Latest news with #DM1
Yahoo
13 hours ago
- Business
- Yahoo
Sarepta Therapeutics Announces Advancement of siRNA Collaboration and Sale of Arrowhead Equity Investment
- Safety review leading to dose escalation and achievement of first predetermined enrollment target in Phase 1/2 study of SRP-1003 for DM1 triggers $100 million milestone payment - Sarepta sells at least $174 million of Arrowhead common stock for cash proceeds, Arrowhead will redeem $50 million of stock from Sarepta to prefund half of $100 million milestone payment due to Arrowhead CAMBRIDGE, Mass., August 13, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has sold 9,265,312 shares of common stock of Arrowhead Pharmaceuticals, Inc. ("Arrowhead") in a privately negotiated block trade. The Company expects to receive at least $174 million in gross proceeds from the block trade. In addition, Sarepta entered into an agreement with Arrowhead pursuant to which it will transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the Company's previously announced $100 million milestone payment obligation. "We are very pleased with the progress of our potentially best-in-class siRNA programs, including the advancement of our SRP-1003 program for DM1, triggering the $100 million milestone payment to our partner, Arrowhead," said Doug Ingram, chief executive officer, Sarepta. "The sale of our equity investment is a strategic decision to help fund this milestone but does not change our conviction in the utility of the siRNA approach and our confidence in the work Arrowhead is doing to apply this technology across several disease states. We look forward to sharing early data from our FSHD and DM1 programs in the second half of this year." The Company's $100 million milestone payment obligation was triggered following a review of the safety data and Arrowhead achieving the first of two predetermined enrollment targets, in the Phase 1/2 clinical study of SRP-1003, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Sarepta expects to release preliminary data from the Phase 1/2 study of SRP-1003 in the second half of 2025. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements In order to provide Sarepta's investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "may," "intends," "prepares," "looks," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our liquidity position, business plans, priorities and research and development programs and technologies; our Phase 1/2 clinical study of SRP-1003; the potential benefits of our technologies and scientific approaches; and expected plans and milestones, including clinical data readouts and milestones expected in 2025 for multiple programs. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, the COVID-19 pandemic and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
14 hours ago
- Business
- Business Wire
Sarepta Therapeutics Announces Advancement of siRNA Collaboration and Sale of Arrowhead Equity Investment
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has sold 9,265,312 shares of common stock of Arrowhead Pharmaceuticals, Inc. ('Arrowhead') in a privately negotiated block trade. The Company expects to receive at least $174 million in gross proceeds from the block trade. In addition, Sarepta entered into an agreement with Arrowhead pursuant to which it will transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the Company's previously announced $100 million milestone payment obligation. 'We are very pleased with the progress of our potentially best-in-class siRNA programs, including the advancement of our SRP-1003 program for DM1, triggering the $100 million milestone payment to our partner, Arrowhead,' said Doug Ingram, chief executive officer, Sarepta. 'The sale of our equity investment is a strategic decision to help fund this milestone but does not change our conviction in the utility of the siRNA approach and our confidence in the work Arrowhead is doing to apply this technology across several disease states. We look forward to sharing early data from our FSHD and DM1 programs in the second half of this year.' The Company's $100 million milestone payment obligation was triggered following a review of the safety data and Arrowhead achieving the first of two predetermined enrollment targets, in the Phase 1/2 clinical study of SRP-1003, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Sarepta expects to release preliminary data from the Phase 1/2 study of SRP-1003 in the second half of 2025. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements In order to provide Sarepta's investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as 'believes,' 'anticipates,' 'plans,' 'expects,' 'will,' 'may,' 'intends,' 'prepares,' 'looks,' 'potential,' 'possible' and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our liquidity position, business plans, priorities and research and development programs and technologies; our Phase 1/2 clinical study of SRP-1003; the potential benefits of our technologies and scientific approaches; and expected plans and milestones, including clinical data readouts and milestones expected in 2025 for multiple programs. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, the COVID-19 pandemic and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading 'Risk Factors' in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Yahoo
30-05-2025
- Business
- Yahoo
PepGen price target lowered to $1 from $3 at BofA
BofA lowered the firm's price target on PepGen (PEPG) to $1 from $3 and keeps an Underperform rating on the shares after the company announced it will be discontinuing development of Duchenne muscular dystrophy programs following 'disappointing' 10mg/kg data for PGN-EDO51. PepGen will now focus developmental efforts on its DM1 program, which the firm thinks 'still needs significant de-risking,' the analyst tells investors. The firm updated its model for Q1 results, cash and share count and removed value for DMD from its model based on the discontinuation. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO Sign in to access your portfolio
Yahoo
30-05-2025
- Business
- Yahoo
PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target
PepGen (PEPG) announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy patients amenable to exon 51 skipping, the company will focus on advancing its 'promising' myotonic dystrophy type 1 program currently in Phase 2 clinical development. The company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities. In the 10 mg/kg cohort of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26%; however, total dystrophin only increased to 0.59% of normal levels. The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. 'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.' PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO PepGen Inc.'s Promising Advances in DMD and DM1 Programs Justify Buy Rating Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
28-05-2025
- Business
- Business Wire
PepGen to Focus on Development of Promising DM1 Program Following 10 mg/kg PGN-EDO51 Update
BOSTON--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies, today announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping, the Company will focus on advancing its promising myotonic dystrophy type 1 (DM1) program currently in Phase 2 clinical development. The Company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities. In the 10 mg/kg cohort (n=4) of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26% (a mean increase of 3.5%); however, total dystrophin only increased to 0.59% of normal levels (a mean increase of 0.36%). The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. 'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.' Paul Streck, MD, MBA, Executive Vice President, Head of R&D of PepGen continued, 'PGN-EDODM1, PepGen's investigational drug in development for DM1, has already demonstrated robust target engagement after a single 10 mg/kg dose in patients that resulted in mean mis-splicing correction of 29% with a favorable emerging safety profile as of February 24, 2025, the most recent safety update. Mis-splicing is the primary driver of DM1 pathology and currently patients have no approved treatment options for this disabling, life-shortening disorder. Going forward, we will focus our resources on advancing the Company's ongoing DM1 clinical program along with our research pipeline.' PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The Company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures. About PGN-EDODM1 PGN-EDODM1, PepGen's investigational candidate in development for the treatment of myotonic dystrophy Type 1 (DM1), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to restore the normal splicing function of MBNL1, a key RNA splicing protein. PGN-EDODM1 is designed to directly address the deleterious effects of cytosine-uracil-guanine (CUG) repeat expansion in the dystrophia myotonic protein kinase (DMPK) transcripts which sequester MBNL1, by binding to the pathogenic CUG trinucleotide repeat expansion present in the DMPK transcripts, disrupting the binding between the CUG repeat expansion and MBNL1. 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 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 PGN-EDO51 and PGN-EDODM1, our plans to discontinue our DMD-related research and development activities, including our CONNECT1-EDO51 and CONNECT2-EDO51 Phase 2 clinical trials, based on results from the CONNECT1 trial, the design and conduct of clinical trials, including our FREEDOM-DM1 Phase 1 trial and FREEDOM2-DM1 Phase 2 trial, and the expected timing for additional data from our FREEDOM Phase 1 trial and initial data from our FREEDOM2 Phase 2 trial. 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; our ability to enroll patients in our clinical trials, including FREEDOM and FREEDOM2; 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; our product candidates, including PGN-EDODM1, 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 and FREEDOM2 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-EDO51 and PGN-EDODM1, 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 or any other investigational therapy will successfully complete clinical development or gain regulatory authority approval.
