Patient-Focused Drug Development Initiative

The Food and Drug Administration (FDA) announced that Alopecia Areata (AA) was one of eight diseases selected to participate in the Patient-Focused Drug Development Initiative (PFDDI) 2016-2017
 Meetings Planned for FY 2016 – 2017

• Psoriasis: March 17, 2016
• Neuropathic pain associated with peripheral neuropathy: June 10, 2016
• Patients who have received an organ transplant: September 27, 2016
• Alopecia areata
• Autism
• Hereditary angioedema
• Neuropathic pain associated with peripheral neuropathy
• Sarcopenia

The FDA will publicly meet with patients who suffer from debilitating conditions without adequate treatment options in the hopes of better understanding their wants and needs in the drug development process.  The meeting will take place in 2016-2017, specific date has not been determined yet.  This can only have a positive impact for disease victims of Alopecia Areata, as the FDA will get a first hand patient perspective of that disease.
CTP-543 is currently in a phase 1 clinical trial for AA, and will commence an efficacy phase 2 trial, first half of 2017.  Concert management has taken the initiative to bring CTP-543 into clinical trials for this disease, which has a very high probability of being the first FDA approved drug for AA.  Next, we will examine the potential market for this indication.  Thank you for reading.

D-Ivacaftor US Patent 9,181,192 B2

This is a new d-ivacaftor patent listed on Concerts website.  This looks like an add on to the original US patent regarding d-ivacaftor for Cystic Fibrosis, except that it has a section that relates to COPD (chronic obstruction pulmonary disease).  The added section of the patent is below.

15. A method of treating COPD in a subject comprising administering to the subject a compound of Formula I or a pharmaceutical composition comprising a compound of Formula I and a pharmaceutically acceptable carrier, wherein the compound of Formula I has the following structural formula:

Concert is currently assessing the possibility of entering clinical trials for COPD, which would be an extremely large potential market to pursue.  The patent runs to 2032.  Thank you for reading.
 

CTP-543 Phase 1 Initiated

Concert Pharmaceuticals has begun dosing in a phase 1 clinical trial with 80 healthy volunteers to assess safety and pharmacokinetics (PK).  From the company press release below.
“Alopecia Areata is a disease that can have devastating effects on patients but which currently lacks approved and effective therapies. We are pleased to broaden Concert’s pipeline of clinical drug candidates with the advancement of CTP-543 into clinical testing. CTP-543 is another example of how we have applied our novel deuterium platform to create medicines that represent new treatment options,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “Recent advances in our understanding of alopecia areata biology, combined with patient data from several investigator-initiated studies, give us confidence that CTP-543 has potential to be an effective treatment. We hope to move this program quickly through Phase 1 evaluation in order to initiate efficacy studies in patients with alopecia areata next year.”

Aclaris Therapeutics acquired the rights, Aclaris Pipeline of a JAK inhibitor, and this is the patent, WO2013149194A that Columbia University through Vixen Pharmaceuticals, sold the rights to Aclaris Therapeutics.

CTP-543 Patent: 2032
Ruxolitinib Patent: 2028

Thank you for reading. 
 

Nuplazid Priced at $23,400 / Year

Based on the Acadia Pharmaceuticals first quarter conference call, pricing for the recently approved Nuplazid for Parkinson's Disease Psychosis (PDP), will be around $23,400 per year, wholesale acquisition cost (WAC), much higher than we anticipated.  We previously relied on company information of around $13,000 per year for Nuplazid. The adjustment higher makes a large valuation difference, and puts the recent price of ($27.50) rather cheap based on a potential buyout scenario. 

1,000,000 With Parkinson's in US.
30% develop PDP or Parkinson's Disease Psychosis at some point.
300,000 total patients with PDP.
50% penetration rate of those with the psychosis.
150,000 the actual number treated for PDP with Nuplazid out of 1 million Parkinson's total victims.
$65.00 = WAC price per tablet per day of treatment.  Equates to around $23,400 per year. 
$32.50 = Sales to Acadia per 34 mg tablet after discounts various distributors.
$11,700 = Per patient ACAD annual revenue, ($32.50 per day * 30 days * 12 months)

$1.755 billion = Total peak revenue to ACAD, (150,000 patients * $11,700 annual revenue to ACAD) U.S. Parkinson's Disease Psychosis only.  If applying a buyout scenario of four times peak revenue = $1.755 x 4 = $7.020b valuation / 130k (shares outstanding) = $ 54.00 share price.  It would take 85,470 (PDP) patients treated with Nuplazid to achieve $1 billion in revenue.  Acadia did pre-marketing, and estimated around 11,000 Parkinson's Disease doctors in the U.S.  Each doctor would have to treat 7.77 (PDP) patients with Nuplazid to achieve the $1b in revenue.

Thank you for reading.
 

Nuplazid Gets FDA Approval

Acadia Pharmaceuticals Nuplazid, was FDA approved on Friday for the indication of Parkinson's Disease Psychosis, and will carry a warning as seen below.
 

IMPORTANT SAFETY INFORMATION AND INDICATION

INCREASED MORTALITY IN ELDERLY PATIENTS WITH DEMENTIA-RELATED PSYCHOSIS
Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. NUPLAZID is not approved for the treatment of patients with dementia-related psychosis unrelated to the hallucinations and delusions associated with Parkinson’s disease psychosis.

QT Interval Prolongation: NUPLAZID prolongs the QT interval. The use of NUPLAZID should be avoided in patients with known QT prolongation or in combination with other drugs known to prolong QT interval including Class 1A antiarrhythmics or Class 3 antiarrhythmics, certain antipsychotic medications, and certain antibiotics. NUPLAZID should also be avoided in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes and/or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and presence of congenital prolongation of the QT interval. 

 
Bottom Line:  Now it's on to commercialization.  Thank you for reading.