Athenex Announces Klisyri® (tirbanibulin) Receives Positive CHMP Opinion from the European Medicines Agency (EMA) for the Treatment of Actinic Keratosis of the Face or Scalp

Rhea-AI Summary

Athenex, Inc. (NASDAQ: ATNX) announced a positive opinion from the European Medicines Agency's CHMP for its partner Almirall regarding Klisyri® (tirbanibulin) for treating actinic keratosis (AK) of the face or scalp. This first-in-class microtubule inhibitor has shown substantial efficacy and safety in Phase III trials. Athenex stands to gain up to $45 million in milestone payments and tiered royalties starting at 15%. The FDA approved Klisyri® in the U.S. in December 2020, positioning it as a key player in a market where AK affects roughly 18% of the European population.

Positive

• Positive CHMP opinion for Klisyri® enhances regulatory approval prospects.
• Athenex can receive up to $45 million in milestone payments and royalties starting at 15%.

Negative

• None.

European Commission Decision Anticipated by July 2021

BUFFALO, N.Y., May 21, 2021 (GLOBE NEWSWIRE) -- Athenex, Inc., (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, announced today that its partner, Almirall (Almirall, S.A., BME: ALM), has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the regulatory approval of Klisyri® (tirbanibulin), indicated for the topical treatment of actinic keratosis (AK) of the face or scalp.

Tirbanibulin is a novel, topical first-in-class microtubule inhibitor with a selective antiproliferative mechanism of action that represents a significant step forward in the treatment of AK due to its short treatment protocol (once daily application for 5 days), proven efficacy, and safety profile.

“We are delighted with the CHMP’s positive opinion on Klisyri® as the decision recognizes the demonstrated benefits of Klisyri®,” said Dr. Johnson Lau, Chief Executive Officer of Athenex. “The positive CHMP opinion is Athenex’s first, and further demonstrates Athenex’s ability to advance a product from drug discovery, through clinical development, and ultimately obtain regulatory approval.”

Our partner Almirall led the U.S. launch of Klisyri® in February and is supporting the launch of Klisyri® in Europe. Under the terms of the license agreement with Almirall, Athenex is eligible to receive up to $45 million in milestone payments associated with launch and expansion into additional indications. The Company is also eligible to receive additional sales-related milestone payments. The terms of the agreement included tiered royalties payable to Athenex starting at 15%, based on annual net sales.

AK is one of the most common diagnoses in dermatology practices and available data suggests that its estimated prevalence is roughly 18% of the population in Europe [1,2]. Treatment is a critical aspect in the management of the disease as it may progress to invasive skin squamous cell carcinoma.

In December 2020, Athenex received approval from the U.S. Food and Drug Administration (FDA) for the commercialization of Klisyri^® (tirbanibulin) in the United States for the topical treatment of actinic AK of the face or scalp.

The two Phase III trials evaluated the efficacy and safety of tirbanibulin ointment 1% in adults with actinic keratosis on the face or scalp and included 702 patients across 62 sites in the United States. Enrollment of patients was controlled to achieve a 2:1 ratio of facial: scalp treatment areas. Patients were randomly assigned in a 1:1 ratio to receive tirbanibulin ointment or vehicle ointment, which was self-administered to a contiguous treatment area of 25cm² on the face or scalp containing 4-8 typical AK lesions once daily for five consecutive days.

Both Phase III trials, KX01-AK-003 and KX01-AK-004, met the primary endpoint, which was defined as 100% clearance of AK lesions at Day 57 within the face or scalp treatment areas, each trial achieving a highly statistically significant result (p<0.0001). In KX01-AK-003, complete clearance was observed in 44% of the patients treated with tirbanibulin versus 5% for those treated with vehicle, and in KX01-AK-004, complete clearance was observed in 54% of the patients treated with tirbanibulin versus 13% for vehicle. Furthermore, tirbanibulin also achieved the secondary endpoint of partial (≥75%) clearance of lesions in each trial (68% of patients receiving tirbanibulin versus 16% receiving vehicle in KX01-AK-003, and 76% versus 20% respectively in KX01-AK-004). Both results were again highly statistically significant (p<0.0001).

In both clinical trials, patient-reported adverse events were mostly transient mild application-site pruritus and application-site pain. No patients experienced a serious adverse event or discontinuation due to tirbanibulin or vehicle in either clinical trial. Signs of local skin reactions assessed by investigators were mostly mild to moderate erythema, flaking or scaling that peaked at day 8 (maximum mean composite local skin reaction score ≤4.3 out of 18 across both trials) and resolved spontaneously in about 2 weeks.

About Klisyri® (tirbanibulin)

Klisyri® (tirbanibulin) is a microtubule inhibitor indicated for the topical treatment of actinic keratosis of the face or scalp. Two phase III studies (KX01-AK-003 and KX01-AK-004) evaluated the efficacy and safety of tirbanibulin ointment 1% (10 mg/g) in adults with actinic keratosis on the face or scalp. The studies achieved their primary endpoint, which was defined as 100% clearance of the AK lesions at Day 57 within the face or scalp treatment areas, each study achieving statistical significance (p<0.0001) on this endpoint [3].

About Actinic Keratosis

Actinic keratosis (AK) or solar keratosis is a chronic and precancerous skin disease that occurs primarily in areas that have been exposed to ultraviolet (UV) 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. It is not possible to predict which AK lesions will develop into squamous cell carcinoma. AK is the most common pre-cancerous dermatological condition [4].

About Athenex, Inc.

Founded in 2003, Athenex, Inc. is a global clinical stage biopharmaceutical company dedicated to becoming a leader in the discovery, development, and commercialization of next generation drugs for the treatment of cancer. Athenex is organized around three platforms, including an Oncology Innovation Platform, a Commercial Platform, and a Global Supply Chain Platform. The Company’s current clinical pipeline is derived from four different platform technologies: (1) Orascovery, based on P-glycoprotein inhibitor, (2) Src kinase inhibition, (3) Cell therapy, and (4) Arginine deprivation therapy. Athenex’s employees worldwide are dedicated to improving the lives of cancer patients by creating more active and tolerable treatments. For more information, please visit www.athenex.com.

Forward-Looking Statements

Except for historical information, all of the statements, expectations, and assumptions contained in this press release are forward-looking statements. These forward-looking statements are typically identified by terms such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “foresee,” “goal,” “guidance,” “intend,” “likely,” “may,” “plan,” “potential,” “predict,” “preliminary,” “probable,” “project,” “promising,” “seek,” “should,” “will,” “would,” and similar expressions. Actual results might differ materially from those explicit or implicit in the forward-looking statements. Important factors that could cause actual results to differ materially include: the development stage of our primary clinical candidates and related risks involved in drug development, clinical trials, regulation, uncertainties around regulatory reviews and approvals; our ability to scale our manufacturing and commercial supply operations for current and future approved products, and ability to commercialize our products, once approved; ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Athenex’s drug candidates, which may not support further development of such drug candidates; risks related to counterparty performance, including our reliance on third parties for success in certain areas of Athenex’s business; our history of operating losses and our need and ability to raise additional capital; uncertainties around our ability to meet funding conditions under our financing agreements and access to capital thereunder; risks and uncertainties inherent in litigation, including purported stockholder class actions; risks and uncertainties related to the COVID-19 pandemic and its potential impact on our operations, supply chain, cash flow and financial condition; competition; intellectual property risks; uncertainties around our ability to successfully integrate acquired and merged businesses in a timely and cost-effective manner and to achieve synergies; risks relating to doing business internationally and in China; the risk of development, operational delays, production slowdowns or stoppages or other interruptions at our manufacturing facilities; and the other risk factors set forth from time to time in our SEC filings, copies of which are available for free in the Investor Relations section of our website at http://ir.athenex.com/phoenix.zhtml?c=254495&p=irol-sec or upon request from our Investor Relations Department. All information provided in this release is as of the date hereof and we assume no obligation and do not intend to update these forward-looking statements, except as required by law.

Athenex Contacts

Investors:

Steve Rubis
Athenex, Inc.
Email: stevenrubis@athenex.com

Daniel Lang, MD
Athenex, Inc.
Email: danlang@athenex.com 

Tim McCarthy
LifeSci Advisors, LLC
Email: tim@lifesciadvisors.com

References

1. Lucas R, McMichael T, Smith W, Armstrong B. Solar ultraviolet radiation: Global burden of disease from solar ultraviolet radiation: World Health Organization, 206.
2. Worldometer. Population of Europe. 2020. Available at https://www.worldometers.info/world-population/europe-population/ Accessed: October 2020.
3. Blauvelt A, Kempers S, Lain E, et al. Phase 3 Trials of Tirbanibulin Ointment for Actinic Keratosis. New England Journal of Medicine. 2021 Feb 11;384(6):512-520.
4. Skin Cancer Foundation. Actinic Keratosis Overview. Available at: https://www.skincancer.org/skin-cancer-information/actinic-keratosis/#:~:text=Actinic%20keratosis%20(AK)%20is%20the,to%20ultraviolet%20(UV)%20radiation January 2021.

FAQ

What is the significance of the positive opinion from the CHMP for Athenex (ATNX)?

The positive opinion paves the way for Klisyri® to receive regulatory approval in Europe, potentially increasing market access and revenue for Athenex.

What are the expected financial benefits for Athenex following the CHMP opinion?

Athenex could earn up to $45 million in milestone payments and tiered royalties based on annual net sales of Klisyri®.

When was Klisyri® approved by the FDA?

Klisyri® was approved by the U.S. FDA in December 2020 for the treatment of actinic keratosis.

What is actinic keratosis and its prevalence in Europe?

Actinic keratosis is a common skin condition affecting roughly 18% of the European population and can progress to skin cancer if untreated.

Who is the partner of Athenex in the Klisyri® project?

Athenex's partner in the Klisyri® project is Almirall, which has been leading its commercial launch.