Saturday, July 22, 2017

BCLI drug NurOwn called promising for stopping ALS. Progresses to Phase 3 worldwide trials



CIRM news release HighLight:
In an earlier Phase 2 clinical trial, involving 48 people with ALS, NurOwn was shown to be safe and well-tolerated. The trial also showed it could halt or reverse disease progression over six months, compared to a placebo.


Phase 3 Clinical Trial Targeting Lou Gehrig's Disease Gets $15.9 Million Investment From Stem Cell Agency

July 20, 2017 Oakland, CA Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a particularly nasty, almost always fatal neurological condition that has no effective treatment and no cure. Now the California Institute for Regenerative Medicine (CIRM) is investing $15.9 million in a therapy to try and change that.
ALS attacks motor neurons, the cells in the brain and spinal cord that control our muscles and movement. Over time the disease  destroys a person’s ability to speak, to swallow, to move, even to breathe.
BrainStorm Cell Therapeutics has developed a therapy they hope will slow down, and potentially even stop, the progression of the disease.
“ALS is a devastating disease with an average life expectancy of less than five years and individuals afflicted with this condition suffer an extreme loss in quality of life,” says Maria Millan, MD, CIRM’s interim President & CEO. “CIRM is partnering with BrainStorm to follow up on the company’s promising Phase 2 trial in ALS. CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurologic condition for which there is currently no cure.”
BrainStorm uses mesenchymal stem cells that are taken from the patient’s own bone marrow. These stem cells are then modified to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by the disease. The CIRM funding will enable the company to test this therapy, called NurOwn, in a Phase 3 trial involving around 200 patients.
In an earlier Phase 2 clinical trial, involving 48 people with ALS, NurOwn was shown to be safe and well-tolerated. The trial also showed it could halt or reverse disease progression over six months, compared to a placebo.
"We are honored to be awarded this CIRM grant, and appreciate the support of CIRM in the development of NurOwn," said Chaim Lebovits, President and CEO of BrainStorm. "This substantial award provides further validation for our technology and clinical program, and recognizes the importance of developing effective treatments for patients afflicted with ALS.” 
The CIRM Board today also awarded $5.79 million to Dr. Ezra Cohen at the University of California, San Diego to develop a genetically modified therapy to attack cancer stem cells. These are cells that are believed to be able to evade standard treatments, such as chemotherapy and radiation, enabling the cancer to return and spread. The funding will enable Cohen to do the research needed to apply for clearance from the Food and Drug Administration for a clinical trial.
Cohen is using what are called specific chimeric antigen receptor T cells, or CAR T cells. The cells are taken from the patient's own blood, then modified so they specifically target cancer stem cells that express a protein called ROR1 on their surface. It’s hoped that after being transfused back into the patient, the CAR T cells will then recognize and kill the cancer stem cells that have ROR1 markers. And because these CAR T cells can remain in the body long after they have been infused, it’s hoped they will prevent cancer recurrence and result in long-term remission.

About CIRM
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.
To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With $3 billion in funding and approximately 300 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information, go to www.cirm.ca.gov


https://www.cirm.ca.gov/about-cirm/newsroom/press-releases/07202017/phase-3-clinical-trial-targeting-lou-gehrigs-disease

Tuesday, January 10, 2017

Clear Demographic Shift Underway & Baby Boomers need Biotechs!

Baby Boomers are already retiring and in 15 years the vast majority will have left the workforce.

What happens as the boomers age? Cancer, strokes, arthritis etc, etc. You get the picture, the biotechs will be counted on a mass scale like never before to treat various forms of diseases.
It will be the old story again "Supply vs. Demand", the politicians can scream all they want about pricing because at the end of the day it will be decided by the Market Demand.

See link for full article: http://money.usnews.com/investing/slideshows/7-stocks-to-buy-for-the-baby-boomer-retirement-wave

The coming Biotech Melt-up will crush many shorts who don't see the writing on the wall ahead of time "Central Banks plans to buy equities".


Current biotechs in focus: 


BCLI 

Alert 7/21/2017 

BCLI is going after ALS and the recent news releases have been surging as it enters the final stage before potential approval.  

CIRM is partnering with Brainstorm to follow up on the Company's promising phase 2 trial in patients with ALS.  CIRM's mission is to accelerate stem cell treatments to patients with unmet medical needs and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurologic condition for which there is currently no cure." Brainstorm is in the advanced stages of planning a Phase 3 clinical trial investigating NurOwn in ALS.  The trial is expected to enroll approximately 200 patients and will be conducted at 6 top ALS clinical sites in the U.S. The primary outcome measure will be the ALSFR-S score responder analysis. The patient population will be optimized to include faster-progressing patients who demonstrated superior outcomes in the NurOwn Phase 2 ALS trial.

ALS is a progressive disease which destroys nerve cells in the brain and the spinal cord. Most ALS patients die from muscle paralysis that stops them from breathing. 

Insurance policies, price, and a look ahead

When a competitor drug Radicava arrives in August, it will come with a list price of nearly $1,100 per infusion, meaning a year’s worth of treatment will be roughly $146,000. So you can see what huge profits are to be had for BCLI upon approval.

Exchange:NASDAQ
Industry: Health Care
Community Rating: Bullish
Brainstorm Cell Therapeutics Inc. Press Releases
 














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VRX
Alert 6/26/17 
Paulson Key to Turning Valeant Around

Paulson:  "The strategic plan to transform Valeant smartly focuses on rebuilding the company's core franchises in ophthalmology, dermatology and gastroenterology while simultaneously using the proceeds from the sale of non-core assets and operating cash flow to de-lever the company. I am fully supportive of the strategy and leadership team at Valeant."
 
VRX stock may never be a $200 per share stock again, but if the market cap gets to even half the five times sales of, say, Johnson & Johnson (NYSE:JNJ) you’re looking at a gain of 500% from current levels (6.22.17). Getting Paulson, the largest stockholder with a 5.9% stake, onto the board is another step in that direction. He’s not a plunger or fast profit guy like Ackman. He became a player after betting right on the 2008 financial crisis.  By Dana Blankenhorn
Read more: Billionaire Hedge Funder John Paulson Joins Valeant Board (VRX) | Investopedia http://www.investopedia.com/news/billionaire-hedge-funder-john-paulson-joins-valeant-board-vrx/#ixzz4l9LiGJ7T


AVEO
Alert 6/23/17 
European Medicines Agency (EMA), has recommended FOTIVDA™ (tivozanib) for approval, If approved by the EC, marketing authorization for tivozanib will be granted in all 28 countries of the Europe.  EUSA Pharma has agreed to pay AVEO up to $394 million in future research and development funding and milestone payments, assuming successful achievement of specified development, regulatory and commercialization objectives, as well as a tiered royalty ranging from a low double-digit up to mid-twenty percent on net sales of tivozanib in the agreement’s territories. Thirty percent of milestone and royalty payments received by AVEO, excluding research and development funding, are due to Kyowa Hakko Kirin (KHK) as a sublicensing fee in Europe. In the United States, the royalty obligation to KHK ranges from the low- to mid-teens on net sales.
In addition, the insiders have been going wild buying millions of shares of $AVEO.

DVAX 
 Odds of FDA approval now at 70% says Biotech Guru, should have a continued runup in July heading to FDA approval. The Cancer Index announced 6/21/17 that it has added DVAX to the index.
 http://www.investopedia.com/investing/william-blair-assigns-70-probability-approval-dynavaxs-heplisav-dvax/
Combination of Dynavax's SD-101 and Keytruda show 100% response in melanoma study;
http://abstracts.asco.org/199/AbstView_199_193149.html

Phase 3 HEPLISAV-B ™ Adult Hepatitis B Vaccine Phase 1/2 Cancer Immunotherapy Phase 2 Asthma Therapy - Partnered with AstraZeneca In Europe, Dynavax manufactures Hepatitis B surface  antigen for HEPLISAV-B.
 

BLCM  
 https://seekingalpha.com/article/4061431-bellicum-pharmaceuticals-lead-drug-shows-91-percent-remission-leukemia-94-percent-survival

MCRB

The FDA has granted SER-109 which is in Phase 3 both Breakthrough Therapy and Orphan Drug Designations.  Combined PT estimates are now at $23.20

https://www.thestreet.com/story/14173915/2/seres-therapeutics-initiates-ser-109-phase-3-study-in-patients-with-multiply-recurrent-c-difficile-infection.html



CORT
Revenues of Corcept surged by 72% as its FDA approved drug gains traction.
https://seekingalpha.com/article/4067653-corcept-therapeutics-cort-ceo-joseph-belanoff-q1-2017-results-earnings-call-transcript



NK & OMER
https://www.benzinga.com/general/biotech/17/06/9609084/2-longs-and-2-shorts-to-watch


LOXO
 https://www.ft.com/content/c2e1e358-5df5-3669-8e6a-fd35e20681e8