– FDA confirmed that the ongoing Phase
1/2 single arm study will serve as pivotal and no additional
randomized, placebo-controlled trial will be required to support
submission of a BLA –
– FDA agreed on the
required efficacy and safety endpoints that will support filing an
accelerated approval BLA for licensure –
– Enrollment and dosing in
the ongoing Phase 2 portion of the study is completed
–
– If approved, PRGN-2012 would
potentially be the first therapeutic for the treatment of RRP, a
serious and difficult-to-treat orphan indication for which the
current standard-of-care is repeated surgeries
–
GERMANTOWN, Md., Aug. 9, 2023
/PRNewswire/ -- Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical
company specializing in the development of innovative gene and cell
therapies to improve the lives of patients, today announced that
the US Food and Drug Administration (FDA) has agreed that the
ongoing Phase 1/2 single arm study (NCT04724980) of the
first-in-class investigational PRGN-2012 AdenoVerse™
immunotherapy for the treatment of recurrent respiratory
papillomatosis (RRP) will serve as pivotal for the purpose of
filing an accelerated approval request for licensure. The FDA also
confirmed no additional randomized, placebo-controlled trial will
be required to support submission of a biologics license
application (BLA). Based on the FDA guidance, the Company
plans to initiate a confirmatory study prior to submission of the
BLA.
The FDA has confirmed that the current primary endpoint for the
ongoing Phase 1/2 study, which is Complete Response rate
(percentage of patients with no surgical interventions during the
12 months following treatment with PRGN-2012), along with an
immunological surrogate marker demonstrating an induction of
HPV-specific T cell responses following PRGN-2012 treatment, is
acceptable for the accelerated approval request.
PRGN-2012 is an innovative therapeutic vaccine with optimized
antigen design that uses Precigen's gorilla adenovector technology,
part of Precigen's proprietary AdenoVerse platform, to elicit
immune responses directed against cells infected with HPV 6 or HPV
11. Gorilla adenovectors have numerous advantages, including the
ability for repeat administration, the inability to replicate in
vivo, which may improve safety, and the ability to deliver a
large genetic payload. The FDA granted PRGN-2012 Breakthrough
Therapy Designation and Orphan Drug Designation for the
treatment of RRP.
Data from the Phase 1 portion of the study showed that 50%
of adult RRP patients (who had ≥3 surgeries to treat the disease in
the year prior to treatment) were "surgery-free" (Complete
Response) after PRGN-2012 treatment during the 12 month follow-up.
All complete responders continue to be surgery-free with a minimum
follow-up of 18 months post-treatment. Precigen has
completed enrollment and dosing in the Phase 2 portion of the study
(N=23) bringing the total number of enrolled patients to 35 at the
recommended Phase 2 dose. Patient follow up is currently ongoing
and data collection is anticipated to be completed by the second
quarter of 2024.
"The eligibility of the Phase 1/2 study, which has already been
fully enrolled and dosed, as the pivotal study to support
accelerated approval has the potential to significantly reduce the
product development time for PRGN-2012. We are thankful for the
FDA's decision, which underscores the importance of bringing
innovative approaches for the treatment of this serious and rare
disease," said Helen Sabzevari, PhD, President and CEO of
Precigen. "I also want to thank the patients who participated in
the study and our investigators, Dr. Clint
T. Allen and Dr. Scott
Norberg from the National Institutes of Health, as well as
the Precigen team."
"The potential of this treatment is tremendously exciting for
RRP patients. The RRP community has only ever had one treatment
option—surgery," said Kim McClellan,
President of Recurrent Respiratory Papillomatosis Foundation. "The
potential to eliminate even one surgery and improve the quality of
our lives would have a profound impact on those living with
RRP."
Precigen: Advancing Medicine with
Precision™
Precigen (Nasdaq: PGEN) is a dedicated
discovery and clinical stage biopharmaceutical company advancing
the next generation of gene and cell therapies using precision
technology to target the most urgent and intractable diseases in
our core therapeutic areas of immuno-oncology, autoimmune
disorders, and infectious diseases. Our technologies enable us to
find innovative solutions for affordable biotherapeutics in a
controlled manner. Precigen operates as an innovation engine
progressing a preclinical and clinical pipeline of
well-differentiated therapies toward clinical proof-of-concept and
commercialization. For more information about Precigen, visit
www.precigen.com or follow us on Twitter @Precigen,
LinkedIn or YouTube.
About Recurrent Respiratory Papillomatosis
(RRP)
Recurrent respiratory papillomatosis (RRP) is a
rare, difficult-to-treat and sometimes fatal neoplastic disease of
the upper and lower respiratory tracts that is caused by infection
with HPV 6 or HPV 11.1-4 RRP is classified based on age
of onset as juvenile or adult. Juvenile-onset disease has an
incidence of 4 per 100,000 and adult-onset RRP has an incidence of
2 to 3 per 100,000. Currently there is no cure for RRP and the
current standard-of-care is repeated endoscopic debulking with
ablation or excision of papillomatous lesions.3,4
Recurrence of papilloma after surgical removal is very common and
repeated procedures are required to debulk and monitor the disease,
which exposes patients to anesthetic and surgical risks, and
emotional distress. RRP morbidity and mortality results from the
effects of papilloma mass on the vocal cords, trachea, and lungs,
which may cause voice changes, stridor, airway occlusion, loss of
lung volume, and/or post-obstructive pneumonia.5
Although rare, one to three percent of RRP cases can transform into
invasive squamous cell carcinoma.6,7
AdenoVerse™ Immunotherapy Clinical
Program
Precigen's AdenoVerse immunotherapy platform
is currently under clinical investigation in a Phase 1/2 study of
PRGN-2009 AdenoVerse immunotherapy alone or in combination with
anti-PDL1/TGF-Beta Trap (bintrafusp alfa) in patients with
HPV-associated cancers (NCT04432597), including oropharyngeal
squamous cell carcinoma (OPSCC), and a Phase 1/2 study of
PRGN-2012 AdenoVerse immunotherapy in patients with recurrent
respiratory papillomatosis (RRP) (NCT04724980). PRGN-2012 has
been granted Orphan Drug Designation and Breakthrough
Therapy Designation in patients with RRP by the FDA.
Additionally, the FDA has cleared the IND to initiate a Phase
2 study of PRGN-2009 AdenoVerse immunotherapy in combination with
pembrolizumab in patients with recurrent or metastatic cervical
cancer.
For patients interested in enrolling in NCI-led clinical
studies, please call NCI's toll-free number 1-800-4-Cancer
(1-800-422-6237) (TTY: 1-800-332-8615), email
NCIMO_Referrals@mail.nih.gov, and/or visit the
website: https://trials.cancer.gov.
AdenoVerse™ Immunotherapy
Precigen's
AdenoVerse immunotherapy platform utilizes a library of proprietary
adenovectors for the efficient gene delivery of therapeutic
effectors, immunomodulators, and vaccine antigens designed to
modulate the immune system. Precigen's gorilla adenovectors, part
of the AdenoVerse library, have potentially superior performance
characteristics as compared to current competition. AdenoVerse
immunotherapies have been shown to generate high-level and durable
antigen-specific T-cell immune responses as well as an ability to
boost these responses via repeat administration. Superior
performance characteristics and high yield manufacturing of
AdenoVerse vectors leveraging UltraVector® technology
allows Precigen to engineer cutting-edge investigational gene
therapies to treat complex diseases.
Trademarks
Precigen, AdenoVerse, UltraVector and
Advancing Medicine with Precision are trademarks
of Precigen and/or its affiliates. Other names may be
trademarks of their respective owners.
Cautionary Statement Regarding Forward-Looking
Statements
Some of the statements made in this press release
are forward-looking statements. These forward-looking statements
are based upon the Company's current expectations and projections
about future events and generally relate to plans, objectives, and
expectations for the development of the Company's business,
including the timing and progress of preclinical studies, clinical
trials, discovery programs and related milestones, the promise of
the Company's portfolio of therapies, and in particular its CAR-T
and AdenoVerse therapies. Although management believes that the
plans and objectives reflected in or suggested by these
forward-looking statements are reasonable, all forward-looking
statements involve risks and uncertainties and actual future
results may be materially different from the plans, objectives and
expectations expressed in this press release. The Company has no
obligation to provide any updates to these forward-looking
statements even if its expectations change. All forward-looking
statements are expressly qualified in their entirety by this
cautionary statement. For further information on potential risks
and uncertainties, and other important factors, any of which could
cause the Company's actual results to differ from those contained
in the forward-looking statements, see the section entitled "Risk
Factors" in the Company's most recent Annual Report on Form 10-K
and subsequent reports filed with the Securities and Exchange
Commission.
References
1 Mounts, P et al.
(1982). "Viral etiology of juvenile- and adult-onset squamous
papilloma of the larynx." Proc Natl Acad Sci U S A 79(17):
5425-5429.
2 Smith, E et al. (1993). "Human papillomavirus
infection in papillomas and nondiseased respiratory sites of
patients with recurrent respiratory papillomatosis using the
polymerase chain reaction." Arch Otolaryngol Head Neck Surg
119(5): 554-557.
3 Derkay, CS et al. (2008). "Recurrent
respiratory papillomatosis: a review." Laryngoscope 118(7):
1236-1247.
4 Derkay, CS et al. (2019). "Update on Recurrent
Respiratory Papillomatosis." Otolaryngol Clin North Am
52(4): 669-679.
5 Seedat, RY (2020). "Juvenile-Onset Recurrent
Respiratory Papillomatosis Diagnosis and Management - A Developing
Country Review." Pediatric Health Med Ther 11: 39-46.
6 Dedo, HH et al. (2001). "CO(2) laser treatment
in 244 patients with respiratory papillomas." Laryngoscope
111(9): 1639-1644.
7 Silver, RD et al. (2003). "Diagnosis and
management of pulmonary metastasis from recurrent respiratory
papillomatosis." Otolaryngol Head Neck Surg 129(6):
622-629.
Investor Contact:
Steven M.
Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
Donelle M.
Gregory
press@precigen.com
Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com
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SOURCE Precigen, Inc.