Royalty Pharma plc (Nasdaq: RPRX) and Ferring Pharmaceuticals today
announced that Royalty Pharma has acquired a synthetic royalty on
US net sales of Ferring’s Adstiladrin® (nadofaragene
firadenovec-vncg) for up to US $500 million comprised of an upfront
payment of US $300 million and a US $200 million milestone payment.
The milestone payment is contingent on certain manufacturing goals
that are expected to be achieved in 2025 for the FDA-approved
intravesical gene therapy that Ferring will make available next
month through an early experience program for the treatment of
adult patients with high-risk, Bacillus Calmette-Guérin
(BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with
carcinoma in situ (CIS) with or without papillary tumors.
Under the terms of the agreement, Royalty Pharma is acquiring a
5.1% percentage royalty on net sales of Adstiladrin in the United
States, which will increase to 8.0% upon payment of the
manufacturing-related milestone. The royalty is expected to end in
the early to mid-2030s.
“This major investment by Royalty Pharma, the largest buyer of
biopharmaceutical royalties and a leading funder of innovation, is
yet another demonstration of the value and confidence in our gene
therapy Adstiladrin to address significant unmet medical needs for
patients. It also highlights its significant potential as a key
growth driver for Ferring, and our commitment to Uro-Oncology,”
said Jean-Frédéric Paulsen, Executive Chairman of Ferring
Pharmaceuticals.
Bladder cancer is the sixth most common cancer in the United
States, with NMIBC representing approximately 75% of all new
bladder cancer cases.i Adstiladrin is a non-replicating adenovirus
vector-based gene therapy for the treatment of adult patients with
high-risk BCG-unresponsive NMIBC. Although BCG remains the
first-line standard of care for people living with high-grade
NMIBC, more than 50% of patients who receive initial treatment with
BCG will experience disease recurrence and progression within one
year, with many developing BCG-unresponsive disease.ii In April
2023, the FDA approved a Prior-Approval Supplement (PAS) to the
Biologics License Application (BLA) for the therapy which enabled
the scale-up of drug substance manufacturing process.
“After several decades of little progress in the field,
Adstiladrin brings a major innovation to patients
with high-risk NMIBC who no longer respond to
current first-line treatment and have few other good options.
Our ambition is for Adstiladrin to become the new standard of care
and the backbone therapy for these patients and to drive research
in other urothelial cancers. This agreement positions us well for
continued significant and sustained investment to further advance
Adstiladrin as the foundation of our leadership drive in
Uro-Oncology,” said Bipin Dalmia, Global Head, Uro-Oncology
Franchise of Ferring Pharmaceuticals.
“We are delighted to partner with Ferring, a research-driven,
global specialty biopharmaceutical company. This investment is
consistent with our strategy of acquiring royalties on innovative
therapies in areas of high unmet patient need,” said Pablo
Legorreta, founder and Chief Executive Officer of Royalty Pharma.
“Adstiladrin is the first gene therapy in our diversified royalty
portfolio. We believe it has blockbuster potential and we are
pleased to provide funding to support the launch of Adstiladrin and
help Ferring reach as many patients as possible with this important
therapy in the United States.”
About Adstiladrin
Adstiladrin® (nadofaragene firadenovec-vncg) is a gene therapy
developed as a treatment for adult patients with BCG-unresponsive
NMIBC. It is a non-replicating adenovirus vector-based gene therapy
containing the gene encoding interferon alfa-2b protein,
administered by catheter into the bladder once every three months.
The vector enters the cells of the bladder wall, releasing the
interferon gene. The internal cell machinery translates the
interferon DNA sequence, resulting in the cells secreting high
quantities of interferon alfa-2b protein, a recombinant analog of
the naturally occurring protein the body uses to fight cancer. This
novel gene therapy approach thereby turns the patient’s own bladder
wall cells into interferon microfactories, enhancing the body’s
natural defenses against the cancer. Nadofaragene firadenovec-vncg
has been studied in a clinical trial program that includes 157
patients with high-grade, BCG-unresponsive NMIBC who had been
treated with adequate BCG previously and did not see benefit from
additional BCG treatment (full inclusion criteria published on
clinicaltrials.gov: NCT02773849).
US FDA approval of Adstiladrin on December 16, 2022 was based on
results of the Phase 3 clinical trial, which met its primary
endpoint with more than half (51%, n=50 of 98; 95% CI 41 to 61) of
patients with carcinoma in situ with or without concomitant
high-grade Ta or T1 disease (CIS ± Ta/T1) achieving a complete
response (CR) by three months. Of the patients who achieved an
initial CR, 46% (n=23 of 50) continued to remain free of high-grade
recurrence at 12 months.
INDICATION
Adstiladrin is a non-replicating adenoviral vector-based gene
therapy indicated for the treatment of adult patients with
high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle
invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with
or without papillary tumors.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: Adstiladrin is
contraindicated in patients with hypersensitivity to interferon
alfa or any component of the product.
WARNINGS AND PRECAUTIONS:
- Risk with delayed cystectomy: Delaying cystectomy in patients
with BCG-unresponsive CIS could lead to development of muscle
invasive or metastatic bladder cancer, which can be lethal. If
patients with CIS do not have a complete response to treatment
after 3 months or if CIS recurs, consider cystectomy.
- Risk of disseminated adenovirus infection: Persons who are
immunocompromised or immunodeficient may be at risk for
disseminated infection from ADSTILADRIN due to low levels of
replication-competent adenovirus. Avoid ADSTILADRIN exposure to
immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer
Adstiladrin by intravesical instillation only. Adstiladrin is not
for intravenous use, topical use, or oral administration.
USE IN SPECIFIC POPULATIONS: Advise
females of reproductive potential to use effective contraception
during Adstiladrin treatment and for 6 months after the last dose.
Advise male patients with female partners of reproductive potential
to use effective contraception during Adstiladrin treatment and for
3 months after the last dose.
ADVERSE REACTIONS: The most common
(>10%) adverse reactions, including laboratory abnormalities
(>15%), were glucose increased, instillation site discharge,
triglycerides increased, fatigue, bladder spasm, micturition
(urination urgency), creatinine increased, hematuria (blood in
urine), phosphate decreased, chills, pyrexia (fever), and dysuria
(painful urination).
You are encouraged to report negative side effects of
prescription drugs to FDA.
Visit www.FDA.gov/medwatch or
call 1-800-332-1088. You may also contact Ferring Pharmaceuticals
at 1-888-FERRING.
About Royalty Pharma plc
Founded in 1996, Royalty Pharma is the largest buyer of
biopharmaceutical royalties and a leading funder of innovation
across the biopharmaceutical industry, collaborating with
innovators from academic institutions, research hospitals and
non-profits through small and mid-cap biotechnology companies to
leading global pharmaceutical companies. Royalty Pharma has
assembled a portfolio of royalties which entitles it to payments
based directly on the top-line sales of many of the industry’s
leading therapies. Royalty Pharma funds innovation in the
biopharmaceutical industry both directly and indirectly - directly
when it partners with companies to co-fund late-stage clinical
trials and new product launches in exchange for future royalties,
and indirectly when it acquires existing royalties from the
original innovators. Royalty Pharma’s current portfolio includes
royalties on more than 35 commercial products, including Vertex’s
Trikafta, Kalydeco, Orkambi and Symdeko, Biogen’s Tysabri, AbbVie
and Johnson & Johnson’s Imbruvica, Astellas and Pfizer’s
Xtandi, GSK’s Trelegy, Novartis’ Promacta, Pfizer’s Nurtec ODT,
Johnson & Johnson’s Tremfya, Roche’s Evrysdi, Gilead’s
Trodelvy, and 11 development-stage product candidates.
About Ferring Pharmaceuticals
Ferring Pharmaceuticals is a research-driven, specialty
biopharmaceutical group committed to helping people around the
world build families and live better lives. Headquartered in
Saint-Prex, Switzerland, Ferring is a leader in reproductive
medicine and women’s health, and in specialty areas within
gastroenterology and urology. Ferring has been developing
treatments for mothers and babies for over 50 years and has a
portfolio covering treatments from conception to birth. Founded in
1950, privately-owned Ferring now employs around 7,000 people
worldwide, has its own operating subsidiaries in more than 50
countries and markets its products in 100 countries.
Learn more at www.ferring.com, or connect with us on Twitter,
Facebook, Instagram, LinkedIn and YouTube.
Advisors
Goodwin Procter LLP and Fenwick & West LLP acted as legal
advisors to Royalty Pharma. Orrick, Herrington & Sutcliffe LLP
acted as legal advisor to Ferring.
Royalty Pharma Forward-Looking Statements
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References
iEuropean Association of Urology. Guidelines for
non-muscle-invasive bladder cancer (TaT1 and CIS). Available at
https://uroweb.org/guidelines/non-muscle-invasive-bladder-cancer
iiBoorjian SA, Alemozaffar M, Konety BR, et al. Intravesical
nadofaragene firadenovec gene therapy for BCG-unresponsive
non-muscle-invasive bladder cancer: a single-arm, open-label,
repeat-dose clinical trial. Lancet Oncol 2021; 22:107–17.
Royalty Pharma Investor Relations and
Communications
+1 (212) 883-6772ir@royaltypharma.com
Ferring Europe
Matthew Worrall+44 7442271811 Matthew.Worrall@ferring.com
Ferring US
Carol Ready+1 973 7657307Carol.Ready@ferring.com
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