FDA rejects Pain Therapeutics’Remoxyer Capsules NDA; Shares Nosedive-Normally thinly traded shares, Pain Therapeutics $PTIE came under heavy selling pressure on Monday after it announced having received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA), seeking approval of abuse-deterrent REMOXYER. In its letter, the FDA asked the drug-maker to provide additional data and take more actions for approval. The management expects that actions would take about a year to complete. Also, the regulator said that Pain’s clinical assessment to support a claim against abuse by chewing is not sufficient and it will need to provide a fresh proposed label to indicate that the results from the clinical trials do not support the claim. This was Pain’s third attempt for approval. Its licensor DURECT $DRRX also felt the heat on Monday.
Viking Therapeutics Announces Positive POC Data for VK0214 in in-vivo model of X-ALD-Shares of Viking Therapeutics Inc. $VKTX edged higher in pre-market trading on Monday after the clinical biopharmaceutical company, which focuses on the development of therapies for metabolic and endocrine disorders, presented a positive proof-of-concept data for VK0214 in in- vivo model of X-linked adrenoleukodystrophy (X-ALD) at the 86th annual meeting of the American Thyroid Association. It said that the study achieved its primary objective.
ERYTECH Concludes Patients Enrollment in Mid-Stage Study of eryaspase-RYTECH Pharma $EYRYY announced competing enrollment for its Phase 2 clinical trial evaluating eryapase, branded as ERY-ASP or GRASPA intended to cure the patients with pancreatic cancer. The topline data of the study is expected in early 2017. The trial enrolled 139 subjects. ERYTECH said that the safety and efficacy profile of eryaspase, as observed in its Phase 2/3 pivotal clinical trial, offers a very effective alternative for the treatment of leukemia patients.
Lipocine's Shares Climb; Mid-Stage Study Candidate LPCN 1111 Shows Promising Results- Lipocine $LPCN shares surged on Monday after it announced encouraging results from a Phase 2b study of its oral testosterone replacement therapy candidate LPCN1111. The open-label, two-period, multi-dose pharmacokinetic trial randomized 60 hypogonadal males into five treatment groups of 12 subjects each. All were treated for 14 days with single and multiple doses of LPCN 1111. The primary objective, along with safety and tolerability, was to determine the Phase 3 dose, which met the primary and secondary endpoints.
SciClone Pharmaceuticals’ Clinical Trial on ZADAXIN Shows Progression in Greater China- SciClone Pharmaceuticals $SCLN announced two major accomplishments for advancing its clinical pipeline in Greater China. The first patient got treated in the Phase 3 study of ZADAXIN in sepsis, a life-threatening infectious disease, for which the treatment has previously showed therapeutic benefit in a Phase 2 clinical trial. The goal of the SciClone-sponsored Phase 3 trial in sepsis is to build on positive data from a smaller, previously conducted trial which showed a survival advantage for sepsis patients treated with ZADAXIN.
GW Pharma Shares Gain Sharply; Rare Epilepsy Clinical Stage 3 Study Shows Promising Results- Shares of GW Pharma $GWPH surged on Monday after the drug-maker announced its second successful Phase 3 trial for Epidiolex, intended to cure a rare form of epilepsy. It was the second randomized, double-blind, placebo-controlled Phase 3 clinical trial of its candidate Epidiolex (cannabidiol) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy.
Mast to Stop Trials on vepoloxamer- Mast Therapeutics $MSTX reeling under last week’s sell-off following vepoloxamer failure in Phase 3 study, announced on Monday that it was winding down the studies and focus on its heart candidate AIR001 instead. AIR001 (sodium nitrite solution for intermittent inhalation) is intended for the patients with heart failure with preserved ejection fraction (HFpEF). Three ongoing Phase 2 studies, two in HFpEF and one in pulmonary hypertension, will stay on course. The winding down of vepoloxamer would lower operating expenses by about 70% from $32mn -$34mn to $9mn-$10mn, the company said. Avoid at all cost.
Abeona Therapeutics Enrolls First Patient in Phase 2 Study of EB-101- Abeona Therapeutics $ABEO enrolled the first patient in the Phase study of EB-101 intended for the patients with Epidermolysis Bullosa. EB-101 is the company's lead gene therapy program for patients suffering with recessive dystrophic epidermolysis bullosa (RDEB), a severe form of epidermolysis bullosa (EB). Commenting on the enrollment, $ABEO’s CEO, Dr. Timothy J. Miller said, "The clinical data on the initial patients showed significant improvement in wound healing and gene expression through 12 months post- grafting treatment.”
Array Bio's encorafenib-binimetinib combo Shows Good Results in late-stage melanoma study- Shares of Array BioPharma $ARRY gained sharply on Monday after it announced positive results from Phase 3 clinical trial, COLUMBUS, evaluating combination of LGX818 (encorafenib) and MEK162 (binimetinib) for the treatment of patients with BRAF-positive, advanced, unresectable or metastatic melanoma.
Daiichi Sankyo Begins Phase 3 Clinical Trial of CS-3150; To Receive $15mn in Milestone- Daiichi Sankyo commenced Phase 3 clinical study of CS-3150 on Japanese patients with high blood pressure, said its partner Exelixis $EXEL. CS-3150 is an oral, non-steroidal, selective mineralocorticoid receptor antagonist, for the treatment for essential hypertension. Under the terms of the agreement, Daiichi Sankyo is eligible to receive $15M in milestone payment from Exelixis as it enrolled the first patient for study. It expects to receive payment in Q4.
Onconova to Design Phase 3 Study for Lead Candidate rigosertib- Onconova Therapeutics concluded its end-of Phase 2 meeting with FDA for its lead candidate rigosertib. The lead product candidate is a combination of oral rigosertib and azacitidine intended for the patients with higher-risk myelodysplastic syndromes (HR-MDS, Onconova (ONTX +1%). It also informed on Phase 3 design. The late-stage randomized, placebo-controlled study will compare the combination of oral rigosertib and azacitidine to azacitidine plus placebo in first-line HR-MDS patients. The primary efficacy endpoint will be overall response rate (ORR), which is a composite of complete and partial responders.
Abbvie Files Marketing Application IMBRUVICA- AbbVie $ABBV filed a supplemental New Drg Application (sNDA) in the U.S. for its drug Imbruvica. In its sNDA, the drug-maker sought approval for using IMBRUVICA (ibrutinib) for the treatment of patients with marginal zone lymphoma, a slow-growing form of non-Hodgkin's lymphoma. If approved, it will be the fifth blood cancer indication for the treatment.
Biogen Submits NDA for nusinersen- Biogen $BIIB finalized its rolling New Drug Application (NDA) with the FDA on Monday, seeking approval for nusinersen intended for the patients with spinal muscular atrophy (SMA). In its approval application, the drug-maker also requested for a Priority Review which, if granted, will shorten the review clock to six months from the usual 10 months. The company intends to file a marketing application in Europe in the coming months.