Myomo, Inc. (NYSE American: MYO)("Myomo" or the "Company"), a wearable medical robotics company that offers increased functionality for those suffering from neurological disorders and upper-limb paralysis, today announced that the Centers for Medicare & Medicaid Services ("CMS"), through their regional contractors known as the DME MACs, have remitted and paid lump sum reimbursements to the Company and are beginning to reimburse orthotics and prosthetics ("O&P") providers for MyoPros delivered to Medicare Part B beneficiaries.
The amounts paid were based on the fees published by CMS for the Company's Healthcare Common Procedures Coding System ("HCPCS") codes L8701 and L8702, which became effective on April 1, 2024. All four regional DME MAC's have remitted lump sum reimbursements, and payments have been received for a number of patients of the Company and O&P providers based on the published fee schedule.
Q1 2024 Revenue: Reported at $92.8 million, a 34.9% increase year-over-year, surpassing the estimated $85.6 million.
Net Loss: Recorded at $35.4 million for Q1 2024, an improvement from a net loss of $53.3 million in Q1 2023, and better than the estimated net loss of $43.01 million.
Earnings Per Share (EPS): Reported at -$0.17, better than the estimated -$0.21.
ORLADEYO Net Revenue: Grew by 30% year-over-year to $88.9 million, driving the majority of the quarter's revenue.
Research and Development (R&D) Expenses: Decreased by 3.9% year-over-year to $46.5 million, reflecting strategic cuts and reallocation.
Selling, General and Administrative (SG&A) Expenses: Increased by 24% year-over-year to $59.4 million, due to expansion efforts.
2024 Financial Outlook: Adjusted full-year ORLADEYO revenue guidance to $390-$400 million, indicating strong performance and market confidence.
Outset Medical, Inc. (Nasdaq: OM) (“Outset”), a medical technology company pioneering a first-of-its-kind technology to reduce the cost and complexity of dialysis, announced today that the U.S. Food and Drug Administration (FDA) has granted 510(k) clearance of TabloCartTM with prefiltration, an innovative optional accessory for the Tablo® Hemodialysis System.
There was no immediate comment from Israel on the Qatar-Egypt brokered deal, but Israeli media outlet N12 reported the deal was “unacceptable” to Netanyahu’s war cabinet.
After the release of the statement, Palestinians erupted in cheers in the sprawling tent camps around Rafah, hoping the deal meant an Israeli attack had been averted.
The news comes hours after Rafah’s population were struck with panic-inducing posters which read: “The IDF is about to operate with force against the terror organisations in the area you currently reside, as the IDF has operated so far.
“Anyone in the area puts themselves and their family members in danger. For your safety, evacuate immediately to the expanded humanitarian area in Al-Mawasi.”
The order to evacuate has been widely condemned by the international community and aid organisations, with UNICEF warning there is “nowhere safe” for 600,000 children in Rafah to go.
A record 8,000 attendees are expected at this week’s27th Annual Meetingof the American Society of Gene & Cell Therapy in Baltimore to discuss the opportunities and challenges facing the industry. Billed as the largest gathering of gene and cell therapy professionals, ASGCT 2024 starts tomorrow and will run through the end of the week, bringing together stakeholders from therapeutic developers and investors to patient advocates, policymakers and regulators.
I, along with Managing Editor Jef Akst, will be on-site, aiming to assess where the field stands now and what near- and longer-term breakthrough technologies are on the horizon. And I have to say, I’m excited. These relatively new modalities have taken biopharma by storm and continue to dominate the news with clinical results demonstrating breakthrough potential in a range of indications and FDA efforts to accommodate the anticipated flood of regulatory applications, as well as safety concerns and manufacturing challenges.
ASGCT President Jeffrey Chamberlain told BioSpace that the annual meeting will have a record 2,000 abstracts this year on a variety of topics.
“This is going to be the largest meeting we’ve ever had,” Chamberlain said. “There are an increasing number of approved therapies and this really gives us a chance to highlight all of those.” He added that there are more than 20 gene therapies that have been approved worldwide—half of those in the United States—and more than 60 cell therapies approved globally with many of those in the U.S.
FDA’s View of Cell and Gene Therapies Is Evolving
On the regulatory front, 2023 was a banner year for cell and gene therapies (CGTs), with seven approvals in the U.S. and one in the European Union. And all signs point to 2024 surpassing last year. This year, the FDA has given the green light to four new CGTs and has several upcoming PDUFA dates for other novel therapies.
Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, will be speaking in two sessions at ASGCT 2024.
On Wednesday morning, Marks will be featured in a fireside chat on regulating CGTs, hosted by Kristin Van Goor, executive director and U.S. head of global regulatory policy and innovation at Takeda Pharmaceuticals. As the FDA’s top biologics regulator, Marks has been an advocate for regulatory flexibility with regard to CGTs for rare diseases.
Van Goor told BioSpace that she and Marks will discuss “all the hot topics from IND to BLA.” Specifically, she continued, “I plan to front-load questions on areas of rapidly evolving policy and precedent.”
Marks will also participate in a Thursday morning fireside chat on global regulatory convergence that includes officials from the Brazilian Health Regulatory Agency and Japan’s Pharmaceuticals and Medical Devices Agency. Marks has previously argued that disparate CGT requirements among different countries—from preclinical tests to manufacturing—make it difficult for patients in certain areas of the world to have access to therapies, particularly for rare diseases.
Basic Science, Translational Research
Beyond clinical and regulatory affairs, Chamberlain said ASGCT 2024’s agenda will also highlight pivotal basic science and genetics—including translational studies—and the researchers who are making breakthroughs.
“We’re going to have some really exciting talks on what’s coming out of the basic science laboratories, what’s moving into more advanced translational work and what is close to clinical trials,” Chamberlain said. “We’re going to really span the whole spectrum.”
In a keynote address on Wednesday morning, Kevin Campbell, director of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center at the University of Iowa’s Carver College of Medicine, will speak about the mechanism of muscular dystrophies and development of therapeutic strategies to restore muscle function.
Campbell has “led the way for years” in understanding the mechanisms underlying several muscular dystrophies, Chamberlain said, and will talk “about the interaction between basic science and the development of gene therapies.”
Another point of discussion will be the Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration between the National Institutes of Health, the FDA, industry and patient groups to help accelerate the delivery of gene therapies for rare diseases. BGTC’s primary goal is to improve understanding of the basic biology of adeno-associated virus (AAV) vectors to optimize bioproduction technologies and increase their therapeutic efficacy.
While AAV vectors are one of the safest platforms and most common tools for gene delivery, there are challenges in producing sufficient quantity and quality of such gene therapy products. An AAV manufacturing workshop will be held Tuesday morning at ASGCT 2024 providing an overview of how early process development decisions (pre-Phase I), including the basics of chemistry, manufacturing, and controls (CMC) considerations for gene therapies, can translate into clinical success.
“There’s a couple of presentations at the meeting this year on new vector technologies. You know, getting away from the same old ones that most people are using,” Chamberlain said. “I think that’s going to be really important—to develop new vectors. And there are a number of exciting talks [at ASGCT 2024] and I think breakthroughs that we’re going to be hearing about in the next year in developing nonviral delivery systems.”
In December 2023, the FDA approved the first gene therapies for sickle cell disease, including the first-ever CRISPR therapy. Gene, epigenetic and RNA editing will also be a big part of the agenda at ASGCT 2024 with several sessions focused on CRISPR technology, as well as the need for greater progress in making in vivo genome editing scalable to treat a lot more patients.
CGT Challenges, Opportunities
ASGCT 2024 comes at a pivotal time for the sector. In January 2024, the FDA pushed for a class-wide boxed warning on all commercial BCMA-directed and CD19-directed CAR T cell immunotherapies due to the “risk of T cell malignancies, with serious outcomes, including hospitalization and death.” The agency continues to investigate 22 reported cases of T cell malignancies in patients who received CAR-T therapies.
“Anytime you hear reports of adverse events or things that are unanticipated it’s a concern we need to pay attention to,” Chamberlain said. “But the number of incidences appears to be quite low . . . you have to look at risk versus benefit.”
Indeed, the safety concerns have not deterred oncologists, drug developers or investors, and Chamberlain noted that ASGCT 2024 received a “very large number of abstracts” for this year’s annual meeting on the development of CAR T cell therapies. He said there is also work on CAR NK cells. CAR T-cell therapies have become a game changer for some patients with blood cancer. Now, Chamberlain said, “People are trying to adapt . . . CAR technologies more towards solid organs.”
Meanwhile, Sarepta’s Elevidys has faced scrutiny not for safety concerns but for questionable efficacy. In a controversial regulatory decision in June 2023, the FDA granted accelerated approval to the gene therapy as the first for Duchenne muscular dystrophy (DMD), despite Elevidys having missed the primary functional endpoint. The regulator’s decision was based on data showing it increased expression of the micro-dystrophin protein, a biomarker the agency determined is “reasonably likely to predict clinical benefit” in DMD patients four to five years of age.
Four months after the approval, however, Elevidys failed a confirmatory trial. Still, Sarepta is persisting with its application seeking full approval and an expanded label for the therapy. The FDA has set a PDUFA date of June 21.
“Sarepta did a fantastic job in figuring out how to scale this up and start to apply it effectively in the clinic—and you know, we’re seeing a lot of progress,” commented Chamberlain, whose lab at the University of Washington designed a micro-dystrophin that Sarepta’s approved therapy uses. “One of the things that we’ve seen is some of the kids that are being treated with this new therapy are doing tremendously well. Unfortunately, others are not doing quite as well, and we’re still trying to get a handle on that.”
Biden’s administration is working overtime to ensure his health care priorities are protected from a potential second Trump White House.
In recent weeks, regulatory agencies have been racing against the clock to finalize some of their most consequential policies, such as abortion data privacy, antidiscrimination protections for transgender patients and nursing home minimum staffing.
At issue is the Congressional Review Act (CRA), a fast-track legislative tool that allows lawmakers to nullify rules even after the executive branch has completed them. The CRA also bars agencies from pursuing “substantially similar” rules going forward, unless Congress orders it.
Rules can be protected if they are finished before the “look-back” window opens in the last 60 legislative days of the 2024 session. But because of the quirks of the congressional calendar, nobody will likely know when that is until after Congress adjourns for the year.
According to a review from the George Washington University, that window has often fallen between May and August, most regularly occurring in July.
Most of the major rules that supporters of the administration were concerned about were published in April. Advocacy groups praised the White House for finalizing regulations they said will protect vulnerable populations.
“The administration is advancing important work with respect to health care, affordability, and access,” said Ben Anderson, deputy senior director of health policy at the left-leaning consumer advocacy group Families USA. “If rules aren’t finalized soon enough in the calendar, then everything’s sort of at risk of being undone by a future Congress.”
Resolutions of disapproval are not subject to filibuster rules and need only a simple majority of the House and Senate to agree in order to pass. If the president signs the resolution, regulations can be undone in days, rather than the months or years it would take going through the normal notice-and-comment period.
If former President Trump wins again and ushers in GOP control of Congress in 2025, the CRA could be a powerful tool to undo the agenda of the Biden White House.
“We don’t know what’s going to happen in November. So I’m not sure that we’d necessarily think about legacy at this point. But what we’re seeing are really important advances to protecting access and affordability for health care,” Anderson said.
The CRA was passed in 1996, a part of then-Speaker Newt Gingrich’s (R-Ga.) “Contract with America.” Republicans have used it more than Democrats, though prior to 2017, Congress had only used it once to repeal a final rule.
“[CRA] wasn’t on people’s radar the way it is now. We were aware of it, but we weren’t thinking about the deadline the way they are now,” said Susan Dudley, former administrator of the Office of Information and Regulatory Affairs under former President George W. Bush.
But during the Trump administration, Congress used the CRA to overturn 16 rules issued toward the end of former President Obama’s term, including one involving family planning grants.
That history has likely led to a scramble among agencies.
Dudley, who is the founder of the George Washington University Regulatory Studies Center, said there were more major rules issued in April than in any other month since 1981.
“I think what we’re seeing that’s different this year is there’s so much more awareness, especially because we know who the nominee is by now, and we know he’s used CRA before. And so I think there’s just much more acute awareness of this deadline,” Dudley said.
Among the rules health care advocates had been pushing hard for were ones that would expand protections under the Health Insurance Portability and Accountability Act, or HIPAA, privacy regulation for people seeking abortions, as well as another to protect LGBTQ patients from discrimination. Both were released last week and could be likely targets of a future Trump administration.
Liz McCaman Taylor, senior federal policy counsel at the Center for Reproductive Rights, said she had been most anticipating the abortion privacy rule, which blocks health providers and insurers from disclosing protected health information to state officials to aid in the investigation, prosecution or suing of someone who sought or provided an abortion.
“It really responds to the moment we’re in post-Dobbs, where people are traveling for care, but also, technology is such that … my health care data travels with me,” McCaman Taylor said. “Your data can follow you, but that could also haunt you in a situation where providers are truly very fearful of the consequences.”
The antidiscrimination rule itself reinstated and expanded protections that had been gutted under a rule from the Trump administration. So even if a rule can’t be overturned with the CRA, there are still other pathways. Lawsuits filed in conservative courts can also nullify the rules, especially if a new administration takes over and decides not to defend a policy it disagrees with.
Dania Douglas, a senior attorney at the National Health Law Program, said she had been concerned that the administration wouldn’t be able to finish key rules before the CRA “look-back period,” especially because of the uncertainty about when it would fall.
But that hasn’t been the case.
“The Biden administration has been doing a lot of work around health care equity … in the last two weeks, so many of the rules that provide these really critical protections have been issued,” Douglas said.
She specifically referenced a rule bolstering antidiscrimination protections in health care for people with disabilities, something that hadn’t been updated in nearly 50 years.
“I think the Biden-Harris administration was very aware of this CRA deadline and worked very hard to try to get these rules out in April … at a time when they think it will hopefully be safe from the CRA look-back period,” Douglas said.
The incarcerated women looked eager as they gathered outside the building where they would attend a course on Black feminism.
The class of 11 convened last month in a room at the Ohio Reformatory for Women (ORW) that was mostly empty except for chairs, with one big desk with a computer on it for an administrator.
“We are all hungry for knowledge,” Amber Swain, one of the inmates, told The Hill.
The course, one of several offered at the prison, will be part of a discussion Monday with a group of Ohio State University professors as the school looks to ramp up its prison education degree program following the implementation of Pell Grants for prisoners last summer.
Mary Thomas, director of Ohio State University’s Ohio Prison Education Exchange Project, told the class the meeting is an opportunity for the professors to learn from them as much as the other way around.
“The Ohio State University just received its accreditation for a new degree site at the Ohio Reformatory for Women. The degree will be a Bachelor of Arts in women’s, gender and sexuality studies. This summer, OSU will submit an application to the U.S. Department of Education for Pell Grant utilization at the site to begin in academic year 2025-2026,” Thomas said. “We are currently exploring funding opportunities to begin next academic year, so we may begin in autumn semester 2024.”
Last summer, the Department of Education announced it would allow Pell Grants for prisoners again, after they were barred from the money since 1994.
Thomas said every individual education program has to get countless approvals — from the prison system, the school they’re working with, the state government and at last the Department of Education — making the process a slow and laborious one.
“I know one program who has been waiting for approval from the [Department of Education] for five months,” she told The Hill.
The effort to get Pell Grants to prisoners started back in 2015 under the Obama administration, when the Second Chance Pell Experiment began. That program gave federal funding to a limited number of incarcerated people to pursue their education.
Then, in 2020, Congress voted to repeal part of the 1994 Pell, allowing for access to the grants to be greatly expanded. When the FAFSA Simplification Act was passed in 2020, it defined what a prison education program would be and what qualifications a program needed to get that title.
The first step toward prisoners getting Pell Grants is for the classes they attend to get approved as a federal prison education program (PEP), and applications for that approval opened in July.
But the Education Department has so far approved only one institution as a PEP: the California State Polytechnic University’s Humboldt’s Bachelor of Arts in communication at Pelican Bay State Prison, though it hopes to approve 50 more by 2025.
A department spokesperson said more resources will become available soon to help programs apply for the PEP.
“The Department expects to have data about the PEP once participating schools offering approved programs have reported information about confined and incarcerated students enrolled in a PEP. The Department expects to be able to report PEP data — at the earliest — fall 2024,” the spokesperson said.
In the meantime, Ohio State has been offering free courses to the women at ORW, who had to go through an interview process to be selected due to high demand.
Swain said she has also taken gender studies, African American history and creative writing courses with OSU, emphasizing the classes will “not only help when I get out” but that they are useful in prison as she “unlearns societal norms.”
The students at ORW range from women who are in prison for just a few years to those with a life sentence. This group has read four to five books on Black feminism since August.
Arianna Cannon, who is incarcerated for 15 years to life, says “anything that we adapt, we pour back into our communities” and that their education “impacts the people we meet and have in our lives.”
Inmates Arianna Cannon and Amber Swain discuss their presentation on discrimination for OSU professors.
Studies have shown education programs reduce recidivism rates, and Thomas emphasized how even those serving life sentences can affect the prison culture and create a more stable environment.
“The expansion of Pell Grants is just beginning to rectify some of the damage caused by the near-total elimination of higher education programs in prison after 1994,” said Marc Howard, a professor of government and law at Georgetown University, during a recent discussion about prison education with the Brookings Institution. “For nearly 30 years, incarcerated people have had extremely limited access to college, leaving them with almost no avenues to pursue a higher education. We can’t even begin to measure the negative impact of decades’ worth of lost opportunities.”
During the ORW class, the women were told by Tiyi Morris, a professor of African American studies at OSU, to split off into groups to discuss what topics they would be presenting at the workshop with the school’s other representatives.
When they pulled the chairs back in a circle, the women had many different ideas, such as discussing mass incarceration, transphobia, homelessness, climate change and classicism, among other subjects.
They sounded anxious about having to present to professors, with one inmate drawing general agreement when she exclaimed, “How am I going to teach a teacher?”
The women broke off into groups again to talk about what “freedom dreaming” meant to them after reading “Freedom Dreams: The Black Radical Imagination” by Robin D. Kelly. Freedom dreaming is defined by a focus on what a person wants the future to be and what tools they need to make that a reality.
Mary Thomas and Tiyi Morris lead the class discussion on how students can combine topics and weave their ideas together for the workshop.
Inmate Danielle Ennis said, “No one has ever asked me that question before,” specifically mentioning the life she wants to see for her daughter.
“I closed my eyes and realized the future of what I can do here and now, but also what I can do in the future,” Ennis said.
