Cash-strapped Acer sees menopause drug flunk phase 2 trial

Acer Therapeutics’ ambitions for menopause drug osanetant have hit a roadblock after the candidate failed to reduce hot flashes in a mid-stage trial, missing the primary endpoint. The news comes at a bad time as the biotech hunts for cash to keep operations going.

Osanetant, also known as ACER-801, was being evaluated as a potential treatment for moderate to severe vasomotor symptoms (VMS) associated with menopause. However, the former Sanofi asset failed in the phase 2 trial’s primary goal of reducing the frequency or severity of hot flashes in postmenopausal women.

As a result, the biotech is pausing the ACER-801 program while the data is reviewed.

“We are surprised and disappointed the phase 2a trial did not meet its efficacy objectives in treating VMS given the extensive body of non-clinical and clinical evidence previously generated,” CEO Chris Schelling said in the release. “We intend to conduct a comprehensive analysis of the totality of the clinical trial data—including the pharmacokinetic data, which has not yet been analyzed—which will inform our path forward for the program, including our collaborations for prostate cancer and post-traumatic stress disorder.”

The phase 2 study involved 49 postmenopausal women aged 40-65, who experienced moderate to severe hot flashes, receiving twice daily 50 mg, 100 mg or 200 mg doses of osanetant or placebo over 14 days, followed by a 14-day safety follow-up assessment. While missing the primary endpoint, Acer pointed out that the drug was well tolerated.

Acer licensed osanetant from Sanofi in 2018. The drug is also in phase 2 trials for prostate cancer and post-traumatic stress disorder.

While ACER-801 is on pause, the company pointed out that it’s continuing to prepare for the commercial launch of the urea cycle disorder drug Olpruva, which received FDA approval at the end of December. There’s also Edsivo, which is undergoing a phase 3 trial for patients with vascular Ehlers-Danlos Syndrome—a rare disorder of the blood vessels and organs—in patients with a confirmed type III collagen mutation.

The only catch is that the plans for both Olpruva and Edsivo are reliant on Acer finding enough cash to keep going beyond the next few months. The biotech is currently on the hunt for additional capital just to take it beyond the early part of the second quarter.

Acer has yet to release its most recent earnings report, but third-quarter results show the company expected the $6.4 million on hand at the end of September to run out before the year’s end. In November, the company made $1.6 million by selling off some shares, but even this minor windfall was only expected to keep the lights on until the end of December.

The latest news has done no favors to Acer’s share price, sending the stock plummeting over 40% to 90 cents a piece in the opening hour of trading on Friday. That puts Acer back in the danger zone of being delisted from the Nasdaq, after the company celebrated regaining compliance with the stock exchange in December.