– First FDA Breakthrough Therapy
Designation granted for AdenoVerse immunotherapy platform;
designation prioritizes PRGN-2012 as a potential treatment for
RRP –
– Designation based on positive Phase
1 clinical data that showed 50% of patients were "surgery-free"
(Complete Response) after PRGN-2012 treatment with a minimum follow
up of 12 months post-treatment –
GERMANTOWN, Md. ,
June 20,
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 granted Breakthrough Therapy Designation for the
first-in-class investigational PRGN-2012 AdenoVerse
immunotherapy for the treatment of recurrent respiratory
papillomatosis (RRP).
FDA's Breakthrough Therapy Designation expedites the development
and review of medicines which are intended to treat serious or
life-threatening diseases, and in which preliminary clinical
evidence demonstrates substantial improvement on clinically
significant endpoints over available therapies.
"This Breakthrough Therapy Designation is the first for
Precigen's AdenoVerse platform and recognizes the immense potential
of PRGN-2012 to change the lives of patients with RRP," said
Helen Sabzevari, PhD, President and
CEO of Precigen. "Standard-of-care for RRP consists of repeated
surgical interventions, and there are currently no approved
therapeutics. The potential of PRGN-2012 to reduce surgical
interventions and improve outcomes for these patients makes us
incredibly proud to receive the FDA's Breakthrough Therapy
Designation. This designation will enable our direct engagement
with senior leadership at the FDA regarding the most efficient
product development pathway, including eligibility for rolling and
priority review of a BLA to support a potential PRGN-2012
registration."
PRGN-2012 incorporates optimized antigen design that uses
gorilla adenovector technology, part of Precigen's proprietary
AdenoVerse platform, to elicit immune responses directed against
cells infected with human papillomavirus type 6 (HPV 6) or HPV type
11 (HPV 11). Gorilla adenovectors have numerous advantages,
including the ability for repeat administration, the inability to
replicate in vivo, and the ability to deliver a large
genetic payload. PRGN-2012 has previously been granted Orphan
Drug Designation in patients with RRP by the FDA.
The Breakthrough Therapy Designation was informed by the
clinical evidence for PRGN-2012 generated in the Phase 1 study
(NCT04724980), which was presented at Precigen's most recent
R&D day, and showed strong response at the recommended phase 2
dose (RP2D) in patients who had an average of 5.8 RRP surgeries
(range 3 – 10) in the year prior to PRGN-2012 treatment. PRGN-2012
treatment resulted in 50% of patients (6 out of 12) in Complete
Response, requiring no post-treatment surgeries with a minimum
follow up of 12 months. PRGN-2012 treatment resulted in a reduction
of surgeries in 83% (10 out of 12) patients in the 12 months
following treatment. PRGN-2012 induced robust de novo
HPV-specific T-cell immune response in RRP patients. PRGN-2012 was
well-tolerated with no dose-limiting toxicities and no
treatment-related adverse events greater than Grade 2. The Phase 1
clinical evidence demonstrating safety and efficacy of PRGN-2012
for the treatment of RRP patients, who have no approved therapeutic
option, provides the foundation for this Breakthrough Therapy
Designation.
PRGN-2012 is currently being evaluated in a Phase 2 study in
adult patients with RRP. Precigen completed enrollment in the Phase
2 study with 23 patients dosed, bringing the total number of
enrolled patients to 35 at the RP2D. Patient follow up is ongoing
as are discussions with the FDA regarding potential rapid
development paths to enable a future submission of a Biologics
License Application (BLA).
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
About PRGN-2012 AdenoVerse Immunotherapy
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. In preclinical models, PRGN-2012 has demonstrated
strong and specific immune response against HPV 6 and HPV 11.
Precigen's PRGN-2012 AdenoVerse immunotherapy is currently under
clinical investigation in a Phase 2 study in adult patients
with RRP (NCT04724980). PRGN-2012 has been
granted Orphan Drug Designation and Breakthrough Therapy
Designation in patients with RRP by the FDA.
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, including the
possibility that the timeline for the Company's clinical trials
might be impacted by the COVID-19 pandemic, 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.