Another week has rolled by, and the biotech sector, being a volatile one, saw some stocks soaring to the moon and some hitting the skids.
vTv Therapeutics Inc. (VTVT) closed the week, up 238%, at $3.35. The Company’s most advanced drug candidate is Azeliragon, which failed in a phase III trial in patients with mild Alzheimer’s disease, dubbed STEADFAST.
Vaxart Inc. (VXRT) gained as much as 173% in the week, thanks to encouraging data from its phase II challenge study of its H1 influenza oral tablet vaccine. The Company’s oral tablet vaccine provided 39% reduction in flu illness compared to 27% for Sanofi’s Fluzone.
AVROBIO Inc. (AVRO) lost over 50% in the week, following clinical data and patient updates from the investigator-sponsored phase I study and the Company-sponsored phase II clinical trial of AVR-RD-01 in Fabry disease.
Now, here are some of the pharma/biotech stocks to watch out for in the week starting October 8.
1. Blueprint Medicines Corp. (BPMC)
Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined subsets of patients with cancer and other debilitating diseases driven by the abnormal activation of kinases.
Avapritinib (BLU-285), BLU-554 and BLU-667 are the Company’s clinical drug candidates.
BLU-285 is being explored in the indications of advanced gastrointestinal stromal tumors and advanced systemic mastocytosis, BLU-554 for the treatment of advanced hepatocellular carcinoma, and BLU-667, a potent and selective inhibitor of RET mutations, fusions, and predicted resistant mutants, is being explored in RET-altered cancers.
On October 6, the Company presented updated data from ARROW, a phase I trial of BLU-667 in advanced RET-altered medullary and papillary thyroid cancers.
According to the data, 90 percent of evaluable patients with medullary and papillary thyroid cancers had radiographic tumor reductions, regardless of RET alteration type or prior multi-kinase inhibitor (MKI) therapy. In addition, the response rate was 62 percent in patients with medullary thyroid cancer treated once daily with BLU-667 at doses of 300 to 400 mg for at least 24 weeks.
BPMC closed Friday’s trading at $69.54, down 2.33%.
2. PTC Therapeutics Inc. (PTCT)
PTC is a biopharmaceutical company focused on the development of drugs for rare disorders.
The Company has two marketed drugs – Translarna and Emflaza – and is expanding its pipeline through in-house innovation and acquisition.
Translarna is approved in the European Union for ambulatory patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. It is not approved in the U.S. yet.
Emflaza is approved in the U.S. for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.
On October 6, the Company announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna.
The data demonstrates that Translarna slowed disease progression in children with Duchenne caused by a nonsense mutation, and also preserved ambulation for up to 5 years compared to natural history.
PTCT closed Friday’s trading at $40.95, up 1.64%.
3. Galapagos NV (GLPG)
Galapagos is a clinical-stage biotechnology company developing medicines for cystic fibrosis, inflammation, and other indications.
The Company’s lead drug candidate is Filgotinib, a highly selective JAK1 inhibitor. Filgotinib is being evaluated in phase III programs in rheumatoid arthritis, Crohn’s disease and ulcerative colitis, and in phase II studies in small bowel Crohn’s disease, fistulizing Crohn’s disease, Sjögren’s syndrome, ankylosing spondylitis, psoriatic arthritis, cutaneous lupus erythematosus, lupus membranous nephropathy and uveitis.
Also in the pipeline are GLPG1690 for idiopathic pulmonary fibrosis, a triple combination therapy for cystic fibrosis, MOR106 for atopic dermatitis, GLPG1972 for osteoarthritis, and a number of preclinical drug candidates for various indications.
Watch out for…
Topline results from part one of FALCON trial, due in Q3, 2018, are awaited. The FALCON trial is a phase II study of the investigational triple combination therapy in cystic fibrosis patients. The FALCON trial was initiated in April of this year.
The investigational triple combination therapy is a combination of GLPG2451, GLPG2222 and GLPG2737.
GLPG closed Friday’s trading at $104.75, down 0.98%.
4. Bausch Health Companies (BHC)
The FDA decision on Bausch Health’s JEMDEL, a topical lotion, proposed for the treatment of plaque psoriasis, scheduled for October 5, is awaited.
JEMDEL, developed by Ortho Dermatologics, a division of Bausch Health, is a topically applied lotion formulation of Ulobetasol, a corticosteroid.
If approved, JEMDEL will be the first high-potency topical steroid treatment for plaque psoriasis with dosing for as long as eight weeks.
BHC closed Friday’s trading at $26.32, down 0.68%.
5. Trevena Inc. (TRVN)
An FDA panel is slated to review Trevena’s New Drug Application for OLINVO (oliceridine) Injection for the management of moderate to severe acute pain on October 11, 2018.
The regulatory agency’s final decision on OLINVO is anticipated on November 2, 2018.
If approved, OLINVO is expected to be a Schedule II controlled substance, which means it has a high potential for abuse.
TRVN closed Friday’s trading at $3.11, up 1.63%.
6. AcelRx Pharmaceuticals Inc. (ACRX)
On October 12, 2018, a panel of outside experts convened by the FDA is scheduled to review AcelRx Pharmaceuticals Inc.’s (ACRX) New Drug Application for Dsuvia, proposed for the management of moderate-to-severe acute pain in medically supervised settings in adult patients.
Dsuvia, formerly known as ARX-04, consists of 30 mcg sufentanil in very small sublingually absorbed tablets that are delivered via a disposable, pre-filled, single-dose applicator.
This is Dsuvia’s second go-around with the FDA. Last October, the FDA had declined to approve Dsuvia, requesting additional data and recommending certain changes to the Directions for Use.
Addressing all the issues, the Company resubmitted the Dsuvia NDA to the FDA in May of this year, which was accepted for review, with a decision date set for November 3, 2018.
AcelRx estimates that the market potential of Dsuvia in the U.S. will be roughly $1.1 billion.
ACRX closed Friday’s trading at $2.91, down 5.83%.
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