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Concert Pharmaceuticals building a strong portfolio of autoimmune and CNS indications

29 Jan Concert Pharmaceuticals building a strong portfolio of autoimmune and CNS indications

Concert Pharmaceuticals (NASDAQ: CNCE), a clinical stage biopharmaceutical Company, announced the receipt of a Notice of Allowance from the U.S. Patent and Trademark Office for patent application No. 16/098,338 related to CTP-543, its lead candidate for the treatment of alopecia areata. The patent covers pharmaceutical compositions of and methods of treating alopecia areata with CTP-543 and will expire in 2037. Furthermore the Company intends to meet with the FDA as it is nearing the end of its phase 2 clinical trials and is planning to initiate a Phase 3 evaluation of CTP-543 in 2020.

CTP-543 is an oral selective inhibitor of Janus kinases JAK1 and JAK2, which has been granted a Fast Track designation by the FDA for the treatment of alopecia areata, an autoimmune disease that causes partial or complete loss of hair. The Company is developing a deuterium-modified form of the JAK 1/2 inhibitor ruxolitinib, that is capable of inhibiting JAKs and has the potential to treat certain immune-mediated diseases, making it one of the first Companies to leverage science about JAK inhibitors to address the dysregulated autoimmune function.

Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals, stated

“Concert is pursuing multiple avenues for securing its patent position for CTP-543. Importantly, this patent covers the clinical doses of CTP-543 that produced the robust results observed in our Phase 2 dose-ranging trial for the treatment of alopecia areata and upon issuance will be Orange Book eligible. As we prepare to advance CTP-543 into pivotal testing in 2020, we look forward to having this new patent as an important element of our IP estate.”

The Company is currently engaged in a Phase 2 Clinical Trial studying the safety efficacy and tolerability of CTP-543 in a wide range of doses in different cohorts of patients with alopecia areata. Alopecia Areata is one of the eight new disease areas that forms a part of FDA’s Patient-Focused Drug Development Initiative (PFDDI) formulated in 2016-2017. The program aims to bring patient perspectives into an earlier stage of product development, through its “voice of the patient” report.

Concert Pharmaceuticals (NASDAQ: CNCE)

Market Cap: $259.75M; Current Share Price: 10.90 USDChart
Data by YCharts

Alopecia Areata is an autoimmune / polygenic skin disease that causes loss of hair on the scalp and face and is estimated to affect over 6.8 million people in the U.S alone. There is currently no cure available for this disorder, with dermatologists using Corticosteroids, minoxidil 5%, Anthralin and Diphencyprone (DPCP) as treatment options. However these cause discomfort and side effects leading to low patient adherence. Alopecia areata is one of eight new disease areas selected by the FDA in 2015 that it will focus on, under its Patient-Focused Drug Development Initiative (PFDDI).

This disorder can cause a severe emotional impact on the patient as it has social implications and can lead to isolation and stigma, it may even adversely affect the ability to form and sustain relationships. The alopecia industry has faced challenges in achieving proof of concepts (PoC), as patient responses to therapy are highly individualized with some of them experiencing spontaneous remission. However recent scientific advancements have shown that new research such as T-cell mediated mechanisms can achieve faster PoC’s.

A report by Grand View Research, estimates that the global alopecia treatment market will reach USD 11.8 Billion by 2024. The rise in disposable incomes, increasing health awareness being made possible by organizations such as National Alopecia Areata Foundation (NAAF) and the American Hair Loss Association, and the rise in research and development efforts will propel the growth in the market. Many branded products are heading towards patent expiry that could attract an onslaught of generics and act as a hindrance for further innovation.

Tropical applications are the most preferred form of treatment because of their ease of use and non-invasive nature; however the introduction of novel treatment options such as Janus kinase inhibitors (JAK) that can stimulate follicle growth with minimal side effects have the potential to change the therapeutic market. The cost of treatment is still deemed prohibitive and can hamper the growth rate of the industry.

Concert Pharmaceuticals is a pioneer, leveraging its proprietary DCE Platform® (deuterated chemical entity), to improve the pharmacokinetic or metabolic properties of approved drugs in the field of autoimmune and central nervous system disorders. The company intends to capitalize on its ability to create deuterated versions of known drugs thereby achieving faster clinical proof-of-concept and clinical success on one hand and enhanced oral bioavailability, improved metabolic profile and longer half-life’s of the compounds on the other.

Image Source: Company

Deuterium is non-radioactive and safe and can be isolated from sea water. It is naturally occurring in abundance, even in the human body, and hence used in human and metabolic studies. It possesses double the atomic mass of hydrogen which enables it to form potent bonds with carbon and makes it an ideal candidate for drugs that are metabolized by pathways characterized by scission of carbon-hydrogen bonds.

It is developing CTP-543 an oral selective inhibitor of certain Janus kinases, known as JAK1 and JAK2 for treatment of Alopecia, by modifying ruxolitinib being marketed in the U.S under the brand name Jakafi® for the treatment of myelofibrosis and polycythemia vera which is currently undergoing Phase 2a clinical trials. In addition it is also working on CTP-692, a novel drug for adjunctive treatment of schizophrenia.

Image Source: Company

The company’s novel DCE Platform® is protected by 114 issued US patents or patent applications that claim deuterated analogs of more than 90 non-deuterated drugs and drug candidates. The company has strategic collaborations with leading biopharmaceutical companies such as Celegene for developing CTP-730, a product candidate containing deuterated apremilast for the treatment of inflammatory diseases, Jazz Pharmaceuticals (NASDAQ: JAZZ) for the development of a once nightly sodium oxybate product and Avanir pharmaceuticals for developing AVP-786 for the treatment of agitation associated with Alzheimer’s disease.

The company also has an Asset purchase agreement with Vertex (NASDAQ: VRTX) for CTP-656, an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator.

Risk Assessment

  • The Company enjoys a strong financial position, with cash and cash equivalents totaling $121.5 M as of September 2019.
  • Its proprietary technology platform has received strong validation from the market, with VX-561 (CTP-656), being picked up for sale in consideration for $160 M in the form of upfront payment with further eligibility of an estimated $90 M in pre-commercial milestones.
  • The Company is planning to initiate an end of Phase 2 meeting with the FDA to discuss CTP-543, and complete the enrollment for a phase 2 trial CTP-692, intended for the treatment of schizophrenia, along with announcing the topline results from the phase 2 study in 2020.
  • Concert Pharmaceuticals is actively seeking out-licensing opportunities for its non-core development, which not only provide additive value but also offer future financial potential.
  • Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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