Avrobio’s Share Price Drops 50 Percent Amid Clinical Trial Updates

Avrobio (NASDAQ:AVRO) announced on Monday (October 1) updates for two of its ongoing clinical trials.

The company released updates on two drugs in ongoing trials: the first drug AVR-RD-01 is a Phase 1 and Phase 2 clinical trial as a Fabry disease treatment. The other drug AVR-RD-02 about to enter a clinical trial as a treatment for Gaucher’s disease. Both are gene therapy drugs the company is developing as one-time treatments.

The first patient involved in the company’s Phase 1 trial for AVR-RD-01 has been approved to stop taking the current standard, an enzyme replacement therapy (ERT), for the disease after reviewing 18-month data.

According to the release, the patient’s average vector copy number reached a healthy level of 0.1. The second patient in the trial had higher (ADP) levels than the diagnostic range for Fabry disease, but currently remains on the ERT treatment.

“We particularly highlight the 18-month data from patient 1 who maintained AGA enzyme activity above the diagnostic range for classic Fabry disease,” said Dr. Aneal Khan, from the Fabry disease Clinical research and Therapeutics team (FACTs) which is conducting the trial. “We received approval to discontinue ERT and observe the impact of gene therapy alone.”

The third patient in the trial received AVR-RD-01 treatment in July. These are the only three patients in the trial, although the company may enroll up to three more to participate.

While the primary objective of this trial is safety, the patients exhibiting promising AGA enzyme activity is another major milestone for the dru No serious side effects have been reported for the drug.

The Phase 1 trial conducted by the FACTs and is investigator-sponsored. Patients enrolled in this trial must have been treated with the current standard of ERT treatment for at least six months before receiving Avrobio’s drug. Patients stop taking the ERT treatment a month before AVR-RD-01 and resume taking it a month after the treatment.

The Phase 2 trial for the same drug and disease indication is sponsored by Avrobio. This trial is an open-label single-arm clinical trial evaluating the efficacy and safety of the drug in eight to 12 treatment-naive patients.

AVR-RD-01 is an ex vivo lentiviral gene therapy. It works by inserting the GLA gene to encode functional α‑galactosidase A (AGA, the enzyme that is deficient in Fabry disease). This enables continuous AGA production and distribution to tissues and organs.

In a presentation, Avrobio said that its AVR-RD-01 Phase 2 clinical trial will expand trial sites to the US and Japan. Current sites include Canada and Australia, with the Phase 1 trial taking place in Canada.

Fabry disease is a genetic defect in the GLA gene, patients are diagnosed mainly in childhood or adolescents. Symptoms of the disease include raison skin lesions, painful episodes of foot and hand pain, difficulty sweating among others. Over 7,000 people have Fabry disease in US, according to the National Fabry Disease Foundation.

Avrobio additionally announced that its drug candidate for Gaucher’s disease received no objections to its clinical trial from Health Canada. With this news, the company plans to initiate its Phase 1/2 trial for the indication in 2019.

Gaucher’s disease is another rare genetic disease currently treated with ERT treatment. Patients with the disease are unable to breakdown certain enzymes allowing glucose fats to build up in the liver. Approximately 6,000 people in the US have Gaucher’s disease, according to the National Organization of Rare Disorders.

Avrobio is a clinical-stage gene therapy company developing drugs to cure rare diseases with a single dose. Aside from these two rare diseases. the company has other drugs in development for Pompe and Cystinosis diseases.

The company’s drug candidates are made using its proprietary Lentiviral-Based Gene Therapy which takes the patient’s own stem cells and modifies them as part of the treatment.

Investor takeaway

Despite the company’s positive news investors did not share the same sentiment. Over the trading period Monday, Avrobio’s stock price dropped over 51 percent to US$25.13 as of market close.

According to TipRanks, Cowen analyst Ritu Baral released a note to investors on Monday reiterating a “Buy” target for the company. TipRanks shows Avrobio has a high estimate of US$47 for the next six months and a low of US$38.

Both press releases have been filled with information, but to remind investors about upcoming milestones. Both ongoing clinical trials for Fabry disease are enrolling patients, the company intends to expand to four countries for the trial by 2020, the US, Japan, Australia and Canada. The company’s Gaucher’s disease trial is also expected to begin its the Phase 1/2 trial in 2019.

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