Incyte Announces Health Canada Conditional Approval of Pemazyre® (pemigatinib) as First Targeted Treatment for Adults with Previously Treated, Unresectable Locally Advanced or Metastatic Cholangiocarcinoma

MONTREAL, Sept. 17, 2021 /CNW/ - Incyte (NASDAQ: INCY) today announced that Health Canada has granted a Notice of Compliance with conditions for Pemazyre^® (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement.

"For appropriate patients with cholangiocarcinoma, the approval of Pemazyre in Canada offers patients hope with a new potential treatment option after their cancer has returned, following the failure of first-line treatment," said Durhane Wong-Rieger, Ph.D., President and CEO, Canadian Organization for Rare Disorders.

"Cholangiocarcinoma is a rare cancer of the bile duct with limited treatment options. As a result, patients face a poor prognosis," said Dr. Vincent Tam, MD, Medical Oncologist, Tom Baker Cancer Centre. "With the approval of Pemazyre, we now have a targeted treatment option for those patients with progressive disease after first-line chemotherapy."

The conditional approval is based on data from the FIGHT-202 study evaluating pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma.  Study results demonstrated that in patients harbouring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate (ORR) of 35.5 percent (primary endpoint) and median duration of response (DOR) of 9.1 months (secondary endpoint) with a median follow-up of 15.4 months.  Adverse events observed included serous retinal detachment (SRD) and hyperphosphatemia.  The most common adverse reactions (incidence ≥15%) are hyperphosphatemia, alopecia, diarrhea, fatigue, nail toxicity, dysgeusia, nausea, constipation, stomatitis, dry mouth, decreased appetite, vomiting, dry eye, arthralgia, abdominal pain, hypophosphatemia, dry skin, edema peripheral, weight decreased, headache, urinary tract infection, dehydration, hypercalcemia, and palmar-plantar erythrodysaesthesia syndrome.

"We are pleased that Health Canada has granted conditional marketing authorization for Pemazyre and Incyte welcomes the opportunity to serve the cholangiocarcinoma community with this much-needed treatment option," said Josée Brisebois, Ph.D.., Head of Medical Affairs, Incyte Biosciences Canada. "Incyte will strive to secure rapid access across Canada to this innovative targeted therapy to patients suffering from this difficult disease."

Cholangiocarcinoma is a rare cancer that forms in the bile duct. It is classified based on its anatomical origin: intrahepatic cholangiocarcinoma (iCCA) occurs in the bile duct inside the liver, and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver.  Patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor.^1,2 The incidence of cholangiocarcinoma varies regionally and ranges between 0.3-3.4 per 100,000 in North America and Europe.¹   FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16% of patients.^3,4,5,6 FGFRs play an important role in tumour cell proliferation and survival, migration, and angiogenesis (the formation of new blood vessels). Activating fusions, rearrangements, translocations, and gene amplifications in FGFRs are closely correlated with the development of various cancers.

About FIGHT-202
The FIGHT-202 is a multi-center, open-label, single-arm, Phase 2 study (NCT02924376) that evaluated the safety and efficacy of pemigatinib – a selective fibroblast growth factor receptor (FGFR) inhibitor – in adult (age ≥18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement.
Patients were enrolled into one of three cohorts – Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genomic alterations) or Cohort C (no FGF/FGFR genomic alterations). All patients received 13.5 mg pemigatinib orally once daily (QD) on a 21-day cycle (two weeks on/one week off) until radiological disease progression or unacceptable toxicity.

The primary endpoint of FIGHT-202 was overall response rate (ORR) in Cohort A, assessed by independent review per RECIST v1.1. Secondary endpoints include ORR in Cohorts B, A plus B, and C; and duration of response (DOR).

For more information about FIGHT-202, visit https://clinicaltrials.gov/ct2/show/NCT02924376.

About FIGHT
The FIGHT (Fibroblast Growth factor receptor in oncology and Hematology Trials) clinical trial program includes ongoing Phase 2 and 3 studies investigating the safety and efficacy of pemigatinib therapy across several FGFR-driven malignancies. Phase 2 monotherapy studies include FIGHT-202, as well as FIGHT-201 investigating pemigatinib in patients with metastatic or surgically unresectable bladder cancer, including with activating FGFR3 mutations or fusions/rearrangements; FIGHT-203 in patients with myeloproliferative neoplasms with activating FGFR1 fusions/rearrangements; FIGHT-207 in patients with previously treated, locally-advanced/metastatic or surgically unresectable solid tumour malignancies harbouring activating FGFR mutations or translocations, irrespective of tumour type. FIGHT-205 is a Phase 2 study investigating pemigatinib plus pembrolizumab combination therapy and pemigatinib monotherapy as first-line treatment for metastatic or unresectable bladder cancer harbouring FGFR3 mutations or rearrangements who are not eligible to receive cisplatin.  FIGHT-302 is a Phase 3 study investigating pemigatinib as a first-line treatment for patients with cholangiocarcinoma with FGFR2 fusions or rearrangements.

About Pemazyre^® (pemigatinib)
Pemazyre is a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement.⁶

In the United States, Pemazyre is approved for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

In Japan, Pemazyre is approved for the treatment of patients with unresectable biliary tract cancer (BTC) with a fibroblast growth factor receptor 2 (FGFR2) fusion gene, worsening after cancer chemotherapy.

In Europe, Pemazyre is approved for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

Pemazyre is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3, which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations.

Pemazyre is marketed by Incyte in the United States, Europe and Japan. Incyte has granted Innovent Biologics, Inc. rights to develop and commercialize pemigatinib in hematology and oncology in Mainland China, Hong Kong, Macau and Taiwan. Incyte has retained all other rights to develop and commercialize pemigatinib outside of the United States.

Pemazyre is a trademark of Incyte.

About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

To learn more about Incyte Biosciences Canada, visit https://incytebiosciences.ca.

Forward-Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the FIGHT clinical trial program and whether Pemazyre might provide a successful treatment option for patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements, contain predictions, estimates and other forward-looking statements. 

These forward-looking statements are based on the Company's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by Canadian regulatory authorities or other regulatory authorities, including the FDA; the Company's dependence on its relationships with its collaboration partners; the efficacy or safety of the Company's products and the products of the Company's collaboration partners; the acceptance of the Company's products and the products of the Company's collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in the Company's reports filed with the Securities and Exchange Commission, including its annual report for the year ended December 31, 2020, and the quarterly report on Form 10-Q for the quarter ended June 30, 2021. The Company disclaims any intent or obligation to update these forward-looking statements.

_______________________________
^1	Banales JM, et al. Nat Rev Gastroenterol Hepatol. 2016;13:261‒280.
^2	Uhlig J, et al. Ann Surg Oncol. 2019;26:1993–2000.
^3	Graham RP, et al. Hum Pathol. 2014;45:1630‒1638.
^4	Farshidfar F, et al. Cell Rep. 2017;18(11):2780–2794.
^5	Ross JS et al. The Oncologist. 2014;19:235–242.
^6	Pemazyre^® (pemigatinib) [Product Monograph]. Wilmington, DE: Incyte; September 2021

SOURCE Incyte Biosciences Canada

Media, Jenifer Antonacci, [phone], [email protected]; Catalina Loveman, [phone], [email protected]; Investors, Christine Chiou, [phone], [email protected]