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Pfizer Receives Breakthrough Therapy Designation from FDA for PF-06651600, an oral JAK3 Inhibitor, for the Treatment of Patients with Alopecia Areata

Phase II data accepted for late-breaker news session at 2018 EADV Congress

Wednesday, September 5, 2018 - 8:50am
EDT

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE:PFE) today announced its investigational oral Janus
kinase 3 (JAK3) inhibitor PF-06651600 received Breakthrough
Therapy designation from the U.S. Food and Drug Administration (FDA) for
the treatment of patients with alopecia areata, a chronic autoimmune
skin disease that causes hair loss on the scalp, face, or body.1,2

The Breakthrough Therapy designation for alopecia areata was supported
by positive results from a Phase 2 study, which will be presented during
the late-breaking news session at the 27th European Academy
of Dermatology and Venerology (EADV) Congress in Paris on September 15,
2018. Currently, there are no FDA-approved treatments for alopecia
areata, which impacts millions of people worldwide and is often
associated with profound psychological consequences.1,2

“We are encouraged by this Breakthrough Therapy designation as it
underscores the potential of our JAK3 inhibitor to address a critical
unmet need,” said Michael Corbo, Chief Development Officer, Inflammation
& Immunology, Pfizer Global Product Development. “We are continuing to
work closely with the FDA on the development process with the goal of
bringing this potential new treatment to patients living with alopecia
areata as soon as possible.”

Breakthrough Therapy designation was initiated as part of the Food and
Drug Administration Safety and Innovation Act (FDASIA) signed in
2012. As defined by the FDA, a Breakthrough Therapy is a drug intended
to be used alone or in combination with one or more other drugs to treat
a serious or life-threatening disease or condition and preliminary
clinical evidence indicates that the drug may demonstrate substantial
improvement over existing therapies on one or more clinically
significant endpoints, such as substantial treatment effects observed
early in clinical development. If a drug is designated as a Breakthrough
Therapy, the FDA will expedite the development and review of such drug.3

Pfizer is also working with the European Medicines Agency (EMA) on the
clinical development program for PF-06651600.

About Alopecia Areata

Alopecia areata is an autoimmune disease, characterized by hair loss,
often patchy, on the scalp, face, or body.1,2 People
suffering from alopecia areata experience symptoms when immune cells
attack healthy hair follicles, causing the hair to fall out, often
starting with smooth, round patches.1,2 The mean age of onset
is between 25 and 35, but it can also impact children and adolescents,
and is seen in both sexes and all ethnicities.1,2 More than
half of patients with alopecia areata experience poor health-related
quality of life and, as a result, the condition may lead to serious
psychological consequences, including high levels of depression and
anxiety.1

About PF-06651600 and Pfizer’s Kinase Inhibitor Leadership

The JAK pathways are believed to play an important role in inflammatory
processes as they are involved in signaling for over 50 cytokines and
growth factors, many of which drive immune-mediated conditions.4
JAK inhibition offers the potential for new advanced treatment options
for these conditions through unique and targeted selectivity.5

“Pfizer strives to continue moving the JAK science forward with
development of multiple JAKis with unique selectivity profiles that are
being evaluated in clinical trials. These inhibitors have the potential
of meeting significant unmet needs in multiple inflammatory conditions,”
said Michael Vincent, Chief Scientific Officer, Pfizer Inflammation &
Immunology.

PF-06651600 is an oral small molecule that selectively inhibits Janus
kinase (JAK) 3.5 PF-06651600 is also under investigation for
the treatment of rheumatoid arthritis, Crohn’s disease and ulcerative
colitis.

Pfizer has established a leading kinase research capability with
multiple unique kinase inhibitor therapies in development. As a pioneer
in JAK science, the Company is continuing to advance several
investigational programs for molecules with novel selectivity profiles,
which, if approved, could potentially deliver transformative therapies
for patients. In addition to PF-06651600, Pfizer has the following
kinase inhibitors in trials across multiple indications:

  • PF-04965842: A selective JAK1 inhibitor in Phase 3 clinical trials for
    the treatment of atopic dermatitis(AD)6; PF-04965842
    received Breakthrough Therapy designation from the FDA for the
    treatment of patients with moderate-to-severe AD
  • PF-06700841: A tyrosine kinase 2(TYK2)/JAK1 inhibitor under
    investigation for the treatment of psoriasis, Crohn’s disease,
    ulcerative colitis and alopecia areata
  • PF-06650833: An interleukin-1 receptor associated kinase 4 (IRAK4)
    inhibitor under investigation for the treatment of rheumatoid arthritis
  • PF-06826647: A TYK2 inhibitor under investigation for the treatment of
    psoriasis and inflammatory bowel disease (IBD)

Working together for a healthier world

®

At Pfizer, we apply science and our global resources to bring therapies
to people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of health care products. Our global
portfolio includes medicines and vaccines as well as many of the world's
best-known consumer health care products. Every day, Pfizer colleagues
work across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared diseases
of our time. Consistent with our responsibility as one of the world's
premier innovative biopharmaceutical companies, we collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, we have worked to make a difference for all who
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DISCLOSURE NOTICE: The information contained in this release
is as of September 5, 2018. Pfizer assumes no obligation to update
forward-looking statements contained in this release as the result of
new information or future events or developments.

This release contains forward-looking information about PF-06651600
and Pfizer’s ongoing investigational programs in kinase inhibitor
therapies, including their potential benefits, that involves substantial
risks and uncertainties that could cause actual results to differ
materially from those expressed or implied by such statements. Risks and
uncertainties include, among other things, the uncertainties inherent in
research and development, including the ability to meet anticipated
clinical trial commencement and completion dates and regulatory
submission dates, as well as the possibility of unfavorable clinical
trial results, including unfavorable new clinical data and additional
analyses of existing data; risks associated with preliminary data; the
risk that clinical trial data are subject to differing interpretations,
and, even when we view data as sufficient to support the safety and/or
effectiveness of a product candidate, regulatory authorities may not
share our views and may require additional data or may deny approval
altogether; whether regulatory authorities will be satisfied with the
design of and results from our clinical studies; whether and when drug
applications may be filed in any jurisdictions for any potential
indication for PF-06651600 or any other investigational kinase
inhibitor therapies; whether and when any such applications may be
approved by regulatory authorities, which will depend on the assessment
by such regulatory authorities of the benefit-risk profile suggested by
the totality of the efficacy and safety information submitted, and, if
approved, whether PF-06651600 or any such other investigational
kinase inhibitor therapies will be commercially successful; decisions by
regulatory authorities regarding labeling, safety and other matters that
could affect the availability or commercial potential of PF-06651600 or
any other investigational kinase inhibitor therapies; and competitive
developments.

A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended December
31, 2017 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors” and “Forward-Looking
Information and Factors That May Affect Future Results”, as well as in
its subsequent reports on Form 8-K, all of which are filed with the U.S.
Securities and Exchange Commission and available at 


www.sec.gov

 and 

www.pfizer.com

.

1 Villasante Fricke AC, Miteva M. Epidemiology and burden of
alopecia areata: a systematic review. Clinical, Cosmetic and
Investigational Dermatology. 2015;8:397-403. doi:10.2147/CCID.S53985.
2
Pratt CH, King LE, Messenger AG, Christiano AM, Sundberg JP. Alopecia
areata. Nature reviews Disease primers. 2017;3:17011.
doi:10.1038/nrdp.2017.11.
3 Food and Drug Administration
Fact Sheet Breakthrough Therapies at https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendmentstotheFDCAct/FDASIA/ucm329491.htm
accessed on January 25, 2018
4 Banerjee, S., Biehl, A.,
Gadina, M. et al. JAK–STAT Signaling as a Target for Inflammatory and
Autoimmune Diseases: Current and Future Prospects. Drugs. 2017;77:
521. https://doi.org/10.1007/s40265-017

5
Telliez JB, Dowty ME, Wang L, Jussif J, Lin T, Li L, et al. Discovery of
a JAK3-selective inhibitor: functional differentiation of JAK3-selective
inhibition over pan-JAK or JAK1-selective inhibition. ACS Chem Biol.
2016;11(12):3442–51. doi:10.1021/acschembio.6b00677.
6 J
Med Chem. 2018 Feb 8;61(3):1130-1152. doi: 10.1021/acs.jmedchem.7b01598.
Epub 2018 Jan 23.



Contact: 

For Pfizer Inc.
Media:
Neha Wadhwa, 212-733-2835
[email protected]
or
Investors:
Bryan Dunn, 212-733-8917
[email protected]