It’s what biopharma CEOs dream of: winning an approval for a drug they’ve helped develop and delivering it to patients. With its PDUFA date slated for Aug. 13, Citius Pharmaceuticals is on that path with Lymphir, an immunotherapy for a rare blood cancer.
Scoring an approval for Lymphir, which treats patients with persistent or recurrent cutaneous T-cell lymphoma, would be a “breakthrough” for Citius, said CEO Leonard Mazur.
Although CTCL does not have a cure, there are a few treatment options already available, such as chemotherapy and radiation. But there is a lot of variability in treatment pathways and how patients will react as the disease moves through its two stages, according to Mazur.
The drug is among a handful of late-stage first-in-class candidates in Citius’ pipeline, which also includes Mino-Lok, a novel antibiotic to treat catheter infections, Halo-Lido, a topical formulation for hemorrhoids, as well as treatments made from its stem cell platform.
The company head describes himself as an “all-in entrepreneur” and has personally invested $22.5 million into the company. Meanwhile, co-founder and executive vice chairman Myron Holubiak slapped down $4 million, according to Mazur.
Putting his own fortune on the line has been part of Mazur’s playbook from the early days of his career when he took out a mortgage on his house to convince another investor to cut a deal in a former role.
“I remember going to meet with a potential investor, I gave the whole presentation,” Mazur said. “And he says, ‘How much are you putting in?’ I said, ‘I'm applying all my years of experience, I've got a great business plan … all this has value.’ He said, ‘I didn't ask you that. How much are you putting in? Because if you're asking me to put money in and go at risk, I'm going to ask you the same.’”
Since then, Mazur has done so “several times over,” and has led a handful of other pharma companies, including Genesis Pharmaceutical, Triax Pharmaceuticals and Akrimax Pharmaceuticals, from startup stage to a sale.
Asset strategy
Citius’ strategy has focused on late-stage drug assets with a potentially shorter path to commercialization. Lymphir, for example, is a reformulation of denileukin diftitox, a protein fusion of interleukin-2 and diphtheria toxin that targets malignant and immunosuppressive T cells.
The drug, which won orphan drug designation from the FDA, was previously approved under the name Ontak before Eisai voluntarily pulled it from the market in 2014 due to a production issue. In 2021, Citius purchased licensing rights to an improved formulation of Ontak for $40 million from Dr. Reddy’s Laboratories, with up to $70 million in development milestones as part of the deal for additional indications in the future.
However, it hasn’t all been smooth sailing for Lymphir. Citius received a complete response letter from the FDA last year, requiring the company to enhance product testing and agree to additional controls, the company said.
“That response letter was something that occurred because we had passed a manufacturing test, but the validation procedure for it wasn't completed,” Mazur said. “And it was going to run several months past the PDUFA date, so we got the complete response letter. We did finish up all the work, we resubmitted everything and we've got our fingers crossed here. ”
Commercialization plans
With the company betting on approval, Citius is ramping up its commercialization strategy. The patient population for CTCL is small, with around 3,000 new cases in the U.S. every year andthe market for Lymphir is between $300 and $400 million, Mazur said.
To fund the launch, Citius spun off Lymphir and its oncology business into another entity, Citius Oncology, with plans to launch it on the Nasdaq under its own ticker as part of an acquisition deal with a special purpose acquisition company, TenX Keane Acquisition. The transaction is expected to close before the August PDUFA date, Mazur said. The deal leaves Citius in control of “close to 90% of the company,” with a valuation of $675 million, he said.
If all goes well and the FDA approves the drug, Lymphir could launch in November, Mazur said. Citius has already brought sales and marketing personnel on board, with preparations ongoing for almost a year.
Lymphir may also have potential as a combination treatment, and the drug is undergoing testing in two investigations — one with the University of Pittsburgh where it's being combined with Keytruda and another at the University of Minnesota where it's being tested with a CAR-T therapy.
Looking forward, Citius is focused on similar asset types for potential drug candidates to acquire, not limited to specific diseases.
“We like smaller specialties … that don't require a giant sales force. Something we can promote [that has] a size advantage to it,” Mazur said.
In general, Mazur said targeting rare diseases with smaller patient populations “does pay off.”
“The pricing is a lot different than it would be if it wasn't a rare disease. That's one part of it, but that's not all. For us, it's also the fact that there's the element that we want to be able to contribute to the betterment of people,” he said.
