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PTC Announcement on Covid-19 Candidate Met With Investor Indifference

Investors have either become overwhelmed by the number of companies entering the quest to address Covid-19 or are having difficulty separating the wheat from the chaff. Either way, the latest entrant to the coronavirus battle was met with a big ho-hum.

On the announcement that it was going to test its early-stage cancer drug against the virus, PTC Therapeutics Inc. (NASDAQ:PTCT) shares gained two cents to just under $50.36.


The New Jersey-based biotech is set to launch a two-step, phase 2 and 3 trial of the leukemia drug PTC299 in the United States, with additional tests requested in Spain and filings planned in Europe, Brazil and Australia, according to an article in FierceBiotech. PTC is entering new territory with the test inasmuch as its main focus is finding treatments for rare diseases.

CEO Stuart Peltz said that by inhibiting a specific human enzyme called DHODH, PTC299 uniquely addresses the two key issues of Covid-19, namely reducing the high viral replication and also selectively lessening the immune response caused by the uncontrolled cytokine storm resulting from the SARS-CoV-2 infection.

It is thought that in many Covid-19 cases, the worst damage may be caused by a deranged immune response to the infection, rather than the virus itself, according to WebMD. In many of the sickest patients, their blood has been found to have high levels of immune proteins called cytokines. Scientists believe these cytokines are evidence of an immune response called a cytokine storm, where the body starts to attack its own cells and tissues rather than just fighting off the virus.

The PTC pipeline targets Huntington's disease, Angelman syndrome, relapsed/refractory acute leukemias, ovarian cancer and other diseases. In May, the company bolstered its rare disease pipeline by purchasing the small biotech Censa Pharmaceuticals and its drug to treat a metabolic disorder

The company currently markets Emflaza, which treats Duchenne muscular dystrophy (DMD), in the U.S. It sells another DMD drug, Translarna, in Europe, and in Latin America the medication Tegsedi, which treats a rare inherited rare inherited condition characterized by abnormal build-up of a protein called amyloid in the body's organs and tissues.

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The company is scheduled to meet with the U.S. Food and Drug Administration to seek approval for the spinal muscular atrophy called risdiplam, which it is partnering on with Roche (RHHBY). The drug could be a blockbuster, but will be going head to head with medications from Biogen Inc. (NASDAQ:BIIB) and Novartis (NYSE:NVS). In May, PTC added to its rare disease portfolio when it purchased the small biotech Censa Pharmaceuticals and its experimental medication for metabolic diseases.

According to CNN Money, the nine analysts offering 12-month price forecasts for PTC have a median target of $55 and a high estimate of $85. They rate the stock a buy.

In the first quarter of the year, PTC sales were more than $68 million, up 25% from a year earlier. The net loss was $112.7 million for the first quarter compared to a net loss of $72.1 million for the first quarter of 2019. Due to Covid-19, the company withdrew its guidance for the year.

Disclosure: The author holds no positions in any of the stocks mentioned in the article.

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This article first appeared on GuruFocus.