Calliditas is one of European biopharma’s big success stories, bringing a rare disease drug to market and commercializing it using its own sales force. Richard Staines from Optimum Strategic Communications spoke with CEO Renée Aguiar-Lucander who outlined the challenges the company has faced on its journey.
Lengthy experience as a life science investor has helped Renée Aguiar-Lucander learn by observation about the requirements of being a biopharma CEO, she told the Optimum Perspectives podcast. This has taught her that the culture of a developing company is vital to its success – and that sometimes it is necessary to take calculated risks.
During her six years as CEO of Calliditas, Aguiar-Lucander has used these learnings to push forward the development and marketing of TARPEYO, which is being commercialized in both the US and Europe. TARPEYO is the first FDA-approved medicine for the rare disease, IgA nephropathy, where deposits of the IgA antibody build up in the kidneys.
IgA nephropathy causes inflammation and damages kidney tissues, resulting in them leaking blood and protein into the urine, potentially leading to complications including high blood pressure and chronic kidney disease which can progress to kidney failure.
TARPEYO works by delivering a dose of the corticosteroid budesonide to the Peyer’s patches, a part of the lymph tissue lining the ileum that produces IgA antibodies.
It is thought that patients with IgA nephropathy are producing defective antibodies and by down-regulating their production in Peyer’s patches with budesonide, the damage they cause is managed.
The FDA granted an accelerated approval of TARPEYO in December 2021, granting a label covering reduction in protein levels in the urine in adults at risk of rapid disease progression.
This was granted in expectation of confirmatory data showing that TARPEYO slows kidney function decline – data that the company plans to share with the FDA in July 2023.
The FDA accelerated approval was followed by a conditional European approval in July 2022 under the brand name Kinpeygo, with partner STADA Arzneimittel AG marketing the drug in the EEA, Switzerland and the UK.
Aguiar-Lucander noted that the company spent a lot of time and effort recruiting commercial, market access and medical affairs personnel ahead of a potential decision from the regulator.
This was something of a gamble given that there was no guarantee of an FDA approval, a daunting situation that’s familiar to most companies in the biopharma industry.
“So once we actually got the approval, that was a fantastic event for the company,” she said.
Aguiar-Lucander stressed the importance of the approval for patients, noting that despite the disease first being described during the 1960s, there was no dedicated medicine until TARPEYO became available.
Calliditas now has data from the NeflgArd trial, having announced topline results in March 2023, showing a statistically significant benefit over placebo in estimated glomerular filtration rate (eGFR) after nine months of treatment with TARPEYO or placebo, and 15 months follow-up off-drug.
After another FDA review Aguiar-Lucander hopes that TARPEYO will be granted full approval with a label referring to the eGFR data that opens it up to a wider group of patients.
She spoke about the complexities around converting prescriptions into commercial sales before reflecting on the story so far, concluding that the company’s transition from a biotech to a pharma company with a marketed product has been possible because of its culture.
A major focus of her efforts has been to create a company with the right culture, despite having offices in both the US and Sweden, she added.
She concluded: “Getting the people to buy into this culture can be more efficient than getting someone who is highly qualified but actually doesn’t really want to play with the other kids.
“Creating that culture is critical and it’s a job that’s never done.”
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