Dosing Complete in Phase 1 Trial of CTI-1601, Synthetic Frataxin for FA

Larimar Therapeutics completed dosing for its single-ascending dose (SAD) Phase 1 clinical trial of CTI-1601, a modified version of frataxin, in the treatment of Friedrich’s ataxia (FA), the company announced.

A safety review committee cleared the trial to continue after evaluating preliminary blinded data from each of the four dosing groups. Based on that data, single subcutaneous (under-the-skin) injections of up to 100 mg of CTI-1601 appear to be well-tolerated. Furthermore, adverse side effects were deemed mild and transient, with no severe side effects observed.

The medication’s safety and tolerability are the main goals of the trial (NCT04176991). Secondary goals include pharmacokinetic and pharmacodynamic analyses of CTI-1601 — how the compound moves through and affects the body, respectively.

“These preliminary Phase 1 findings represent a critical step forward in developing CTI-1601 as a potential frataxin replacement therapy for patients with FA and we look forward to its continued advancement through the clinic,” Nancy M. Ruiz, MD, chief medical officer of Larimar, said in a press release.

Frataxin is critical to the proper function of mitochondria, membrane-enclosed organelles within cells that generate the energy needed for all cellular processes. Mutations in the FXN gene lead to a defective frataxin protein. The resulting mitochondrial abnormalities causes the nerve and muscle damage that underlies FA.

The modified frataxin of CTI-1601 is designed to enter the mitochondria, where it is expected to ease FA symptoms and halt or slow disease progression by restoring frataxin in healthy levels.

Eligible participants from the SAD trial and other FA adult patients may enroll in a second Phase 1, multiple-ascending dose (MAD) trial (NCT04519567), which will examine multiple subcutaneous infusions of ascending CTI-1601 doses.

The first two of three groups have been dosed in this trial, with dosing of the third expected to begin early in 2021. Larimar expects to report unblinded top-line data from both trials in the second quarter of 2021.

Patients completing either of these trials also are eligible for an open-label extension trial, expected to begin in the second half of 2021.

Finally, while the trials described above involve patients 18 and older, Larimar plans to begin another MAD trial for FA patients younger than 18, also in the second half of 2021.

CTI-1601 has won rare pediatric disease, fast track, and orphan drug designations from the U.S. Food and Drug Administration, and orphan drug designation from the European Commission.

“That our Phase 1 program remains on track for topline data in the second quarter of 2021 despite the industry-wide challenges posed by COVID-19 is a testament to the talent and unwavering commitment of our employees, partners, and investigators,” said Carole Ben-Maimon, MD, president and CEO of Larimar.