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Access to quality, consistent care is imperative for patients with diabetes to maintain their health. Having an ongoing care plan is the best way for patients to avoid complications — one of the most common being diabetic foot ulcers, which can lead to the partial or whole amputation of the foot.

The Centers for Disease Control and Prevention’s most recent data on the diabetes-related amputations, from 2016, shows that there were 4.9 lower-extremity amputations per 1,000 diabetic adults in the U.S. Among diabetic people who have had a lower-extremity amputation, more than half may end up having an amputation of the opposite extremity within five years, according to the agency.

The American Diabetes Association announced an initiative last week aimed at tackling this issue. The nonprofit launched the Amputation Prevention Alliance — a three-year effort to decrease the number of diabetes-related amputations in the country — along with five partner organizations.

Those partners include diabetic foot care provider Podimetrics, wound care company Advanced Oxygen Therapy and membership-based medical society Critical Limb Ischemia Global Society, as well as medical device companies Abbott and Cardiovascular Systems.

The majority of diabetes-related amputations are preventable, according to Jon Bloom, Podimetrics’ CEO and co-founder. 

As diabetes advances within a patient, they may experience nerve damage called diabetic neuropathy, in which damaged tissue struggles to heal. Peripheral neuropathy — nerve damage that leads to sores on the feet and legs first — is the most common form of diabetic neuropathy, according to Bloom. The hands and arms can also be affected later, he said.

Patients at risk for diabetic foot ulcers might not even feel a sore forming on their foot. When this is the case, the sore continues to get worse and does not heal well due to increased blood sugar levels, Bloom pointed out. These sores often advance to require diabetic amputation — an issue Bloom said is “simply not talked about enough.”

To combat the public health issue of preventable diabetes-related amputations, the alliance will prioritize patient education about the signs and symptoms associated with diabetic foot ulcers and diabetic neuropathy. It will also work with providers to ensure their patients with complex diabetes have regular check-ups, including appointments with a podiatrist that are focused primarily on foot health, Bloom said.

The Amputation Prevention Alliance plans to advocate for needed policy changes, one example of important legislation being the The Amputation Reduction and Compassion Act. The bill, which was introduced in Congress last year, would provide coverage of peripheral artery disease (PAD) screening for at-risk beneficiaries under the Medicare and Medicaid programs without cost-sharing requirements.

“By expanding coverage for PAD screening, the bill would help prevent vulnerable individuals from developing serious complications from PAD, which can lead to amputation,” Bloom said. “The ARC Act would also prohibit the use of amputation without the completion of testing to determine if alternative options could be utilized to benefit the patient and establish a PAD education program.”

The alliance has also formed a clinical advisory working group to produce recommendations mapping to policy change, clinician education programs and improved patient engagement, according to Bloom. 

The initiative will focus on reaching providers and patients in communities facing disproportionately high rates of amputations and amputated-related mortality, such as the Black and Latino communities. Black people in the U.S. face amputation rates up to four times higher than White Americans, and Latino people are 50% more likely to have an amputation, according to the announcement.

To Bloom, the alliance’s success will be determined by one metric: whether or not it can reduce the more than 154,000 amputations that occur every year in the U.S.

Photo: gustavofrazao, Getty Images

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Healthcare is an industry designed to provide essential services and care for patients to improve their health and well-being. Over generations, the expertise of medical professionals has allowed the industry to continuously innovate and meet the ever-changing demands and needs of patients — ranging from spikes in mental health to safety concerns regarding an in-person doctor’s visit during a global pandemic. Most recently, the Covid-19 pandemic has shown the agility of the healthcare industry to innovate, as we have experienced a surge of medical startups emerging to accommodate new demands and gaps in the industry with digital innovations. There are a number of digital-first methods that improve the healthcare experience for patients, from remote patient monitoring to tech-enabled patient care, virtual reality in the operating room, and more.

As digital health companies solidify their services and technologies as a preference for many, and innovations continue to be developed, it’s critical for healthcare startups to understand the needs of patients and physicians. Companies can hear those important perspectives during innovation development for a successful product or treatment that ultimately propels the healthcare industry.

Improving R&D with the voice of patients and physicians

Many new medical tools and technologies being developed ultimately end up in a doctor’s office, prompting the need for startups to collaborate with physicians during the research and development phase. By engaging physicians in this critical stage, startups can ensure physicians feel confident in adopting and implementing the new innovations within their own practice.

Beyond verifying medical accuracy, physicians can provide exclusive insights to help navigate any number of strategic issues or healthcare challenges that may be unseen to a startup, as well as inform any gaps in a new tool or technology — such as a potential feature or add-on recommendation — based on what they are seeing in the field.

Complementing their medical expertise, physicians also serve as the voice of the patient; with a growing emphasis on tools and technologies being developed specifically for patient use and improved health outcomes, physicians are a valuable asset in informing startups with unique patient perspectives during the R&D phase. Physicians are in constant communication with patients, which provides them with a front row seat to their evolving healthcare needs and desires, as well as feedback or concerns regarding industry innovations at large. With an inside look into what patients need, and the ability to identify consistent trends among patient groups, physicians can share invaluable insights to startups that reflect today’s patient and help optimize new tools or technologies.

Maximizing go-to-market strategies

While the healthcare landscape is known for constant change, these transformations have created a new type of patient. Increasingly, patients are much savvier and more engaged with their personal healthcare journeys; and they are investing the time to become better informed about any health product, tool or treatment. As such, startups should approach their innovations and market strategy with the understanding that patients will do their own research and read the fine print before buying into a new product or technology.

While physicians can ensure medical accuracy during the R&D phase, moving forward these experts can help maximize startups’ go-to-market strategy. Particularly in healthcare, it can be challenging to simplify a new product or technology into layman’s terms without removing any critical medical information pertaining to use. Physicians can help startups identify key messages to include in communication and product materials, as well as common questions or concerns that may arise from patients to include in an FAQ. A physician’s — and startup’s — goal is to help patients, and by enlisting the support of physician expertise, startups can ensure easy-to-understand, transparent, and factual language within their go-to-market strategy to best reach patients.

Fuel startups with leading experts

Often, companies are challenged in sourcing the right expertise for a new project. And as the healthcare digital innovation market continues to grow at a rapid pace, time is of essence — however, startups cannot compromise real science. Thankfully, a national network of expert physicians can be found in one location.

By applying a gig economy model to healthcare innovation, flipMD from GoodRx is lowering the barrier for physicians and businesses to connect and collaborate through an easy-to-use online platform. Whether the latest innovation is in the research and development phase or ready to go to market, the on-demand marketplace for physicians allows startups to remain at the forefront of medicine and conserve their time in identifying the right expertise by posting a job with unique parameters to recruit experts from a broad range of specialties.

In today’s evolving healthcare industry and amid rising consumer demand, it’s important for startups to collaborate with physicians to successfully innovate while staying on track. Discover how to fuel your startups’ momentum by collaborating with expert physicians.

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An independent committee that advises the FDA on cancer drugs has weighed in on medicines from Spectrum Pharmaceuticals and Oncopeptides, issuing negative votes on both.

First up was Spectrum’s poziotinib, which was developed to treat advanced non-small cell lung cancer (NSCLC) characterized by HER2 exon 20 insertion mutations, a rare genetic signature that drives an estimated 2% to 4% of NSCLC cases.

FDA staff flagged safety concerns at the proposed dose 16 mg dose. In clinical testing, 57% of patients experienced dose reductions and 85% of patients had grade 3 or grade 4 adverse events. They also noted that Boston-based Spectrum is seeking accelerated approval at that once-daily 16 mg dose, but the planned confirmatory study will be at 8 mg twice daily. Last Thursday, the committee voted 9 to 4 that the therapy’s benefits do not outweigh its risks. An FDA decision for the drug is due by Nov. 24.

Following Spectrum, Oncopeptides faced the advisory committee regarding its multiple myeloma drug, Pepaxto. The FDA awarded Pepaxto accelerated approval last year, but months later issued an alert that noted a clinical trial found an increased risk of death associated with the drug. Oncopeptides then stopped marketing the drug in the U.S.

FDA staff pointed to results in a confirmatory trial for Pepaxto, which did not meet the goal of progression-free survival, a measure of how long patients live without their cancer worsening. Staff also added that overall survival was worse in the Pepaxto group. Results showed a higher total number and percentage of deaths in the Pepaxto arm compared to the group given a standard multiple myeloma treatment. Regulators in Europe reached different conclusions about the drug, approving it last month. It is marketed there as Pepaxti. But the FDA advisory committee remained unconvinced by the company’s analyses and the European approval. On the question of whether the drug’s benefits outweigh its risks, the committee answered “no” by a 14 to 2 vote.

The standard qualifier for every FDA advisory committee meeting is that the regulator is not required to abide by the committee vote, but it usually does. If that holds true for Spectrum and Pepaxto, the chances don’t look good for a favorable decision from the FDA.

Here’s a look at other recent regulatory news.

—An experimental gut microbiome therapy from Ferring Pharmaceutical won the backing of an FDA advisory committee, which voted 13 to 4 that the treatment’s data are adequate to support its efficacy. Switzerland-based Ferring designed its therapy, RBX2660, to reduce the recurrence of C. difficile infection (CDI). The committee also voted 12 to 1 with one abstention on the question of whether the data are adequate to support the safety of the Ferring therapy in those 18 and older following antibiotic treatment for recurrent CDI.

—Children have been excluded from many clinical trials due to what the FDA now says was a “misperception that excluding them from research was in fact protecting them.” That thinking is changing and the FDA has issued draft guidance setting out an ethical framework for including and protecting children in clinical trials. The draft is open to public comment for the next three months before the FDA finalizes the guidance.

—The FDA placed a partial clinical hold on a pivotal study testing Viaskin, a peanut allergy patch in development by DBV Technologies. That study has not yet started but France-based DBV said the agency has asked for adjustments to the statistical analysis of the patch’s adhesion, among other changes. According to DBV, the FDA said these modifications are needed for the study to support a future biologics license application.

—Eli Lilly cancer drug Retevmo received FDA approval for treating advanced solid tumors characterized by a RET gene fusion. The accelerated approval covers the treatment of cancers that have progressed following at least one prior treatment. Retevmo won its initial accelerated approval in 2020 for the treatment of three RET-driven cancers: non-small cell lung cancer (NSCLC), thyroid cancer, and medullary thyroid cancer. With the latest tumor agnostic approval, the 2020 accelerated approval in NSCLC has been converted to a traditional one.

Clinical trials of a Merck HIV drug that were paused by the FDA last year can now resume, but at a lower dose. Merck also said it will no longer pursue development of the HIV drug, islatravir, for HIV prevention. The FDA placed multiple tests of the drug on full or partial holds after the observation that some patients treated with islatravir developed lower levels of two types of immune cells.

—Larimar Therapeutics was also able to resolve a clinical hold. Last year, the regulator paused a dose-ranging clinical trial evaluating the biotech’s treatment for Friedreich’s ataxia, a rare neuromuscular disorder, after deaths were reported in a monkey study. Those deaths all happened in monkeys given the highest dose of the drug.

The lift of the full clinical hold comes with the imposition of a partial hold. The planned Phase 2 trial now has a requirement that the FDA review data from a lower 25 mg dose group before the study escalates to a higher dose in the second cohort. Larimar said it expects to begin this study in the fourth quarter of this year; data are expected in the second half of 2023.

Photo by FDA

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Nursing homes became an elder care flashpoint during the Covid-19 pandemic, and while some of the problems were brought on by the virus, others reflect longstanding issues and a lack of regulatory oversight, industry stakeholders told members of Congress this week.

Nursing home workers, policy experts and relatives of people who died were among those who testified before the Select Subcommittee on the Coronavirus Crisis, which held a hearing on Wednesday.

Dr. David Grabowski, professor of healthcare policy at Harvard Medical School, described “dire conditions” in nursing homes in 2020. The pandemic “lifted the veil on nursing home care in America,” he said.

Grabowski shared research with the committee concluding that Covid-19 outbreaks in nursing homes were largely a function of where in the country a nursing home was located versus other specifics about the facility. 

“This does not suggest there was nothing that could have been done to prevent Covid outbreaks,” Grabowski said. “Rather, it suggests that policymakers needed to adopt a system-level approach to address this problem,” Grabowski said. 

Adelina Ramos, a certified nursing assistant at a nursing home in Rhode Island, described the conditions in the nursing home in the spring of 2020. She recounted to the committee that while working with residents who couldn’t eat, drink, get out of bed or go to the bathroom without help, and who required oxygen to be changed every 15 minutes, she had to “make impossible choices about which residents to help” because the facility was understaffed. 

Ramos is now a member of a nursing union, which she attributes to improved working conditions, including paid sick leave and better health insurance. But she said it’s up to the government to look at this struggling industry and provide oversight. 

“We want guidelines to ensure that we have safe staffing levels more often,” Ramos told the committee. 

“The majority of nursing home workers are women and people of color and we are often called unskilled and uneducated,” Ramos said. “Our jobs are devalued and it’s disgraceful that after two-and-a-half years of a daily pandemic we are still treated this way and we are fed up with the lack of respect from nursing home owners and lawmakers, so our workforce change needs to happen now.” 

Racism and structural inequities affect both staff and nursing home residents alike, according to witness Dr. Jasmine Travers, assistant professor of nursing at NYU.

She cited research showing that halls with any black residents experience significantly more Covid infections and deaths than homes with no black residents. This was a problem highlighted during the pandemic, but not created by it. 

“Beyond the pandemic when compared to their white counterparts Black or Latino residents are likelier to experience pressure ulcers falls and under treatment for pain, ordered anti-psychotics, put in restraints and are less likely to receive preventative care,” Travers said. Residents who identify as LGBTQ+ and are living with dementia often do not receive required care, Travers said, because of limited staff knowledge, training and a failure to hire staff that is “culturally congruent” to residents. 

She recalled the pungent smells that “stung” her nose when visiting nursing homes during the pandemic because the Centers for Medicare and Medicaid Services waived inspection requirements.

“I urge the subcommittee to recognize that older adults do not want to stop living, although they might need help living,” Travers said.

Several witnesses blasted the failure of former New York Gov. Andrew Cuomo to address the crisis in nursing homes, saying he was preoccupied with a book deal. One committee member, Rep. Steve Scalise, R-Louisiana, asked the witnesses for statistics on whether New York nursing homes were pressured to not follow CDC guidelines, after hearing many accounts about Cuomo’s failure to address nursing homes in the state while he was in office. 

Though none of the witnesses had statistics on hand about the direct impact of the governor’s orders on nursing homes, one New Yorker described how the virus affected his family.

Daniel Arbeeny, a Brooklyn resident, said in one week in April 2020 four of his family members died from Covid-19, including his father, uncle, and two cousins. Three were in nursing homes. Arbeeny cited guidance he received from the nursing home where his father was that said he needed to bring his father home, where it would safer there than the nursing home. He said the nursing home had been instructed by New York regulators to readmit Covid-19 positive residents to their facility even if they were unequipped. 

“It was like a hurricane. I have no other way to describe how my family came together and made a plan,” Arbeeny said, recalling when he received the news from the nursing home that his father would be safer at home. “We were in a race for our lives and we knew it,” though his father eventually died at 88 from the virus while at home a week later.

Rep. Scalise said he would find answers and statistics about directives from the New York regulators that influenced the decisions affecting those like Arbeeny and his family, and would hold to account the authorities that abandoned New York nursing homes. 

The John A. Hartford Foundation, based in New York City, is a private, nonpartisan, national philanthropy dedicated to improving the care of older adults. The foundation released a statement following the hearing saying it was “a critical step toward accountability and action to improve America’s nursing homes.”

Photo: Tempura, Getty Images

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The Children’s Mercy Kansas City Research Institute announced on Monday that it is building what it calls the “most advanced genomic sequencing system in the world.” 

This claim refers to the work Children’s Mercy is doing through its Genomic Answers for Kids (GA4K) program. The health system rolled out the program three years ago with the goal of collecting genomic data and health information for 30,000 children and their families — it expects to create a database of at least 100,000 genomes over seven years. GA4K is already enjoying a significant milestone: providing 1,000 children with rare disease diagnoses based on its genome sequencing.

The program is unique because it uses 5-base genomic sequencing. This is a novel technology that combines multiple genomic technologies that have been used before into a single test, Dr. Tomi Pastinen, the director of Children’s Mercy Kansas City’s genomic medicine center, said in an interview.

“What 5-base genomic sequencing allows us to immediately do in the clinical space is replace multiple, usually successive genetic tests that are carried out on a patient into a single combined test and speeds up the analysis of multiple different types of genetic diseases,” he said.

Beyond the ability to diagnose all types of genetic diseases, 5-base genomic sequencing opens up our ability to “read the genome beyond the currently interpreted clinical genome,” Dr. Pastinen added. 

Genomic sequencing tests currently interpret only the protein coding part of the genome, which Dr. Pastinen claimed makes up only about 2% of the human genome. The use of 5-base genomic sequencing unveils the 98% of the genome that is currently not clinically analyzed. This is important because 60% cases involving children in which physicians suspect genetic disease remain unsolved with current genetic testing methods, Dr. Pastinen said.

On average, about 30-40% of pediatric rare disease cases receive a diagnosis, according to Dr. Pastinen. He said 5-base sequencing can help that percentage inch closer to half.

Dr. Pastinen claimed that the database being built for GA4K is the first of its kind for a couple reasons. The first is its scale, and the second is its comprehensiveness. While there have been pediatric rare disease studies that focus on specific indications, this program collects data from as many pediatric patients as possible who have been evaluated for an unsolved disease, Dr. Pastinen said.

This data comes from Children’s Mercy patients, as well as patients at the 17 partner institutions that take part in GA4K, two of which include NYU Langone Health and the University of Nebraska Medical Center, Dr. Pastinen said. These partners send their patients’ genomic samples for testing at Children’s Mercy.

“The program is unique because of the depth of data per patient — and because it’s dynamic,” Dr. Pasinen said. “And what that means is that we actually query the genome over the lifetime of the patient. The patient and their medical record lives with us. If there are changes in the medical record or changes in our understanding of the genome, we update the analysis in real time and actually share the data in real time with the scientific community and physician scientists.”

The 5-base sequencing used in GA4K expands upon previous work conducted by pediatric hospitals to better understand rare diseases in children. For example, San Diego-based Rady Children’s Institute for Genomic Medicine has also been a pioneer in the space, with a strong precision medicine program that deploys rapid genomic sequencing to quickly and accurately diagnose patients as early as possible. This program was championed by Dr. Stephen Kingsmore, who served as director of Children’s Mercy’s center for pediatric genomic medicine from 2011-2015.

Photo: Andy, Getty Images

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When Biogen’s experimental treatment for amyotrophic lateral sclerosis (ALS) missed the main goal of a pivotal study last year, the drugmaker said further analysis could yield better results. Additional data now show that treatment over a longer period of time led to improvement on several measures of the neuromuscular disorder.

The latest clinical trial results were published Wednesday in the New England Journal of Medicine. The data lend additional support for the drug, tofersen, which has been submitted for FDA review and is expected to receive a regulatory decision in early 2023.

Tofersen is an antisense oligonucleotide, a type of drug comprised of small pieces of DNA or RNA. The Biogen drug addresses a subset of ALS patients whose disease is driven by mutations to the SOD1 gene. These mutations lead to abnormal versions of the SOD1 protein believed to contribute to motor neuron dysfunction and cell death. Tofersen is designed to bind to and degrade SOD1 messenger RNA, which in turn reduces synthesis of SOD1 protein.

The main goal of the randomized, placebo-controlled Phase 3 test of the drug was to evaluate ALS patients on various functional measures at 28 weeks. According to results reported last October, the observed patient improvement was not enough to show statistical significance. However, investigators at the time noted that the results showed reductions in SOD1 protein and neurofilaments, filaments found in neurons that are considered a potential biological indicator of neurodegenerative disease.

Biogen did not give up on the drug. In June, the company presented data analysis  in which the randomized study at 28 weeks was combined with an open-label extension study at 52 weeks. In that new 52-week analysis, Biogen reported slower declines in measures such as respiratory function and muscle strength in those who started on tofersen earlier—the patients who received the study drug at the start of the study compared with those who started on placebo and were switched over to tofersen at week 28 to begin the extension study. These are the results that are now published in the New England Journal of Medicine.

Timothy Miller, co-director at the ALC Center at the Washington University School of Medicine and the principal investigator of the tofersen clinical trial, said in a prepared statement that in addition to the lowering of SOD1 protein, the drug led to “substantial lowering of neurofilament levels, which I interpret as potentially slowing the underlying disease process.” Miller added that looking at the results in the later time points in the open-label extension study show “meaningful clinical benefit.” The New England Journal of Medicine article notes that comparisons of earlier initiation of tofersen versus delayed initiation are still being evaluated in the extension stage of the clinical trial.

The 52-week data were part of Biogen’s submission seeking FDA approval. The FDA accepted that application in July, setting a Jan. 25, 2023 target date for a regulatory decision. At the time, the agency said it planned to convene an advisory committee meeting to discuss the application. The date for that meeting has not yet been set.

The published data for Biogen’s ALS drug come as an ALS drug from Amylyx Pharmaceuticals is making its way through regulatory review. Two weeks ago, an FDA advisory panel voted 7-2 in support of recommending approval of that company’s experimental ALS treatment, AMX0035. An FDA decision for the Amylyx drug is due by Sept. 29.

Meanwhile, another ALS drug developer, BrainStorm Cell Therapeutics, is taking a shot at FDA approval. Last year, the FDA called out Brainstorm’s analysis of its ALS drug, Nurown, saying that that in addition to missing the main clinical trial goal, the results fell did not show patient benefit. Nevertheless, Brainstorm said last month that it plans to seek FDA approval of its ALS therapy.

Photo: Adam Glanzman/Bloomberg, via Getty Images

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People taking their mental health into their own hands via the use of psychedelics. Recent college graduates entering the workforce without ever stepping foot in an office. Individuals who make investment decisions based on Reddit. 

These newly emerging populations represent the forces that are shaping the future of health today, and healthcare organizations that pay attention to these new pivotal groups will stay relevant in a post-pandemic world.

These new populations are best exemplified by the archetypal personas I met Monday at an interactive event in Chicago. The event, titled Inspiration Experience: Renaissance 2022,” took place Monday at the Oliver Wyman’s Health Innovation Summit. Billed as an “immersive experience,” it was more an interactive event that featured presentations from actors and members of Oliver Wyman’s health and life sciences team.

The event summarized Oliver Wyman’s consumer research and explored four major trends that will play critical roles in accelerating digitization within major industries such as healthcare, technology, science, technology, manufacturing and education. The presentations also introduced new names for emerging consumer and employee populations, such as “digital bloomers” and “citizens of the metaverse.”

To conduct the research that informed the interactive event, Oliver Wyman surveyed more than 100,000 respondents across nine countries from August 2020 to December 2021. The countries were the U.S., U.K., Mexico, Brazil, Germany, Italy, Spain, France and China.

Dr. Who? Dr. Me

In this section, we met three characters who embodied the country’s changing health behaviors. The first was Ruchir, a young man using ketamine therapy to treat his depression. After years of unsuccessful treatments — including SSRIs (selective serotonin reuptake inhibitors) and transcranial magnetic stimulation — he began the psychedelic therapy and is finally seeing progress for his condition.

Though his mental health is improving, he is plagued with endless back-and-forths with his health plan, begging it to cover his treatment given it is the only thing that is working for him. It is doubly frustrating for him given the therapy has been proven to be effective. He is also bothered by the fact that ketamine therapy was not widely available at mental health facilities. 

Next, we met Hannah, an expecting mother who is on her way to meet with her doula. This is Hannah’s first pregnancy, and she knows she needs a doula to help guide her through the emotional and oftentimes overwhelming process of preparing for childbirth. She is grateful that she is able to afford the doula, but expresses resentment and confusion as to why her insurer didn’t cover the services. Research proves that expectant mothers who are matched with a doula have better birth outcomes — Hannah felt like her health plan didn’t prioritize her best interest of her child’s health. 

Jay, the final character, is an athlete focused on optimizing his physical performance. He is willing to try every new and existing technology available to strengthen the full spectrum of his wellbeing. However, he said that when he brought up sleep tracking technology and the other wearables that he used to understand his health to his physician, she was dismissive of his efforts to learn from his health data. 

Each of these characters represents a society that is becoming more educated and aware of “alternative” therapies and ways to measure their health. They demonstrate a frustration that more and more patients are feeling: Why should I listen to my doctor when they don’t even know all of my options? The presentation recommended that healthcare companies respond to these consumer trends by keeping their operating models up to date as new research and care delivery breakthroughs continue to emerge.

Influencers over Institutions

As exemplified by Ruchir, Hannah and Jay, patients have more health information at their fingertips than ever before. Oliver Wyman’s research revealed that people gather the bulk of this information through the Internet. It also found that distrust in experts and corporate organizations is at a high. Rather than turning to institutions, people prefer to get their information — whether it be health advice, political guidance or briefings on current events — from influencers and friends on social media, according to Oliver Wyman’s health and life sciences team.

Investors are not immune to this trend as exemplified in the concept of the “hivemind investor,” a persona representing those whose investment decisions are driven by social media. This character entered mainstream consciousness in January 2021, when the “WallStreetBets” subreddit rallied around stocks that traditional investors on Wall Street had expected to do poorly, such as Gamestop and AMC. Oliver Wyman’s research showed that this community-based approach to investing is unlikely to go away soon, so startups and companies preparing for their IPOs should be aware of this trend.

I also met the “specter of disinformation” as part of the experience. I did this by looking into a mirror — it was meant to reveal the skeptic that lives inside all of us. The presentation reminded me that in a world in which we curate our own information from the vastness of the Internet, it’s imperative that we be discerning when consuming news online. 

I was shown various TikToks and Instagram reels in which users spouted their expertise on disease outbreaks and health maintenance. Some of these videos were more outlandish than others — such as the one that argued raw milk is the only healthy coffee additive — but they all contained health information that was questionable and unvetted. It was then revealed that the accounts I was watching all had more followers than the Centers for Disease Control and Prevention. More Instagram followers, that is — so far the CDC doesn’t even have a TikTok account.

People are more likely to trust information when it is delivered by someone they can relate to, the research showed. For a growing number of people, a TikToker uploading content from their home is much more relatable than Anthony Fauci delivering information in a suit. This is a prime area where healthcare companies need to innovate — they are still struggling to handle disinformation and its effects on their customers, Oliver Wyman’s presenters said.

Mobilizing the Metaverse  

Just like social media isn’t going away, neither are digital modes of care delivery. In this session, I learned about two distinct groups: digital bloomers and citizens of the metaverse. 

Digital bloomers are people, mainly those over age 45, who entered the digital ecosystem because of the pandemic. Until then, they did things the analog way and saw no reason to change. Now, this group is becoming increasingly willing to digitize necessities, such as their healthcare interactions, banking and grocery shopping, the research revealed.

The presentation suggested that healthcare companies need to capitalize on this newly emerged group by scaling up their digital offerings and focusing on convenience for the end user. This becomes even more imperative when we think about reaching citizens of the metaverse, a term which refers to people who are willing to participate in a wholly virtual world without hesitation. 

As more and more people become comfortable with the metaverse, healthcare companies have the opportunity to tailor their products and services toward the convergence of virtual and physical reality. This could be as simple as offering more telehealth visits, or as complex as creating virtual reality experiences for physical therapy. During the interactive portion of this session, I was given the chance to take part in various VR experiences, such as a physical therapy session in which patients slash virtual balloons with virtual swords to help regain muscle strength.

The Great Renegotiation

In addition to new consumer preferences, healthcare companies also need to start adapting to new types of employees that have entered the workforce over the past couple years. For example, the workforce has seen an influx of workers who are virtual natives, meaning they graduated during the pandemic and have since taken jobs where they work almost exclusively remotely. 

Without knowing it, this group of young people have reinvented what a white collar job can look like. Many prefer remote work, enjoying the fact that they rarely have to leave the comfort of their homes and pets. Others are unsure whether they enjoy it, finding it strange to have never met your boss in person, according to the research. 

This group has a complicated relationship with returning to the office. In the survey, 87% said they do (or would) enjoy going to the office, and 86% said they would quit or look for another job if they were required to return to the office full-time. As a 2020 graduate and virtual native myself, I can attest to this — my peers and I have mixed feelings on remote work. Conducting more research to understand this population’s unique wants and needs will help healthcare companies discover ways to improve white-collar retention and build employee loyalty, the presentation recommended.

Another employee group that has emerged during the pandemic is blue collar workers who taught themselves new skills that helped them land a white collar job. As these workers shift to white collar positions and blue collar baby boomers continue to retire, the blue collar labor shortage is being exacerbated, according to the research. This will force healthcare companies to fully embrace automation and figure out how humans and machines can best work alongside each other.

During this presentation, I saw two actors have a conversation as Gen Z employees. They worked as call center employees at a regional health plan, and they were on their lunch break. The women talked about remote work concerns and how they were unsure how to advocate for better pay. One of these characters also said she “didn’t even understand” which benefits her employer was offering, claiming that the company needed to do a better job of educating her about them.

From these bemused young workers to hivemind investors to psychedelic explorers, all of these new personas are worth healthcare companies’ attention, according to Oliver Wyman’s presenters. Successful healthcare companies must continually reassess their strategies to keep up with these changing consumer and employee preferences — or they may fall behind.

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heart, doctor, cardiac

Gene therapy developer Rocket Pharmaceuticals is on track to seek FDA approval for its lead program but it’s also taking care to keep its pipeline stocked. The company has agreed to acquisition of Renovacor, a biotech developing a gene therapy for a genetically driven form of heart failure.

According to financial terms announced Tuesday, the all-stock deal represents an equity value of $53 million, or an implied value of $2.60 for each Renovacor share. That’s a 36.8% premium over the closing price of Renovacor’s closing stock price on Monday but a steep drop from a year ago, when the company went public via a SPAC merger and saw its shares new shares trade on the New York Stock Exchange for more than $10 apiece.

Cambridge, Massachusetts-based Renovacor develops gene therapies for genetically driven cardiovascular diseases. The company emerged in 2019 with an $11 million Series A round of financing for preclinical development of a gene therapy that addresses BLCL2-associated athanogene 3 (BAG3) mutations that lead to dilated cardiomyopathy, a severe form of heart failure. Renovacor was founded by Arthur Feldman, a cardiologist and professor of medicine at Temple University.

Lead Renovacor program REN-001 uses an adeno-associated virus (AAV) to deliver to cells a healthy version of the BAG3 gene. In preclinical testing, Renovacor said its gene therapy led to the production of functional BAG3 protein and improvement in cardiac function. Human testing is the next step. The company has said it expects to submit an investigational new drug application in the second half of this year to support a Phase 1/2 clinical trial. The Renovacor pipeline includes discovery-stage gene therapies addressing BAG3 mutations as well as the expression and function of that gene. The company has also expanded its research to include genetically driven arrhythmogenic cardiomyopathy.

“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy,” Rocket CEO Gaurav Shah said in a prepared statement.

Rocket’s cardiac gene therapy research focuses on Danon disease, a weakening of the heart muscle caused by mutations to the LAMP2 gene. The Cranbury, New Jersey-based company’s Danon program, RP-A501, is currently in Phase 1 testing. In a research note sent to investors, William Blair analyst Raju Prasad said the prevalence of BAG3-associated dilated cardiomyopathy is estimated to be as high as 30,000 patients in the U.S., a figure that is expected to grow with more genetic testing and disease awareness. He added that Renovacor brings synergies to Rocket, as both companies are using AAV-9 vectors to pursue genetically defined targets.

Rocket’s most advanced program, RP-L201, has reached pivotal Phase 2 testing for leukocyte adhesion deficiency-1 (LAD-1), a rare disorder caused by mutations to the gene that encodes CD18, a protein that helps white blood cells stick to blood vessels. Children born with LAD-1 are susceptible to fungal and bacterial infections that can become life-threatening. In May, Rocket reported data showing 100% survival in seven patients 12 months after infusion with the gene therapy. In its report of second quarter 2022 financial results last month, Rocket said it expects to file an application seeking FDA approval of its LAD-1 gene therapy in the first half of 2023.

The boards of directors of both Rocket and Renovacor have approved the acquisition, but approval by shareholders of both companies is still needed. The deal is expected to close by the first quarter of next year.

Photo: BrianAJackson, Getty Images

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graphic design of a liver

Genfit has a drug candidate on track toward clinical testing for a rapidly progressing and potentially fatal complication of chronic liver disease. By acquiring Versantis, it adds another drug that works in a different way and is further along in development for the same disorder.

Lille, France-based Genfit announced Monday that it has agreed to pay 40 million Swiss francs (about $41.4 million) to buy Versantis, a privately held Swiss biotech focused on developing liver disease drugs. In addition to the upfront payment, Versantis shareholders could earn up to 65 million Swiss francs (about $67.3 million) tied to clinical trial progress and regulatory approval of its lead asset.

The lead disease target of Zurich, Switzerland-based Versantis is acute-on-chronic liver failure (ACLF), a syndrome that can develop in patients whose chronic liver disease has progressed to cirrhosis. In addition to liver failure, this condition can lead to the failure of other organs such as the brain, kidneys, heart, and lungs. ACLF can rapidly progress to a coma and then death. According to Genfit, an estimated 137,000 patients are hospitalized with ACLF in the U.S. each year. The condition has no FDA-approved therapies.

The most advanced Versantis program, VS-01, employs liposomes, which are tiny, spherical vesicles that can be used as transport vehicles. The company says its drug clears toxic metabolites from the body, extracting them from the blood and taking them into the abdominal cavity, where they are taken up by liposomes that are then drained from the body. In a Phase 1b study, Versantis reported that its drug was safe and well tolerated by the 12 study participants, who experienced no dose-limiting toxicities. A 60-patient Phase 2 test is set to begin in the fourth quarter of this year. In addition to ACLF, the drug has potential application as a treatment of urea cycle disorder. The FDA has granted the drug a rare pediatric disease designation in this indication. In Europe, the Versantis drug candidate has orphan drug designation.

Genfit’s approach to developing an ACLF therapy involves repurposing nitazoxanide, or NTZ for short. This older drug is used to treat diarrhea and intestinal inflammation caused by parasites. Genfit is looking to apply the drug to liver and fibrotic diseases. The company said a meeting with the FDA is scheduled to discuss clinical trial plans for NTZ in ACLF following encouraging Phase 1 data that informs potential dose adjustments in patients with cirrhosis and liver impairment.

The Versantis acquisition is consistent with the liver disease focus that Genfit said it would maintain following the high-profile 2020 Phase 3 failure of elafibranor, a drug that the biotech was developing for the fatty liver disease non-alcoholic steatohepatitis (NASH). The company abandoned its pursuit of the drug for NASH, but restructured and focused development of the small molecule in primary biliary cholangitis, a different rare liver disorder. Late last year, Genfit licensed elafibranor’s global rights to Ipsen for €120 million up front. According to the licensing agreement, Genfit is still responsible for Phase 3 development of elafibranor until completion of that study’s double-blind treatment period.

Speaking on a conference call Monday, Genfit CEO Pascal Prigent said that the Versantis acquisition is a logical continuation of the rare liver diseases corporate strategy that the company started at the end of 2020. The Ipsen deal provided Genfit with cash to finance acquisitions, with an eye on assets that are in the clinic or close to clinical development. The Versantis acquisition brings Genfit programs for other liver disorders. VS-02 is in preclinical development for the treatment of chronic hepatic encephalopathy, a nervous system disorder triggered by advanced chronic liver disease. Versantis is also developing TS-01, a point-of-care diagnostic in development for at-home measurement of ammonia in the blood, which is the primary cause of hepatic encephalopathy.

“We believe Versantis’s portfolio of programs gives us exactly what we are looking for,” Prigent said. “After the acquisition, Genfit will have multiple promising programs in rare liver diseases, and will be a global leader in ACLF, a therapeutic area that we feel is both important and underserved.”

The acquisition agreement could yield more cash from the sale of an FDA priority review voucher. Under this FDA program, regulatory approval of a drug for a rare disease can lead to the award of a voucher that grants speedy review of another rare disease drug. This program is intended to incentivize rare disease drug development and the rare pediatric disease designation granted to VS-01 in urea cycle disorder makes it eligible for a voucher if the drug is approved.

A company awarded a voucher can apply it toward one of its own drugs, but many companies opt to sell these vouchers, fetching prices of $100 million or more. Under the acquisition agreement, Versantis is eligible to receive one third of the net proceeds from the sale of a voucher. If Genfit decides to use the voucher for one if its own programs, Versantis would be entitled to one third of the fair market value of the voucher.

Genfit expects to close the Versantis acquisition in the fourth quarter of this year.

Photo: eranicle, Getty Images

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Ten months after Acelyrin landed $250 million for mid-stage clinical development of an in-licensed immunology drug that could compete against blockbuster Novartis and Eli Lilly drugs, the biotech has raised $300 million more.

The new cash follows the report over the summer of positive Phase 2 data for izokibep, an antibody mimetic that blocks interleukin-17A. Los Angeles-based Acelyrin plans to use the latest fundraising haul to fund Phase 3 tests of its drug in psoriatic arthritis and axial spondyloarthritis.

Acelyrin’s Series C round of funding was the biggest biotech cash infusion of the past week. Here’s a recap of other notable financings:

Capstan Therapeutics emerged from stealth with $165 million raised to date as it works to advance toward the clinic with cell therapies engineered inside a patient’s body. The startup, which has operations in San Diego and Philadelphia, is based on research from University of Pennsylvania scientists with expertise in cell therapy and messenger RNA.

—Radiopharmaceuticals developer RayzeBio closed a $160 million Series D financing, which the company will apply toward more clinical tests of its lead asset, RYZ-101. A Phase 1b study is underway in neuroendocrine tumors; the company says Phase 3 tests could begin as early as next year. The FDA has also cleared the company to begin testing the radiopharmaceutical in non-small cell lung cancer.

Nimbus Therapeutics, a biotech company whose drug discovery approach is based on computational techniques, raised $125 million to back clinical development of programs in autoimmune diseases and oncology. One of those programs is blocks an enzyme called TYK2, which the Cambridge-based biotech is developing for moderate-to-severe plaque psoriasis and active psoriatic arthritis. The biotech is chasing Bristol Myers Squibb drug Sotyktu, whose recent FDA approval made it the first approved TKY2 inhibitor for moderate-to-severe plaque psoriasis.

—ATP, a venture capital firm formerly known as Apple Tree Partners, has merged three of its portfolio companies to form Galvanize Therapeutics, which is now backed by $100 million. San Carolos, California-based Galvanize is developing a pulsed electric field energy technology to treat various disorders including chronic bronchitis, cardiac arrhythmias solid tumors, as well as drug delivery. The new financing is a Series B round led by Fidelity Management & Research Company with participation from Intuitive Surgical, ATP, and Gilmartin Capital.

Forge Biologics, a company that operates as a gene therapy contract manufacturer while also developing its own therapies, closed a $90 million round. Columbus, Ohio-based Forge will use the new capital to expand its offerings to clients and bolster its manufacturing platform. The Series C financing was co-led by Drive Capital and Aisling Capital with an additional investor that remains undisclosed.

—SparingVision, a biotech developing gene therapies to treat inherited eye disorders, has raised €75 million. The Paris-based company will use the cash to begin clinical testing of two therapies, SPVN06 and SPVN20. SparingVision also plans to advance CRISPR-based gene-editing programs covered under a partnership with Intellia Therapeutics. The Series B round of financing was co-led by Jeito Capital and UPMC Enterprises. 4Bio Capital, Bpifrance, the RD Fund, and Ysios Capital also participated.

—Pretzel Therapeutics launched with $72.5 million to finance development of technology that modulates mitochondria, the energy-producing components of cells. Mitochondrial dysfunction is associated with more than 50 diseases, both rare and common. Waltham, Massachusetts-based Pretzel’s platform takes three approaches to affect mitochondrial function: genome correction, genome expression modulation, and mitochondrial control. Arch Venture Partners and Mubadala Capital led Pretzel’s Series A round.

Novome Biotechnologies raised $43.5 million for its gut microbiome approach to treating disease. The South San Francisco-based biotech’s lead program is in mid-stage testing for hyperoxaluria, in which people lack an enzyme needed to break down a compound in leafy greens called oxalate. Novome’s experimental treatment is comprised of bacteria engineered to break down oxalate. Tencent led Novome’s Series B financing.

—Carver Biosciences, a new company founded by Princeton University molecular biologist Cameron Myhrvold, emerged with an unspecified amount of seed financing led by Khosla Ventures. Boston-based Carver is developing antivirals that employ the CRISPR gene-editing system along with the RNA-directed Cas13 enzyme to target respiratory viruses. Myhrvold, whose research included developing Cas13-based technologies for studying viral host RNAs, founded Carver last year and will serve as the chair of the biotech’s scientific advisory board.

Picture: Feodora Chiosea, Getty Images

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