Home Care In Sarepta, LA

When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. But they didn't think it was anything serious.

"He would fall a lot. Not like a lot a lot. But he had trouble climbing playground equipment. He fell down the stairs at his pre school a couple of times," says his mother, Susan Finazzo, 40, who lives in Miami.

Chase's younger brother, Dylan, was more agile. But he started walking a little late.

"We're not thinking anything of it. He's just got some weak ankles. He's got some little tiny little orthotics for toddlers," she says. "He's sees an orthopedic surgeon. We've got PT. We're all over it."

So Susan and her husband Chris Finazzo were stunned when doctors told them both boys had Duchenne muscular dystrophy. While rare, the disease is the most common inherited neuromuscular disorder among children. It affects an estimated 10,000 to 12,000 children in the U.S.

The disease, which almost exclusively affects boys, destroys muscles. Most boys end up in wheelchairs before they become teenagers. Eventually, their hearts and lungs give out. Most people with the disease die in their 30s or 40s. It's incurable.

"It's absolutely devastating," says Susan Finazzo. "You end up going through a mourning process. You're mourning the loss of the life that your child should have had."

Finazzo couldn't help but wonder of her children: "Are they going to be able to go to college? Are they going to live that long? Are they going to be able to fall in love? Have children of their own? You thought you were going to have soccer practices. And now you're wondering: How much time am I going to have with them?"

Finazzo and her husband started giving their sons steroids to help them even though the drugs can cause side effects and can't slow down the disease. They also started looking for better options.

Finally, they volunteered Chase, who's now 8, and Dylan, who's now 5, for a study testing an experimental gene therapy for Duchenne muscular dystrophy.

The gene therapy works by infusing trillions of harmless viruses that has been genetically modified to ferry a gene to patients' muscles. The gene produces a miniature version of a protein called dystrophin, that boys with muscular dystrophy are missing or don't have enough of. The hope is this "micro-dystrophin" will at least help slow the progression of the disease.

"I hope that this can extend their lives a little bit. At the end of the day as a Duchenne parent, you don't even care about the wheelchair anymore. You don't care about the not walking," Finazzo says. "I just want them to be alive longer. I want them to be alive long enough to enjoy what life is. So if this moves the needle in their life expectancy — even if it does it for a day — it's worth it."

But there's an intense debate about this. Sarepta Therapeutics, the Cambridge, Mass., company that developed the treatment, wants the Food and Drug Administration to approve the gene therapy largely based on how much micro-dystrophin it produces in patients' muscles — without direct evidence that's actually helping alleviate symptoms and prevent disease progression.

The FDA has convened a committee of independent advisors to consider whether to recommend the approval on May 12.

The approval would come through a process known as accelerated approvals. That lets the FDA approve promising treatments quickly before strong evidence is available that the therapy helps, as long as companies promise to then conduct follow-up studies to prove it works.

"We're dealing with a very serious rare disease. A deadly disease. Every day matters to these patients," says Douglas Ingram, Sarepta's CEO. "This could be the most important therapy so far developed for children with Duchenne muscular dystrophy. We have, at least from our perspective, the perfect opportunity to use the accelerated approval pathway."

But this accelerated approval process is controversial because some companies fail to follow-through on their promises to confirm their treatments work. A drug approved this way to prevent premature birth was recently withdrawn after being found useless.

Sarepta's gene therapy for muscular dystrophy would be the first gene therapy approved through the accelerated approval process. And the request has reportedly triggered intense debate within the FDA.

Sarepta has yet to complete the required follow-up studies for three other treatments focused on dystrophin for muscular dystrophy previously approved through the accelerated approval process, according to Dr. Reshma Ramachandran, who studies drug approvals at the Yale School of Medicine.

That leaves open the question of whether micro-dystrophin levels are an accurate way to assess treatments for the the disease, she says.

"We're still left uncertain whether or not these very expensive treatments actually yield a meaningful clinical benefit, or if we're just simply still guessing," Ramachandran says.

"This question's been looming since 2016: Is dystrophin an appropriate proxy measure for clinical benefit for Duchenne muscular dystrophy. That's still a looming question. And the fact that it's, you know, seven years later and we still don't have an answer is a bit appalling," she says.

If it turns out not to help, Ramachandran worries the approval could discourage the development of other treatments that could work and preclude patients from being eligible for those therapies.

And then there's the issue of cost. Though the company hasn't released expected costs yet, other recently approved gene therapies have cost as much as $3 million to treat each patient.

The costs, which may not always be covered by insurance, could drain money families could otherwise use for much-needed treatment, such as physical therapy and in-home care-givers, she says.

"That's real harm," she says.

Dr. Glen Nuckolls at the National Institute of Neurological Disorders and Stroke said he couldn't comment directly on Sarepta's request. But he too expressed reservations about relying on a surrogate marker.

"There's certainly data to suggest that you start making dystrophin it's going to result in regeneration of the tissue and increased strength. But I think the relationship of what exact level is needed, we'd probably like to have some more data on that," Nuckolls says.

But Ingram, Sarepta's CEO, says waiting for more data would come with a cost.

"During that period of waiting, some six to 800 kids will die. Some 600 to 800 kids will be consigned to a power wheelchair for the rest of their lives. Six hundred to 800 kids will be consigned to permanent ventilation," Ingram says. "All of these kids ... will be irreparably damaged in ways we won't be able to reverse."

The company says it has preliminary data indicating the gene therapy is helping patients, and already has a big study to confirm those finding well underway.

The company's request is being supported by groups like the Muscular Dystrophy Association.

"Surrogate endpoints like dystrophin measurements allow more access," says Dr. Barry Byrne from the University of Florida, the association's chief medical advisor. "An analogy would be a drug that lowers cholesterol is expected to have an improvement in cardiovascular mortality. So it's a similar analysis."

"We believe that it is slowing the disease and hopefully stabilizing these patients," says Debra Miller, who heads CureDuchenne, a patient advocacy group that helped fund Sarepta. "We have high hopes that at least for many years we'll be able to arrest the downward progression of this disease through gene therapy."

For her part, Susan Finazzo knows it's possible the drug won't help her sons, but she's hopeful.

"I'm just so excited that we even got the opportunity to take part in this [study]. Because the majority of the kids can't. So this is this is an amazing opportunity," she says.

Each year at her sons' birthdays, she's reminded of how little time they have to help them. "Birthdays are especially hard because they're bitter sweet," Finazzo says. You're so happy but then you're also knowing that that's one less year with them. That's just a reminder of the clock's that ticking."

The digital version of this story was edited by Carmel Wroth; broadcast edited by Scott Hensley.

Copyright 2023 NPR. To see more, visit https://www.npr.org.

We believe that the stock price of Sarepta Therapeutics (NASDAQ: SRPT), a biotech company focused on the discovery and development of RNA-targeted therapeutics and gene therapy for the treatment of rare diseases, is undervalued at current levels of $71. SRPT stock is down 20% off the March 2020 bottom compared to the S&P which has moved 85%. SRPT stock has significantly underperformed the broader markets, primarily due to a development around its pipeline. Earlier in January 2021 the company announced an unfavorable outcome from clinical trials for one of its drugs, SRP-9001, used for treating Duchenne muscular dystrophy, and SRPT stock crashed 52% in a single trading session following the announcement. However, now that the stock has corrected (down 40% in the last one year) despite revenue growing 42% y-o-y over the last four quarters, we believe SRPT stock is likely to see higher levels. Our dashboard ’Buy Or Fear Sarepta Therapeutics Stock’ provides the key numbers behind our thinking.

Looking at a longer time period, SRPT stock is actually down 35% from levels seen toward the end of 2018. The decline in stock price over the last two years or so can be attributed to unfavorable changes in the company’s P/S multiple. The company’s revenues have trended higher, rising 79% from $301 million in 2018 to $540 million in 2020, led by higher demand for its DMD treatments. While the company posted strong revenue growth over the recent years, it also issued more shares, resulting in a 18% jump in total shares outstanding. As such, on a per share basis, revenue grew 53% from $4.54 in 2018 to $6.93 in 2020. Despite the strong RPS growth, the company’s P/S multiple has actually contracted, falling from levels of over 24x seen between 2018 and 2020 to 10x currently.

Outlook

2020 has been a solid year for Sarepta’s revenue growth, courtesy of its DMD treatments. The development around SRP-9001 surely is negative for the company, as there will likely be a two year delay in the study, and a treatment from Pfizer PFE -0.6% is now expected to take the lead. However, Sarepta has a large pipeline with 42 programs, and SRP-9003, used to treat a type of muscular dystrophy, could potentially be a blockbuster treatment, after the company announced positive results from clinical trials. Also, the U.S. FDA has approved Amondys 45, a drug used to treat certain patients with DMD. Amondys is the third drug from Sarepta, after Exondys 51 and Vyondys 53, all used for the treatment of DMD, and these combined cover 30% of the DMD patients in the U.S.

Looking forward, Sarepta will likely see an increase in demand for its DMD treatments, given the market is large, and it is expected to grow at a CAGR of 11% over the next few years. While the Covid-19 related lockdowns had earlier impacted expansion of several drugs in 2020, due to a delay in new patient enrollments, the trend has largely reversed over the recent months. Now that 40% of the U.S. population has already received at least one dose of a Covid-19 vaccine, the U.S. in particular is fast approaching toward normalcy. That said, any further recovery in the economy and its timing hinge on the broader containment of the coronavirus spread. We are now seeing a large spike in Covid-19 cases in some of the countries, such as India and Brazil, owing to the new variants of the virus. Our dashboard Trends In U.S. Covid-19 Cases provides an overview of how the pandemic has been spreading in the U.S. and contrasts with trends in Brazil and Russia.

Finally, looking at the valuation, we believe that SRPT stock is undervalued at the current levels of $71. Currently, investors seem to be worried about the pipeline and revenue dependency only on DMD treatments. However, going by the numbers, Sarepta’s revenues are estimated to grow a solid 65% between 2020 and 2022. This means that on a per share basis, the company’s revenue will grow from $6.93 in 2020 to around $11.45 in 2022. At the current price of $71, SRPT stock is trading at just 9x its 2021 and 6x its 2022 RPS, compared to levels of 24x seen over the recent years, implying that SRPT stock is undervalued currently. To some extent the decline in the P/S multiple is justified given the delay in SRP-9001, but the selling now appears to be overdone, and we believe the stock will likely see a strong rebound in the near term.

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While SRPT stock may see higher levels, 2020 has created many pricing discontinuities which can offer attractive trading opportunities. For example, you’ll be surprised how counter-intuitive the stock valuation is for Catalent vs. Emergent Biosolutions.

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/EIN News/ -- -- Agreement leverages Sarepta’s leadership in gene therapy for neuromuscular and cardiovascular diseases and Dyno’s CapsidMap artificial intelligence platform to design AAV vectors --

CAMBRIDGE, Mass., May 11, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, and Dyno Therapeutics, Inc., a biotech company applying artificial intelligence (AI) to gene therapy, today announced an agreement to develop next-generation Adeno-Associated Virus (AAV) vectors for muscle diseases, using Dyno’s CapsidMap™ platform.

AI and machine learning technologies have the potential to deliver enhanced vectors for gene therapies. Dyno’s proprietary CapsidMap platform opens up new ways to identify novel capsids – the cell-targeting protein shell of viral vectors – that could offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing.

“Sarepta’s world-leading gene therapy engine is founded on three pillars: developing a broad portfolio of programs to treat rare diseases; our first-in-class manufacturing expertise; and investment in advancing and further improving the science of gene therapy to help patients in need of more options. To that end, our agreement with Dyno provides us with another valuable tool to develop next-generation capsids for gene therapies to treat rare diseases,” said Doug Ingram, Sarepta’s President and Chief Executive Officer. “By leveraging Dyno’s AI platform and Sarepta’s deep expertise in gene therapy development, our goal is to advance next-generation treatments with improved muscle-targeting capabilities.”

Under the terms of the agreement, Dyno will be responsible for the design and discovery of novel AAV capsids with improved functional properties for gene therapy and Sarepta will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. If successful, Dyno could receive over $40 million in upfront, option and license payments during the research phase of the collaboration. Additionally, if Sarepta develops and commercializes multiple candidates for multiple muscle diseases, Dyno will be eligible for additional significant future milestone payments. Dyno will also receive royalties on worldwide net sales of any commercial products developed through the collaboration.

“This agreement is a major step forward in our plan to realize the potential of Dyno’s AI platform for gene therapies to improve patient health. We are excited to work with Sarepta to create gene therapies with improved properties to address a range of muscle-related diseases,” stated Dyno’s CEO and co-founder Eric D. Kelsic, Ph.D. “The success of the gene therapies developed through this collaboration with Sarepta will rely on AI-powered vectors that allow gene therapies to be safely and precisely targeted to the muscle tissue.”

About CapsidMap™ for Designing AAV Gene Therapies By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next-generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

Dyno’s technology platform builds on certain intellectual property developed in the lab of George Church, Ph.D., who is Robert Winthrop Professor of Genetics at Harvard Medical School (HMS), a Core Faculty member at Harvard’s Wyss Institute for Biologically Inspired Engineering, and a co-founder of Dyno. Several of the technical breakthroughs that enabled Dyno’s approach to optimize synthetic AAV capsid engineering were described in a November 2019 publication in the journal Science, based on work conducted by Dyno founders and members of the Church Lab at HMS and the Wyss Institute. Dyno has an exclusive option to enter into a license agreement with Harvard University for this technology.

About Dyno TherapeuticsDyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. The company’s proprietary CapsidMap™ platform is designed to rapidly discover and systematically optimize superior Adeno-Associated Virus (AAV) capsid vectors with delivery properties that significantly improve upon current approaches to gene therapy and expand the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit www.dynotx.com for additional information.

About Sarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Sarepta Therapeutics Forward-looking StatementsThis press release contains "forward-looking statements." 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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the potential of artificial intelligence and machine learning technologies to deliver enhanced vectors for gene therapies; the potential of the CapsidMap™ platform to offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing; the agreement between Sarepta and Dyno Therapeutics providing a valuable tool to develop next-generation capsids for gene therapies to treat rare disease; the parties’ goal to advance next-generation treatments with improved muscle-targeting capabilities; the parties’ responsibilities under the agreement and potential payments to Dyno Therapeutics; and the potential of AI-powered vectors to allow gene therapies to be safely and precisely targeted to the muscle tissue.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and Dyno Therapeutics may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development. In particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement; the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates; possible limitations of company financial and other resources; manufacturing limitations that may not be anticipated or resolved for in a timely manner; regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2019 and 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.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta's 2019 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

ContactsFor Sarepta: Investors: Ian Estepan, 617-274-4052, [email protected]Media: Tracy Sorrentino, 617-301-8566, [email protected]

For Dyno:Kathryn MorrisThe Yates Network[email protected]914-204-6412

MINDEN, La. -- A Sarepta man, convicted on two charges of rape, may never see freedom again.

District Judge Michael Craig on Monday sentenced Michael Duck, 54, to life in prison without benefit of probation, parole or suspension of sentence for the first degree rape of a child under 13. He also gave Duck a consecutive sentence of 38 years hard labor for one count of second-degree rape.

Webster Parish Assistant District Attorney Hugo Holland pointed out that Duck must be convicted to life for the first-degree rape; however, he added it would be up to the judge to determine sentencing for the other rape conviction.

Defense attorney Eric Johnson asked the judge to consider making the sentences concurrent.

“The court required a pre-sentence investigation, and saw in 2010 you were found guilty of possession of Schedule II, making you a second and third felony offender,” Craig told Duck. “You didn’t testify, and you were given the opportunity to respond in pre-sentencing and declined.”

Craig went on to point out that by the sworn testimonies, the offenses were separate and subsequent, multiple times over a period of time with both victims.

“Based on your actions, the damage you did to these two girls for the rest of their lives, and the damage you did to other girls that you are not going to be held accountable, that is going to impact them,” the judge continued. “You impacted five or six young girls – and those are the ones we know about– for the rest of their lives.

“Not to mention their families,” he continued. “Because they are all related to you in some form or fashion. It will have a ripple effect in your family.”

Duck was arrested in 2016, following an extensive investigation by the Webster Parish Sheriff’s Office when one of the minor victims contacted the WPSO and reported that her stepfather had been sexually assaulting her.

During the course of the investigation, multiple other family members revealed they were also sexually assaulted by Duck.

The jury heard testimony from Detective Teresa Rogers and Detective Heather Boucher, both experts in the fields of clinical psychology, DNA analysis and sexual assault examinations as well as from multiple other victims who came forward during the investigation.

Duck has 30 days to appeal his sentence.

Duck’s son, Michael Dragland, 35, was also sentenced Monday on two counts of second-degree rape to which he enter a guilty plea.

“I believe if you had gone to trial and been convicted, your sentence would’ve been much greater than it’s about to be,” Craig told Dragland in court. “This sentence is being handed down pursuant to a plea agreement, and I’m going to honor that plea agreement.”

Dragland will serve 20 years at hard labor, two of those years to be served without benefit of probation, parole or suspension of sentence.