Wednesday, April 22, 2015

Deuterated Drugs For Hematologic Diseases

Concert Pharmaceutical is actively pursuing patent applications for deuterated drugs for hematologic diseases.  The company has run a pre-clinical drug trial for the deutered lenalidomide (Concert drug named CTP-221), and is pursuing patents related to Rigosertib, a drug that is used by Onconova (ONTX) in clinical trials. Each drug is primarily directed toward some form of  Myelodysplastic Syndrome (MDS) or AML.
Lenalidomide was approved in 2005 for low to intermediate-1 risk MDS with deletion 5-q chromosomol abnormality.
Rigosertib has been used in several clinical trials as both monotherapy and as co-therapy, intravenously and orally, primarily for intermediate-2 to higher risk MDS, and for patients who have failed prior therapies. For some of these patients who have failed previous therapy (refractory MDS), the prognosis is less than 12 months survival, and the drug side effect profile is quite adverse.  
I really like the direction CNCE is taking in pursuing patents for hematological indications such as intermediate-2 to higher risk MDS, where there remains an un-met need for longer and better tolerated drug therapies.  I am looking one to two years out into the future direction of the company, and believe Concert may be able to get both of these deutered hematological drugs into clinical trials for different MDS patients in 2016, after all patents have been secured.  At this time we do not know if CTP-221 will ever make clinical trials due to competing patent issues.  Thank you for reading.

Friday, April 17, 2015

Concert Pharmaceuticals: JZP-386

JZP-386 is the deutered enhanced version of sodium oxybate or commercial drug known as Xyrem, which is manufactured by Jazz Pharmaceuticals.  The drug was approved in 2002 for cataplexy in narcolepsy, and 2005 for excessive daytime sleepiness (EDS) in narcolepsy.  The patents run from 2019 through 2024 at the latest.  Xyrem is administered twice daily as a liquid dose, once before bed and again after about four hours of sleep. Xyrem has a side effect profile that includes headaches, dizziness, nausea, and potentially more serious side effects in higher doses such as convulsions, respiratory depression, coma and death, according to Xyrem prescribing information.   Concert is currently in a phase 1 trial to evaluate the deutered Xyrem version called JZP-386, compared to Xyrem, with a readout on the trial any day now.  The goal of the deutered version JZP-386, could be once daily dosing, lower dosing, or a reduction in side effects that currently exists with Xyrem. Concert would have a chance to receive royalties that run from mid single digits to low double digits, not to exceed 20% on a country-by-country basis, and licensed product-by-licensed product basis. The patent for JZP-386 runs from 2030 to 2032, U.S. EU, and Japan. Thank you for reading.


Sunday, April 12, 2015

What is CTP-221

CTP-221 is the deutered enhanced drug for lenalidomide, or commercial drug known as Revlimid, which is manufactued by Celgene.  The drug targets indications in Myelodysplastic Syndrome (MDS) and Multiple Myeloma amongst others, and is expected to produce revenue up to seven billion annually for the company. This is the second Celgene drug after CTP-730 that Concert may consider for clinical trials. The patent for Revlimid runs through 2026. CTP-221 has shown to be approximately 5-6 fold greater potency in animal models with similar antitumor effects.  Thank you for reading.
 

Friday, April 3, 2015

What is CTP-730

CTP-730 is the deutered enhanced version of Otezla (Apremilast), that is manufactured by Celgene.  The drug was approved in March of 2014 for psoriatic arthiritis, and again in September of 2014 for moderate to severe plaque psoriasis. The patent for Otezla runs until 2024 US and EU.  The drug is forecasted to bring in around $2 billion in revenue by 2020. The typical dose is 30 mg twice daily, and the drug is known to exhibit side effects such as diarrhea, nausea, and headache. A deuterated version could potentially reduce the dosage to once daily, improve the efficacy, and side effect profile.
Concert is currently in phase 1 testing to explore the safety and pharmacokinetics profile compared to placebo with a full readout around September 2015.  Once the trial is complete a one time $8 million milestone will be awarded to Concert from Celgene. More importantly, will be the trial results....the first in human trials for CTP-730.  The relevance for increased efficacy, or safety with a once a day tablet, would be valuable to Celgene, not to mention the increased patent life that the D-Apremilast version would have, to 2030. Thank you for reading.  
                                                                                       

Wednesday, April 1, 2015

Deuteration Validation

On March 30th, Teva Pharmaceuticals agreed to acquire Auspex Pharmaceuticals for $3.5 billion, or around $100.00 per share.  A few points to consider as it relates to Concert Pharmaceuticals, which is one of our holdings.

1. This acquisition instills confidence in the science of deuterium-based chemistry.  Both Auspex and Concert are applying deuterium to existing drugs to potentially enhance their efficacy, half-life or safety profile.
2. In 2011 Concert assigned patent rights for the deuterated drug Pirfenidone to Auspex.  Under the agreement, if a change in control of ASPX, Concert would be due 1.44% of the purchase price, in this case, approximately $50 million.
3.  After completing a recent secondary offering for up to $46 million and considering the newly acquired $42 million, the company should hold approximately $154 million, after retiring $7 million in debt.  Thank you for reading.