Weekly Medical Stock Spotlight: 3 big winners and 2 total duds.

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1) ProQR Therapeutics NV (Nasdaq:PRQR) led advancing issues on the Labor Day-shortened week, soaring 107% over the week to $15.80 on Friday (August 31, 2018). The upward move came after the clinical-stage biotech focused on RNA medicines that treat rare genetic diseases reported data from a Phase 1/2 clinical trial.

Based in Leiden, the Netherlands, ProQR is testing its compound called QR-110 as a hopeful treatment for LCA10, a genetic mutation that usually leads to childhood blindness. Use of QR-110 led to improvements in vision of the majority of patients when measured using visual acuity and the mobility course performance tests. Specifically, about 60% of patients showed a clinically meaningful response to the drug after three months of treatment.

A secondary study also showed that the efficacy effects of the drug persisted beyond three months. The drug was well-tolerated by study participants, and no serious adverse events were reported. A pivotal Phase 2/3 trial is set to start in the first half of 2019.

Steve’s Take:

The 52-week trading range is $2.75-$17.55.

Shares were trading up another 19% Monday (September 10, 2018) at $18.75

Based upon 3 analysts offering recommendations, the current rating on ProQR is Strong Buy.

The Barchart Technical Opinion rating is a 100% Buy with a strengthening short-term outlook on maintaining the current direction.

Longer term, the trend strength is Maximum. Long-term indicators fully support a continuation of the trend.

The market is approaching overbought territory. Be watchful of a trend reversal.

2) Elsewhere, Arrowhead Pharmaceuticals Inc. (Nasdaq:ARWR) rocketed 34% to $19.74 after the company presented early clinical data showing promising results for its drug to treat the chronic hepatitis B virus.

Pasadena, CA-based Arrowhead, with its research staff in Madison, WI, said its RNA interference (RNAi) drug, ARO-HBV, showed substantial reduction in the virus after three monthly injections were given to eight patients who received a low dose version of the drug. The initial results were disclosed Thursday at the World Gastroenterologists Summit in Auckland, New Zealand, and Arrowhead officials said they will present more complete data later this year. It is the company’s third generation of an RNAi drug being developed as a potential cure for patients with chronic hepatitis B.

Analyst Y. Katherine Xu, of the William Blair investment firm, said the results are the “best knockdown of the virus” seen so far by an RNA interference drug.

Steve’s Take:

The 52-weel trading range is $2.65-$22.39.

Shares were trading up another 2% Monday at $20.16.

Based on 6 analysts offering recommendations, the current rating on Arrowhead is Strong Buy.

The Barchart Technical Opinion rating is an 88% Buy with a strongest short-term outlook on maintaining the current direction.

Longer term, the trend strength is maximum. Long-term indicators fully support a continuation of the trend.

Weekly #Medical #Stock Spotlight 3 big winners: @ProQR @ArrowheadPharma #recropharma and 2 total duds: #iovance @SangamoTx #biotech #Pharmaceutical Click To Tweet

3) And Recro Pharma Inc. (Nasdaq:REPH) surged 28% to $8.15 after reporting a positive outcome of its meeting with the FDA about its NDA for intravenous meloxicam that was rejected last May.

Based on the discussion with the FDA, the Malvern, PA-based company now plans to submit a revised NDA by the end of this month. Suggestions offered by the FDA are steering the company to include revised labelling language. The May CRL included an ad hoc analysis of the trial and reports on secondary endpoints that undermined the candidate’s efficacy claims.

Meloxicam is a sustained cyclooxygenase (COX-2) inhibitor that leads to subsequent reduction in prostaglandin biosynthesis (i.e., the formation of chemical compounds by living organisms or cells). This chemical property confers the drug with analgesic, anti-inflammatory and antipyretic actions.

Steve’s Take:

The 52-week trading range is $4.78-$13.05.

Shares were giving back 7% Monday (September 10, 2018) at $7.55.

Based on 4 analysts offering recommendations, the current rating is Strong Buy.

The Barchart Technical Opinion rating is a 64% Buy with a weakening short-term outlook on maintaining the current direction.

Longer term, the trend strength is average. Long-term indicators mostly agree with the trend.

4) But cancer immunotherapy company Iovance Biotherapeutics Inc. (Nasdaq:IOVA) tanked 29% to $12.65 in response to a presentation at the H.C. Wainwright Global Investor Conference in New York City last week.

Investors apparently weren’t thrilled with the San Carlos, CA-based company’s clinical update for its mid-stage tumor-infiltrating lymphocyte (TIL) therapy, C-144-01, in patients with advanced skin cancer who have been previously treated with an anti-PD-1 and anti-CTLA-4 checkpoint inhibitor. The top headline is that C-144-01 failed to produce an objective response rate on par with Bristol-Myers Squibb Co.’s combo therapy consisting of Opdivo and Yervoy for this indication (40% vs. 50%).

Leading into this update, however, Wall Street was hoping that C-144-01 would prove to be a superior therapy to Bristol’s cocktail. While it’s still early for C-144-01, this less-than-stellar result may force the company to rethink its clinical strategy.

Steve’s Take:

The 52 week trading range is $6.60-$19.90

Shares were trading off another 2% Monday (September 10, 2018) at $12.35.

Based on 7 analysts offering recommendations, the current rating is Strong Buy.

However, the Barchart Technical Opinion rating is a 72% Sell with a strengthening short-term outlook on maintaining the current direction.

Longer term, the trend strength is Maximum. Long-term indicators fully support a continuation of the trend.

The market is approaching oversold territory. Be on the lookout for a trend reversal and buying opportunity.

5) And Sangamo Therapeutics Inc. (Nasdaq:SGMO) skidded 25% to $13.75 following the biotech’s interim update from a Phase 1/2 clinical study evaluating SB-913 in treating Hunter syndrome, which is also known as mucopolysaccharidosis type II (MPS II).

Richmond, CA-based Sangamo’s update disappointed investors because SB-913 didn’t demonstrate solid efficacy. There are two primary reasons why the SB-913 interim update was such a letdown for investors. Primarily, the study is the first to use zinc-finger nuclease (ZFN) in vivo (inside the body) gene editing in humans. There were high expectations that Sangamo’s ZFN approach would be overwhelmingly effective. That wasn’t the case.

The other reason is that SB-913 is one of three Sangamo ZFN therapies that are currently in early-stage clinical studies. A failure in treating Hunter syndrome could hint at problems for prospects using ZFN to treat hemophilia B and MPS I.

Steve’s Take:

The 52-week trading range is $11.30-$27.50.

Shares were trading up 6% Monday (September 10, 2018) at $14.55.

Based on 7 analysts offering recommendations, the current rating of 6 is Strong Buy and 1 Hold.

On the other hand, the Barchart Technical Opinion rating is a 48% Sell with a weakest short-term outlook on maintaining the current direction.

Longer term, the trend strength is average. Long-term indicators fully support a continuation of the trend.

The market is approaching oversold territory. Be watchful for a trend reversal and buying opportunity.

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