Immunology

When Roivant Sciences CEO Matt Gline held a meeting with pharma giant Roche at the J.P. Morgan Healthcare Conference in early 2023, he expected a brief one-on-one conversation with a member of the Swiss pharma’s team.

Instead, 13 people showed up to the meeting “and it evolved from there,” Gline said in an October interview, days after Roche announced a $7 billion deal to buy Roivant subsidiary Telavant, picking up a promising drug candidate for inflammatory bowel disease, or IBD.

The deal, fostered by Telavant chairman and CEO Frank Torti, was a case of good science and good business colliding, Torti said.

“There’s a lot of value in this ecosystem for doing things that really matter,” Torti said. “And that’s where we focus.”

Telavant came about in 2022 as a result of a joint venture between Roivant and Pfizer. While Roche will have commercialization rights for the drug, called RVT-3101, in the U.S. and Japan, Pfizer will sell it outside of those countries.

The deal is exactly what Roivant, founded by former CEO Vivek Ramaswamy in 2014, is all about — reading the industry tea leaves to find valuable drugs and conduct business deals like the one with Roche, Gline said.

“The sort of market property that makes us successful is that other people have to make strategic decisions based on what’s going on in their world,” Gline said.

Gline sees Roivant as “agnostic to therapeutic category” and focused on finding the right biologic areas that can produce a return on investment with minimal risk.

“If we said we’re only interested in deals in a certain therapeutic area, that indication would be very narrowly limiting our aperture,” Gline said.

Making of a deal

RVT-3101 is in a class of drugs called TL1A directed antibodies, which have drawn wide interest. Another company developing TL1A antibodies, Prometheus Therapeutics, was purchased this year by Merck & Co. for $10.8 billion.

“People forget how fast this class evolved,” Torti said. “When Prometheus put out data about a year ago, it validated the class — we followed that, and suddenly now you have two positive Phase 2 studies.”

Making that kind of bet on a growing drug class is how Roivant anticipates where the market is headed and stays ahead of the competition, Torti said.

“Very rapidly, this went from a class that people were kind of interested in and following to a class that would look like it was going to transform the treatment of IBD,” Torti said. “And so with that transformation, every step of the way, we have had a lot of strategic inbound — we heard from a lot of folks in that process and had back-and-forth conversations with a number of parties over a number of months, which culminated in this deal.”

With Roche coming out on top after the monthslong conversation process, the pharma giant pulled in an asset in a therapeutic area that had eluded them once before. Roche last year pulled the plug on etrolizumab, an antibody the company was developing for Crohn’s disease. RVT-3101 could bring Roche back into the game.

And Gline believes Roche is the right company to make the most of RVT-3101.

“Roche’s credentials developing drugs probably speak for themselves at some level, and what we liked about it is we thought they saw a big opportunity to benefit patients certainly with IBD but potentially beyond IBD,” Gline said.

Opportunity in change

Immunology is a lucrative market with multiple blockbusters such as Humira, which topped the list of bestselling drugs for half a decade before it began to see competition this year. But that doesn’t mean there aren’t opportunities for improvement, Gline said.

“These things ebb and flow in terms of market dynamics and how crowded the space is and what pharma companies are doing, but scientifically, we’re just getting started here,” Gline said. “There has been this Cambrian explosion in the anti-inflammatory world, and there’s a huge category of diseases, some rare and some more prevalent, where there’s white space opportunity, and we’re just getting good at it.”

For Roivant, seeing a shift at companies like Roche means opportunity, Gline said. Roche, like other pharma companies its size, is looking to revamp its drug pipeline due to trial failures and near-term patent cliffs for blockbusters like Actemra.

By watching pharmas like Roche are doing, Gline said a company like Roivant can profit by matching their needs.

“These companies are smart, thoughtful and they make good decisions about what they’re interested in — but they are large, sophisticated, complicated machines that can’t turn quickly,” Gline said. “And when they’re making changes, invariably, there’s a long tail of stuff that falls out.”

But Gline doesn’t see Roivant the way some of its critics have: as a “discard” company buying up and pursuing projects started elsewhere.

“Discard, shelf programs — these are all misnomers for it,” Gline said. “TL1A was nobody’s discard. TL1A was an incredibly important scientific program that was an oddly shaped puzzle piece that didn’t quite fit Pfizer’s strategic plan.”

So when a company like Roivant finishes development of a program that doesn’t fit elsewhere, everyone wins, Gline said.

“We’re building a business, and the deals like the one we’ve just announced are once in a lifetime,” Gline said. “When big pharma companies are moving slowly, putting a lot of their blue chips and muscle behind what they’re trying to do, sometimes the best course is something we happen to have — and that’s a good position to be in.”

The executives behind autoimmune drug developer Prometheus Biosciences are back, armed with more than $400 million to launch their latest venture, a biotechnology startup known as Mirador Therapeutics.

Based in San Diego, Mirador looks similar to its predecessor, which Merck & Co. acquired roughly a year ago for nearly $11 billion. Like Prometheus, Mirador intends to develop drugs for inflammatory and fibrotic diseases. And like many biotech startups, the company intends to do so with a precision approach, using a combination of genetic insights, large datasets and tailored diagnostics to identify drug targets and patients who might be most likely to benefit from treatment.

"The journey was unfinished," said Mirador CEO Mark McKenna, Prometheus’ former leader, in an interview with BioPharma Dive.

Prometheus’ work led to a drug aimed at a regulatory protein, TL1A, that’s implicated in inflammatory bowel disease. Its acquisition came amid a rush of dealmaking for new types of medicines for inflammatory conditions, which are typically treated with more blunt forces that tamp down the immune system. Following Prometheus’ buyout, Roche and Sanofi each bought into similar TL1A medicines from other developers.

According to McKenna, though, Prometheus didn’t “finish the mission” it had originally taken on. So he brought many members of the team back together to try to build a company focused on precision therapies for autoimmune diseases, leaning on their experience and a wealth of data to develop new medicines for inflammatory conditions of the gut, lung and skin.

“It’s much broader than what we were doing before,” McKenna said.

Mirador isn’t saying which conditions it’ll go after first, or which drug targets it’s identified. McKenna only revealed that those targets have “strong genetic ties” to the diseases it’s interested in. But he envisions advancing multiple new drug prospects over the next 18 months and also indicated the company, with its large bankroll, may do deals to bring in more advanced candidates as well.

“Speed is the new currency in biotech,” he said. “We demonstrated it before and we can do it again.”

The company’s Series A round was led by Arch Venture Partners and includes a long list of investors such as OrbiMed, Fairmount, Fidelity Management and Sanofi. It is the largest private financing raised by a biotech startup so far in 2024, and one of the sector’s most sizable Series A rounds in recent years.

The financing comes at a time when private investment in biotech appears to be picking up after a multiyear slump. Industry insiders interviewed by BioPharma Dive earlier this year indicated that there’s been a recent surge in investments, particularly in newer companies. In March, three other biotech startups raised funding rounds of at least $150 million.

A surge of investments has carried more advanced companies, too. In a recent report, analysts at the investment bank Jefferies wrote that public biotech stock fundings are on pace for their best quarter in three years. Initial public offerings are also on their fastest trajectory, in terms of total dollars raised, since 2021, according to BioPharma Dive data.

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By Thermo Fisher Scientific

A new class of therapy focused on CAR-engineered natural killer (NK) cells is now emerging in early trials to help fill gaps in chimeric antigen receptor T-cell therapy (CAR-T cell therapy) – comparable enough to CAR-T to build off existing manufacturing approaches, but distinct in ways that really matter for wider access and better safety.

We spoke with thought leaders from the product engineering side of cell therapy on the state of the field, what’s coming next, and why now is the right time to be excited about CAR-NK therapy.

What is CAR-NK therapy and how does it work?

Like T cells, NK cells are lymphocytes – NK accounts for 5-20% of them. But unlike T cells, which must first be primed to respond, NK cells are part of the innate immune system responsible for the body’s first line of defense.

“They’re already killer cells. But if you can make them even more functional and targeted to a specific cancer, the therapy may not require as many doses or as many cells,” said Erica Heipertz, a cell biologist at Thermo Fisher Scientific who specializes in NK cell research.

NK therapy does not appear to carry the same side effects or risk of graft-versus-host disease (GVHD) even in allogeneic form, and it requires none of the immune HLA mapping and matching that can take precious time and make it difficult to source a donor.

All of this adds up to another exciting option for treating blood cancers, and there’s early hope that NK cells may migrate more easily than T cells to the solid tumor microenvironment.

There’s never been a more thrilling time to reconsider the simple savvy of the NK cell. The biology is there, the tools for manipulation are there, and the combined potential is incredible.

“The field really started with autologous cell therapy, and now we have ten years or so of research in that space,” said Xavier de Mollerat du Jeu, Senior Director of Research and Development in Cell and Gene Therapy at Thermo Fisher Scientific.

It has given NK cell therapy research and manufacturing a nice head start.

The cell therapy instrument manufacturing landscape has had time to improve from the early days of DIY ‘garage workshop’ style setups of individual instruments to factory-scale, fit-for-purpose, integrated manufacturing systems.

Even with these tools, scaling up cell therapy production to meet demand is no small task. If autologous therapy is the only option, it may be an insurmountable task. The average autologous CAR-T therapy course requires 3-8 weeks of manufacturing time, multiplied by some 60,000 patients in need annually.

From the patient’s perspective, that ‘vein-to-vein’ waiting period between cell collection and infusion can be more time than they have to spare.

“If we can figure out a way to have a bank of these cells cryopreserved and available as a patient needs it? All you’d have to do is thaw and inject the therapy. That’s the big dream,” said Heipertz. “Patients won’t have to wait weeks and weeks for a biotech company to harvest and expand and engineer their own cells to give their treatment. Cell therapy should be something that one day is as readily available as a pill.”

NK cell expansion innovation

“Whatever we can do to either speed up expansion time or make therapies more effective so that fewer cells are needed – that’s where the field is likely going. And unlike T cells, NK cells don’t really stick around for very long in the body. So it will take multiple doses and quite a large number of cells,” said Heipertz.

Building CAR-NK manufacturing infrastructure for the future

Cell therapy is so much more individualized that it presents an entirely new scale of operations, and tools need to be miniaturized to adapt.

“We need instruments that are integrated, automated, and have smaller footprints, so that you can put more into a facility and produce more therapy for the market. There are only six cell therapies approved on the market right now, but we’re projected to have as many as 25 annually in the next 5 years or so,“ de Mollerat du Jeu said.

That’s where next-generation, automated, closed-system instruments come in. It’s all part of a strategy is to build “fit-for-purpose” tools designed for this industry that cut out exposure to the environment and, with it, risk of exposing vulnerable patients to contamination.

Looking ahead towards CAR-NK therapy and beyond

It’s difficult to overstate how “astonishing” the results of cell therapy feel for the people who benefit, and for those who witness it as the miraculous revival of a gravely ill friend or family member.

We need to build better infrastructure and set the stage for a cresting wave of innovation – a revolutionary mix of accessible “off-the-shelf” therapies, new modalities of treatment (like NK cell therapy) mixed with improved applications of old, and immunotherapies that we may not have even uncovered yet.

The clue came, as they often do, from an unexpected source. In September 2022, a small study of five people in Germany pointed to a new direction for the high-profile field of cell therapy.

The study showed a cellular medicine could drive a tough-to-treat form of lupus into remission. While early, the finding suggested a technique used to create powerful cellular treatments for cancer might also work against autoimmune conditions like lupus, too.

“It’s very early days, but it’s exciting for the field to see this possibility,” said cell therapy pioneer and University of Pennsylvania immunologist Carl June, in an interview at the time.

The biopharmaceutical industry took note. Since the paper was published, a number of drugmakers have revealed plans to develop cell therapies for lupus, and nearly a dozen are now in clinical testing.

Updated results from that German study, presented at a medical meeting in December, indicate they may be right to invest. Among 15 people who had lupus or one of two other autoimmune diseases and hadn’t responded to prior treatment, all were in remission after receiving cell therapy. Some responses have lasted two years.

“It’s the tip of the iceberg, but it looks extraordinary,” said Andy Plump, Takeda Pharmaceutical’s head of research, in a recent interview. “The hope is that this can expand to not just other refractory autoimmune diseases, but to more common diseases.”

Challenges remain. Current cell therapies for cancer are associated with side effects that in rare cases can be fatal, a risk that’s easier to accept for people with deadly tumors than those with chronic health conditions. They’re also complex to produce and administer, and only available at major treatment centers, limiting their potential for wider use. Some believe newer “off-the-shelf” alternatives may be a better fit.

Answers could come within the next few years from clinical trials just getting underway. At least a dozen studies run by startups, biotechnology companies and large pharmaceutical giants are either recruiting participants or soon will be. Here’s where the field stands:

How is lupus treated now?

Lupus is a chronic autoimmune condition that occurs when the body attacks itself. The disease leads to pain and inflammation in many parts of the body, from the skin and joints to internal organs like the kidneys or lungs. About 1.5 million people in the U.S. and 5 million worldwide have a form of the disease, according to the Lupus Foundation of America, a patient advocacy group. For reasons that aren’t quite clear, many of those affected are women of childbearing age. Researchers aren’t certain what triggers the disease in the first place, either.

Lupus has several types, the most common of which is known as systemic lupus erythematosus, or SLE. It affects multiple organs and can involve a constellation of symptoms, making it tough to diagnose. Lupus’ characteristic ‘flares’ arrive unpredictably and can last days or weeks. While most people can manage their disease and live a long life, the condition can result in kidney failure or death from heart-related complications.

There is no cure. Available treatments can help manage symptoms and counteract the immune response associated with the disease. They include repurposed anti-inflammatory or immunosuppressive therapies, steroids or antimalarials.

More recently, three medicines specifically developed for lupus — GSK’s Benlysta, Aurinia Pharmaceuticals’ Lupkynis and AstraZeneca’s Saphnelo — have reached market. Roche’s cancer drug rituximab is sometimes used off-label as well, despite disappointing clinical results.

All have limitations, however. Not everyone responds to treatment and patients can continue to have flare-ups, requiring them to try different treatments. Additionally, drugs that blunt the immune system, like steroids, can leave patients vulnerable to infection.

How could cell therapy be used?

Six so-called CAR-T cell therapies are approved in the U.S. to treat leukemia, lymphoma and multiple myeloma. When they work, these therapies can lead to long-lasting benefit.

They’re made by genetically modifying a patient’s immune cells to spot proteins found on the surface of cancerous B cells. These cells, which normally make protective antibodies, are also implicated in an array of autoimmune diseases. In lupus, for example, B cells go haywire and make antibodies that damage healthy tissue.

CAR-T therapies wipe out B cells, whether cancerous or not. That effect led Georg Schett and colleagues at the Friedrich Alexander University, Erlangen-Nurnberg, to test CAR-T therapy in people with lupus.

Schett’s team published a case report in The New England Journal of Medicine in 2021, following up one year later with results in four more study volunteers, which were presented in Nature. They found the drug depleted B cell counts and drove participants’ disease into remissions that persisted without using other drugs.

Notably, treated individuals still responded to vaccines and their levels of infectious disease antibodies “largely remained intact,” wrote analysts at the investment bank William Blair, in a recent report. Side effects were mild.

When B cells did regenerate, disease symptoms didn’t return, which the authors suggested could support the idea that CAR-T therapy “reboots” the immune system.

To drugmakers, the findings indicate cellular therapies, if engineered right, could potentially cure some autoimmune diseases.

“There's a huge potential for patients to really benefit,” said Samit Hirawat, the chief medical officer of Bristol Myers Squibb, in a recent interview.

Yet it’s still unclear how long responses will last, or whether people may need to be re-treated later. The results may not be replicated as CAR-T therapies are studied in large groups of people with lupus, either.

CAR-T also involves a chemotherapy “conditioning” regimen to prepare patients for therapy. And treatment can cause serious immune and neurological side effects that, in an autoimmune disease setting, “will not be acceptable to patients and physicians,” wrote William Blair analysts.

More seriously, the Food and Drug Administration recently issued safety warnings for all six available CAR-T therapies, following a review of reports of patients developing secondary malignancies after treatment.

Those risks will require developers to mitigate CAR-T’s known safety issues. Companies working on adapting the therapies say their goal is to fine-tune the approach for use in autoimmune diseases.

“I think the whole industry is asking: how can we work on the benefit-risk profile and the conditioning process so that, ideally, you can [treat patients] earlier and earlier and not wait until they have severe, refractory disease?” said Victor Bulto, head of Novartis’ U.S. division.

Which companies are working on cell therapies?

About a dozen companies have launched trials testing cell therapies in lupus, according to a federal database. At least another six have received Food and Drug Administration clearance to begin initial studies and could soon follow, while others still have disclosed plans for testing.

Two cell therapy leaders, Novartis and Bristol Myers Squibb, started Phase 1 trials last year. They’re each testing newer versions of the products they brought to market for cancer, tweaking them to cut manufacturing times, boost safety and raise potency.

Bristol Myers’ Hirawat noted how Schett’s research suggests developers can deliver benefit in lupus with lower and, theoretically, safer doses than in cancer. That’s partly because there are fewer B cells to target in lupus than there are cancerous cells in lymphoma or leukemia.

“We don't anticipate, or want to see, the same safety profile as you saw in malignancies,” he said.

Alongside Bristol Myers and Novartis is Kyverna Therapeutics, a well-funded biotech startup that filed for an initial public offering earlier this month. Kyverna’s lead program was licensed from the National Institutes of Health and, like that of its larger rivals, targets the protein CD19.

Also in testing are Gracell Biotechnologies, which AstraZeneca acquired last year, Cartesian Therapeutics and startups Cabaletta Bio and ImmPACT Bio. Behind them are an array of cell therapy companies that have said they will go after lupus and other autoimmune diseases, like Fate Therapeutics, CRISPR Therapeutics and Autolus Therapeutics.

China-based biotechs and academic researchers are investing in lupus research, too. A federal database shows nearly a dozen academic or industry-backed trials underway, including studies run by JW Therapeutics, Shanghai GeneChem and Chongqing Precision Biotech.

Some that haven’t publicly announced trial plans, like Takeda, are interested: “Even though we're very far behind, we still think that we have a path,” Plump said.

One reason: Not everyone is pursuing the same approach. Some companies have cell therapies that target BCMA, another protein on B cells, as well as CD19. Some are looking at different targets. And others are changing out components to boost potency, or are tweaking conditioning regimens.

Meanwhile, companies like Artiva Biotherapeutics, Fate, and Sana Biotechnology are trying approaches that rely on the cells of healthy donors. These so-called allogeneic treatments have struggled as cancer therapies. But some developers claim they may have an edge over personalized, or “autologous,” treatments in autoimmune conditions. Here, they say, the advantages of donor-derived therapies like cheaper production costs or lower rates of side effects will be more important.

“Autologous cell therapy for autoimmune [disease] is going to be pretty challenging,” said Carlo Rizzuto, managing director at Versant Ventures, a venture firm that invested in allogeneic cell therapy developer Century Therapeutics. “I do think that allogeneic off-the-shelf approaches, especially the stem cell-based approaches, will have an advantage.”

Cell therapies could also face competition from newer antibody drugs designed to better deplete B cell counts. Roche and Johnson & Johnson, for instance, have drugs in mid- or late-stage testing for lupus.

“We will have to see how science evolves across all these modalities and see which one works best for which patients,” said Bulto, of Novartis.

What’s next?

Several clinical trial readouts this year and next could build on Schett’s study. The tests underway are enrolling patients with SLE or lupus nephritis, when the condition affects the kidneys. Some developers are recruiting people with other autoimmune conditions as well, which could provide more clues as to how broadly cell therapy might be applied.

Kyverna presented early results from a lupus nephritis study last year and is recruiting patients. A fuller readout could come in 2025, according to a federal database. The company is planning to test its treatment in systemic sclerosis, myasthenia gravis and multiple sclerosis, too.

Novartis shared preliminary findings last year in a study testing its therapy, YTB323, in SLE. More data are expected in the second half of 2024. Bristol Myers should provide an early look at its program’s potential this year, too, and intends to expand into MS, myositis and other conditions.

Cabaletta has said it will share results from patients with lupus or myositis in the first half of 2024. ImmPACT and Sana could also report initial data in 2024.

Others, such as Fate, Nkarta and CRISPR intend to start dosing patients in 2024. Startups with different approaches, like Luminary Therapeutics, could follow afterwards.

Some of these efforts will need to bear fruit for investment to continue at such a torrid pace. CAR-T’s potential in lupus alone “can't sustain this massive energy” in the industry, Plump said. “There are pieces that we're just starting to work through, but it's an area of a lot of excitement.”

A new research paper offers the most thorough look yet at a study that sparked the drug industry’s recent experimentation with cell therapy for autoimmune diseases.

The paper, which was published in The New England Journal of Medicine on Feb. 21, comes from a team of scientists at the Friedrich Alexander University, Erlangen-Nurnberg in Germany. They detail how 15 people with severe autoimmune conditions experienced either disease remission or a sharp reduction in symptoms after an infusion of a kind of cell therapy known as CAR-T. 

The patients, who had tough-to-treat forms of lupus, inflammatory myositis or systemic sclerosis, have since been followed for a median of 15 months. None have needed medications they typically used to manage their conditions, and all treatment-related side effects were judged by investigators to be mild or moderate. 

The results were presented at a medical meeting in December, but the NEJM paper gives fuller detail of the study findings.

“The findings are remarkable,” wrote John Isaacs, a professor of clinical rheumatology at Newcastle University, in an editorial also published Feb. 21. Isaacs wasn’t involved in the study.

The paper adds to research suggesting CAR-T therapy, a method of engineering immune cells to fight cancer, might be applied to autoimmune disease. The idea is that CAR-T treatments, rather than destroying tumors, could eliminate the antibody-producing B cells that have malfunctioned in many autoimmune disorders. 

Though the study in Germany is small and lacks a placebo control, its results have already influenced a wide range of drugmakers. Since 2022, biotechnology and pharmaceutical firms have begun about a dozen clinical trials testing cell therapies in lupus. At least six others have received Food and Drug Administration clearance to begin testing. 

Drugmakers are also evaluating CAR-T therapies in other autoimmune conditions like myasthenia gravis and multiple sclerosis.

“There’s a huge potential for patients to really benefit,” said Samit Hirawat, chief medical officer of Bristol Myers Squibb, one of the companies developing a CAR-T treatment for autoimmune disease, in a recent interview. 

The new NEJM results highlight the approach’s promise, as well as the questions developers will have to answer in testing. 

The study suggests CAR-T may induce a “reset” of patients’ autoimmune disease. In the months following treatment, participants’ B cell networks were reconstituted, and the number of destructive “autoantibodies” associated with inflammatory disease diminished. Participants have gone up to two years after their B cells were replenished without signs of a relapse. Other markers of disease have also disappeared. 

“Even though it is premature to judge whether these patients are indeed cured from their autoimmune disease … CAR T cells at least appear to be able to achieve sustained disease- and drug-free remission,” the study authors wrote. 

However, due to the trial’s small size and scope, it’s unclear how long the benefits from treatment will last, whether people might need to be retreated, or if any long-term health issues arise.

When used in cancer, for example, CAR-T has in rare cases been associated with secondary malignancies. 

Published research has also linked depleted B cell counts to poor health outcomes from COVID-19, Isaacs noted. Multiple study participants contracted COVID-19 after therapy in the autoimmune disease study.

The reduction — but not elimination — of some protective and vaccine-related antibodies raise the question of whether people will need new shots, the authors wrote. 

Additionally, developers must figure out the best chemotherapy “conditioning” regimen to prepare patients for treatment. Investigators in the German study used a pair of drugs that are typically administered before CAR-T therapies. But they noted how the field will need to determine “whether and what doses are needed” in an autoimmune disease setting, where the expectations for a drug’s safety profile are different than with deadly cancers.

If those problems can be solved and “extended follow-up reinforces the current data, the benefit-to-risk ratio is likely to prove acceptable to both physician and patient, at least in certain cases of refractory disease,” Isaacs wrote. 

“Nonetheless,” he cautioned, “such therapy is in its early days, and there remains much to observe and understand.”

The Food and Drug Administration in October approved Pfizer’s Velsipity to treat ulcerative colitis, making it the second pill of its type cleared for use in inflammatory bowel disease, the company said. Velsipity enters a market with several oral and injectable drugs which block the immune response that causes the disease, including one in its class, Bristol Myers Squibb’s Zeposia.

Pfizer acquired the medicine through its $6.7 billion buyout of Arena Pharmaceuticals in 2021. The big drugmaker hopes Velisipity, which slows the entry of white blood cells into the bloodstream, can also work in other immune-related conditions like Crohn’s disease, alopecia areata and eczema.

Pfizer expects to add $25 billion in revenue by 2030 from new products acquired through biotech buyouts and licensings. The additional revenue will help cushion the company against revenue declines from its COVID-19 products as well as loss of patent protection for older drugs.

In clinical trials, Velsipity helped 27% of patients taking it go into remission at 12 weeks and 32% at 52 weeks, significantly higher than the 7% of patients who received placebos at both time points. The pill also showed a benefit on other endpoints Pfizer studied, such as improvement based on measurements taken through an endoscope.

Pfizer set a list price of $6,164 for a 30-day supply of the once-daily pill, or about $75,000 a year. That is in line with oral treatments like Pfizer’s own Xeljanz and AbbVie’s Rinvoq, and less than the $100,000 annual price Bristol Myers set for Zeposia.

Zeposia was first approved as a multiple sclerosis drug, allowing it to command a higher price but one that is “not suitable” for digestive diseases like ulcerative colitis, Evercore ISI analyst Umer Raffat wrote in a note to clients.

Velsipity and Zeposia are a type of drug called S1P receptor modulators, while Xeljanz and Rinvoq work differently, inhibiting enzymes known as JAKs. The latter class has been dogged by warnings about complications for blood clots, potentially making the S1P drugs an attractive alternative.

Other treatments are biologic drugs that require frequent injections. The least costly new drug for ulcerative colitis is Takeda’s Entyvio, priced at about $50,000 a year, although others are priced at significantly more than $100,000 a year.

“Because of the unpredictable nature of UC, people living with the disease can cycle through several different treatments over time,” said Michael Chiorean, co-director of the IBD Center at Swedish Medical Center and an investigator in Pfizer’s clinical trials, in a statement. “Patients may also be apprehensive about using injectable therapies, like biologics.”

In February, Johnson & Johnson said one of its most closely watched experimental medicines appears to have positive effects on two autoimmune diseases, providing further support to a drug that, by the company’s estimates, could eventually generate billions of dollars in annual sales.

J&J didn’t release any data, but rather said the drug hit the main goals of a mid-stage clinical trial testing it in patients with Sjögren's disease as well as a late-stage study focused on generalized myasthenia gravis, a rare condition known in short as gMG. The company plans to present more detailed results from both studies at upcoming medical meetings, and to engage with regulators about the path to approval in gMG.

Named nipocalimab, the drug has over the past year demonstrated what J&J calls “clinical efficacy” across four diseases caused by autoantibodies — a type of immune system foot soldier that attacks the body’s own proteins. Nipocalimab is among the most advanced in a new class of therapies that treat autoimmune disorders by blocking “FcRn,” a protein that keeps the most common antibody found in people from degrading.

In gMG, autoantibodies interrupt the communication between nerve cells and muscles, leading to muscle weakness in the eyes, face and limbs. J&J said that, in its study, patients on nipocalimab experienced a significant reduction in the severity of their symptoms over a roughly six-month period, compared to those who were given a placebo.

The same was true in the Sjögren's study, which J&J said was the first ever to test an anti-FcRn therapy against this condition and have positive results. Sjögren's is often characterized by dry eyes and mouth, though it can affect various organs like the kidneys and lungs as well as cause joint pain similar to arthritis. The National Institutes of Health estimate that between 1 million and 4 million people in the U.S. have the disease.

Together, the two trials could help build the case for a therapy J&J believes to be a possible blockbuster. In a December presentation, the company listed nipocalimab as one of seven experimental drug programs in its pipeline that have the potential to reach over $5 billion in yearly sales.

Previously, J&J disclosed high-level results from two additional trials of nipocalimab. One found the drug worked as intended in certain patients with hard-to-treat rheumatoid arthritis. The other enrolled pregnant adults at high risk for severe hemolytic disease of the fetus and newborn, or HDFN, a rare, serious blood disorder that can lead to life-threatening anemia in the fetus.

In that open-label study, J&J said nipocalimab met the main goal, as the majority of participants who received it had a live birth at or after 32 weeks of gestation, without the need for an infusion of red blood cells into the fetus during their pregnancy.

The Food and Drug Administration has not yet approved any therapies for HDFN or disease-modifying treatments for Sjögren's. In gMG, meanwhile, it has cleared AstraZeneca’s Soliris and, in late 2021, Argenx’s Vyvgart — the first ever anti-FcRn medicine to get a nod from the agency. More recently, the FDA also greenlit AstraZeneca’s Ultomiris and UCB’s Zilbrysq and Rystiggo.

For J&J and its dealmakers, adding nipocalimab to that list would be validating, since the company spent $6.5 billion to acquire the drug’s former developer, Momenta Pharmaceuticals, in 2020.

Biotechnology startup Cour Pharmaceuticals has raised $105 million to advance a portfolio of medicines for autoimmune diseases.

The startup’s funding was co-led by Alpha Wave Ventures and Lumira Ventures and involved large drugmakers Roche, Pfizer and Bristol Myers Squibb. The cash will support Cour’s plans to develop nanoparticle drugs that can “reprogram” the immune systems of people with inflammatory disorders, said CEO John Puisis.

Cour already has drugs in mid-stage testing for celiac disease and the rare liver disorder primary biliary cholangitis that are partnered with Takeda and Ironwood Pharmaceuticals, respectively. The funding will help generate proof-of-concept data for its wholly-owned programs for myasthenia gravis and Type 1 diabetes.

Typically, biotech startups are far from clinical testing when they raise their first venture funding. But Cour, which was spun out around research at Northwestern University more than a decade ago, traveled a different path.

In 2015, the company partnered with Takeda to develop a drug for celiac and, four years later, sold full rights to the Japanese pharmaceutical giant. In 2021, Ironwood secured an option to buy Cour’s next drug, for PBC.

Those deals traded away some of the potential financial upside to the company’s two first medicines. But they also gave Cour cash without it giving up equity, as well as time to advance other programs “in a non-traditional way,” said Puisis, who co-founded the biotech.

“From the outside looking in, comparatively, we may appear more like a later-stage Series B or Series C” company, he said.

Cour aims to stand out with a new approach in fields that have recently grown crowded. A number of cell therapy developers are targeting myasthenia gravis and Type 1 diabetes, two autoimmune conditions that require chronic therapy. Many are already in testing with treatments meant to deliver lasting benefit.

Cour hopes for long-lasting effects, too, but via a different method. It’s developed a nanoparticle to carry proteins the body has mistakenly identified as harmful. The particles are absorbed by specialized cells that "reintroduce" those proteins to the T cells responsible for the body's adaptive immune response.

Because of the way the nanoparticles are designed, Cour says, the cellular reintroduction convinces the T cells to break off their attack, reestablishing immune tolerance of the misidentified proteins. Effectively, the approach is “reprogramming the immune system" without compromising its overall function, added Puisis.

The company does have some early evidence to support its claims. Phase 2 results for its celiac treatment were published in the journal Gastroenterology in 2021. After treatment with the drug, now called TAK-101, patients were evaluated for celiac disease and “many would be below the threshold for diagnosis,” Puisis said. Preclinical research has also been published in the Journal of Immunology.

But Puisis acknowledges those findings are early. It’s unclear how long the effects of Cour’s therapies might last.

“We are still learning about the duration of effect,” Puisis said, but “we are very encouraged by the data generated to date.”

Gwendolyn Wu contributed reporting.