Friday, July 31, 2015

Three Is Fine

Let me explain.......Three is the number of indications (in house or licensed out) that I believe Concert Pharmaceutical could have in pivotal phase 3 clinical trials in 2016.  The three indications are as follows.

1.  AVP-786 - Agitation in patients with dementia of the Alzheimer's type.
2.  CTP- 499 - For diabetic nephropathy (kidney disease or kidney damage that occurs in people with diabetes).
3.  AVP -786 - Adjunctive therapy in patients with major depressive disorder with an inadequate response to anti depressant treatment.  

AVP-786 has two phase 3's for agitation listed in the clinical government documents, but not ready to recruit just yet.  I expect the recruitment to be in progress by September end.  
CTP-499 for diabetic nephropathy is in negotiations for a Special Protocol Agreement with the FDA. Once that is achieved the company would then look for a partnership to license out and split costs of the lengthy phase 3 clinical trial.  
AVP-786 for major depressive disorder, will complete their phase 2 clinical trial by mid year 2016. If the results are good, I would expect Avanir (Otsuka) to file for a pivotal phase 3 clinical trial or trials by the end of 2016.  

The optimistic view, is that the company could potentially have five indications (a combination of in house and licensed out) in phase 3 clinical trials in 2016.  This could happen if CTP-730 and CTP-656 whom both will have completed phase 1 clinical trials in 2015, apply for the 505(b)(2) New Drug Application path, and be able to move right to phase 3 pivotal clinical trials in 2016. 

Bottom Line:  Concert is having a very productive year, both in advancing clinical trials, and by increasing their cash position by about $100 million, from a combination of a secondary offering, and a $50 million windfall from a previous agreement with Auspex Pharmaceutical.  The company should have three pivotal phase 3 clinical trials running in 2016, with the optimistic view that they could potentially have up to five indications in phase 3 in 2016.  Thank you for reading.  

Contact:  portfoliomgt1@gmail.com

Sunday, July 26, 2015

Allergan Buys NMDA drug for $560 Million

Allergan says yes to CNS (central nervous system) and NMDA (N-methyl-D-aspartate) modulating drugs for depression, with the purchase of private company Naurex for $560 million.  We previously wrote about NMDA drugs and Naurex here, NMDA Receptor Modulators.  NMDA drugs are rapidly gaining press as mono and adjunct therapy for depression patients.  Avanir is currently in a phase 2 clinical trial with their NMDA modulator AVP-786, as an adjunctive therapy in patients with Major Depressive Disorder, with an inadequate response to anti depressant treatment. Avanir will complete their phase 2 clinical trial in the first half of 2016.  Naurex has already tested NRX-1074 as an IV as adjunctive in a phase 2 study for patients with Major Depressive Disorder.  Next, the company will test an oral NRX-1074 in a planned phase 2 clinical trial as mono-therapy for Major Depressive Disorder (MDD).

Bottom Line:  At this point we have no idea how NRX-1074 oral will perform, compared to their completed intravenous phase 2 clinical trial for patients with Major Depressive Disorder (MDD).  Avanir with drug AVP-786, is most likely two years ahead of NRX-1074 in the clinical trial process.  Thank you for reading.      

Contact: portfoliomgt1@gmail.com

Thursday, July 23, 2015

CTP-730: Potential Milestone and Royalty Winner

We originally wrote about CTP-730 here, What is CTP-730.  Today Celgene held it's second quarter conference call this morning.  This is what Scott Smith head of Global Inflammation and Immunology had to say regarding the launch of Otezla, and the future pipeline of indications that Otezla could potentially get approved for.  
Scott Smith President, Global Inflammation and Immunology
"Thank you, Jackie.  Q2 was a great quarter for Celgene I and I.  During the quarter, we saw significant acceleration of prescriptions and revenues for OTEZLA in the U.S. and strong initial uptake in the early launch countries internationally. We also made progress on indication expansion for OTEZLA advancing a global Phase III program in Behcet's Disease and Phase II studies in atopic dermatitis and ulcerative colitis.
Now turning to OTEZLA, were seeing a substantive uptick in revenues and demand in the U.S. Total prescriptions far outpace the recent launch analogs in the I & I space and currently measure over 4,500 TRx' per week based on the latest data. Revenues for the quarter grew to $90 million worldwide and we're tracking well in line with internal plans. We're very encouraged at the progress we're seeing outside of the U.S., both in Canada and in early launch countries in the EU.  After only five months, we're outpacing all recent launches in Germany.  Still very early in the launch, but this initial success helps reinforce the global value proposition of OTEZLA and the need for novel approaches to the treatment of I & I disease.
The trends in the U.S. are supported by positive launch metrics.  While the PSA launch continues to make strong and steady progress, the launch of psoriasis indication has fueled much of the recent acceleration.
Access to new therapies is a critical component of success in the market.  And it's important to note that over 70% of OTEZLA prescriptions in pre-biologic patients are being approved on first pass.  Total U.S. patient share for OTEZLA in psoriasis surpassed ALLERA some months ago and passed ENBREL's overall patient share in June.  The source of business in psoriasis continues to be heavily weghted towards the pre-biologic sector with 75% of patients coming to OTEZLA from topical therapy or no therapy at all over the past 12 months".

Bottom Line:  CTP-730 is Concert Pharmaceutical's deutered enhanced Otezla, that just finished a phase 1 clinical trial.  CTP-730 has been licensed to Celgene, with future milestone, and royalty payment potential for Concert.  Otezla has had a very strong launch, and could expand into Behcet's Disease, atopic dermatitis, and ulcerative colitis. Thank you for reading.
Contact:  portfoliomgt1@gmail.com

Saturday, July 18, 2015

Rexulti & AVP-923 Safety Profile Comparison

When prescribing drugs for the elderly population with neurological conditions, it is important to recognize the side effect profile for this fragile population.  There is not an FDA approved drug for the treatment of agitation in patients with dementia of the Alzheimer's type.  Otsuka Pharmaceuticals has a total of five phase 3 clinical trials that have been in progress, or about to start.  The two drugs that are addressing this indication are Rexulti (Brexpiprazole), and AVP-923 (AVP-786). Rexulti is a joint 50% ownership between Lundbeck and Otsuka.  AVP-923 (AVP-786) is 100% owned by Otsuka, with a milestone and royalty licensing agreement with Concert Pharmaceuticals. The side effect comparison between Rexulti and AVP-923 is below.

Rexulti (Brexpiprazole)
In clinical trials here phase 3 Schizophreniathe 2 mg group of Rexulti exhibited the following side effect profile.
Insomnia 13.4%
Headache 9.3%
Agitation 8.6%
In clinical trials here phase 3 Depression, the 2 mg group of Rexulti exhibited the following side effect profile.
Weight Increase 8.0%
Akathisia 7.4%

The newly approved Rexulti for schizophrenia and depression, has the following safety label information below.

"Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk (1.6 to 1.7 times) of death compared to placebo (4.5% vs. 2.6%, respectively).  Although the causes of death varied, most of the deaths appeared to be cardiovascular (e.g., heart failure, sudden death) or infectious (e.g.,pneumonia) in nature. Rexulti is not approved for the treatment of patients with dementia-related psychosis".

AVP-923 (AVP-786)
In a phase 2 clinical trial here AVP-923 Phase 2 Results for patients with agitation with dementia of the Alzheimer's type, the following side effect profile was exhibited.
Falls 8.6%
Diarrhea 5.9%
Urinary Tract Infection 5.3%

Falls had a baseline skew, that had more patients in the treatment group (20% to 12%) than the placebo group, at the start of the trial.  In addition from that phase 2 clinical trial, there were no new cardiovascular safety signals and no clinically significant changes in QTc observed in the study.  These results above are consistent with several other clinical trials that Avanir has run with AVP-923, for traumatic brain injury, or stroke here Prism IIwith diarrhea being the most commonly reported adverse event.

Bottom Line:  Otsuka has advanced the deutered version of AVP-923, (known as AVP-786) into three phase 3 clinical trials for the treatment of agitation in patients with dementia of the Alzheimer's type, to begin soon.  The company also has two ongoing clinical trials in progress with Rexulti for the same patients, that started in 2013. The patent for Rexulti runs until 2027 US, and until 2025 EU.  Thank you for reading.

Contact:  portfoliomgt1@gmail.com

Saturday, July 11, 2015

FDA Approves Rexulti (Brexpiprazole)

Late Friday the FDA approved Otsuka and Lundbeck's atypical antipsychotic drug Rexulti (Brexpiprazole), as mono therapy for Acute Schizophrenia, and as adjunct therapy for Major Depressive Disorder. We previously wrote about Brexpiprazole here Lundbeck Clinical Trials Update .  Otsuka, the maker of Abilify has been under pressure to replace lost sales of the world's number one selling drug Abilify (Top Selling Drugs) in 2014, due to patent expiration in 2015. Rexulti (Brexpiprazole) has been the drug to replace Abilify for several similar CNS indications.  Under the marketing agreement Lundbeck (co-developer and co-commercialization) will get 45% of Brexpiprazole U.S. net sales, and 50% net sales in Europe.  The patent for Rexulti runs until 2027 US, and 2025 in the EU. The label for Rexulti is below.

INDICATIONS and IMPORTANT SAFETY INFORMATION for REXULTI ®(brexpiprazole)
INDICATIONS
REXULTI is indicated for:
  • Use as an adjunctive therapy to antidepressants in adults with major depressive disorder
  • Treatment of schizophrenia in adults
IMPORTANT SAFETY INFORMATION
Increased Mortality in Elderly Patients with Dementia-Related Psychosis
Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk (1.6 to 1.7 times) of death compared to placebo (4.5% vs 2.6%, respectively). Although the causes of death were varied, most of the deaths appeared to be cardiovascular (e.g., heart failure, sudden death) or infectious (e.g., pneumonia) in nature. REXULTI is not approved for the treatment of patients with dementia-related psychosis.

Rexulti (Brexpiprazole) deemed the second generation Abilify, is getting a similar safety label as Abilify, even though the side effect profile between the two drugs varies, with Rexulti showing improvement in some areas such as akathisia.  It will be interesting to see what kind of label AVP-786 gets for the Treatment of Agitation in Patients with Dementia of the Alzheimer's Type.  AVP-786 is about to enter phase 3 clinical trials in September. Thank you for reading.

Contact:  portfoliomgt1@gmail.com

Sunday, July 5, 2015

Aquarius Biotechnologies

Private company Aquarius Biotechnologies was acquired by Matinas Biopharma on January 30th, 2015 here, Matinas Biopharma Acquires Aquarius Biotechnologies. This was a good acquisition for Matinas as it gives the company a more broad and differentiated pipeline of indications.  Aquarius specializes in a lipid based chochleate drug delivery system.  Their goal is to improve current drugs for anti-fungal; like Amphotericin (MAT2203), and for bacteria in the body; like Amikacin (MAT2501), with their chochleate technology.  The strong point of the technology is in it's oral delivery, which is an improvement over currently available standard of care intravenous (IV), and the safety profile on a comparable basis.
Aquarius (now Matinas) is not the only company that is targeting bacterial and fungal indications.  AstraZeneca has also funded a start-up with $40 million here Entasisfor Gram-negative infections including Acinetobacter baumannii, Pseudomonas aeruginosa, and Enterobacteriaceae.  Also, a single dose oral therapy for susceptible and drug-resistant Neisseria gonorrhoeae, which is currently in a phase 2 clinical trial with drug EXT0914. Thank you for reading.

Contact:  portfoliomgt1@gmail.com

Thursday, July 2, 2015

Orkambi Gets FDA Approval

Today the FDA approved the combination of Vertex's Lumacaftor and Kalydeco (Orkambi) for cystic fibrosis people, 12 years and older, with two copies (homozygous) of the F508del mutation. We originally wrote about Orkambi here Vertex Pharmaceutical Passes Advisory Committee, when they were front and center with the FDA advisory committee.

Orkambi has been appoved for CF patients despite the relatively small improvement in FEV1 in two F508del clinical studies.  How high is the bar set for other companies to get clinically significant improvement over Kalydeco as mono therapy for the G551D mutation, and Orkambi combination therapy, for patients 12 years and older, with two copies of the F508del mutation?

G551D Through 24 weeks Kalydeco 150 mg bid
10.6% and 12.5% FEV1 improvement vs. placebo
G551D Through 48 weeks Kalydeco 150 mg bid
10.5% and 10.0% FEV1 improvement vs. placebo
3,000 Worldwide Patients

F508del Through 24 weeks Lumacaftor - Kalydeco 400/250 mg
2.6% and 3.0% FEV1 improvement vs. placebo
26,000 U.S. + EU Orkambi Patient Potential

These are the FEV1 (forced expired volume) numbers that other companies will be considering as significant, to improve current therapy for CF patients.  The side effect profile was mild, as a high percentage of patients completed the Vertex clinical trials.  The annual wholesale acquisition cost (WAC) will be priced at around $259,000 per patient for the Orkambi combination.  Thank you for reading.

Contact:  portfoliomgt1@gmail.com

Saturday, June 27, 2015

NMDA Receptor Modulators for Depression

NMDA (N-mythel-D-asparate) roots track back to a drug known as Ketamine or street name Special K, with hallucination effects.  Ketamine is an NMDA receptor antagonist originally used as an anaesthetic.  The glutamate receptor subtype known as (NMDA) plays a central role of modulating brain activity in the central nervous system, such as synaptic transmission, synaptic plasticity, and excitotoxicity. Pharmaceutical firms have been developing NMDA type drugs for over two decades, and a few including Memantine, and Nuedexta are being marketed for other indications outside of depression.
There have been several studies modulating the NMDA receptor, that have shown efficacy in patients with treatment-resistant depression.  A trial performed by Avanir Pharmaceuticals for pseudobulbar affect with drug AVP-923 (Nuedexta), showed efficacy in a subset of patients for depression using the Beck Depression Inventory II or BDI-II. Data showed that the dextromethorphan (an NMDA receptor antagonist, sigma 1 agonist) / quinidine combination was effective in patients with BDI-II scores greater than 18 at inclusion with AVP-923 30/10, and was associated with a statistically significant improvement of (p=0.03). What's also significant is that major depression was an exclusion into the trial.  Avanir is now in a phase 2 clinical trial with AVP-786, for major depressive disorder patients as adjunct to current antidepressant here NCT02153502.  I think the chances of success are high, based on how the dextromethorphan/quinidine combination performed in the subset of patients in prior trials.
Private company Naurex, is also working on NMDA drugs for depression patients. The company has displayed good data in early clinical trials using IV, and soon to be launched an oral NMDA drug for depression.  Thank you for reading.

AVP-786 is Concert Pharmaceutical's deutered enhanced version of AVP-923.

Contact:  portfoliomgt1@gmail.com

Tuesday, June 23, 2015

AVP-786 Clinical Trials

AVP-786 is the deutered version of AVP-923, an Avanir drug that has been FDA approved for pseudobulbar affect (PBA) since 2010. Since then AVP-786 has expanded out to other indications, such as alzheimer's, depression, and now residual schizophrenia. Below is a list of current and clinical trials that are close to being initiated. Otsuka acquired Avanir in 2014.

NCT02153502
Phase 2 Major Depressive Disorder (Adjunct)
NCT02477670
Phase 2 Residual Schizophrenia
NCT02442778
Phase 3 Agitation in patients with dementia of Alzheimer's
NCT02442765
Phase 3 Agitation in patients with dementia of Alzheimer's
NCT02446132
Phase 3 Long term, extension study agitation in patients with dementia of Alzheimer's

AVP-786 is the Concert Pharmaceutical deuterated version of AVP-923.

Contact: portfoliomgt1@gmail.com

Friday, June 19, 2015

Triglyceride-Lowering Agents

Omega - 3 fatty acids sourced from fish oil have been shown to be beneficial to patients with high triglycerides. In 2004 while at Reliant Pharmaceuticals, current Matinas CEO Rongen and other employees led the FDA approval and launch of Lovaza (omega 3-acid-ethyl esters) for severe "hypertriglyceridemia greater than 500 mg/dL".  In 2007 GSK (Glaxo Smith Kline) purchased privately held Reliant Pharmaceuticals for $1.65 billion.  Lovaza at the time of acquisition was the only drug approved by the FDA for very high triglycerides, ranging from greater than 500 to 2000 triglyceride level.

In July 2012.  Amarin Pharma received U.S. FDA marketing approval for Vascepa, also referred to as AMR-101 in the severe triglyceride range of greater than 500 and higher, same as Lovaza.  In clinical trials, Vascepa also lowers LDL-C, Lovaza does not.


Vascepa Phase 3 Trial Results:  
- Phase 3 12 week "Marine" clinical trial results 500 and greater TG, with or without statin,  4g / day -45.4% reduction TG, and LDL -2.3%.
Phase 3 12 week "Anchor" clinical trial results 200 - 500 (73% in treatment arm had diabetes), and on statin therapy, 4g / day, -17.5% TG (median reduction from baseline) and +1.5% LDL.
Amarin is also in a clinical trial here called REDUCE-IT for patients with high triglycerides.  The study is a time to first event, which in this case happens to be a first major cardiovascular event.  

Matinas is developing MAT9001 initially for very high triglycerides in the range of 500 to 2000.  In a head to head clinical trial with Vascepa (200 - 400), MAT9001 had a 33.2% reduction compared to 10.5% for Vascepa (p value = .001).  Also reduced LDL by 2.4% vs. 4.3% for Vascepa.  The trial was well designed, as the same patients were used twice, first with MAT9001, than there was a washout period, and those patients were tested again with Vascepa. The company feels that the greater than 500 triglyceride level is a good first step to pursue for FDA approval, prior to the high 200 - 499 level.

Lovaza generic is being prescribed for patients in the very high TG levels of  500 - 2000. Below are two phase 3 trials below that led to FDA approval for these patients and in Europe as a supplement to the diet for the treatment of elevated levels of triglycerides. 

Pivotal Studies of Omega-3-Acid Ethyl Esters (Lovaza) For the Treatment of Very High Triglyceride Levels
Study 1 (Harris)Study 2 (Pownall)
No. of patients4240
Inclusion criteriaTG 500–2,000 mg/dLTG 500–2,000 mg/dL
TreatmentEsters 4 g daily or corn oil placeboEsters 4 g daily or corn oil placebo
Duration16 weeks6 weeks
ResultsChange from baseline results of Esters/placeboChange from baseline results of Esters/placebo
Total cholesterol↓15% / ↓2%↓10% / 0%
Triglycerides↓45% / ↑15%↓39% / ↑8%
LDL-C↑31% / ↓5%↑17% / ↓4%
HDL-C↑13% / 0%↑6% / ↓6%
LDL-C = low-density lipoprotein-cholesterol; HDL-C = high-density lipoprotein-cholesterol; TG = triglyceride.
From the two phase 3 trials above. Lovaza did a nice job of reducing triglyceride levels of 45% and 39%, but the drug raised the LDL-C levels in both trials by 31% and 17%.  This was not seen in the phase 1/2 clinical trial with either MAT9001 or Vascepa in the 200 - 500 triglyceride range.  
Bottom Line:  Matinas Biopharma  has a unique agent to reduce triglycerides, and has shown in a head to head phase 1 / 2 clinical trial that it held a significant advantage over Vascepa for patients in the 200 - 400 range.  Next step is to initiate a phase 3 clinical trial for patients with severe triglycerides, in the range of 500 - 2000.  Thank you for reading.

Friday, June 5, 2015

Matinas Biopharma ($1.33)

Longshot dejour.  Matinas Biopharma is an interesting bulletin board stock that has sound management and a drug that could make it through FDA approval within the next few years depending on how many phase 3 trials the FDA requires.  From Matinas Biopharma website...."Matinas BioPharma is deveolping MAT9001 for the therapeutic applications in dyslipidemia field, with severe hypertriglyceridemia (TG greater than 500 mg/dL) as the primary indication. Based on promising early data, the company filed an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2014. Matinas Biopharma plans to provide further details on its development program for MAT9001 as well as its plans to commence its Phase 3 registration program in the near future".

Quick Facts:
57 million shares outstanding
66 Million Market Cap
13 Million in Cash
39M Warrants Outstanding

Intellectual Property:
MAT 9001 2033  Expiration
MAT 8800 2033  Expiration
MAT 2203 2027  Expiration
MAT 2501 2027  Expiration

Bottom Line:
Matinas will announce the start of a phase 3 clinical trial (will last approximately one year) to commence in 2015 for lead drug MAT9001, for patients with high triglycerides, reading over 500 mg/dL.  Based on a head to head comparison in a phase 1 trial compared with Vascepa, the phase 3 trial could prove to be statistically significant.  Whether the FDA requires two phase 3 trials, remains to be seen.  The patent for MAT9001 extends to 2033. Thank you for reading.

Contact: portfoliomgt1@gmail.com

Tuesday, June 2, 2015

Concert Pharmaceutical: ($16.91)

Quick technical look at Concert Pharmaceuticals with a daily chart below. Everyday the stock closes higher, a new all-time closing high is achieved.
The out performance is evident when shown on a chart that has the XBI Small Cap Biotechnology Fund, and the S&P 500 below over a five day period.  
Bottom Line:  Funds are adding shares, as they see future value at these prices.
Thank you for reading.

Monday, May 25, 2015

AVP-923 and AVP-786

Avanir Pharmaceuticals with drug AVP-923 completed a very successful phase 2 trial for symptoms of agitation in Alzheimer's patients.  Avanir now under the Otsuka umbrella, is conducting two phase 3 trials with the deuterated version of AVP-923, known as AVP-786 for the same patients.  So what are the drug dose differences and exclusion criterias between the successful phase 2 AVP-923 clinical study, and the AVP-786 phase 3 clinical trials for the treatment of agitation in patients with dementia of the Alzheimer's type?

AVP-923  NCT01584440
Phase 2 for the treatment of agitation in patients with dementia of the Alzheimer's type.
Dextromethorphan 20 mg / Quinidine 10 mg
Dextromethorphan 30 mg / Quinidine 10 mg

Exclusion Criteria:
  • Patient has other type of dementia (e.g., vascular dementia, frontotemporal dementia, Parkinson's disease, substance-induced dementia).
  • Patients with co-existent clinically significant or unstable systemic diseases that could confound the interpretation of the safety results of the study (e.g. malignancy, poorly controlled diabetes, poorly controlled hypertension, unstable pulmonary, renal or hepatic disease, unstable ischemic cardiac disease, dilated cardiomyopathy, certain cardiac conduction abnormalities including QTc prolongation, or unstable valvular heart disease).
  • Patients with myasthenia gravis.
AVP-786   NCT02442765NCT02442778
Phase 3 for the treatment of agitation in patients with dementia of the Alzheimer's type.
Dextromethorphan 28 mg / Quinidine 4.9 mg
Dextromethorphan 18 mg / Quinidine 4.9 mg

Exclusion Criteria:
  • Patient has dementia predominantly of non-Alzheimer's type (e.g., vascular dementia, frontotemporal dementia, Parkinson's disease, substance-induced dementia)
  • Patients with co-existent clinically significant or unstable systemic diseases that could confound the interpretation of the safety results of the study (e.g., malignancy, poorly controlled diabetes, poorly controlled hypertension, unstable pulmonary, renal or hepatic disease, unstable ischemic cardiac disease, dilated cardiomyopathy, or unstable valvular heart disease)
  • Patient with myasthenia gravis
The difference in the exclusions from the phase 2 AVP-923 trial and the deutered version using AVP-786 is that QTc prolongation, which was an exclusion in the phase 2 is not listed in the current phase 3 trials.  The reduction in the dose of quinidine to 4.9 mg from 10 mg could be the prime reason for the two new phase 3 trials not including QTc prolongation as an exclusion criteria.  I expect these two phase 3 trials to recruit at a much faster rate, than the AVP-923 trial achieved. I also expect that patients currently taking AVP-923 for other indications, will eventually switch to the deutered version AVP-786, which has a similar PK profile to AVP-923, favorable dosing, and has a longer patent that extends to 2028-2030. Thank you for reading.

Contact:   portfoliomgt1@gmail.com

Friday, May 22, 2015

What is a 505(b)(2) New Drug Application

A 505(b)(2) application differs from a 505(b)(1) new drug application, in that the process to approval could potentially be quicker with less expense.  The 505(b)(2) process takes drugs that have already been approved and makes small modifications to them. Typically a company will perform a phase 1 bridging study to compare the systemic levels of the proposed drug product and the reference product.  A bridging study also allows a company to reference the safety and efficacy information that is known for the original drug.
There are three advantages for a company that pursues a 505(b)(2) application.
1.  It is a relatively lower risk process because the original drug may have already been proven to be safe.
2.  The entire process is lower cost.
3.  The pathway to approval can be quicker because of the fewer studies required.
Companies such as Auspex, and Concert Pharmaceuticals, who attempt to improve the metabolic, safety, or efficacy profile with the use of deuteration, could benefit greatly by following the pathway of a 505(b)(2) submission application to get to market quicker, with less expense.  As those drugs get approved quicker, with less expense, the benefit should fall to the patient in need, and our medical healthcare system that provides insurance coverage.  Thank you for reading.

Contact: portfoliomgt1@gmail.com

Saturday, May 16, 2015

What is CTP-656

CTP-656 is the name of Concert Pharmaceuticals deutered enhanced version of Vertex's Ivacaftor, (commercially known as Kalydeco).  Ivacaftor was initially approved for Cystic Fibrosis patients with various mutations, and soon to be approved again as a combo with Lumacaftor known as Orkambi, for patients who have two copies of the F508del mutation 12 years and older. The hope is that CTP-656 enhances therapy for patients by improving the metabolic profile, reduce dosage to once daily, or ameliorate the drug interaction profile.
Concert ran pre-clinical studies on two candidates for their lead potentiator D9 and D18 in this PDF here Pre-Clinical D-Ivacaftor.  Then entered a first in human clinical trial to assess which deuterated analog (D9 or D18) would advance based on PK data here Phase 1 NCT02392702.  That phase 1 trial will continue to evaluate the chosen deuterated analog in single and multiple dosages against placebo (Kalydeco) on a pharmacokinetic basis with a readout end of 2015 or early 2016. Even though the deuterated potentiator candidate has been chosen from the first part of the phase 1 trial in May, the CEO plans on holding off on the data until a medical conference abstract is presented in the near future by the lead scientist. The only indication on how the data looks was expressed by CEO Tung as "very happy" with the data.  Thank you for reading.

Contact: portfoliomgt1@gmail.com 
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