Just weeks after Homology Medicines (NASDAQ:FIXX) received investigational new drug clearance from the US Food and Drug Administration (FDA) for its gene therapy candidate, HMI-102, the company revealed on Tuesday (May 14) that the regulatory agency has now granted the candidate fast track designation.
HMI-102, which is a one-time gene therapy drug to treat adults with phenylketonuria (PKU), received the fast track nod from the FDA as PKU is considered a rare and serious genetic disease.
According to the FDA, fast track designation is primarily awarded to drugs or biologics that aim to treat life-threatening and serious diseases and have the potential to deal with unmet medical needs.
In the case of PKU, the condition occurs when patients are born without the ability to break down phenylalanine (Phe), which is amino acid found in a wide range of foods and drinks containing protein and strong sweeteners. Should the condition go untreated, it could leave patients with severe neurological damage.
“We believe FDA’s decision to grant Fast Track designation is a positive step forward for the development of HMI-102, which is designed to treat the underlying cause of this disease and allow freedom from a restrictive diet, the current standard of care,” Arthur Tzianabos, Ph.D, president and CEO of Homology Medicines, said in a press release.
Tzianabos continued, stating that his company has been working over the last three years on pushing its dual gene therapy and gene editing platform towards providing care to patients with PKU and other rare conditions.
While the FDA granted the investigational new drug application for HMI-102 in April, the company received orphan drug designation for the candidate in the US and the European Union for use of the human hematopoietic stem cell derived adeno-associated virus AAVHSC15 to treat PAH deficiency. Severe deficiency of the PAH gene is the main cause of PKU.
Homology Medicines is currently looking to start pheNIX, its Phase 1/2 trial for adult patients with PKU, and expects initial clinical data sometime this year.
Tuesday’s announcement comes on the heels of the company revealing its Q1 2019 financial results on Monday (May 13). In its results, Homology Medicines highlighted a net loss of US$23.29 million for the quarter as compared to US$10.2 million for the same quarter in 2018.
The company noted that its HMI-103 candidate, which is an in vivo gene editing candidate for child patients with PKU, has also begun investigational new drug-enabling studies.
Since Monday’s open, shares of Homology Medicines have increased 1.66 percent to close at US$22.65 on Tuesday. The company currently has a “strong buy” ranking on TipRanks based off three analyst ratings with an average price target of US$32.50, a high estimate of US$36 and a low of US$29.
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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.