1st Patient Dosed in Trial of IPF Therapy Candidate Taladegib

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by Marta Figueiredo PhD |

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A Phase 2 clinical trial evaluating Endeavor BioMedicines’ experimental oral therapy taladegib (ENV-101) in people with idiopathic pulmonary fibrosis (IPF), has dosed its first patient.

The trial (NCT04968574) is enrolling adults, ages 40 and older, with mild-to-moderate IPF at one Australian site, with at least three more expected to open in the country.

“There are currently no approved therapies that stop the progression of fibrosis [tissue scarring] or treat the underlying causes of the disease,” John Hood, PhD, Endeavor’s co-founder, CEO, and chairman, said in a press release.

“This Phase 2 study now underway will help us understand how taladegib may help to stop or even reverse one of the most devastating pulmonary diseases by targeting … the underpinnings of the disease rather than solely the symptoms patients face,” Hood added.

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Taladegib is an orally available small molecule that works by suppressing the Hedgehog pathway. Hedgehog is a signaling cascade involved in wound healing and in the activation of  myofibroblasts — the main cellular drivers of lung fibrosis — that is overly active in the lungs of IPF patients.

According to Endeavor, previous data from 192 people given taladegib showed the therapy safely and effectively blocks the Hedgehog signaling and subsequent myofibroblast activation. As such, taladegib is expected to halt, or even reverse, the lung fibrosis that characterizes IPF.

Early this year, Endeavor raised $62 million in funding to support the launch of two Phase 2 trials to test taladegib in IPF patients.

The ongoing multi-center Phase 2 trial is evaluating the therapy’s safety and efficacy in up to 60 patients with mild-to-moderate disease. Participants will be assigned randomly to receive an oral tablet of either taladegib (30 patients) or a placebo (30 patients), once a day, for 12 weeks (about three months).

Patients in the taladegib group will receive a starting 200 mg-daily dose that may change across the study. Participants will not be allowed to receive standard IPF treatments, such as Ofev (nintedanib) and Esbriet (pirfenidone), two weeks before and during the trial.

After completing the treatment period, all patients will be followed for an additional one-and-a-half months.

The trial’s main goals are to assess the frequency and severity of adverse events and of clinical laboratory abnormalities, as well as changes in patients’ key vital signs. Secondary goals include using several validated measures to assess changes in lung function and shortness of breath.

Pending results from this study, Endeavor may launch a second Phase 2 trial in 2022 to test taladegib in combination with standard IPF therapies.

In the release, the company also announced the appointment of Srikanth Pendyala, MD, as its chief medical officer.

“With [Pendyala’s] extensive background in [therapy] development, paired with his expertise in academic medicine, we are confident that his experience will be invaluable as we advance Endeavor’s pipeline of precision medicine treatments,” Hood said.

“Endeavor’s forward-thinking approach to precision therapies used to treat pulmonary disease and cancer has already exhibited immense potential since the company launched,” Pendyala said. “I look forward to working with the team and helping propel forward Endeavor’s pipeline of medicines for patients with significant unmet medical needs.”

Endeavor also is evaluating taladegib as a potential therapy for types of cancer where the hedgehog pathway is implicated.