recent corporate highlights for the quarter ended March 31, 2025. 'Our EDO platform's unique ability to efficiently deliver oligonucleotides into the nuclei of cells is the foundation of our differentiated pipeline, with promising clinical data from two programs underscoring what we believe to be the broad potential of our technology,' said James McArthur, PhD, President and CEO of PepGen. 'This is a pivotal and catalyst-rich 12 months for PepGen and for our DM1 and DMD clinical programs. Following the highly encouraging initial results from our FREEDOM-DM1 study reported in February, demonstrating potentially best-in-class splicing correction after a single therapeutic dose, we look forward to sharing data from the 15 mg/kg cohort in the second half of the year. We also expect to report dystrophin production data from the 10 mg/kg cohort of our CONNECT1-EDO51 study in patients with DMD in the third quarter. Collectively, we believe these data will build on the body of evidence supporting our platform's promise. Additionally, during the first quarter, we have undertaken various measures to extend our cash runway while prioritizing delivering on our key clinical milestones.' Recent Program Updates PGN-EDODM1: Myotonic Dystrophy Type 1 (DM1) Phase 1 FREEDOM-DM1 Single Ascending Dose (SAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: In February, the Company reported positive initial clinical data from the 5 mg/kg and 10 mg/kg dose cohorts from the ongoing FREEDOM trial. At day 28 following a single dose, PGN-EDODM1 was observed to have a favorable emerging safety profile and robust, dose-dependent splicing correction in the 5 and 10 mg/kg dose cohorts. PepGen expects to report safety, 28-day splicing and functional benefit data from the 15 mg/kg cohort during the second half of 2025. Phase 2 FREEDOM2-DM1 Multiple Ascending Dose (MAD) Randomized, Placebo-Controlled Clinical Trial of PGN-EDODM1: The Company expects to report results from the 5 mg/kg cohort of the FREEDOM2 trial in the first quarter of 2026. PGN-EDO51: Duchenne Muscular Dystrophy (DMD) Phase 2 CONNECT1-EDO51 Open-Label MAD Clinical Trial of PGN-EDO51: PepGen expects to report additional safety as well as dystrophin production data from the 10 mg/kg cohort of the CONNECT1 trial in DMD patients in the third quarter of 2025. Phase 2 CONNECT2-EDO51 MAD Clinical Trial of PGN-EDO51: In March 2025, the Company announced its voluntary decision to pause the Phase 2 CONNECT2 trial in DMD patients until it can review results from the 10 mg/kg cohort in the ongoing Phase 2 CONNECT1 trial. Corporate Updates In March 2025, PepGen announced that Lisa Wyman and Mitchell H. Finer, PhD, joined the Company's Board of Directors. Each are industry veterans bringing decades of executive and operational experience in life sciences to the Company. In March 2025, the Company gave two oral presentations and presented five posters at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. The oral presentations featured data from the ongoing CONNECT1 clinical trial in DMD and the FREEDOM clinical trial in DM1. In May 2025, the Company made an oral presentation and presented two posters at the 2025 Myotonic Dystrophy Foundation (MDF) Conference. PepGen also presented at the 5 th Edition of Euro-DyMA's Pharma Day, held in conjunction with the MDF Conference. These presentations highlighted the recently announced FREEDOM clinical data in DM1, as well as proof-of-mechanism preclinical results for PGN-EDODM1. Financial Results for the Three Months Ended March 31, 2025 Cash, Cash Equivalents and Marketable Securities were $97.8 million as of March 31, 2025. Based on currently planned operations, the Company believes that its existing cash, cash, equivalents, and marketable securities will be sufficient to fund its operations for at least 12 months from the date of this release. Research and Development Expenses were $25.4 million for the three months ended March 31, 2025, compared to $14.7 million for the same period in 2024. General and Administrative Expenses were $5.9 million for the three months ended March 31, 2025, compared to $5.1 million for the same period in 2024. Net Loss was $30.2 million, or $(0.92) basic and diluted net loss per share, for the three months ended March 31, 2025, compared to $18.0 million, or $(0.63) basic and diluted net loss per share, for the same period in 2024. PepGen had approximately 32.7 million shares outstanding on March 31, 2025. Upcoming Potential Milestones 3Q 2025: CONNECT1-EDO51 data from 10 mg/kg cohort expected 2H 2025: FREEDOM-DM1 data from 15 mg/kg cohort expected 1Q 2026: FREEDOM2-DM1 data from the 5 mg/kg cohort expected About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of DM1, utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic oligonucleotide that is designed to restore the normal splicing function of MBNL1, a key RNA splicing protein. DM1 is a progressively disabling, life-shortening genetic disorder. DM1 is estimated to affect 40,000 people in the United States, and over 74,000 people in Europe. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations for the treatment of patients with DM1. About PGN-EDO51 PGN-EDO51, PepGen's investigational candidate in development for the treatment of DMD, utilizes the Company's proprietary EDO technology to deliver a therapeutic oligonucleotide that is designed to target the root cause of this devastating disease. PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a truncated, yet functional dystrophin protein. The U.S. Food and Drug Administration has granted PGN-EDO51 both Orphan Drug and Rare Pediatric Disease Designations for the treatment of patients with DMD amenable to an exon-51 skipping approach. About PepGen PepGen is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases. PepGen's EDO platform is founded on over a decade of research and development and leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. Using these EDO peptides, we are generating a pipeline of oligonucleotide therapeutic candidates designed to target the root cause of serious diseases. For more information, please visit Follow PepGen on LinkedIn and X. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by words such as 'aims,' 'anticipates,' 'believes,' 'could,' 'estimates,' 'expects,' 'forecasts,' 'goal,' 'intends,' 'may,' 'plans,' 'possible,' 'potential,' 'seeks,' 'will,' and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential and safety profile of our product candidates, including, based on early data, PGN-EDODM1 and PGN-EDO51, the expected timing for additional data reports from our FREEDOM Phase 1 trial and CONNECT1 Phase 2 trial and initial data from our FREEDOM2 Phase 2 trial, ongoing and planned regulatory interactions, and our financial resources and expected cash runway. Any forward-looking statements in this press release are based on current expectations, estimates and projections only as of the date of this release 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. These risks and uncertainties include, but are not limited to risks related to: delays or failure to successfully initiate or complete our ongoing and planned development activities for our product candidates, including PGN-EDODM1 and PGN-EDO51; our ability to enroll patients in our clinical trials, including FREEDOM, FREEDOM2 and CONNECT2; that our interpretation of clinical and preclinical study results may be incorrect, or that we may not observe the levels of therapeutic activity in clinical testing that we anticipate based on prior clinical or preclinical results, including for PGN-EDODM1 and PGN-EDO51; our product candidates, including PGN-EDODM1 and PGN-EDO51, may not be safe and effective or otherwise demonstrate safety and efficacy in our clinical trials; adverse outcomes from our regulatory interactions, including delays in regulatory review, clearance to proceed or approval by regulatory authorities with respect to our programs, including clearance to commence planned clinical studies of our product candidates, or other regulatory feedback requiring modifications to our development programs, including in each case with respect to our FREEDOM, FREEDOM2, CONNECT1 and CONNECT2 clinical trials; changes in regulatory framework that are out of our control; unexpected increases in the expenses associated with our development activities or other events that adversely impact our financial resources and cash runway; and our dependence on third parties for some or all aspects of our product manufacturing, research and preclinical and clinical testing. Additional risks concerning PepGen's programs and operations are described in our most recent annual report on Form 10-K and quarterly report on Form 10-Q that are filed with the SEC. PepGen explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. This release discusses PGN-EDODM1 and PGN-EDO51, investigational therapies that have not been approved for use in any country and is not intended to convey conclusions about their efficacy or safety. There is no guarantee that PGN-EDODM1, PGN-EDO51 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval. Expand
Yahoo
29-01-2025
- Business
- Yahoo
Scholar Rock Announces the Appointment of Industry Leader Lisa Wyman as Chief Technical and Quality Officer and Planned Leadership Transition
- Lisa Wyman brings more than 20 years of leadership and functional experience to new role of Chief Technical and Quality Officer on the Executive Team at Scholar Rock - Ted Myles, Chief Operating Officer & Chief Financial Officer, will continue to operate through transition phase to a new organizational structure CAMBRIDGE, Mass., January 29, 2025--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, announced today that Lisa Wyman has been appointed to the new role of Chief Technical and Quality Officer (CTQO) to support the Company's ongoing growth and evolution to becoming a fully integrated biopharma company as it prepares for its first expected drug approval and commercial launch in 2025. Ms. Wyman brings more than 20 years of leadership and functional experience in technical and quality operations, including executive team responsibilities in her most recent roles at Generate:Biomedicines and Acceleron Pharma. She joins Scholar Rock's Executive Team, reporting directly to Jay Backstrom, M.D., MPH, President and Chief Executive Officer. As the Company transitions to a new organizational structure, the role of Chief Operating Officer (COO) is being eliminated, and the Company has initiated a search for a Chief Financial Officer (CFO). Ted Myles, COO & CFO at Scholar Rock, will remain with the Company through mid-March, and then serve as a Senior Advisor for six months, during the leadership transition. "I am delighted to have Lisa join Scholar Rock and further fortify our readiness to bring apitegromab to the market for patients and continue to expand our technical and quality operations for our earlier stage clinical pipeline," said Dr. Backstrom. "We are at a critical and exciting stage in our evolution from a historically R&D focused company, to planning for our first anticipated commercial product launch. Lisa's leadership and technical expertise are instrumental for us to achieve our mission to discover, develop, and deliver therapies to improve people's lives and make a meaningful difference for people living with serious diseases." Dr. Backstrom added, "I am grateful to Ted for his many contributions to Scholar Rock during a very important period of organizational growth and transformation and helping us on our path to becoming a fully integrated biopharma company. On behalf of the Board of Directors and the entire Executive Team, I would like to thank Ted for his strong leadership and his continued support through this transition period. We wish him continued success in his next industry leadership role." Ms. Wyman joins Scholar Rock from Generate:Biomedicines where she served as Chief Technical Operations Officer, and a member of the Company's Executive Leadership Team. While at Generate, she was responsible for establishing and building out the global technical operations organization to support its pursuit of developing a broad portfolio of medicines for patients. Her role included leading multi-faceted teams across Chemistry, Manufacturing, and Controls (CMC), technical development, quality, supply operations. Prior to Generate, Ms. Wyman served as the Senior Vice President of Technical Operations and Quality, and member of the Executive Committee at Acceleron, where she helped to transform the enterprise from a research and development-focused company to a fully integrated biotech, which led to the eventual sale of the company to Merck & Co. in 2021 for over $11 billion. At Acceleron, she led the CMC strategy for the accelerated development of sotatercept (marketed as WINREVAIR) for pulmonary arterial hypertension and supported the launch of Acceleron's first commercial product Reblozyl. Before joining Acceleron, she had roles of increasing responsibility at Mersana Therapeutics, Shire, and Boston Scientific. She graduated from Ithaca College with a degree in biology and earned a master's degree in engineering management from Tufts University. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at and follow @ScholarRock and on LinkedIn. The efficacy and safety of apitegromab has not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock's future expectations, plans and prospects, including without limitation, Scholar Rock's expectations regarding its progress and plans for apitegromab and the ability of any individual to affect the performance of the Company. The use of words such as "may," "could," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. All such forward-looking statements 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. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 3 trial of apitegromab, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; Scholar Rock's ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock's nonclinical and preclinical studies and clinical trials; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock's ability to obtain, maintain and protect its intellectual property; the success of Scholar Rock's current and potential future collaborations; Scholar Rock's dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; Scholar Rock's ability to manage expenses and to obtain additional funding when needed to support its business activities; its ability to establish or maintain strategic business alliances; its ability to receive priority or expedited regulatory review or to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in the United States of apitegromab in the fourth quarter of 2025; as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock's subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock's views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. View source version on Contacts Scholar Rock: Investors Rushmie NofsingerScholar Rockrnofsinger@ ir@ 857-259-5573 Media Molly MacLeodScholar Rockmmacleod@ media@ 802-579-5995 Sign in to access your portfolio
