– Designation is an important step toward
bringing this potentially life-changing therapy to European
patients –
– EU Orphan Drug Designation is the first
regulatory designation for a gorilla adenovector based
immunotherapy outside of the United
States for the Company –
GERMANTOWN, Md., Jan. 16,
2024 /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 European Commission (EC) has
granted Orphan Drug Designation for the Company's first-in-class
investigational medicine PRGN-2012 for the treatment of recurrent
respiratory papillomatosis (RRP). RRP is a rare, difficult-to-treat
and sometimes fatal neoplastic disease of the upper and lower
respiratory tracts caused by human papillomavirus type 6 (HPV 6) or
HPV type 11 (HPV 11). PRGN-2012 received Orphan Drug Designation
from the United States Food and Drug Administration (FDA) and
Precigen was the first company to receive Breakthrough Therapy
Designation and an accelerated approval pathway from the
FDA for an RRP treatment.
PRGN-2012 is an innovative therapeutic vaccine with optimized
antigen design that uses 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, and the ability to deliver a large genetic payload.
Orphan Drug Designation in the European Union (EU) is granted by
the EC based on a positive opinion issued by the European Medicines
Agency (EMA) Committee for Orphan Medicinal Products. Medicines
intended for the treatment, diagnosis or prevention of seriously
debilitating or life-threatening conditions that affect fewer than
five in 10,000 people in the European Union (EU) are eligible for
the designation. The Orphan Drug Designation allows companies
certain regulatory, financial and commercial incentives to develop
medicines for rare diseases where there are no satisfactory
treatment options.
"Outside of the US, our commissioned research suggests that
there are more than 125,000 patients diagnosed and living with RRP,
a rare and debilitating disease which currently has no approved
therapeutic treatment option and is treated through repeated
surgical intervention," said Helen
Sabzevari, PhD, President and CEO of Precigen. "The ongoing
Phase 1/2 study for PRGN-2012 in the
United States has shown the immense potential for this
therapy. Results from the Phase 1 study showed significant clinical
benefit with 50% of patients remaining surgery-free after more than
two years following treatment. Additionally, PRGN-2012's favorable
safety profile and ease of administration are potentially paradigm
shifting for both patients and their treating physicians. This
designation is an important next step as we work to expand this
novel therapeutic to the EU in hopes of benefiting the unmet needs
of RRP patients."
PRGN-2012 is currently under investigation in a Phase 1/2
pivotal single-arm study in adult patients with RRP in the United States (clinical trial identifier:
NCT04724980). A Phase 2 data presentation is anticipated in the
second quarter of 2024 and a planned Biologics License
Application (BLA) submission under an accelerated approval pathway
with the FDA is anticipated in the second half of 2024. Commercial
readiness preparations are underway for a potential launch in
the United States in 2025.
About RRP
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. 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
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 X @Precigen, LinkedIn or
YouTube.
Trademarks
Precigen, AdenoVerse 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, product candidate approval and
commercialization 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.
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
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.
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