Clementia Completes Phase 3 Move Trial Patient Enrolment

In some cases enrolling patients, especially in trials with rare diseases, can be one of the most challenging parts of clinical trials.

Clementia Pharmaceuticals (NASDAQ:CMTA) announced on Thursday (August 16) it completed enrolment for the Phase 3 Move trial with palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva (FOP).

As the enrolment of this trial is four months ahead of schedule, the company anticipates conducting two interim data analyses in 2019.

The first analyses will take place after the company’s first 35 patients undergo their 12-month whole body CT scans, which are expected to take place in Q2 2019.

The second analyses will take place when each enrolled patient has finished the same scans, which is expected in the second half of 2019. The company also expects 24-month results from this study in Q4 2020.

FOP is a very rare disabling disorder characterized by heterotopic ossification (HO), or bone that forms outside of the normal skeleton muscles, tendons or soft tissue. HO progressively restricts movement by locking joints leading to a cumulative loss of function, progressive disability and increased risk of early death.

About one in two million people worldwide are diagnosed with FOB, with several hundred reported according to the US National Library of Medicine.

The disease is caused by a mutation in the ACVR1 gene resulting in excessive signally the bone morphogenetic pathway which controls the bone growth and development. There is currently no approved treatment for FOP.

Palovarotene is an RARγ agonist in development for this disease and multiple osteochondromas (MO), dry eye disease and others. Data from the Phase 2 trial shows the drug is able to significantly reduce the development of new bone growth in abnormal places or HO for FOP patients.

In preclinical models, the drug was additionally found to prevent the formation of osteochondromas in multiple exostoses. It received Orphan Drug status for FOP and MO from the US Food and Drug Administration (FDA), and Fast Track and Breakthrough Therapy designations for FOP. The drug was also granted orphan status for the treatment of FOP in the European Union.

For the Move trial, patients receive a daily single dose of palovarotene with the option for increased dosing during a flare-up. The primary endpoint for the trial is the annualized change in the new HO volume assessed by whole body scan compared to data from Clementia’s natural history study data from the same time period.

Clementia currently has just palovarotene in development, and continues to discover other indications for the drug. Though the company was founded in 2011 and maintains the same management team, it’s only been on the public market since last year in August.

Investor takeaway

From the announcement Thursday, Clementia’s share price increased 16.78 percent to US$9.88 as of market close.

According to TipRanks, the most recent analyst report was made yesterday by B.Riley FBR analyst Madhu Kumar for a “Buy” rating at US$16.

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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.