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Thursday, December 8, 2016

21st Century Cures Act A Big Win For Revive Therapeutics

The passage of the 21st Century Cures Act by the U.S. Senate earlier this week is a big positive for Revive Therapeutics (RVV.V) and a validation of the company's business model. The Act, which passed through U.S. congress with an overwhelming majority, 94-5 in the senate and 326-24 in the house, is designed to facilitate U.S. FDA approval of new drugs and medical devices and bring innovative medicines to patients faster.

Specifically, the Act enhances current FDA programs like breakthrough designation, accelerated approval, and fast track status, and directs the U.S. FDA to make decisions based on the use of “real world evidence” for approval of new indications for existing drugs. Companies can now include things like observational studies, patient input, anecdotal data, and other retrospective research analysis as part of its petition for U.S. approval. This greatly reduces the cost and time to market compared to conducting large, prohibitively expensive randomized clinical trials and should increase the odds of success reformulations and label expansions of known therapeutic agents.

Did Congress Just Kick Off A New Bio Bubble?

On December 7, 2016, the U.S. Senate passed the 21st Century Cures Act. President Obama said he plans to sign the bill into law as soon as it hits his desk. The Act, which is designed to facilitate FDA approval of new drugs and medical devices, is a major win for the Pharmaceutical and Medical Device industry, and its supporters such as PhRMA and AdvaMed. Industry spokespersons praise the Act as a way bring about more innovation and get treatments to patients faster.

The bill passed by an overwhelming 94-5 and 326-24 majority. The measure would benefit people with mental illness and chronic diseases, biomedical researchers, pregnant women, hospitals, children with diabetes, people addicted to opioid drugs, children who are bullied, and those who are gravely ill (source: NY Times). And importantly, the measure includes no provisions to rein in prescription drug prices.

Wednesday, December 7, 2016

ORY-1001 Phase 1 Data At ASH In AL Looks Good For Oryzon, Roche

Oryzon Genomics (MADX: ORY) is a clinical stage biopharmaceutical company headquartered in Barcelona, Spain. The company is a leader in the development of epigenetics-based therapeutics. Epigenetics is the study of modifications that occur in DNA that results in activation or deactivation of gene expression without alteration in DNA sequence. The company is applying epigenetics to drug discovery and development in the area of cancer and neuroinflammatory / neurodegenerative diseases.

One of Oryzon's lead drug candidates is ORY-1001, a potent and highly selective Lysine Specific Demethylase 1 (LSD1) inhibitor under development for the treatment of acute myeloid leukemia (AML) and small cell lung cancer (SCLC). Oryzon has made significant progress with ORY-1001 over the past year and just recently presented Phase 1 data at the American Society of Hematology (ASH) meeting earlier in the week. Roche, the company's development and commercialization partner, will take over further clinical studies. In fact, Roche noted during Oryzon's analyst / investor presentation at ASH that they have initiated a trial in SCLC with ORY-1001 (to be called RG-6016). Below is a quick review of ORY-1001 and the Phase 1 data.

Thursday, December 1, 2016

Cerecor Phase 2 Failure Opens Door for Competitors

On November 29, 2016, Cerecor, Inc. (CERC) reported top-line results from a Phase 2 study examining CERC-301 as an adjunctive therapy for the treatment of depression. The study failed to show a statistically significant separation from placebo. In fact, the placebo was superior to the 12 mg dose of CERC-301 using the Bech-6 subset of the Hamilton Depression Scale. The higher dose of 20 mg CERC-301 did show some signs of efficacy, but the results were not meaningful.

CERC-301 is an oral, NR2B specific, NMDA receptor antagonist. I've been keenly interested in biopharma research targeting the NMDA receptor for the treatment of resistant depression (TRD). Along with behemoths like J&J and Allergan, small biopharma players such as VistaGen Therapeutics (VTGN) and Relmada Therapeutics (RLMD) are developing NMDA receptor targeting small molecules for TRD. The failure of CERC-301 is relevant to both VistaGen and Relmada.

Wednesday, November 30, 2016

Vitality Biopharma Taking Cannabinoid Research To The Next Level

Sales of both medical marijuana and adult legal marijuana are soaring. Total marijuana sales in 2014 were less than $5 billion. A story in USAToday notes the number is expected to approach $23 billion in 2020 (1) due to decriminalization and the increasing recognition of its therapeutic utility within the medical community. Twenty-nine states plus the District of Columbia have medical or legalized marijuana laws in place (2). Millions of Americans use marijuana to provide symptom relief from dozens of indication, including cancer, glaucoma, Parkinson's, multiple sclerosis, epilepsy, seizures, wasting syndrome, Crohn's disease, PTSD, and chronic pain.

There are three cannabinoid products approved in the U.S. These products hold the same therapeutic potential as marijuana, yet IMS Health estimates sales totaled only $133 million in 2014. Sales are restrained by several well-known limitations, including poor oral bioavailability, erratic pharmacokinetics, inconvenient administration, short duration of action, poor tolerability, and psychoactive side effects. 

California-based Vitality Biopharma, Inc. (VBIO) has a proprietary glycosylation technology for producing cannabinoid prodrugs. The technology was crafted by the company's research with Stevia and natural sweeteners in 2015; and to date, Vitality has produced more than 25 novel cannaboside molecules; several composition of matter patent applications are pending. Below is an introduction to Vitality Bio, a look at the company's pipeline and the medical marijuana industry, and why investors should put this name on their radar.

Monday, November 28, 2016

Interview With Neurologist and ALS KOL Sheds Light on Brainstorm's Potential With NurOwn

BrainStorm Cell Therapeutics (NASDAQ: BCLI) is a biopharmaceutical company developing adult stem cell therapies for a range of neurodegenerative diseases, with an initial focus on Amyotrophic Lateral Sclerosis (ALS). The company has a proprietary technology that uses a novel differentiation protocol such that mesenchymal stem cells (MSCs) are converted into neurotrophic factor-secreting cells (MSC-NTF cells, NurOwn®). BrainStorm then returns the MSC-NTF cells to the patient at or near the target area for treatment.

In July 2016, BrainStorm reported top-line data from a Phase 2 double-blind, placebo controlled multi-center study with NurOwn® for the treatment of ALS. The company is currently in the process of finalizing the full data analysis from this trial and plans to submit the results for presentation at a medical conference during the first half of 2017. I believe results for the Phase 2 study were very positive; yet, the stock has not reacted accordingly and there seems continued investors confusion around the story. In the meantime, additional studies are being planned to advance NurOwn® into multi-dose and registration trials.

The recently completed Phase 2 study took place at three prestigious medical centers in the U.S.: Mayo Clinic, The University of Massachusetts Medical School, and the Massachusetts General Hospital. To give investors a better sense of the data and the path forward for Brainstorm, I decided to speak with one of the Principal Investigators from the Phase 2 study, Merit E. Cudkowicz, MD, MSc. Dr. Cudkowicz is a Professor of Neurology at Harvard Medical School, Chief of Neurology at the Massachusetts General Hospital and Director of the ALS Multidisciplinary Clinic.

Monday, November 21, 2016

Matinas Initiates Important Phase 2 Study With MAT2203

On November 21, 2016, Matinas BioPharma Holdings, Inc. (MTNB) announced the commencement of dosing in a Phase 2 clinical study examining MAT2203 in patients with vulvovaginal candidiasis (VVC). MAT2203 is an orally-administered, lipid-crystal nanoparticle formulation of amphotericin B. This is an important step for the company's lead pipeline candidate and should provide further clinical evidence of MAT2203 activity against candida infections. Below is a quick review of MAT2203 and my thoughts on why this VVC study is so important.

Wednesday, November 16, 2016

RedHill Expects An Eventful Next Few Months

On November 14, 2016, RedHill Biopharma Ltd. (NASDAQ: RDHL) reported financial results and provided an update on operations for investors following the end of the third quarter 2016. Management has outlined a number of major events expected over the next several months. I believe these could act as catalysts to drive the shares higher in 2017. In the meantime, the company is actively engaged in business development activities and well-capitalized, with $40.5 million in cash on hand and no debt as of September 30, 2016. Below is a quick review of some of the upcoming events for the company.