US stocks look set to open higher on Thursday as investors tried to shake off concerns surrounding global trade. Today’s biggest movers and shakers on the Street belong to biotech firms Proteostasis Therapeutics Inc (NASDAQ:PTI) and Solid Biosciences Inc (NASDAQ:SLDB). Let’s take a closer look:
Proteostasis Therapeutics’ Cystic Fibrosis Drug Gets Orphan Status, Shares Soar
Clinical-stage biopharmaceutical company Proteostasis Therapeutics said its experimental drug PTI-428 received an orphan drug designation from U.S. health regulators for the treatment of cystic fibrosis (CF), sending its shares up 21%.
The FDA grants orphan drug designation to drugs that may provide a significant therapeutic advantage over existing treatments and target conditions affecting 200,000 or fewer patients in the United States every year.
According to the GVR, the global CF therapeutics market is expected to reach USD 13.9 billion by 2025. The rising prevalence of CF indicates the rising demand for treatment alternatives, thereby uplifting the market.
Some other drugs that are under clinical studies are Vertex Pharmaceuticals’ (NASDAQ:VRTX) VX-445 in combination with tezacaftor and ivacaftor (phase 3), ProQR Therapeutics’ (NASDAQ:PRQR) QR-010 (phase 1 – on hold), AstraZeneca’s (NYSE:AZN) AZD5634 (phase 1), and Polyphor’s POL6014 (phase 1).
Proteostasis CEO commented, “This is the second important regulatory designation PTI-428 has been granted from the FDA this week, highlighting our amplifier’s potential to provide clinical benefit in the treatment of CF.”
A Major Clinical Setback Is Wrecking Havoc on Solid Biosciences Stock
Shares of life science company Solid Biosciences are in free fall on Thursday.
The company announced that the FDA placed a clinical hold on the company’s lead microdystrophin gene transfer candidate for the treatment of Duchenne muscular dystrophy (DMD).
The hold reportedly stems from a cases of unexpected adverse reaction in one of the patients on the treatment. Specifically, the first patient dosed in the clinical trial was a non-ambulatory adolescent who received 5E13 vg/kg of SGT-001 on February 14, 2018. Several days after administration the patient was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation. The patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests. The patient responded well to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.
Solid Biosciences’ shares are down by 57% at 8:46 a.m EST on heavy volume in response to this news.
Chardan analyst Gbola Amusa has recently initiated a Buy rating on SLDB with a $40 price target.
Amusa wrote, “We initiate coverage on Solid Biosciences with a Buy rating (PT$40), on the potential for SGT-001 as a best-in-class medicine for the progressive, fatal muscular degeneration and weakness of Duchenne muscular dystrophy (DMD). DMD is a rare, X-linked disease with an incidence of roughly 1/3,500 male births1 , which we calculate yields approximately 22,000 prevalent DMD cases in the United States. 2 DMD patients are born with normal muscle function. However, because of mutations in the gene encoding dystrophin, patients eventually present with muscle weakness between ages 3 and 5. With disease progression, afflicted individuals are typically wheelchair-bound by the ages of 9 to 13 and see early mortality in their 20s to 40s from cardiac or lung complications. The standard of care for DMD includes corticosteroids, which are the only treatments shown to slow muscle degeneration and extend ambulation (for only 2 years or so), while producing numerous side effects. Due to limited clinical evidence, other products (e.g. Exondys 51) are only conditionally approved in the US and Europe.”