Home Japanese values PharmaEssentia Announces Top Results from Phase I Clinical Trial of KX01 for Actinic Keratosis (AK) in Japan on Behalf of Subsidiary PharmaEssentia Japan KK

PharmaEssentia Announces Top Results from Phase I Clinical Trial of KX01 for Actinic Keratosis (AK) in Japan on Behalf of Subsidiary PharmaEssentia Japan KK

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Statement

1.Date of occurrence of the event:2022/04/15
2.New drug name or code:KX01 Ointment 1%
3.Indication:Actinic Keratosis (AK)
4.Planned development stages:
To complete the phase III clinical trial, and later to apply for the
marketing authorization of KX01 for the treatment of adults with AK
in Japan.
5.Current development stage:
(1)Application submission/approval/disapproval/each of clinical trials
   (include interim analysis):
   Announcement of the top-line results of the phase I clinical trial of
   KX01 for the treatment of adults with AK in Japan.
   A.Study design
     a.Study title:
       Skin Irritation and Phototoxicity Study of KX01 Ointment 1% in
       Japanese healthy male subjects
       (Study Protocol Number: B21-101; ClinicalTrials.gov Identifier:
       NCT05245578)
     b.Objectives:
       To assess skin irritation and phototoxicity following a single
       application of KX01 Ointment 1% and placebo by simple-patch test
       and under open conditions in Japanese healthy male subjects.
     c.Phase of development:Phase I clinical trial
     d.Name of investigational drug:KX01 Ointment 1%
     e.Indication:Actinic Keratosis
     f.Endpoints:
       The primary objective is to assess skin irritation following a
       single application of KX01 Ointment 1% and placebo by simple-patch
       test and under open conditions in Japanese healthy male subjects.
       In addition, the safety of KX01 Ointment 1% will be assessed by the
       evaluation of any adverse events (AE) reported during the study.
     g.Number of subjects:20 Japanese healthy male subjects.
   B.The statistical results (including but not limited to P value) and
     statistical significance (including but not limited to whether
     statistical significance is achieved) of the primary and secondary
     endpoints. If the Company cannot disclose statistical data due to
     other important reasons, the reasons should be stated.
     The primary endpoint: Of the 20 subjects on the investigational
     product, 13 had 41 treatment-related AE during the trial. Pruritus
     was the most common AE (total 37 events in 13 subjects), and no
     phototoxicity-related AE occurred. All AE were mild, no moderate or
     severe AE, and no Serious Adverse Events occurred.
     The data from this study showed a favorable safety profile for KX01
     ointment 1%. The safety results support the continuation of the phase
     III clinical trial of KX01 to treat Actinic Keratosis in Japan.
   C.If statistical results of the phase III clinical trial of the new drug
     is disclosed, the marketing plan of the Company:N/A
   D.The results of a single clinical trial (including the statistical
     P-values of t the primary and secondary endpoints and whether they
     are statistically significant) are not sufficient to fully reflect
     the success or failure of the launch of the new drug. Investors are
     advised to exercise caution and conduct thorough evaluation.
(2)Risks and mitigation measures to be taken by the company upon
   disapproval of the competent authority:N/A
(3)Strategies to be taken by the company upon approval by the competent
   authority:
   To complete the phase III clinical trial, and later to apply for the
   marketing authorization of KX01 for the treatment of adults with AK
   in Japan.
(4)Accumulated investment expenditure incurred:
   In consideration of the future marketing strategy and to protect the
   rights of the company and investors, no public disclosure will be made
   for the time being.
6.Upcoming development plan:To complete the phase III clinical trial in Japan.
(1)Estimated date of completion:
   The phase III clinical trial is expected to be completed by the third
   quarter of 2023. The timeline will be adjusted according to the actual
   progress of the clinical trial.
(2)Estimated responsibilities: None.
7.Market situation:
Actinic Keratosis (AK) is a chronic and precancerous skin disease that
occurs primarily in areas that have been exposed to ultraviolet radiation
for a long period of time. It is usually found on the face, ears, lips,
bald scalp, forearms, the posterior part of the hands, and lower legs.
The mortality rate of AK is not high; however, it is most likely to
progress to malignant and skin cancer in the long term if left untreated.
According to Taipei Chang Gung Hospital's dermatology column, the medical
statistics show that 1 in 10 AK would progress to skin cancer. Since it is
not possible to predict which AK lesions will develop into squamous cell
carcinoma, all lesions should be treated by dermatologists.
The prevalence of AK increases with age and is more common in people with
lighter skin. In Japan, the prevalence rate of AK is about 414 per 100,000
people; in Okinawa, it is about 1,238 per 100,000 people. AK is treated
with topical creams, photodynamic therapy, cryotherapy, CO2 laser and
surgical excision.
8.Any other matters that need to be specified:
(1)The Company is granted exclusive rights of KX01 for treating AK in
   Taiwan, Japan, and Korea from Athenex, Inc.
(2)KX01 is approved for the topical treatment of AK of the face and scalp
   in adults and launched in the U.S., EU, and the U.K.
(3)The Company has completed the submission of the New Drug Application
   for KX01 treating AK to TFDA on November 22, 2021. Since the U.S. FDA
   and EMA have approved KX01 for AK, the Company's application for
   Abbreviated Review of NDA for KX01 was approved by TFDA, and the review
   time was shortened from the standard 360 days to 180 days.
9.New drug development requires long process, vast investments and with
no guarantee in success which may pose investment risks.The investors
are advised to exercise caution and conduct thorough evaluation.: