22 Mar Is a cure for Alopecia Areata on the horizon? New research shows promise
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.
Grand View Research Inc. 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.
We take a look at some companies which are striving to address the needs of the patients in this field:
Concert Pharmaceuticals (NASDAQ: CNCE)
Market Cap: $378.35M; Current Share Price: 16.08 USD
The company is a pioneer that uses 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 leverage 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.
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.
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 (NASDAQ: CELG) 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, acquired by Otsuka Pharmaceuticals Co. Ltd., for developing AVP-786 for the treatment of agitation associated with Alzheimer’s disease.
The company also has an asset purchase agreement with Vertex for CTP-656, an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator.
As per the company’s 2018 financial results, its cash and cash equivalents stood at $153.3 million, while revenues were at $10.5 million as of 31 December 2018.
Aclaris Therapeutics (NASDAQ: ACRS)
Market Cap: $244.09M; Current Share Price: 5.98 USD
Aclaris is a biotechnology company with focus on creating solutions for areas of dermatology that is lacking in FDA-approved therapies especially in skin and hair related disorders. The company’s pipeline consists of drug candidates for treatment of Alopcia Areta, Vitiligo, Common warts, Atopic Deramatitis, Rheumatoid Arthritis, Psoriasis, etc. that seek to unlock the potential of Kinome, a subset of human genome, to control dysregulation and/or mutations of cells.
Its proprietary KINect™ platform can identify drug candidates with the help of its chemical library of kinase inhibitors, a structure based drug design and a custom kinase assay. The companies lead drug candidate for Vitiligo is ATI-502, a JAK1/JAK3 Inhibitor and ATI-1777, a JAK1/JAK3 Inhibitor that are currently in Phase II clinical trial and IND enabling studies respectively. Its lead product candidate for treatment of Alopecia Areata is ATI-502 a topical JAK1/JAK3 inhibitor for which the company filed an Investigational New Drug application with the FDA in 2017. It is currently engaged in Phase II clinical trials.
It presently markets two products, eskata (hydrogen peroxide) topical solution, 40% and Rhofade (oxymetazoline HCI) cream, 1% that it acquired from Allergan Sales, LLC.
The company is currently seeking partnerships for in-licensing commercial-stage or developmental-stage aesthetic dermatology treatments and out-licensing rights for its investigational protein kinase inhibitors for non-dermatology indications along with out-licensing commercial rights outside of the United States for seborrheic keratoses and common warts. It also wants to collaborate with research partners for its KINect™ platform for drug discovery for treatment of autoimmune diseases and for new applications of its cysteinome-targeted chemical library representing 60% of the kinome.
It’s fully owned subsidiary, Confluence Discovery Technologies, works as a contract research organization for drug discovery and early development research partner for various pharmaceutical and biotech companies in areas such as biochemistry, enzymology, biochemistry, immunology to name a few, using tools like computational, bioanalytical and medicinal chemistry.
Aclaris enjoys a strong intellectual property rights portfolio consisting of over 150 patents issued or pending in U.S and Worldwide.
As per the company’s Q3, 2018 financial results, cash and cash equivalents balance stood at $134 million. In addition, it made a public offering of 9,941,750 shares of its common stock at a price to the public of $10.75 per share for total gross proceeds of approximately $106.9 million.
Incyte (NASDAQ: INCY)
Market Cap: $17.65B; Current Share Price: 82.45 USD
The company aims to be a global leader in the field of immunotherapy and manufactures Jakafi® for the treatment of polycythemia vera and myelofibrosis. Incyte’s growth strategy is based on its discovery capabilities of both small and large molecule modalities to target diverse mechanisms, including: JAK, PI3Kδ, PD-1, IDO1, OX40, and GITR.
The company has entered into license, development and commercialization agreement with Eli Lily and Company (NYSE: LLY) for its baricitinib, a JAK inhibitor and other compounds for treatment of inflammatory and autoimmune diseases. It is currently conducting Phase II/III trials for severe alopecia areata.
It also has a collaboration and licensing agreement with Novartis for its JAK inhibitor ruxolitinib and certain compounds for hematologic and oncology indications. It has also granted Novartis (NYSE: NVS) worldwide exclusive development and commercialization rights for its c-MET inhibitor compound capmatinib. Novartis has the rights for research, development and commercialization of ruxolitinib for GVHD outside the United States. In addition, the company has an agreement with Agenus Inc for developing immuno-therapeutics using Agenus Inc.’s proprietary Retrocyte Display antibody discovery platform.
Merus N.V. (NASDAQ: MRUS) (bispecific antibodies utilizing Merus’ proprietary Biclonics® technology platform), Calithera Biosciences, Inc. (NASDAQ: CALA) (CB-1158 small-molecule arginase inhibitor, in hematology and oncology) and MacroGenics, Inc. (NASDAQ: MGNX) (MGA012, an investigational monoclonal antibody that inhibits (PD-1) are some of its other collaborations.
It has an extensive portfolio of drugs consisting of 17 molecular targets and 19 clinical candidates under various stages of development for treatment of numerous disorders such as acute and chronic graft vs. host disease, thrombocythemia, Non-small cell lung cancer (with osimertinib), cholangiocarcinoma solid tumors, vitiligo, atopic dermatitis, Sjögren’s syndrome to name a few.
As per the company’s year-end financial results for 2018, total product-related revenues were at $1.7 billion, with Jakafi contributing an estimated $1.4 billion of the total revenue.
Switch Biotech LLC
Its proprietary technology, SWITCHBIOTECH DATA (Drug Analysis Target Analysis), enables it to rapidly match drug candidates to disease targets with a focus on existing compounds for whom patents for dermatological applications can be filed under second medical use. Switch is presently focused on solutions for Psoriasis, Acne Vulgaris, Actinic Keratoses, Atopic Dermatitis, Cutaneous Ulcers and Onychomycosis (nail fungus). Its in-depth understanding of the molecular biology of the skin enables it to revolutionize traditional pharmaceutical compounds and biotechnology treatment approaches.
Its unique drug identification and development process makes use of its in-house database catalogs of 370,000 relevant substances and employs its proprietary data mining to match biological and compound data, a re-profiling approach that cuts down the drug discovery process to only a fraction of time taken by traditional approaches. This allows the company to expedite the process of acquiring a proof of concept (PoC) in a cost-effective manner and caters to the needs of mid-sized pharmaceutical companies seeking clinically validated drugs. Its re-profiling strategy can also be used for filing second medical use patents while preventing the off-label use of generic compounds.
The company’s pipeline consists of drug candidates for treatment of psoriasis, atopic Dermatitis, alopecia and fibromyalgia. The company has been able to narrow down the genetic pathway called the androgen metabolism, whose dysfunction leads to androgenic alopecia. It aims to prolong the hair cycle, stop the miniaturization of the hair follicle, or block the signals from the dermal papilla that inhibit hair growth and for this purpose, it has gathered all the relevant components and technologies required for a successful androgenic alopecia research program.
Biologics MD, Inc.
A preclinical therapeutic development company, BiologicsMD™, is focused on offering highly targeted treatment of hair loss diseases and severe bone disorders by using a series of recombinant fusion proteins with potent stimulatory effects. Its lead compound BMD-1141 is a first-in class active domain of parathyroid hormone that is linked with a collagen binding domain. It is conducting preclinical studies for restoration of hair growth in alopecia areata, androgenetic alopecia, and chemotherapy-induced alopecia.
Biologics uses its indepth knowledge and expertise of hair cycles and growth phases to work on a pipeline of targeted therapeutics that offer Hair Cycle Stimulation, a novel approach to restoration of growth. Its lead candidate for alopecia areata BMD-1141 has shown to stimulate beta-catenin in hair follicles through activation of the PTH/PTHrP receptor thereby stimulating growth and preventing hair loss.
Its pipeline consists of drugs for treatment of bone disorders such as spinal fusion procedures in lieu of autologous bone grafts and pharmacokinetics like BMD-3151, a fusion of a parathyroid antagonist for the prevention and treatment of breast cancer metastasis.
The company holds patents for method of use for promoting bone growth using fusion protein of collagen binding-domain and parathyroid hormone in U.S as well as in Japan. It also holds a patent for “BMD-2341”, a protein that can promote hair growth or prevent hair loss.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.