In a tough fundraising space, cell therapy biotechs pursuing autoimmune indications review staffing to ensure the right expertise is in place to tackle the new disease area.
As cell therapy biotechs pivot from oncology to autoimmune disease, they face a big question: Can your cancer researchers run a lupus trial?
Not necessarily, experts told BioSpace. And without the right teams in place, this segment of the biotech world may further put off potential investors to an area of innovation that is already facing a tough fundraising environment.
“You have to be really careful,” Jakob Dupont, executive partner with Sofinnova Investments, told BioSpace. “Do you have the right team to actually run a lupus trial or rheumatoid arthritis trial, or do you have oncologists sitting at your company who are now going to try to get into the mindset of running a lupus trial—which is very different.”
These companies are following the science, which is now suggesting CAR T therapies could help patients with autoimmune disorders. Arcellx, which has an extensive oncology pipeline, just received clearance to test its investigational multiple myeloma therapy anito-cel in myasthenia gravis. Allogene, also largely focused on oncology, is working on filing an investigational new drug (IND) application for CRISPR-based therapy ALLO-329 in an unnamed autoimmune disorder in the first quarter of 2025, with proof-of-concept data expected by the end of that year.
As companies make this pivot to autoimmunity, they’re confronting some tough decisions about staffing, said Eric Celidonio, senior managing partner at Sci.Bio Recruiting. It’s not just cell therapy, either. Some biotechs working on other modalities that began with both autoimmune and oncology are finding that one focus is best in this environment. Celidonio pointed to biotechs Odyssey Therapeutics and Kymera Therapeutics, which have both shed their cancer programs. Kymera last week moved its resources from oncology to immunology, leaving two programs to advance only with an outside partnership after Phase I.
This all points to a growing demand for experienced autoimmune personnel, Celidonio predicted. It also has implications for getting funding.
The fundraising environment in biotech is still very uncertain, so venture capitalists and other investors like to see names they recognize or high levels of expertise, Dupont explained. If your cell therapy company has cancer experts looking to move into immunology, that may raise some eyebrows.
“The company has to be really built well with the right team to yield results, because drug development is hard,” Dupont said. “We scrutinize: Who’s the leadership? Who are the people running the clinical trials, and do they have deep expertise?”
A Thin Line
A major difference between cancer and autoimmune disease clinical trials is that the participating sites will likely be different. If you have prior relationships with cancer centers, your operation teams will have to reassess, and you may have to switch out your contract research organization to get access to the right patients, Dupont said.
“The other concern becomes that if you don’t have all those connections because you pivoted from oncology to autoimmune, then you can’t enroll any of your studies to get that data in a reasonable time frame.”
Patient characteristics can also be really different, Dupont continued, with different subsets of the disease that need to be included or not depending on what you’re trying to do. “That can be all the difference between success and failure.”
For Poseida Therapeutics, leadership will be everything in the transition to autoimmune, a spokesperson for the company told BioSpace. For example, CEO Kristin Yarema has deep roots in the disease area thanks to previous experience at Amgen, Novartis and more.
“Moving into autoimmune disease has involved reactivating the network, bringing in new talent, and identifying individuals within the company who had relevant experience but weren’t utilizing it when the focus was solely on oncology,” the spokesperson said in an email.
While the company is still very much committed to cancer, it is working on IND-enabling studies for P-BCMACD19-ALLO1—a CAR T therapy that was originally developed for multiple myeloma—in autoimmune. The biotech has not designated which diseases the program will target, but Yarema previously told BioSpace that those involving plasma cells and other types of B cells, such as multiple sclerosis, myasthenia gravis and systemic sclerosis, could be candidates.
The company has been “very intentional” about how it enters this new space, the spokesperson said. “We continue to assess our capabilities to ensure that we have the right skillsets at the right time as we undertake IND-enabling studies of P-BCMACD19-ALLO1 in autoimmune disease indications.”
The same process is happening at Arcellx, which officially decided to pursue autoimmune about a year ago, according to Chief Medical Officer Chris Heery.
“You have to go recruit the right people,” Heery told BioSpace. “You’ve got to build up the total amount of workforce to be able to manage the complexities of getting a clinical trial started,” such as writing an effective trial protocol.
At the same time, senior leadership needs more than just a crash course in the new disease area to be able to execute such a huge shift, he added. “It’s not like we want to make these decisions and go forward and not have the same strategic insights that we did with another indication like multiple myeloma.”
The hiring process for these new roles can help build some of the knowledge needed, he said. While interviewing candidates, the Arcellx team has been reading more published studies on autoimmune disease and talking to clinicians to build up baseline knowledge in this space.
The company has hired some “really great people” to support the new direction, Heery said, but the companystill has more learning to do. “Now we feel like we’re ready for myasthenia. It doesn’t mean we’re ready for all autoimmune disease,” he said. “We think if we’re going to go into something, we want to be really good at it.”
Finding the Right Fit
Oftentimes these staffing shifts can be subtle. Cullinan Therapeutics, which recently received FDA clearance for human testing of CD19 T cell engager CLN-978 in patients with moderate to severe systemic lupus erythematosus (SLE), has also brought in some new talent with the clinical, regulatory and research experience needed in immunology to get the program off the ground, but also turned to immunology experts already on staff.
Kymera Therapeutics CEO Nello Mainolfi told BioSpace that the switch from oncology to immunology is expected to have minimal impact on employees. The company has had an eye toward the disease area for a long time and has been developing capabilities in-house in preparation. But the biotech will be making key investments in areas of development such as the clinical team, Mainolfi said.
Kyverna Therapeutics, meanwhile, has always been an autoimmune disease company, so rather than find new experts in that disease area, the company did some shuffling in the C-suite to bring on veterans with deep experience commercializing CAR T. Cell therapy veteran Warner Biddle was appointed as CEO in September, replacing Peter Maag, who saw the company through its $319 million upsized IPO earlier this year.
While Biddle’s background is in cancer, he most recently served as head of Gilead unit Kite Pharma, which has some of the most successful cell therapy products on the market today. The biotech also snagged fellow Kite alum Christi Shaw as a board member.
In announcing the CEO swap, Kyverna said that Biddle is the “right fit” for the company’s future, starting with KYV-101 for B cell–driven autoimmune diseases such as lupus nephritis and myasthenia gravia.
“Kyverna today sits on the precipice of its next chapter, as KYV-101 is progressing rapidly into later stages of development and embarking on a path to market,” said Ian Clark, board chairman, in a statement. “This introduces new and exciting opportunities, which Peter and the board recognized the company must evolve to meet.”
Editor’s Note: Shawna Williams contributed reporting to this story.
