Latest news with #eDSP
Yahoo
3 days ago
- Business
- Yahoo
Quince Therapeutics Exceeds 75% Enrollment in Pivotal Phase 3 NEAT Clinical Trial in Ataxia-Telangiectasia
SOUTH SAN FRANCISCO, Calif., June 03, 2025--(BUSINESS WIRE)--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, today announced that its pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial in the rare neurodegenerative disease Ataxia-Telangiectasia (A-T) has reached an important milestone as enrollment now exceeds 75% with a total of 65 participants randomized in the study's targeted 86 participants in the six to nine year-old primary analysis population. Key Phase 3 NEAT clinical trial enrollment highlights to date include: A total of 76 participants have been enrolled, including 65 participants in the six to nine year-old primary analysis population and 11 participants aged 10 years or older. Study powering based on currently enrolled participants is approximately 80%. The rate of screening and randomization activities has materially increased with the recent activation of all study sites. A total of 108 NEAT participants have been screened to date with a low screen failure rate of 10%. All 37 NEAT participants to date have elected to transition to the NEAT open label extension (OLE) study (NCT06664853/IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study. Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2026. Quince was granted FDA Fast Track designation for the company's eDSP System for the treatment of patients with A-T based on the potential for eDSP to address a high unmet medical need. NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince's lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T. Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. About Ataxia-Telangiectasia A-T is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. There are currently no approved therapeutic treatments in any global market for A-T. About eDSP for A-T eDSP is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient's own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment. eDSP leverages Quince's proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient's own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince's AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. About Quince Therapeutics Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on the company and its latest news, visit and follow Quince on social media platforms LinkedIn, Facebook, X, and YouTube. Forward-looking Statements Statements in this news release contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the "safe harbor" created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as "believe," "may," "should," "expect," "anticipate," "plan," "believe," "estimated," "potential," "intend," "will," "can," "seek," or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including plans and the ability to enroll participants, impact of closing enrollment, conduct, and/or complete current and additional studies; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP; planned regulatory agency submissions, including NDAs, and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company's market opportunity. Forward-looking statements are based on Quince's current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled "Risk Factors" in the company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 13, 2025, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law. View source version on Contacts Media & Investor Contact: Stacy RoughanQuince Therapeutics, President, Corporate Communications & Investor Relationsir@ Sign in to access your portfolio
Business Wire
3 days ago
- Business
- Business Wire
Quince Therapeutics Exceeds 75% Enrollment in Pivotal Phase 3 NEAT Clinical Trial in Ataxia-Telangiectasia
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, today announced that its pivotal Phase 3 NEAT (N eurological E ffects of e DSP on Subjects with A - T; NCT06193200 / IEDAT-04-2022) clinical trial in the rare neurodegenerative disease Ataxia-Telangiectasia (A-T) has reached an important milestone as enrollment now exceeds 75% with a total of 65 participants randomized in the study's targeted 86 participants in the six to nine year-old primary analysis population. Key Phase 3 NEAT clinical trial enrollment highlights to date include: A total of 76 participants have been enrolled, including 65 participants in the six to nine year-old primary analysis population and 11 participants aged 10 years or older. Study powering based on currently enrolled participants is approximately 80%. The rate of screening and randomization activities has materially increased with the recent activation of all study sites. A total of 108 NEAT participants have been screened to date with a low screen failure rate of 10%. All 37 NEAT participants to date have elected to transition to the NEAT open label extension (OLE) study (NCT06664853 / IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study. Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2026. Quince was granted FDA Fast Track designation for the company's eDSP System for the treatment of patients with A-T based on the potential for eDSP to address a high unmet medical need. NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince's lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T. Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. About Ataxia-Telangiectasia A-T is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. There are currently no approved therapeutic treatments in any global market for A-T. About eDSP for A-T eDSP is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient's own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment. eDSP leverages Quince's proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient's own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince's AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. About Quince Therapeutics Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on the company and its latest news, visit and follow Quince on social media platforms LinkedIn, Facebook, X, and YouTube. Forward-looking Statements Statements in this news release contain 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the 'safe harbor' created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as 'believe,' 'may,' 'should,' 'expect,' 'anticipate,' 'plan,' 'believe,' 'estimated,' 'potential,' 'intend,' 'will,' 'can,' 'seek,' or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including plans and the ability to enroll participants, impact of closing enrollment, conduct, and/or complete current and additional studies; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP; planned regulatory agency submissions, including NDAs, and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company's market opportunity. Forward-looking statements are based on Quince's current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled 'Risk Factors' in the company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 13, 2025, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.
Yahoo
05-05-2025
- Business
- Yahoo
Oppenheimer Predicts Up to ~840% Surge for These 2 ‘Strong Buy' Stocks
Wall Street wrapped up the week with a bang, as a solid jobs report and a possible thaw in US-China trade tensions gave investors plenty to cheer about. Discover companies with rock-solid fundamentals in TipRanks' Smart Value Newsletter. Receive undervalued stocks, resilient to market uncertainty, delivered straight to your inbox. The S&P 500 notched its longest bull run in nearly two decades, surging for a ninth consecutive day and fully erasing the losses triggered by President Trump's early-April 'Liberation Day' tariff blitz. Watching the current situation from Oppenheimer, chief investment strategist John Stoltzfus believes that the markets are finally finding their footing after months of uncertainty. 'US equities that had been pretty much locked in worry-worry mode for much of the time since February 19 when the S&P 500 hit its most recent record high found reason to change direction not just on a lessening of the day-to-day tariff war worry, but also on better-than-expected earnings growth… We remain positive on equities with current conditions suggesting we are right about where we should be considering the changes in stateside trade policy that are underway, and the degree of uncertainty change brings,' Stoltzfus opined. Stoltzfus' colleagues among the Oppenheimer stock analysts are running with this positive outlook and advising investors to buy two stocks they believe are primed for major gains – one of which could skyrocket as much as 840%. And they're not alone. TipRanks data shows the broader analyst community is firmly behind these names, handing out 'Strong Buy' ratings and projecting massive upside potential. Let's take a closer look at what makes these stocks so compelling. Quince Therapeutics (QNCX) We'll start with Quince Therapeutics, a late-stage biotech firm taking an innovative approach to rare disease treatment by harnessing the patient's biology. Its lead program, eDSP (formerly EryDex), is a novel formulation of dexamethasone, a well-established corticosteroid valued for its anti-inflammatory power. While dexamethasone is effective, it's also known to cause serious adverse effects, such as adrenal gland suppression, especially with prolonged use. eDSP, however, leverages Quince's proprietary AIDE technology to encapsulate the drug within a patient's own red blood cells, aiming to preserve its efficacy while reducing those side effects. That delivery method – AIDE, or Autologous Intracellular Drug Encapsulation – is where Quince's innovation stands out. Instead of traditional drug delivery routes, AIDE employs red blood cells from the patient to carry therapeutic agents through the bloodstream. This method offers several built-in advantages: improved tolerability, extended circulation time, reduced immune response, and broader tissue exposure. By leveraging the body's natural carriers, AIDE enables the drug to remain active and better tolerated until it reaches its destination. On the clinical trial side, eDSP is currently undergoing a Phase 3 NEAT study in the treatment of ataxia-telangiectasia, or A-T. This is a rare, inherited pediatric disease caused by mutations in the ATM gene that controls cell homeostatic and cell division functions. The gene mutation causes a neurodegenerative and immunodeficiency disorder. Quince plans to enroll 86 A-T patients from ages 6 to 9 years, and another 20 patients aged 10 years or older. Currently, 61 patients are enrolled. Topline results are expected to be ready for release in 4Q25, and assuming a positive outcome, the company plans to make regulatory submissions to the FDA and EMA during 2026. The company is also preparing a Phase 2 clinical trial to assess eDSP as a prospective treatment for Duchenne muscular dystrophy (DMD), a severe inherited condition caused by mutations in the dystrophin gene on the X chromosome. These mutations result in a lack of dystrophin protein, which is essential for muscle function. The company plans to initiate this study by year-end. With shares trading at just $1.06 and a major catalyst on the horizon, Oppenheimer analyst Leland Gershell sees QNCX as deeply undervalued with substantial upside. 'We like the setup into QNCX's Phase 3 results in ataxia-telangiectasia (A-T)… We see a $1B+ global opportunity for lead candidate EryDex [eDSP] and project $200M in 2031 US sales. Our enthusiasm for development and commercial success is driven by our KOL checks, prior clinical data, and a pivotal design we see as heavily derisked. Upside potential stems from EryDex's prospects to meaningfully improve the standard of care in Duchenne muscular dystrophy (DMD), a second indication on which we have good visibility. The company's drug/device platform could serve a growing number of rare disorders over time and offers durable market exclusivity,' the analyst opined. Looking ahead, Gershell emphasizes the attractive setup: 'We see favorable risk-reward and encourage investors to build a position. We would expect positive results in NEAT to yield considerable stock upside potential and enable the company to strengthen its capital base by attracting strong interest from dedicated healthcare institutional investors.' Gershell backs his bullish stance on QNCX with an Outperform (i.e., Buy) rating and a $10 price target, implying a massive ~840% upside over the next 12 months. (To watch Gershell's track record, click here) Overall, the stock earns a unanimous thumbs up from the analyst consensus, with 3 recent Buy reviews supporting a Strong Buy rating. The average price target of $6.67 implies a potential gain of 529% from current levels. (See QNCX stock forecast) Sarepta Therapeutics (SRPT) The next stock catching Oppenheimer's attention is Sarepta Therapeutics, a cutting-edge biotech firm specializing in precision genetic medicine. With a strong focus on rare diseases, Sarepta has emerged as a leader in developing breakthrough treatments for Duchenne muscular dystrophy. Backed by a robust pipeline, Sarepta is advancing a wide range of drug candidates currently in human clinical trials, with multiple gene therapy programs in the mix. The latest expansion of that pipeline comes through a collaboration with Arrowhead Pharmaceuticals, announced last November. The deal gives Sarepta exclusive global rights to seven of Arrowhead's programs – four already in clinical stages and three in preclinical development. These include drug candidates with potential as best-in-class siRNA treatments for myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). While there is much to say about Sarepta, perhaps the most notable issue involves its commercial drug, Elevidys. Approved in June 2023, Elevidys is the first, and currently only, gene therapy approved for the treatment of DMD. The big news on this front came in March of this year, when Sarepta reported the death of a patient who had been treated with Elevidys. The cause of death was acute liver failure. Although liver damage is a known potential side effect of Elevidys and other gene therapies in its class, this marked the first reported fatality associated with the drug. As a result of the reported patient death, the European Medicines Agency (EMA) has temporarily paused several ongoing clinical trials of Elevidys. Conducted by Sarepta in collaboration with Roche, these trials aim to support label expansion of the already approved drug. Enrollment and dosing have been halted while Sarepta and Roche review the data and work to determine the exact cause of the fatality. Looking at the financial side, Sarepta's latest earnings report – covering 4Q24 – shows the company generating strong revenue and turning a profit. The top line came in at $658.4 million, up 66% year-over-year and beating the forecast by $27.37 million. This total included $638.2 million in net product revenue, a 75% increase from the prior year. At the bottom line, Sarepta reported non-GAAP EPS of $1.90, more than double the 4Q23 figure – although it fell 16 cents short of expectations. Investors won't have to wait long for the next update, with Q1 earnings set to be released on Tuesday, May 6. In his coverage of Sarepta for Oppenheimer, analyst Andreas Argyrides sees plenty of reasons to feel optimistic about the stock, despite the headwind of the tragic patient death. 'Feedback from a KOL call with a leading neurologist supports ELEVIDYS' favorable benefit/risk profile as the only gene therapy approved in DMD. Overall, the KOL was not surprised by the first patient death following treatment with ELEVIDYS, since acute liver injury is a known side effect of AAV-based gene therapies. While another fatality is possible, he sees little risk of ELEVIDYS being pulled or restricted, expects an expanded label for children <4-y/o, and his overall perception remains unchanged. We view the recent selloff in the stock, pricing in revisions to ELEVIDYS sales, as overdone, driven by initial fears following the death and EMA's temporary clinical hold and see an opportunity for a rebound potentially supported by the share repurchase of up to $500M… We believe SRPT is investing considerable resources to become the leader in gene therapy and muscular dystrophies in particular,' Argyrides stated. Argyrides goes on to rate Sarepta's shares as Outperform (i.e., Buy), with a $184 price target that points toward a one-year gain of ~190%. (To watch Argyrides' track record, click here) Wall Street is largely in agreement. SRPT holds a Strong Buy consensus rating based on 22 recent analyst reviews, including 18 Buys and 4 Holds. With shares currently trading at $63.51, the $148.25 average price target suggests a 133% upside over the next year. (See SRPT stock forecast) To find good ideas for stocks trading at attractive valuations, visit TipRanks' Best Stocks to Buy, a tool that unites all of TipRanks' equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment. Disclaimer & DisclosureReport an Issue Sign in to access your portfolio
Business Insider
03-05-2025
- Business
- Business Insider
Oppenheimer Predicts Up to ~840% Surge for These 2 ‘Strong Buy' Stocks
Wall Street wrapped up the week with a bang, as a solid jobs report and a possible thaw in US-China trade tensions gave investors plenty to cheer about. Protect Your Portfolio Against Market Uncertainty The S&P 500 notched its longest bull run in nearly two decades, surging for a ninth consecutive day and fully erasing the losses triggered by President Trump's early-April 'Liberation Day' tariff blitz. Watching the current situation from Oppenheimer, chief investment strategist John Stoltzfus believes that the markets are finally finding their footing after months of uncertainty. 'US equities that had been pretty much locked in worry-worry mode for much of the time since February 19 when the S&P 500 hit its most recent record high found reason to change direction not just on a lessening of the day-to-day tariff war worry, but also on better-than-expected earnings growth… We remain positive on equities with current conditions suggesting we are right about where we should be considering the changes in stateside trade policy that are underway, and the degree of uncertainty change brings,' Stoltzfus opined. Stoltzfus' colleagues among the Oppenheimer stock analysts are running with this positive outlook and advising investors to buy two stocks they believe are primed for major gains – one of which could skyrocket as much as 840%. And they're not alone. TipRanks data shows the broader analyst community is firmly behind these names, handing out 'Strong Buy' ratings and projecting massive upside potential. Let's take a closer look at what makes these stocks so compelling. Quince Therapeutics (QNCX) We'll start with Quince Therapeutics, a late-stage biotech firm taking an innovative approach to rare disease treatment by harnessing the patient's biology. Its lead program, eDSP (formerly EryDex), is a novel formulation of dexamethasone, a well-established corticosteroid valued for its anti-inflammatory power. While dexamethasone is effective, it's also known to cause serious adverse effects, such as adrenal gland suppression, especially with prolonged use. eDSP, however, leverages Quince's proprietary AIDE technology to encapsulate the drug within a patient's own red blood cells, aiming to preserve its efficacy while reducing those side effects. That delivery method – AIDE, or Autologous Intracellular Drug Encapsulation – is where Quince's innovation stands out. Instead of traditional drug delivery routes, AIDE employs red blood cells from the patient to carry therapeutic agents through the bloodstream. This method offers several built-in advantages: improved tolerability, extended circulation time, reduced immune response, and broader tissue exposure. By leveraging the body's natural carriers, AIDE enables the drug to remain active and better tolerated until it reaches its destination. On the clinical trial side, eDSP is currently undergoing a Phase 3 NEAT study in the treatment of ataxia-telangiectasia, or A-T. This is a rare, inherited pediatric disease caused by mutations in the ATM gene that controls cell homeostatic and cell division functions. The gene mutation causes a neurodegenerative and immunodeficiency disorder. Quince plans to enroll 86 A-T patients from ages 6 to 9 years, and another 20 patients aged 10 years or older. Currently, 61 patients are enrolled. Topline results are expected to be ready for release in 4Q25, and assuming a positive outcome, the company plans to make regulatory submissions to the FDA and EMA during 2026. The company is also preparing a Phase 2 clinical trial to assess eDSP as a prospective treatment for Duchenne muscular dystrophy (DMD), a severe inherited condition caused by mutations in the dystrophin gene on the X chromosome. These mutations result in a lack of dystrophin protein, which is essential for muscle function. The company plans to initiate this study by year-end. With shares trading at just $1.06 and a major catalyst on the horizon, Oppenheimer analyst Leland Gershell sees QNCX as deeply undervalued with substantial upside. 'We like the setup into QNCX's Phase 3 results in ataxia-telangiectasia (A-T)… We see a $1B+ global opportunity for lead candidate EryDex [eDSP] and project $200M in 2031 US sales. Our enthusiasm for development and commercial success is driven by our KOL checks, prior clinical data, and a pivotal design we see as heavily derisked. Upside potential stems from EryDex's prospects to meaningfully improve the standard of care in Duchenne muscular dystrophy (DMD), a second indication on which we have good visibility. The company's drug/device platform could serve a growing number of rare disorders over time and offers durable market exclusivity,' the analyst opined. Looking ahead, Gershell emphasizes the attractive setup: 'We see favorable risk-reward and encourage investors to build a position. We would expect positive results in NEAT to yield considerable stock upside potential and enable the company to strengthen its capital base by attracting strong interest from dedicated healthcare institutional investors.' Gershell backs his bullish stance on QNCX with an Outperform (i.e., Buy) rating and a $10 price target, implying a massive ~840% upside over the next 12 months. (To watch Gershell's track record, click here) Overall, the stock earns a unanimous thumbs up from the analyst consensus, with 3 recent Buy reviews supporting a Strong Buy rating. The average price target of $6.67 implies a potential gain of 529% from current levels. (See QNCX stock forecast) Sarepta Therapeutics (SRPT) The next stock catching Oppenheimer's attention is Sarepta Therapeutics, a cutting-edge biotech firm specializing in precision genetic medicine. With a strong focus on rare diseases, Sarepta has emerged as a leader in developing breakthrough treatments for Duchenne muscular dystrophy. Backed by a robust pipeline, Sarepta is advancing a wide range of drug candidates currently in human clinical trials, with multiple gene therapy programs in the mix. The latest expansion of that pipeline comes through a collaboration with Arrowhead Pharmaceuticals, announced last November. The deal gives Sarepta exclusive global rights to seven of Arrowhead's programs – four already in clinical stages and three in preclinical development. These include drug candidates with potential as best-in-class siRNA treatments for myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). While there is much to say about Sarepta, perhaps the most notable issue involves its commercial drug, Elevidys. Approved in June 2023, Elevidys is the first, and currently only, gene therapy approved for the treatment of DMD. The big news on this front came in March of this year, when Sarepta reported the death of a patient who had been treated with Elevidys. The cause of death was acute liver failure. Although liver damage is a known potential side effect of Elevidys and other gene therapies in its class, this marked the first reported fatality associated with the drug. As a result of the reported patient death, the European Medicines Agency (EMA) has temporarily paused several ongoing clinical trials of Elevidys. Conducted by Sarepta in collaboration with Roche, these trials aim to support label expansion of the already approved drug. Enrollment and dosing have been halted while Sarepta and Roche review the data and work to determine the exact cause of the fatality. Looking at the financial side, Sarepta's latest earnings report – covering 4Q24 – shows the company generating strong revenue and turning a profit. The top line came in at $658.4 million, up 66% year-over-year and beating the forecast by $27.37 million. This total included $638.2 million in net product revenue, a 75% increase from the prior year. At the bottom line, Sarepta reported non-GAAP EPS of $1.90, more than double the 4Q23 figure – although it fell 16 cents short of expectations. Investors won't have to wait long for the next update, with Q1 earnings set to be released on Tuesday, May 6. In his coverage of Sarepta for Oppenheimer, analyst Andreas Argyrides sees plenty of reasons to feel optimistic about the stock, despite the headwind of the tragic patient death. 'Feedback from a KOL call with a leading neurologist supports ELEVIDYS' favorable benefit/risk profile as the only gene therapy approved in DMD. Overall, the KOL was not surprised by the first patient death following treatment with ELEVIDYS, since acute liver injury is a known side effect of AAV-based gene therapies. While another fatality is possible, he sees little risk of ELEVIDYS being pulled or restricted, expects an expanded label for children <4-y/o, and his overall perception remains unchanged. We view the recent selloff in the stock, pricing in revisions to ELEVIDYS sales, as overdone, driven by initial fears following the death and EMA's temporary clinical hold and see an opportunity for a rebound potentially supported by the share repurchase of up to $500M… We believe SRPT is investing considerable resources to become the leader in gene therapy and muscular dystrophies in particular,' Argyrides stated. Argyrides goes on to rate Sarepta's shares as Outperform (i.e., Buy), with a $184 price target that points toward a one-year gain of ~190%. (To watch Argyrides' track record, click here) Wall Street is largely in agreement. SRPT holds a Strong Buy consensus rating based on 22 recent analyst reviews, including 18 Buys and 4 Holds. With shares currently trading at $63.51, the $148.25 average price target suggests a 133% upside over the next year. (See SRPT stock forecast) To find good ideas for stocks trading at attractive valuations, visit TipRanks' Best Stocks to Buy, a tool that unites all of TipRanks' equity insights.
