Prana Granted First MSA Orphan Drug Designation from FDA

- January 31st, 2019

Prana revealed Thursday that the FDA has granted it the first orphan drug designation to treat Multiple System Atrophy for its lead molecule.

The first ever orphan drug designation to treat Multiple System Atrophy (MSA) has been granted by the US Food and Drug Administration (FDA) to Prana Biotechnology (NASDAQ:PRAN,ASX:PBT) for its lead molecule, the company revealed on Thursday (January 31).

According to the press release, Prana’s lead molecule, called PBT434, has demonstrated it can minimize the accumulation of alpha-synuclein, preserve neurons and boost motor functions in animal models which have the disease.

MSA is a rare progressive neurological disorder similar to Parkinson’s disease. The company’s orphan drug application for PBT434 was based off of its proposed use as a treatment for the disorder, both in terms of medical and scientific reasoning.

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Thursday’s announcement comes following a strategic investment of US$7.5 million to Prana from Life Biosciences, a Boston-based longevity company, in late December. According to December’s release, Prana will use the funds to facilitate its drug development programs, which includes PBT434.

“This recognition, in conjunction with the recent investment from Life Biosciences, positions us strongly to accelerate the development of PBT434 for this devastating condition,” Dr. David Stamler, chief medical officer of Prana, said in Thursday’s release.

A Phase 1 clinical trial for the lead molecule is currently being conducted by Prana, which the company projects will have completed sometime this year.

According to the National Institute of Neurological Disorders and Stroke, MSA impacts roughly 15,000 to 50,000 Americans with symptoms appearing in people over the age of 50. Signs of MSA include fainting, heart rate issues, erectile disfunction and bladder control. Motor impairments range from tremors, rigidity, loss of muscle coordination speech and walking difficulties.

In addition to MSA, Prana says PBT434 has “excellent potential” to treat Progressive Supranuclear Palsy (PSP). PSP is an uncommon brain disorder that impacts walking control, balance, speech, swallowing, vision, mood and behavior and thinking.

Shares of Prana responded favorably to the FDA’s orphan drug designation of PBT434. Its NASDAQ-listed stock rose 10.57 percent over Thursday’s trading period to close the session at US$1.57. On the ASX, Prana’s share price increased from AUD$0.41 to AUD$0.42.

Prana currently has a “Sell” ranking on TradingView, with nine against, 10 neutral and an additional nine ranking the company as a “Buy.” On TipRanks, the company has no current analyst ratings or price targets. However, the most recent analyst rating was given in March 2018 by George Zavoico from B. Riley FBR, who gave a “Buy” position to Prana with a US$4 price target.

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

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