The Vaccines and Related Biological Products Advisory Committee will meet June 18 to discuss Moderna’s seasonal flu vaccine mRNA-1010 after the FDA initially refused to accept the application in February.

Moderna’s flu vaccine will face an FDA advisory committee this week as the mRNA company continues to walk a tumultuous path toward approval. But the initial documents are out, and the regulator and biotech appear to have reached alignment.

In February, the FDA shocked the biopharma world by issuing a refusal to file letter to Moderna for the seasonal mRNA flu shot mRNA-1010, now known as mFLUSIVA. At the time, then–Center for Biologics Evaluation and Research (CBER) director Vinay Prasad intervened in the application and said the company failed to support the case for approval with an adequate and well-controlled trial. In particular, the FDA took issue with the fact that Moderna initially did not use a high-dose vaccine as a comparator for the older populations, which is standard of care.

Days later, the FDA reversed course and allowed the application to proceed, setting a decision date for August. The approval request was narrowed to focus on specific age groups. The agency later set a meeting of the Vaccines and Related Biological Products Advisory Committee for June 18 to discuss the shot.

The documents for that meeting are now available, and Jefferies told investors that the regulator appears to have softened its stance toward Moderna’s application.

Moderna is seeking full approval for the shot’s use in adults ages 50–64 and an accelerated approval for adults 65 and older. For the latter indication, Moderna will have to conduct a post-marketing study to secure a full approval at a later date.

The FDA seems to have determined that the immunogenicity data submitted support the efficacy of mRNA-1010 in people over 65, according to Jefferies. The regulator noted no major safety issues or imbalances in adverse events or deaths between groups that received treatment versus placebo. The shot also showed superior relative vaccine efficacy as compared to the standard dose vaccine comparator in patients 50 to 64 years old.

To sum up, the FDA said no major efficacy deficiencies were identified, with the studies showing that mRNA-1010 “met all prespecified sequential success criteria,” according to the FDA document.

But the FDA flagged for the adcomm to review a few uncertainties, including evidence gaps as the data available are only from one flu season. Efficacy has also not yet been established in immunocompromised individuals and very frail older adults, the agency added.

On safety, the FDA flagged the relatively short dataset—just six months of follow-up—which may not reveal any rare adverse events like myocarditis or neurologic events that have been tied to Moderna’s original mRNA-based COVID-19 vaccine Spikevax. In terms of other adverse events, including anemia and urinary tract infections, people who took Moderna’s shot showed a higher rate than controls.

To address these concerns, Moderna recommended the bifurcated approach to the approval—with a promised confirmatory study to verify benefit in the older population.

The adcomm will vote on two questions: whether the benefits of mFlusiva outweigh its risks for the prevention of influenza disease in the two different age groups: adults 50 through 64 years and adults 65 and older.

FDA advisory committee recommendations are non-binding, but the agency typically follows the advice of these outside experts. Under the Trump administration, fewer adcomms have been held, but since a recent leadership shakeup that saw Commissioner Marty Makary depart, more have been scheduled.

Moderna preps for launch

The stakes are high for Moderna, which has an ambitious plan to market more than seven products by 2027–2028 across respiratory, oncology and rare disease. The company, which has repeatedly reported negative earnings, is looking to improve revenue by 10% year-over-year for $1.9 billion in 2026, Jefferies said.

The FDA’s refusal-to-file letter threatened to blow open Moderna’s plans to breakeven by 2028, with mRNA-1010 representing a potential $1 billion revenue opportunity.

But the company seems to be more upbeat, revealing on Tuesday a shake-up in the C-suite. President Stephen Hoge’s role was expanded to include oversight of leadership across R&D, manufacturing and commercial operations. Moderna has also hired Ester Banque as chief commercial officer, as the company prepares for three product launches next year and in 2028.

The CCO role has been filled by CEO Stephan Bancel since the 2023 departure of Arpa Garay.

While biopharma’s overarching mission is to develop innovative medicines to improve patient outcomes, for these six people, the motivation came from much closer to home.

In February, Doug Ingram dropped a bombshell on the industry: He would be stepping down from his post as CEO of Sarepta Therapeutics after a career spanning more than three decades.

Ingram’s difficult decision was driven by what he called a “fairly shocking and ironic twist of fate.”

“As you know, in late 2024 we entered into a partnership with Arrowhead, and we gained access to a number of very promising therapies, including SRP-1003, for a devastating disease, DM-1,” he said. Shortly after the deal, that same disease came knocking on Ingram’s door, afflicting two members of his immediate family.

Leaving Sarepta, Ingram said, will help him pour more of his focus at home.

By the end of the year, Sarepta will be under new management, though there has been no word on Ingram’s replacement yet.

Ingram’s situation is heart-breaking but by no means unheard of. In fact, biopharma is replete with examples of devastating diagnoses driving ardent drug development and entrepreneurship. It’s unclear yet where this personal bout with DM1 will take Ingram, but if he decides to return to biopharma and focus his efforts on this disease once again, there will be paths open to him, forged by many others who were steered to biopharma by personal motivations.

BioSpace looks at six such cases, where drug development was spurred by a mission far more individualized than profit and shareholder value or the benefit of entire patient communities.

‘Deeply personal’ investment adds to Quiver’s arrows

Around one in 4,000 children is born with a specific chromosomal aberration that will leave them stunted, both physically and developmentally. Babies with the disease, called Dup15q, may appear floppy and could have trouble feeding and be behind on key motor milestones. Kids with Dup15q also suffer from learning and intellectual disabilities.

In rare cases, Dup15q leads to sudden death. There is no approved treatment for this disease.

That’s where Quiver Bioscience comes in. Based in Massachusetts, the biotech is working on an antisense oligonucleotide that targets the UBE3A gene, heightened expression of which has been linked to Dup15q, the company said in a March 2025 news release. A precision approach to reduce UBE3A has “disease-modifying potential” for this condition, Quiver noted.

Last month, Quiver received a “targeted investment” from a family office in Argentina, specifically to help the company take its oligonucleotide program forward. But instead of wanting to see the money grow, this placement was much more personal: One of the family members also has Dup15q.

“As the father of a daughter living with this condition, this is not an ordinary investment — it is a deeply personal commitment to every family navigating this journey every single day,” José A. Porta, president of the family office, said in a prepared statement.

In an email to BioSpace, Quiver declined to provide additional details regarding the Porta family’s investment, including how much money the company received. The support, however, will allow the biotech to carry out preclinical activities for its antisense oligonucleotide, including candidate selection and safety assessments, opening the path toward clinical development, according to its April news announcement.

Parents launch Solid in search of cure for son

Solid Biosciences was founded in 2013 by husband-and-wife team Ilan and Annie Ganot. Their goal: Find a cure for their son Eytani, who had been diagnosed with Duchenne muscular dystrophy (DMD).

At the time of Solid’s establishment, there were no therapies for Duchenne on the market yet. The first Duchenne drug to win the FDA’s approval—Sarepta Therapeutics’ exon-skipper Exondys 51—was still three years away, and even then, it left a big portion of the patient population unserved. Exondys 51 is only indicated for patients who are amenable to exon 51 skipping, which represents around 13% of all patients with Duchenne.

Indeed, not long after their son was diagnosed, the Ganots realized that exon skipping therapies and those that boost dystrophin expression—a key deficiency in Duchenne—wouldn’t lead to meaningful and profound symptom improvements that parents like them would want for their kids, Ilan told Rare Disease Advisor in 2022.

The Ganots decided that only a gene therapy could give the relief they were looking for.

Solid is working toward this goal with SGT-003, which makes use of a genetic construct that expresses the microdystrophin protein, according to the company’s website. This could help compensate for the dystrophin deficiency in patients with Duchenne. To deliver the genetic payload, Solid is using a proprietary adeno-associated virus (AAV) vector that selectively targets cardiac and skeletal muscles while avoiding the liver—a profile that could lend the asset better efficacy and safety. Sarepta’s now-approved Duchenne gene therapy Elevidys was rocked last summer by patient deaths relating to liver complications from the AAV vector technology.

SGT-003 is being tested in two studies: the Phase 1/2 INSPIRE trial and the Phase 3 IMPACT study. INSPIRE has shown robust microdystrophin expression and early signs of muscle improvements. Solid is working with the FDA for a potential accelerated pathway for SGT-003. IMPACT is set to start this year.

Elpida born from parents’ quest to save son

As in the case of Solid, Elpida Therapeutics was born out of a couple’s desire to save their son.

In 2019, Terry and Georgia Pirovolakis were told that their son Michael had SPG50, a neurodegenerative and developmental disorder that, with fewer than 100 known cases worldwide, is classified as “ultra-rare.” There is no cure for SPG50, only treatments to manage its symptoms.

SPG50—also known as spastic paraplegia 50—is caused by mutations to the AP4M1 gene, which under healthy conditions encodes a part of a protein that helps move molecules along inside cells. Children must inherit an altered version of this gene from both of their parents for the disease to manifest, causing developmental delays, poorly formed muscles and lower-limb paralysis.

Searching for relief for their son Michael, the Pirovolakises decided that they needed to develop their own gene therapy, leading to the launch of Elpida in May 2023. The company’s lead program is MELPIDA, which delivers a functioning copy of the AP4M1 gene.

Michael became the first-ever patient to receive MELPIDA in 2022, around three years after he was diagnosed—and the results were promising. At about a year after receiving the gene therapy, Michael showed improvements in limb spasticity and achieved notable development and motor gains, according to a June 2024 publication in Nature Medicine.

MELPIDA was also largely safe for Michael, and the gene therapy was able to keep SPG50 at bay, preventing disease progression. The asset is in Phase 3 development, according to Elpida’s website.

CEO-Dad enters biotech to develop treatment for kids

Joining the roster of parents going into biopharma to save their kids is John Crowley—and of those that made it to this list, he might be the most well known and his fight the most documented. Crowley’s story even became the backbone for the 2010 film Extraordinary Measures, starring Harrison Ford and Brendan Fraser.

In 2000, Crowley helped establish Novazyme Pharmaceuticals with the goal of developing a treatment for Pompe disease, a rare metabolic disorder that afflicted two of his children. Patients with Pompe suffer from muscle weakness and have enlarged hearts and livers, often leading to other complications. Left unchecked, the condition can leave children debilitated or lead to dead.

Indeed, the prognosis for Crowley’s kids was dire, according to reporting by Rare Disease Advisor last month: They wouldn’t survive past two years.

Roughly a year into its operations, Novazyme got acquired by Genzyme for $225 million. Despite being a hefty vote of confidence for his mission, this deal posed a crucial conundrum for Crowley: Being an employee, he couldn’t enroll his kids into the company’s clinical trial, Fierce Pharma reported last year.

But Crowley would not be stopped and ultimately stepped down from Genzyme. His gamble worked—as did the investigational therapy. His children were enrolled in the trial and experienced improved muscle strength and normalized heart sizes.

Today, this therapy is marketed under the brand name Lumizyme. Sanofi owns the rights to it after a 2011 deal with Genzyme worth $20 billion.

Crowley, meanwhile, has gone on to establish Amicus Therapeutics, still keeping his sights set on advancing therapies for rare diseases. After a $4.8 billion acquisition in December, Amicus today operates as a subsidiary of BioMarin.

Crowley is also currently serving as CEO of the Biotechnology Innovation Organization (BIO) after rising as an executive leader in biotech.

Mom launches EveryONE Medicines to honor daughter’s life

A parent’s perseverance sometimes ends in heartbreak.

That’s the case for EveryONE Medicines, which closed up shop in March. After cofounding the company in early 2021 with the goal of developing individualized precision treatments for patients with rare diseases, Julia Vitarello publicly debuted EveryONE in 2024.

EveryONE’s mission is deeply personal to Vitarello. Her daughter, Mila, suffered from Batten disease, an umbrella term that refers to a group of genetic disorders involving the toxic buildup of waste in a child’s brain cells. Symptoms include vision loss, muscle weakness and seizures, alongside developmental and cognitive problems.

Batten disease is fatal and often leads to death early in a patient’s life.

Vitarello worked hard to avert this fate for her daughter and enlisted researchers at the Boston Children’s Hospital to come up with a cure, C&EN reported in 2024. This effort resulted in an antisense oligonucleotide which they called milasen, designed to address the specific genetic mutation driving Mila’s condition.

The treatment helped improve Mila’s condition, reducing her seizures—but it wasn’t enough to save her. Mila was diagnosed at age 6 and died at 10.

Today, despite EveryONE folding, Vitarello remains involved in rare disease development. She is the chairwoman and CEO of Mila’s Miracle Foundation, a non-profit that works with academics, government agencies and companies to foster the development of individualized medicines. Vitarello also serves as a steering committee member of the N=1 Collaborative, a network of organizations and experts advancing personalized treatments.

Cure Rare Disease honors founder’s late brother

Capping off this list is Cure Rare Disease, a non-profit company that works to develop therapies for patients who had been “previously deemed untreatable,” according to its website.

The group was established by Rich Horgan with the goal of developing a treatment for his brother, Terry, who had been diagnosed with Duchenne muscular dystrophy. The group got very close to their goal and in October 2022 they were able to dose Terry with a CRISPR-based gene therapy made specifically for him to address the rare genetic mutation that drove his condition.

A month later, however, Terry died. Cure Rare Disease suggested on its website that the team was just too late, noting that Terry’s death “would have been avoided had the treatment been accessible sooner.” Research since has however pointed the finger at the viral vector used to deliver the gene therapy, which triggered a powerful immune reaction.

Regardless of the cause of his brother’s death, Rich Horgan has continued his mission of developing therapies for other rare diseases. Alongside Duchenne, and in collaboration with researchers, policy experts and other foundations, Cure Rare Disease is also working on programs for limb-girdle muscular dystrophy, spinocerebellar ataxia and adenylosuccinate synthase 1-related myopathy.

Weeks after Boehringer Ingelheim and Eli Lilly retracted billions of dollar in German commitments, the nation’s government is reportedly changing a contentious element of its planned healthcare reforms.

The German government is dropping plans to introduce variable discounts on medicines after receiving pushback from industry, Reuters reported Monday.

An anonymous government source told the publication the government will replace variable discounts with fixed reductions to help drugmakers plan for the shift. The original proposal tied the discount rate to Germany’s overall spending on medicines and to healthcare revenues, limiting visibility into the figure. Details of the size of the new, fixed discounts have yet to emerge.

The report comes less than two weeks after Boehringer Ingelheim and Eli Lilly pulled back from planned investments in Germany in response to the government’s proposed healthcare reforms. BioSpace did not receive an immediate response from Lilly to questions about whether the company will reconsider its actions in light of the government’s proposed move to a fixed discount rate. Boehringer, meanwhile, replied that it has “no comment” to the same questions.

Hundreds of jobs and billions of euros rest on the outcomes of the companies’ decisions. At Boehringer, concerns over the proposed German reforms led management to ax plans to invest €900 million ($1 billion) from 2027 to 2030 to expand its infrastructure in the country.

On the day Boehringer disclosed its investment U-turn, Lilly CEO Dave Ricks revealed that his company was halving a planned investment in a German plant. Lilly originally intended to invest €2.3 billion ($2.7 billion) in a German injectable manufacturing site to support the supply of its GLP-1 drugs Zepbound and Mounjaro.

The healthcare reforms drove Lilly to scale back its investment. Under the revised plan, Lilly will open the site as planned next year but run the plant at half the intended capacity. The change will involve a halving of the site’s planned headcount, which was originally 1,000. Lilly’s revised plan reflects Ricks’ belief that Germany’s reforms would make it the least supportive country in Europe.

Last week, Pfizer CEO Albert Bourla applied further pressure to the German government. Bourla told the government that Pfizer was reviewing its “external engagements as well as the timing, scope and future prioritization ⁠of certain planned investments in Germany,” Reuters reported.

The series of actions by drugmakers echoes the industry’s response to a drug pricing and market access dispute in the U.K. The long-running argument escalated in September when Merck pulled out of a $1.3 billion project and AstraZeneca paused plans to invest around $270 million in an R&D site. The U.K. later acceded to some of the industry’s requests in its trade deal with the U.S.

After the FDA flagged patient deaths linked to Amgen’s rare disease drug Tavneos and called for its voluntary removal, the pharma recruited an independent data analysis from Duke researchers to help build the case for the drug’s continued market approval.

Amgen has requested a hearing with the FDA over the agency’s insistence that Amgen pull its rare inflammatory disease drug Tavneos from the market—and this time, the company is bringing in help from the Duke Clinical Research Institute.

The pharma has asked Duke researchers to conduct an “independent and fully blinded re-adjudication” of data from the ADVOCATE trial, according to a June 1 letter to the FDA, which was posted online on Thursday. ADVOCATE is the Phase 3 study that Tavneos’ original developer ChemoCentryx used to support its approval in ANCA-associated vasculitis in October 2021. Amgen acquired ChemoCentryx for $4 billion just under a year later.

“Withdrawal of approval of TAVNEOS would be neither in the best interest of patients nor consistent with the statutory criteria,” Jay Bradner, Amgen’s executive vice president for R&D, wrote in the company’s letter to the FDA. Duke’s re-adjudication of ADVOCATE data began in February 2026 and the pharma plans to submit results, alongside other “detailed data,” to the FDA by June 29.

Alongside the Duke analysis, Amgen has listed in its letter more than a dozen ongoing or recently completed studies to support Tavneos’ overall benefit-risk profile.

The FDA first called for the voluntary withdrawal of Tavneos in January, raising concerns about “the process followed by ChemoCentryx” to evaluate the primary endpoints in ADVOCATE for nine of the 331 patients enrolled, according to an Amgen release in February. The regulator also pointed to liver toxicities in the context of Tavneos’ overall risk-benefit profile.

Amgen refused the regulator’s request, leaving Tavneos on the market.

The agency continued to apply pressure, however, and in late March put out a safety alert flagging eight deaths and 76 cases of drug-induced liver injury (DILI) that have “reasonable evidence of a causal association with Tavneos.” Seven of the 76 cases involved biopsy-confirmed vanishing bile duct syndrome (VBS), all of which necessitated hospitalization and three of which contributed to the total tally of eight deaths.

While the FDA at the time conceded that liver toxicities have been “identified in premarket clinical trials and described in product labelling,” potentially fatal DILI and VBS “represent new safety concerns.”

Last month, Japan’s Kissei Pharmaceutical, which is Amgen’s partner in charge of Tavneos’ Japanese distribution, said that 20 patients on the drug have died since the product launched in 2022. Most of the deaths were attributed to DILI and VBS, though Kissei took care to say that it remains unclear if Tavneos was the direct cause of the fatalities.

The European Medicines Agency has also launched a review of Tavneos, similarly flagging “questions regarding the data integrity” of ADVOCATE, according to a January release.

The RNA-based medicine is one of a handful of antibody-oligonucleotide conjugates that Novartis acquired last October when it took over neuromuscular-focused Avidity Biosciences.

Novartis’ $12 billion gambit for Avidity Biosciences late last year is starting to pay off, with the pharma reporting promising biomarker findings for its investigational RNA medicine in patients with facioscapulohumeral muscular dystrophy.

In the Phase 1/2 FORTITUDE study, Novartis enrolled 90 patients to receive the antibody-oligonucleotide conjugate delpacibart braxlosiran (del-bax) or placebo. Aside from safety, the trial’s primary outcome is the treatment effect on KHDC1L, a plasma disease biomarker for facioscapulohumeral muscular dystrophy (FSHD).

Without disclosing specific data, Novartis in a Thursday news release said that FORTITUDE’s biomarker cohort met its primary and key secondary endpoints. KHDC1L levels were reduced in patients treated with del-brax, suggesting “strong target engagement,” the pharma noted.

Del-brax also elicited a drop in creatine kinase concentrations, which Novartis explained is indicative of “reduction in muscle damage” in treated patients.

These findings “replicate the target engagement and downstream muscle protection seen with del-brax in earlier dose-escalation cohorts,” Nazem Atassi, global head of neuroscience and gene therapy development at Novartis, said in a prepared statement.

A previous FORTITUDE readout covering earlier dosing cohorts showed that patients on del-brax improved on the 10-meter walk-run test at 12 months compared to placebo. Similarly, those given del-brax performed better on the timed up-and-go test as well as on quantitative muscle and upper limb function assessments at 12 months while placebo controls deteriorated.

“We are now evaluating the totality of the biomarker and clinical data and look forward to discussions with global regulatory agencies,” Atassi said on Thursday, though Novartis hasn’t yet specified filing plans for the asset. The pharma, meanwhile, has launched the Phase 3 FORTITUDE-3 trial of del-brax in FSHD, aiming to enroll around 200 patients and looking at the therapy’s effects on muscle performance.

FSHD is a rare and progressive neuromuscular disease that manifests as a life-long muscle wasting, pain, fatigue and disability. The condition, affecting some 45,000 to 87,000 people across the U.S. and Europe, is caused by the abnormal expression of the DUX4 protein, which in turn activates genes that are toxic to muscles. Del-brax addresses this disease mechanism by combining the specificity of antibodies and the precision of oligonucleotides to target and suppress the expression of DUX4, according to Avidity’s website.

The asset came to Novartis through the pharma’s $12 billion takeover of Avidity in October last year, alongside two other RNA-based therapies: del-zota, being proposed for Duchenne muscular dystrophy, and del-desiran, being trialed for myotonic dystrophy type 1. The pharma also won over a clutch of preclinical programs for other rare neuromuscular disorders.

Novartis closed the Avidity acquisition in February.

After the FDA flagged patient deaths linked to Amgen’s rare disease drug Tavneos and called for its voluntary removal, the pharma recruited an independent data analysis from Duke researchers to help build the case for the drug’s continued market approval.

Amgen has requested a hearing with the FDA over the agency’s insistence that Amgen pull its rare inflammatory disease drug Tavneos from the market—and this time, the company is bringing in help from the Duke Clinical Research Institute.

The pharma has asked Duke researchers to conduct an “independent and fully blinded re-adjudication” of data from the ADVOCATE trial, according to a June 1 letter to the FDA, which was posted online on Thursday. ADVOCATE is the Phase 3 study that Tavneos’ original developer ChemoCentryx used to support its approval in ANCA-associated vasculitis in October 2021. Amgen acquired ChemoCentryx for $4 billion just under a year later.

“Withdrawal of approval of TAVNEOS would be neither in the best interest of patients nor consistent with the statutory criteria,” Jay Bradner, Amgen’s executive vice president for R&D, wrote in the company’s letter to the FDA. Duke’s re-adjudication of ADVOCATE data began in February 2026 and the pharma plans to submit results, alongside other “detailed data,” to the FDA by June 29.

Alongside the Duke analysis, Amgen has listed in its letter more than a dozen ongoing or recently completed studies to support Tavneos’ overall benefit-risk profile.

The FDA first called for the voluntary withdrawal of Tavneos in January, raising concerns about “the process followed by ChemoCentryx” to evaluate the primary endpoints in ADVOCATE for nine of the 331 patients enrolled, according to an Amgen release in February. The regulator also pointed to liver toxicities in the context of Tavneos’ overall risk-benefit profile.

Amgen refused the regulator’s request, leaving Tavneos on the market.

The agency continued to apply pressure, however, and in late March put out a safety alert flagging eight deaths and 76 cases of drug-induced liver injury (DILI) that have “reasonable evidence of a causal association with Tavneos.” Seven of the 76 cases involved biopsy-confirmed vanishing bile duct syndrome (VBS), all of which necessitated hospitalization and three of which contributed to the total tally of eight deaths.

While the FDA at the time conceded that liver toxicities have been “identified in premarket clinical trials and described in product labelling,” potentially fatal DILI and VBS “represent new safety concerns.”

Last month, Japan’s Kissei Pharmaceutical, which is Amgen’s partner in charge of Tavneos’ Japanese distribution, said that 20 patients on the drug have died since the product launched in 2022. Most of the deaths were attributed to DILI and VBS, though Kissei took care to say that it remains unclear if Tavneos was the direct cause of the fatalities.

The European Medicines Agency has also launched a review of Tavneos, similarly flagging “questions regarding the data integrity” of ADVOCATE, according to a January release.

The RNA-based medicine is one of a handful of antibody-oligonucleotide conjugates that Novartis acquired last October when it took over neuromuscular-focused Avidity Biosciences.

Novartis’ $12 billion gambit for Avidity Biosciences late last year is starting to pay off, with the pharma reporting promising biomarker findings for its investigational RNA medicine in patients with facioscapulohumeral muscular dystrophy.

In the Phase 1/2 FORTITUDE study, Novartis enrolled 90 patients to receive the antibody-oligonucleotide conjugate delpacibart braxlosiran (del-bax) or placebo. Aside from safety, the trial’s primary outcome is the treatment effect on KHDC1L, a plasma disease biomarker for facioscapulohumeral muscular dystrophy (FSHD).

Without disclosing specific data, Novartis in a Thursday news release said that FORTITUDE’s biomarker cohort met its primary and key secondary endpoints. KHDC1L levels were reduced in patients treated with del-brax, suggesting “strong target engagement,” the pharma noted.

Del-brax also elicited a drop in creatine kinase concentrations, which Novartis explained is indicative of “reduction in muscle damage” in treated patients.

These findings “replicate the target engagement and downstream muscle protection seen with del-brax in earlier dose-escalation cohorts,” Nazem Atassi, global head of neuroscience and gene therapy development at Novartis, said in a prepared statement.

A previous FORTITUDE readout covering earlier dosing cohorts showed that patients on del-brax improved on the 10-meter walk-run test at 12 months compared to placebo. Similarly, those given del-brax performed better on the timed up-and-go test as well as on quantitative muscle and upper limb function assessments at 12 months while placebo controls deteriorated.

“We are now evaluating the totality of the biomarker and clinical data and look forward to discussions with global regulatory agencies,” Atassi said on Thursday, though Novartis hasn’t yet specified filing plans for the asset. The pharma, meanwhile, has launched the Phase 3 FORTITUDE-3 trial of del-brax in FSHD, aiming to enroll around 200 patients and looking at the therapy’s effects on muscle performance.

FSHD is a rare and progressive neuromuscular disease that manifests as a life-long muscle wasting, pain, fatigue and disability. The condition, affecting some 45,000 to 87,000 people across the U.S. and Europe, is caused by the abnormal expression of the DUX4 protein, which in turn activates genes that are toxic to muscles. Del-brax addresses this disease mechanism by combining the specificity of antibodies and the precision of oligonucleotides to target and suppress the expression of DUX4, according to Avidity’s website.

The asset came to Novartis through the pharma’s $12 billion takeover of Avidity in October last year, alongside two other RNA-based therapies: del-zota, being proposed for Duchenne muscular dystrophy, and del-desiran, being trialed for myotonic dystrophy type 1. The pharma also won over a clutch of preclinical programs for other rare neuromuscular disorders.

Novartis closed the Avidity acquisition in February.

Takeda eyes an FDA run for its investigational psoriasis pill after the drug elicited total skin clearance in more than 35% of patients at 16 weeks—more than 2.5 times that in controls taking Bristol Myers Squibb’s Sotyktu.
Takeda’s investigational oral psoriasis drug significantly outperformed Bristol Myers Squibb’s Sotyktu at achieving complete skin clearance in a Phase 3 psoriasis study, setting the Japanese pharma up for a regulatory filing in the coming months.

Treatment with Takeda’s daily pill zasocitinib resulted in complete skin clearance in over 35% of treated patients at 16 weeks, the company said in a Thursday release. This effect was “more than 2.5 times the response rate for [BMS’ Sotyktu],” Chinwe Ukomadu, head of the Gastrointestinal & Inflammation Therapeutic Area at Takeda, said in a prepared statement. Zasocitinib’s separation of efficacy from Sotkytu was evident as early as week 8, Ukomadu added.

Zasocitinib also beat out Sotyktu in terms of key secondary outcomes, including PASI 90 response, which refers to at least a 90% improvement in skin lesions.

“These findings reinforce the promise of zasocitinib to deliver rapid and durable skin clearance in a convenient once-daily pill,” Ukomadu said.

Takeda bought zasocitinib from Nimbus Therapeutics in December for $4 billion, beating out “almost every biopharmaceutical company,” Andy Plump, Takeda’s head of R&D, told Fierce Biotech in January 2024. The drug, designed with AI, works by targeting the TYK2 protein and maintaining IL-23 inhibition for 24 hours, which gives it its therapeutic potential for inflammatory conditions.

In December last year, Takeda said that “on average about 30 percent” of patients treated with zasocitinib across two late-stage studies achieved completely clear skin, while more than half reached PASI 90.

These findings “de-risk regulatory approval,” according to a Dec. 19 note from Jefferies, which forecasted at the time that the oral psoriasis market could top $5 billion by 2030. The data, the firm added, “validate Zasocitinib’s potential to redefine the oral psoriasis market.”

Takeda provided more data from two studies of zasocitinib in March, touting total skin clearance rates ranging from 25.2% to 33.4% at week 16. These figures were significantly better than in comparators on placebo or Amgen’s Otezla.

With Thursday’s head-to-head outcomes, Takeda said that it is on track to file a drug application for zasocitinib with the FDA and other global health authorities “starting this fiscal year.” The pharma’s fiscal year runs from April 1 through March 31 the following calendar year.

Biotechs are benefitting from the AI tech frenzy and inflation, but validated pipelines and careful planning are still key to the recent record-setting IPOs, experts say.
Kailera Therapeutics’ $625 million biotech IPO record did not stand for long, as Parabilis stole the crown on Wednesday with a $670 million Nasdaq debut. These new records have generated much excitement across biotech and pharma, but experts say we shouldn’t get too used to them.

“These transactions are getting completed. It’s a really big change from the environment just a year ago, or two years ago, where you basically couldn’t give biotech new issues away,” said Les Funtleyer, healthcare portfolio manager at family office E Squared.

Now, there’s an overall market frenzy thanks to massive AI IPOs from Open AI and Anthropic. That’s trickling into biotech stocks to some extent, according to Funtleyer, but “these are paling in comparison with the AI games.” Peptide-maker Parabilis emerged with an enterprise value of $2.65 billion, according to S&P Capital IQ. Anthropic, on the other hand, broke records with a nearly $1 trillion valuation in May.

Nevertheless, Parabilis and Kailera’s debuts were biotech big, which suggests a huge appetite for biotech IPOs. In fact, Funtleyer says there’s a backlog of private biotechs at E Squared that are waiting to go public.

“I take all of this as a very positive sign,” Funtleyer said. He does not think that the IPO scene is going to heat up to the red-hot inferno that characterized the post 2021-period when many biotechs went public without validated data—only to disappoint investors or even go bankrupt. But he does see how biotech market watchers could think that the market could become over-saturated given the “recency bias” of the past few large IPOs.

“Your natural inclination is to almost think, oh my goodness, is this the end of the world? But there’s no indication that that is the case at the moment,” he said. “It’s actually when people get too comfortable, too complacent, that’s when you kind of think that something bad is going to happen. . . . In my judgment, the market hasn’t gotten complacent yet.”

The higher raises are really company specific, Funtleyer insisted. Larger IPOs going forward will be on a case-by-case basis, just as Kailera and Parabilis have been.

George Thampy, a biotech IPO and investor relations advisor, wrote on LinkedIn that Parabilis in particular has a star team of executives and researchers—including CEO Mathai Mammen and scientist Greg Verdine—driving the raise.

The company also went into the IPO with $800 million raised across private rounds. Once all is tallied from the underwriters and a $75 million concurrent private placement from partner Regeneron, the Parabilis IPO is likely to hit $845 million, according to Thampy.

Thampy also pointed to the fact that Parabilis raised 25% more than the midpoint estimate—the industry standard for initial IPO pricing—landing on the Nasdaq at $20. This “tells me that there was a torrent of new demand after the public flip, and that the order book was significantly more oversubscribed,” Thampy wrote.

The same kind of excitement seemed to usher Kailera through the IPO roadshow, according to Chief Commercial Officer Jamie Coleman. She told BioSpace last week that the company visited hundreds of investors, which underscored the interest in the obesity biotech’s pipeline. The company was touting a late-stage pipeline loaded with assets in the obesity space that was already partly derisked with data from China.

Kailera’s final tally after underwriters’ options hit $718.8 million, according to PitchBook data.

Both Parabilis and Kailera likely also benefitted from inflation, as everything is getting more expensive, Funtleyer noted.

But there were also fundamental differences between the two IPO leaders, Pitchbook Senior Analyst Ben Zercher said. “Unlike Kailera, which formed in 2024 and quickly stockpiled private capital before going public, Parabilis took a longer and far less linear road,” he told BioSpace in an email.

Parabilis began in 2017 as FogPharma, riding out what Zercher called “the post-pandemic biotech winter” before rebranding as Parabilis. The company then enjoyed a successful 2025 thanks to lead asset zolucatetide, which is being tested in several cancers including a Phase 2 program for desmoid tumors. Parabilis was able to prep for the IPO with a crossover round of $305 million in January, plus a lucrative partnership with Regeneron. Then zolucatetide received fast track and orphan drug status in March, which provided solid footing for the IPO.

Both companies’ advanced pipelines are key to the valuation and records, according to Funtleyer. He does not expect to see preclinical biotechs generating raises in the $600 million range anytime soon.

“If you start to see preclinical companies coming public with multi-billion dollar valuations, I think that’s when you start to look at the panic button—but there’s no indication that that’s the case now,” Funtleyer said.

There could be market disruptions due to macro factors, but for now the excitement is refreshing, according to Funtleyer.

On Thursday, Kardigan priced a more reserved IPO, seeking to raise $373.3 ⁠million with a valuation of $1.4 billion. One of BioSpace’s NextGen honorees, the company just announced proof-of-concept Phase 2 data in March, showing “clinically meaningful” changes in blood pressure for tonlamarsen.

“With the biotech window reopened, the volume of IPOs reflects a backlog of quality companies that kept building through the biotech funding downturn rather than a wave of hype,” Zercher said. “Where the pandemic-era class sold preclinical optionality, Parabilis and the 2026 cohort are being priced on de-risked clinical programs with clear regulatory paths.”

The newly public biotechs will now have to maintain that excitement and keep their share prices up, which could encourage other companies to go out. Thampy predicted that Parabilis would spike when the stock began trading on Tuesday because of the demand prior to the public flip. Sure enough, the shares were worth $31.60 at close—a 58% increase from the opening price.

Other companies have been less fortunate. Kailera’s shares have fallen 24% since debuting on April 17. Funtleyer wants to see how Parabilis trades over the next month as a guide for companies waiting in the wings.

“Biotech is coming back to life, which is great, but ultimately it really comes down to the individual issues,” Funtleyer said. “At a certain point, the company’s got to stand on its own.”