Dive Brief:

• The Food and Drug Administration has rejected a gene therapy developed by Rocket Pharmaceuticals for a rare type of inherited immunodeficiency, the biotechnology company disclosed Friday.
• According to Rocket, the FDA asked for “limited additional” information on what’s known as chemistry, manufacturing and controls — the data detailing the quality and consistency of the final drug product. The company said it has met with the agency to “align” on the scope of that request so it can move quickly to secure an eventual approval. 
• “It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access,” Rocket CEO Gaurav Shah said in the company’s statement.

Dive Insight:

The FDA doesn’t release complete response letters, meaning descriptions of the agency’s feedback come only from the company involved. Still, Wall Street analysts who spoke with Rocket are convinced the rejection is a speed bump, rather than a dead-end for the drugmaker’s gene therapy, called Kresladi. 

“We strongly suspect that Kresladi’s eventual approval is really just a matter of when — not if,” wrote Dae Gon Ha, an analyst at Stifel, in an investor note.

The FDA had already postponed its decision on Kresladi by three months due to a change in the reviewer overseeing Rocket’s application, meaning the agency couldn’t extend its evaluation of the application further. The complete response letter “strikes us as a reluctant last option for the FDA rather than a reflection of any significant deficiencies with the submission package,” Ha wrote. 

Analysts at William Blair and at Chardan outlined similar views in notes to clients Friday. “We ultimately view the CRL as more related to FDA staffing constraints than Kresladi’s critical quality attributes,” wrote William Blair’s Sami Corwin.

Kresladi is built from a patient’s own stem cells, which Rocket genetically modifies to deliver a functional copy of the gene that’s mutated in the condition the therapy treats. Known as severe leukocyte adhesion deficiency-I, the disease weakens the body’s ability to fight infections. Infants born with it often experience bacterial and fungal infections that require hospitalizations and survival beyond childhood is uncommon without a successful bone marrow transplant. 

All nine patients treated in Rocket’s small study of Kresladi have survived through the duration of the trial’s follow-up, now between 18 to 42 months. Data showed declines in the incidence of significant infections follow treatment, as well as signs of resolution of disease-related skin lesions, per Rocket. 

Even if the FDA’s issues with Rocket’s applications are easily resolved, the rejection is another setback for a field that’s faced notable clinical, regulatory and business hurdles. Manufacturing remains a challenging area for developers, as experienced by Abeona Therapeutics, which saw its skin disease cell therapy rejected by the FDA in April.

Biotechnology companies aren’t the only ones running into issues. Both Daiichi Sankyo and AbbVie have recently received manufacturing-related complete response letters from the FDA for drugs they submitted for approval.

Dive Brief:

• Advisers to the Centers for Disease Control and Prevention on Thursday unanimously recommended Merck & Co.’s recently approved pneumococcal vaccine for use in adults aged 65 years and older and in younger adults with certain health conditions who haven’t previously received a shot.
• The recommendation puts Merck’s vaccine, called Capvaxive, in the same bucket as Pfizer’s Prevnar 20 shot and Merck’s earlier pneumococcal products Vaxneuvance and Pneumovax. Capvaxive was approved by the Food and Drug Administration earlier this month to prevent invasive pneumococcal disease and pneumonia.
• Pfizer’s Prevnar franchise has long been dominant in the pneumococcal market. Merck hopes to compete with Vaxneuvance and Capvaxive, although it’s not clear how much share it might yet win.

Dive Insight:

Pneumococcal disease can lead to severe infections in the lungs such as pneumonia or meningitis. Older adults and people with certain health conditions are at higher risk of disease.

Capvaxive is Merck’s latest entrant in the pneumococcal vaccine market and offers broad coverage against 21 types of the bacteria that causes pneumococcal disease. Eight of those serotypes are not covered by other shots.

The CDC panel specifically voted to recommend a single Capvaxive dose in adults older than 65 with no prior pneumococcal vaccination or whose vaccination history is unknown. Adults aged 19 to 64 years old who have certain underlying health conditions and no previous record of receiving a pneumococcal conjugate vaccine can also receive the shot, the advisers indicated.

The panel postponed discussion of lowering the age-based recommendation for pneumococcal vaccination to 50 from 65, however. They expect to take that question up at an October meeting.

“When they pick this decision back up in October, we believe the committee will lower the age for vaccination to 50 and recommend both Capvaxive and Prevnar 20,” wrote Leerink Partners analyst Daina Graybosch in a note to clients. “This, as in the current market, will put the companies on equal footing and put Capvaxive sales in the hands of Merck’s commercial organization.”

Another pneumococcal vaccine developer, Vaxcyte, predicts substantial growth in the overall market, forecasts annual sales to reach about $13 billion by 2027, up from $8 billion today.

Total sales for Merck’s Vaxneuvance and Pneumovax 23 totaled over $1 billion in 2023. Pfizer’s Prevnar franchise generated over $6 billion in sales.

Merck previously said Capvaxive would be available as soon as late July.

Dive Brief:

• 2seventy Bio is shrinking further, announcing Thursday the sale of a hemophilia A research program, along with associated technology rights, to partner Novo Nordisk.
• 2seventy and Novo first teamed up in 2019 to develop a gene editing therapy for the bleeding condition. Under Thursday’s deal, Novo will pay $38 million in cash to acquire the therapy, which is in preclinical testing. 2seventy staff who have been working on the program will join Novo.
• “Novo Nordisk has been a valued partner over the past five years, and we are confident that under their leadership, the promise of developing a new treatment approach for patients living with hemophilia A will continue to progress,” said 2seventy CEO Chip Baird in a statement.

Dive Insight:

2seventy is the product of Bluebird Bio splitting in half nearly three years ago. The road since then has been difficult for 2seventy, which inherited Bluebird’s cancer drug pipeline. A weak financing market for biotechnology companies made raising fresh funding difficult, while the initial launch of Abecma, a cell therapy for multiple myeloma developed with Bristol Myers Squibb, was hamstrung by tight supply.

Last September, longtime Bluebird CEO Nick Leschly stepped down as head of 2seventy, part of a restructuring that claimed about 180 jobs. Five months later, 2seventy sold off much of its pipeline to Regeneron Pharmaceuticals for cheap in a deal that sent 150 2seventy employees to the larger company.

Thursday’s deal tightens 2seventy’s focus even more narrowly on Abecma, which recently won an expanded approval for earlier multiple myeloma use in the U.S.

In addition to the hemophilia A program, Novo is also acquiring rights to 2seventy’s in vivo gene editing technology outside of its use in cancer and gene editing for autoimmune disease cell therapies.

The hemophilia program in question is built around a gene editing technology known as megaTALs that the companies envisioned using to insert a functional gene into the genome of liver cells. The target gene, known as Factor VIII, is mutated in people with hemophilia A.

While Novo is best known for its diabetes and obesity medicines, the company is active in hemophilia research and recently disclosed updated results for a preventive therapy it’s developing.

“We are devoted to developing therapies with a curative outlook, including our continued development of a next-generation in vivo genome editing program aiming to offer individuals living with Hemophilia A a lifetime free of factor replacement therapy,” said Karina Thorn, Novo’s head of global nucleic acid therapies research, in a statement.

Shares in 2seventy rose by nearly 5% in Thursday morning trading.

Dive Brief:

• Shares of Moderna, Pfizer and GSK fell as advisers to the Centers for Disease Control and Prevention discussed updated guidelines for RSV vaccination at a Wednesday meeting.
• The expert panel recommended adults over 75 years should receive a shot for RSV, or respiratory syncytial virus, as should those between 60 and 74 years who are at higher risk of severe disease. The recommendation, which the CDC adopted, replaces the shared clinical decision-making guidance for all adults over 60 that was put in place last year. 
• The committee postponed discussion of vaccine recommendations for adults aged 50 to 59 years old, which could potentially delay GSK’s plans to expand its shot’s use in that age group after a recent Food and Drug Administration approval.

Dive Insight:

The new vaccination recommendations could shape the market for RSV shots in the U.S. Notably, the CDC is not yet recommending that anyone who previously was vaccinated for RSV get another dose. 

The guidelines for adults between 60 and 74 years old are now more narrow. While people in this age group who are not high risk of RSV disease can still get a vaccine, the changed recommendations might affect whether a shot is covered by insurance. The list of relevant risk factors is broad, however, including cardiovascular disease, lung disease and neurologic or neuromuscular conditions.

Committee members also reviewed at the Wednesday meeting safety data on a neurological side effect called Guillain-Barré syndrome, or GBS, that’s been reported in very rare cases following vaccination GSK’s Arexvy and Pfizer’s Abrysvo. No cases have been reported with Moderna’s newer vaccine Mresvia.

While the rate associated with Abrysvo was higher than with Arexvy, an FDA presentation noted that the analyses were “mixed and highly uncertain.” Still, Evercore ISI analyst Umer Raffat wrote that the advisers gave a “huge pass” to Pfizer. 

Shares in Moderna were hit particularly hard as the company presented updated data on its shot’s efficacy over time. After 18 months, Mresvia’s protection against RSV disease with two or more symptoms fell to 50%, lower than with GSK’s Abrysvo. 

Moderna has been banking on winning a share of the market with its mRNA-based shot, which is available as a more convenient pre-filled syrigne. The vaccine was approved in May.

Dive Brief:

• Novo Nordisk will end a Phase 3 clinical trial of a hypertension drug it bought for up to $1.3 billion last year because treatment didn’t appear to be working, the company said Wednesday.
• The decision was based on an interim data analysis by study monitors, which revealed the drug, called ocedurenone, met the trial’s “futility criteria,” indicating treated participants didn’t experience a greater drop in blood pressure than those given placebo.
• The company will take an accounting charge of 5.7 billion Danish kroner, or about $817 million, on its second quarter earnings to offset the loss of valuation represented by ocedurenone. The charge will lower Novo’s operating profit guidance by 6 percentage points from the 22% to 30% it forecast in May.

Dive Insight:

Novo bought ocedurenone from Singapore-based KBP Biosciences, one of a string of acquisitions made as it seeks to put profits from its diabetes and obesity drugs Ozempic and Wegovy to work.

When Novo announced the deal, KBP had already begun a Phase 3 trial of ocedurenone in people with chronic kidney disease and hypertension that couldn’t be controlled with at least two medications. Results from a Phase 2 study of 162 people had shown ocedurenone lowered blood pressure in that population by 10 points more than a placebo.

The larger Phase 3 study, intended to confirm the earlier findings, enrolled 652. The decision to terminate the trial was based on an interim analysis investigators set up before it began, which involved measuring blood pressure after 12 weeks of treatment for a difference between study volunteers taking ocedurenone and those on placebo.

Had the drug shown signs of benefit at 12 weeks, the study would have evaluated blood pressure again between 48 and 52 weeks to determine how well it worked.

Novo said it is still evaluating next steps for ocedurenone in other indications. When the two companies agreed on the deal, Novo executives had said they intended to initiate Phase 3 trials in cardiovascular disease and other kidney disease settings.

People with chronic kidney disease and hypertension often take multiple drugs to lower blood pressure, including medicines called angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, diuretics, calcium channel blockers or beta blockers. Ocedurenone is a type of diuretic called a mineralocorticoid receptor antagonist — a similar mechanism of action to Bayer’s recently approved Kerendia.

Ozempic and GLP-1 drugs like it became household names due to their potent weight loss benefits, and are now proving useful in a slew of other chronic conditions.

Women are noticing other benefits, too, including some that may be flying under pharmaceutical companies’ radars. Reports have emerged of unexpected pregnancies among women on GLP-1 treatment, and researchers have mused of possible effects on fertility or on hormonal disorders as well.

Melanie Cree, a pediatric endocrinologist at Children’s Hospital Colorado, is one physician looking into this question. Working with her colleagues, she conducted a randomized trial testing semaglutide, the ingredient in Novo Nordisk’s Rybelsus and Wegovy, in women and adolescents with polycystic ovary syndrome, or PCOS.

Cree said this research “provides evidence of what these medications can do for PCOS, reproductive measures and periods specifically.” While results from Cree’s first trial aren’t yet published, they were encouraging enough to spur Cree to launch another, larger trial testing the link between weight loss, metabolic changes and reproductive function in people with PCOS.

PCOS is a set of symptoms caused by a hormonal imbalance, usually overproduction of testosterone, that disrupts ovulation. The condition leads to cysts developing in the ovaries, which in turn changes an individual’s menstrual cycle and may result in infertility. People with PCOS can also develop insulin resistance, increasing the risk of Type 2 diabetes and other cardiometabolic issues. The disorder is typically diagnosed during adolescence.

There is no standard treatment for PCOS, and diagnosis can often be time-consuming and difficult. The World Health Organization estimates 70% of affected women remaining undiagnosed globally.

“[Researchers] haven’t pinpointed the reason or background etiology for the condition,” said Pardis Hosseinzadeh, an assistant professor of gynecology and obstetrics at Johns Hopkins School of Medicine. “So obviously, when that’s not fully discovered yet it gets hard to treat PCOS, because it affects many different systems in the body.”

Depending on symptoms, doctors may recommend different medications or other interventions, such as weight management or birth control that helps with ovulation, mood changes and hormonal acne.

“Studies like mine and my colleagues’ are very important because there are currently no FDA-approved medications for PCOS,” Cree said.

Weight loss can improve symptoms of PCOS and is therefore usually an objective of treatment. Often, metformin, a diabetes medication, is prescribed, but other lifestyle interventions or even bariatric surgery may also be used.

“One of the primary reasons I started looking at GLP-1’s is because there is this tight association between weight, PCOS hormones and PCOS periods,” Cree said.

Endocrinologists and doctors helping women with PCOS are well aware of the difference weight loss can make in menstrual cycles and hormone regulation. Last year, the American Society for Reproductive Medicine, or ASRM, updated its guidelines for treating PCOS, which had been the same since 2018. The new guidelines now note the potential for GLP-1 drugs like semaglutide to help control weight. 

Chau Thien Tay, an endocrinologist at Monash Health who contributed to the new guidelines, told BioPharma Dive that weight loss therapies were considered in the previous framework. The inclusion of GLP-1s this time around “simply reflects a shift in the therapeutic landscape guided by recent advancements and clinical evidence,” she wrote.

With her new study, Cree aims to study whether PCOS symptoms are eased with weight loss, regardless of method, or whether GLP-1 drugs specifically can help with hormonal disorders.

The 10-month trial, which aims to recruit 80 girls and women between 12 and 35 years old, will test the effects of injected semaglutide on ovulation. It will also assess whether age or metabolic and hormonal changes could predict the response to GLP-1 treatment.

Dive Brief:

• Novo Nordisk will spend $4.1 billion to build a new U.S. factory in North Carolina, the latest multibillion-dollar investment by the Danish drugmaker to expand production of its fast-selling weight loss and diabetes drugs Wegovy and Ozempic.
• Funding for the planned factory, announced Tuesday, comes as Novo plans to allocate $6.8 billion towards manufacturing this year. The investments are designed to help the company expand access to its obesity medicines and fend off competition from Eli Lilly.
• Novo last year budgeted $3.9 billion on production capacity expansions. It added even more bandwidth earlier this year, when its controlling shareholder agreed to buy contract manufacturing company Catalent and sell three plants to Novo in a separate transaction. Meanwhile, Lilly has committed $9 billion to facilities in Indiana.

Dive Insight:

Wall Street analysts believe the market for weight loss drugs like Wegovy and Zepbound could surpass more than $100 billion a year by the 2030s. But even now, manufacturers of marketed products are struggling to keep pace with demand. Novo limited access to the lower, “starter” doses of Wegovy for months, while some doses of Lilly’s Zepbound and Mounjaro are currently in shortage.

Demand is expected to grow in the years ahead, as Novo and Lilly bring their medicines into new indications and broaden insurance coverage. Novo showed in testing that Wegovy can help reduce the risk of cardiovascular events, winning Medicare coverage for some people. Lilly could similarly win over Medicare with recent results showing Zepbound can ease obstructive sleep apnea.

The two companies are also testing their medicines in other common conditions, like the metabolic disease known as MASH, heart failure and even Alzheimer’s.

In the meantime, Lilly and Novo are pouring considerable resources into drug production. The $9 billion Lilly is spending on facilities in Indiana is the largest single investment in its history. Now Novo is following suit with plans to significantly increase its production footprint in North Carolina.

Novo already has a large presence in the state, with three production plants there. But the new plant in Clayton will double its existing square footage. Foundational work has already begun on a 56-acre site, with construction expected to be finalized between 2027 and 2029. The facility will specialize in what’s known as fill-finish, or sterilizing and standardizing medicines and containers before filling and sealing them with drug products.

When completed, the new facility will add 1,000 new jobs to the roughly 2,500 the company already employs in the region.

The facility is the second in the U.S. the company has added this year. The Catalent transaction hands Novo a plant in Indiana, as well as manufacturing sites in Italy and Belgium.

In addition to its investment in Indiana, meanwhile, Lilly is buying a plant in Wisconsin.

Dive Brief:

• Novo Nordisk on Sunday disclosed detailed clinical trial results for an experimental hemophilia treatment dubbed Mim8, showing once-weekly and once-monthly doses of the antibody drug controlled bleeding in people with the more common “A” form of the disorder.
• The data, which were presented at the International Society on Thrombosis and Haemostasis Annual Congress in Thailand, fill in a positive picture for Mim8’s effectiveness and safety. Novo had said in May that the Phase 3 trial, called Frontier-2, succeeded and shared topline findings.
• Among people who had not previously been on preventive treatment, researchers reported zero bleeds in 86% of study participants who received once-weekly Mim8, and 95% of those given the once-monthly dose. Those figures were 66% and 65%, respectively, among people in the trial who had prior preventive treatment.

Dive Insight:

The success of Ozempic and Wegovy, Novo’s drugs for diabetes and weight loss, have transformed the Danish drugmaker into the pharmaceutical industry’s second largest. Not surprisingly, analysts and Wall Street investors are now laser focused on that portion of the company’s business.

But Novo continues to develop drugs for rare blood disorders, and has placed emphasis on the potential of Mim8. It’s also invested in sickle cell disease, acquiring Forma Therapeutics two years ago and advancing another drug it licensed in 2018.

The recent results for Mim8 are the first from a Phase 3 trial program that includes three other studies beyond Frontier-2.

Novo enrolled 254 people with hemophilia A into Frontier-2, including those with and without “inhibitors,” which can prevent standard drugs from effectively clotting blood. The one-year study compared Mim8 to either no prophylaxis among those not on preventive treatment, or to prior coagulation factor prophylaxis among those who were.

In the former group, a once-weekly dose of Mim8 reduced the average annualized bleeding rate by 97%, while the once-monthly dose led to a 99% reduction. That translated to an average of 0.45 treated bleeds per patient year among the once-weekly group and 0.20 among the once-monthly patients. In the control arm of people who did not receive preventive treatment, the average rate was 15.75 treated bleeds per patient year.

Patients in the study who previously were on preventive treatment went through a “run-in” phase of the study, and then were given one of the two Mim8 doses. The average rate of treated bleeds was 2.51 per patient year for those on once-weekly dosing, versus 4.83 on their prior prophylaxis, and 1.78 for those on the once-monthly, versus 3.10 on their prior treatment.

There were no thromboembolic events or related serious side effects reported in the trial, Novo said.

“With Mim8, we have the potential of offering a substantial proportion of patients the prospect of zero bleeds and convenient dosing flexibility to fit their lifestyles and needs,” said Martin Holst Lange, head of development for Novo, in the company’s Sunday statement.

Novo plans to submit an approval application for Mim8 toward the end of the year, and will disclose more trial data for the drug at upcoming medical meetings.

A “bispecific” antibody, Mim8 is designed to bridge Factors IXa and X to replace the Factor VIII that’s missing in people with hemophilia A.

If approved, Mim8 would likely compete with Roche’s Hemlibra, which is also a bispecific anitbody and holds approval in the U.S. as a routine preventive treatment for people with hemophilia A. Dosing is flexible to either once-weekly, bi-weekly or once-monthly. Roche reported sales of 4.1 billion Swiss francs last year, or about $4.6 billion, using an average U.S. dollar exchange rate for 2023.

Today, a brief rundown of news from Altimmune, Jiangsu Hengrui, Eli Lilly and others that you might have missed from over the weekend, when the American Diabetes Association held its annual meeting.

After an early setback for Altimmune’s obesity shot pemvidutide, the biotechnology company appears to be back on track with additional data. The dual-acting drug, which stimulates the hormones GLP-1 and glucagon, helped participants in the Momentum trial lose an average of 16% of their body weight at the highest dose given, according to full trial results presented at ADA. That weight loss at 48 weeks is in line with what Novo Nordisk’s Wegovy produced at 68 weeks. The company also pointed to pemvidutide’s preservation of lean body mass, which could help differentiate it if approved. Trial investigators described the shot as “well-tolerated,” although early data cuts had revealed that nearly one-quarter of patients dropped out due to side effects. — Jonathan Gardner

An obesity drug Jiangsu Hengrui Pharmaceuticals recently licensed to a new startup company showed weight loss in a Phase 2 trial that appears “very competitive” to other drugs in development, wrote Cantor Fitzgerald analyst Prakhar Agrawal in a Sunday note to clients. Treatment with the “dual agonist” drug, code-named HRS9531, led to weight loss at 24 weeks of up to 17% among participants given the highest dose. The startup now developing it is backed by $400 million raised from investors Bain Capital Life Sciences, RTW Investments, Atlas Venture and Lyra Capital. — Ned Pagliarulo

Back in April, Eli Lilly released summary data on studies of weight loss medicine Zepbound in people with obesity and sleep apnea. A fuller presentation of results at the ADA meeting, also published in The New England Journal of Medicine Friday, revealed data on “secondary” measures. Among them was a finding that 42% of trial volunteers taking Zepbound alone and half of those taking it while using a breathing machine saw resolution of their condition when measured by sleep apnea events and daytime sleepiness scores. Lilly also said it has submitted the data to the Food and Drug Administration. An approval could allow for Medicare coverage of Zepbound for people with obesity and sleep apnea. — Jonathan Gardner

Three people with Type 1 diabetes achieved independence from insulin one year following treatment with a stem cell-derived cell therapy being developed by Vertex Pharmaceuticals, according to data presented at the ADA meeting on Friday. Researchers also reported the elimination of severe hypoglycemic events in these three individuals, who all had HbA1C levels below the recommended 7%. Nine other study participants with shorter follow-up also appear to be benefiting from treatment, showing blood sugar control and endogenous insulin secretion. — Ned Pagliarulo

Amylyx Pharmaceuticals is buying an experimental, blood sugar-regulating medicine from Eiger Biopharmaceuticals, which is currently selling assets as part of its Chapter 11 bankruptcy proceedings. Eiger’s medicine acts on a protein tied to GLP-1 — the hormone at the center of a revolution in obesity and diabetes care. On Friday, Amylyx agreed to acquire all the rights to the drug for $35 million, plus some other, smaller costs. Earlier this year, the company pulled its closely watched treatment for ALS from the market, and has been trying to recover. — Jacob Bell

Today, a brief rundown of news from Belharra Therapeutics, AstraZeneca, Regenxbio and Tasyha Gene Therapies.

Sanofi will work with biotechnology startup Belharra Therapeutics to develop small molecule drugs for inflammatory diseases, the companies announced Tuesday. Belharra will get $40 million in upfront and near-term payments to start the deal, through which it will use what it describes as “next-generation chemoproteomics” to identify immune drug prospects. The alliance is the second for Belharra, which launched last year with $130 million in funding and a research pact in place with Genentech. — Ben Fidler

AstraZeneca’s bid to expand use of its new breast cancer drug Truqap has fallen short. In the Capitello-290 trial in “triple negative” breast cancer, Truqap plus chemotherapy didn’t help people live longer than did a placebo and chemotherapy. The drug is approved to treat people with hormone-receptor positive, HER2-negative breast cancer if they have certain genetic alterations, a setting where it recorded $50 million in sales in the first quarter of 2024. The U.K.-based drugmaker expects data from a trial in prostate cancer later this year. — Jonathan Gardner

A gene therapy from Taysha Gene Therapies helped improve motor function in four people with the rare neurodevelopmental disorder Rett syndrome, the biotechnology company said Tuesday. The data are from two adult and two pediatric patients enrolled in a Phase 1/2 trial of Taysha’s therapy, called TSHA-102. One of the treated adults sat up unassisted for the first time in over a decade, while researchers observed symptom benefits in the other participants. Taysha plans to next test higher doses of its therapy. — Ned Pagliarulo

Regenxbio got agreement from the Food and Drug Administration on details of an accelerated approval application for a gene therapy the company is developing for the neurodegenerative disease Hunter syndrome. According to Regenxbio, the agency endorses the company’s plans to use a neurological protein as a surrogate marker “reasonably likely” to predict clinical benefit. It’s also discussed with Regenxbio the design of a confirmatory trial that would start next year. The biotech plans to start submitting its application in the third quarter. Dubbed RGX-121, the therapy would be its first wholly owned treatment to reach market. — Ben Fidler

The FDA has partially halted testing of an antibody-drug conjugate BioNTech licensed last year from China-based biotech MediLink Therapeutics. The FDA is concerned that higher doses of the drug, which is being evaluated against breast and lung tumors, may lead to “unreasonable and significant risk of illness and injuries,” according to a regulatory filing from BioNTech. MediLink has stopped enrolling new patients in a Phase 1 trial in the U.S. while it addresses the FDA’s concerns. — Ben Fidler