Azer-Cel Safety Profile was Significantly
Improved Compared to Prior Cohorts in Patients Dosed Using
Optimized Product at Lower Intensity Lymphodepletion; No Grade 3 or
Greater Allogeneic CAR T Related Adverse Events Were Observed
Azer-Cel Achieved 83% Overall Response Rate
(ORR), 61% Complete Response (CR) Rate with 55% Durable Response
Greater Than or Equal to Six Months Among Evaluable CAR T Relapsed
Subjects (n=18)
Upcoming Azer-Cel Meeting with FDA in June to
Align on Potential Phase 2 Study in CAR T Relapsed Diffuse Large
B-cell Lymphoma (DLBCL); Focus on Trial Design, Size, and
Endpoints
PBCAR19B Stealth Cell Proof of Concept
Achieved; Designed to Enable Expansion and Persistence by Delaying
Host Rejection Through Immune Cloaking
PBCAR19B Stealth Cell Achieved 71% ORR with No
Grade 3 or Greater Allogeneic CAR T Related Adverse Events; Most
Compelling Signal in Third Line DLBCL (n=5) with 80% ORR and 60% CR
(MRD-)
Company to Host Webcast and Conference Call
Today at 8:30 AM ET
Precision BioSciences, Inc. (Nasdaq: DTIL) a clinical stage gene
editing company developing ARCUS®-based in vivo gene editing and ex
vivo allogeneic CAR T therapies, today announced program updates
across its allogeneic CAR T pipeline. The Company highlighted new
interim clinical data for its lead candidate, azercabtagene
zapreleucel (azer-cel), as a potential first-in-class allogeneic
CD19 CAR T for the growing CAR T relapsed patient population with
DLBCL. The Company also provided the first clinical update on
PBCAR19B stealth cell, which is in development as a potential
best-in-class allogeneic CAR T therapy for patients with relapsed
or refractory (r/r) non-Hodgkin lymphoma (NHL), with primary focus
on DLBCL.
“Over the last two years, we have pursued a deliberate,
multi-faceted approach in the development of our CAR T programs
with the aim of bringing off-the-shelf therapies to patients. In
the process, we have built one of the most extensive data packages
for an allogeneic product, with the goal of tailoring azer-cel for
patients who have relapsed following autologous CAR T treatment,”
said Michael Amoroso, Chief Executive Officer at Precision
BioSciences. “With the updated clinical data presented today,
including safety, efficacy, and durability, we believe we have
identified the recommended azer-cel dosing regimen to discuss in a
clinical meeting with the U.S. Food and Drug Administration (FDA)
to align on next steps.”
Mr. Amoroso continued, “In addition to azer-cel, we are also
advancing our CD19-targeted program, PBCAR19B stealth cell, which
incorporates an immune cloaking approach designed to allow greater
CAR T expansion and persistence. Last year, we applied
platform-wide manufacturing optimizations using ARCUS for CAR T
insertion for both of our clinical candidates. We believe the
optimized stealth cell product resulted in preliminary efficacy and
safety on par with autologous CAR T in the r/r DLBCL setting. The
interim data highlighted today supports further investigation of
the stealth cell product candidate in DLBCL patients. Looking
ahead, we will continue to evaluate the durability of response in
the stealth cell patients and seek potential partnerships in this
larger, earlier line setting.”
Azer-cel as a Potential First-in Class
Allogeneic CAR T Product Candidate for CD19+ CAR T Relapsed
Patients
As of May 30, 2023, we observed continued high response rates
with an acceptable safety profile in r/r NHL patients. Among all
evaluable subjects (n=61), ORR was 58% with 41% achieving a CR,
across all doses and lymphodepletion regimen.
- Activity was most compelling among the azer-cel treated
subjects who had relapsed following autologous CAR T therapy
(n=18); ORR was achieved in 83% of subjects and 61% achieved
CR.
- In CAR T relapsed evaluable subjects (n=11), 55% had ongoing
durable responses for ≥ 6-months.
- In the most recent CAR T relapsed cohort receiving optimized
Dose Level 4b with FluCy7501 (n=5), 60% ORR was achieved, and 66%
of evaluable patients achieved a full molecular remission (MRD-)
which may be predictive of durability.
- No Grade 3 or greater cytokine release syndrome (CRS), immune
effector cell-associated neurotoxicity syndrome (ICANS), infection
or graft versus host disease was observed in the most recent
cohort.
“Azer-cel continues to demonstrate promising results in DLBCL
patients who relapsed following CAR T, and we are encouraged by the
high overall response rates with molecular remissions in this
patient setting,” said Alan List, M.D., Chief Medical Officer at
Precision BioSciences. “Based on this dataset, azer-cel has the
potential to improve outcomes in this large and growing population
with high unmet need. Also based on these results, a clinical
meeting with the FDA has been scheduled in June to discuss next
steps for azer-cel in the CAR T relapsed setting. We look forward
to providing updates on the path forward in the near future.”
PBCAR19B Stealth Cell as a Potential
Best-in Class Allogeneic CAR T Product Candidate for Earlier Line
CAR T Naïve CD19+ DLBCL
PBCAR19B is Precision’s anti-CD19 targeting allogeneic CAR T
candidate designed to evade immune rejection by host T cell and NK
cells with a single ARCUS gene edit to insert a CD19 CAR transgene,
knock-down beta-2 microglobulin, and insert an HLA-E transgene. The
treatment goal of this program is to potentially displace
autologous CAR T in the 2nd line DLBCL setting.
As of May 30, 2023, in Phase 1 results we observed an acceptable
safety profile with high overall response rates among all evaluable
subjects with evidence of molecular remission (MRD-) and
preliminary durability at Dose Level 2 (540 million cells)2.
- Out of seven evaluable subjects at Dose Level 2, five with
DLBCL and two with mantle cell lymphoma, PBCAR19B achieved 71% ORR
and 43% CR rate.
- In DLBCL patients, ORR was achieved in 80% of subjects and 60%
achieved a CR (MRD-).
- No Grade 3 or greater cytokine release syndrome (CRS), immune
effector cell-associated neurotoxicity syndrome (ICANS), infection
or graft versus host disease was observed.
- PBCAR19B stealth cell achieved proof of concept and appeared to
be effective in delaying recovery of host T- and NK-cells.
- PBCAR19B stealth cell dosed at 540M cells + FluCy750 has been
established as the dosing regimen for further investigation in
DLBCL patients.
“We are impressed with our PBCAR19B stealth cell construct which
appeared to delay host rejection through immune cloaking. Stealth
cell achieved a high response rate, especially in DLBCL subjects
with a high frequency of MRD- complete responses, and acceptable
safety with our improved manufacturing process at Dose Level 2 in
the Phase 1 study,” said Dr. List. “The next steps for stealth cell
will be to await further durability data and seek thoughtful
partnership for ongoing development in the earlier line DLBCL
setting.”
Company-Hosted Webcast and Conference Call
Information
Precision will host a conference call and webcast today, May 31,
2023, at 8:30 AM ET. The dial-in conference call number is (800)
715-9871 and the conference ID number for the call is 4729500.
Participants may access the live webcast, and accompanying
presentation materials, as well as the archived webcast on
Precision’s website in the Investors section under Events &
Presentations:
https://investor.precisionbiosciences.com/events-and-presentations.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology
company dedicated to improving life (DTIL) with its novel and
proprietary ARCUS® genome editing platform. ARCUS is a highly
precise and versatile genome editing platform that was designed
with therapeutic safety, delivery, and control in mind. Using
ARCUS, the Company’s pipeline consists of several in vivo gene
editing candidates designed to cure genetic and infectious diseases
where no adequate treatments exist and multiple ex vivo clinical
candidates. For more information about Precision BioSciences,
please visit www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including, without limitation,
statements regarding the clinical development and expected efficacy
and benefit of our product candidates, the expected timing of
updates regarding our allogenic CAR T and in vivo programs, the
expected timing of regulatory processes, expectations about our
operational initiatives and business strategy, expectations around
partnership opportunities, and expectations about achievement of
key milestones. In some cases, you can identify forward-looking
statements by terms such as “aim,” “anticipate,” “approach,”
“believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,”
“intend,” “look,” “may,” “mission,” “plan,” “possible,”
“potential,” “predict,” “project,” “pursue,” “should,” “target,”
“will,” “would,” or the negative thereof and similar words and
expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. These statements are neither promises nor
guarantees, but involve number of known and unknown risks,
uncertainties and assumptions, and actual results may differ
materially from those expressed or implied in the forward-looking
statements due to various important factors, including, but not
limited to: our ability to become profitable; our ability to
procure sufficient funding and requirements under our current debt
instruments and effects of restrictions thereunder; risks
associated with raising additional capital; our operating expenses
and our ability to predict what those expenses will be; our limited
operating history; the success of our programs and product
candidates in which we expend our resources; our limited ability or
inability to assess the safety and efficacy of our product
candidates; the risk that other genome-editing technologies may
provide significant advantages over our ARCUS technology; our
dependence on our ARCUS technology; the initiation, cost, timing,
progress, achievement of milestones and results of research and
development activities and preclinical and clinical studies; public
perception about genome editing technology and its applications;
competition in the genome editing, biopharmaceutical, and
biotechnology fields; our or our collaborators’ ability to
identify, develop and commercialize product candidates; pending and
potential product liability lawsuits and penalties against us or
our collaborators related to our technology and our product
candidates; the U.S. and foreign regulatory landscape applicable to
our and our collaborators’ development of product candidates; our
or our collaborators’ ability to advance product candidates into,
and successfully design, implement and complete, clinical or field
trials; potential manufacturing problems associated with the
development or commercialization of any of our product candidates;
our ability to obtain an adequate supply of T cells from qualified
donors; our ability to achieve our anticipated operating
efficiencies at our manufacturing facility; delays or difficulties
in our and our collaborators’ ability to enroll patients; changes
in interim “top-line” and initial data that we announce or publish;
if our product candidates do not work as intended or cause
undesirable side effects; risks associated with applicable
healthcare, data protection, privacy and security regulations and
our compliance therewith; our ability to obtain orphan drug
designation or fast track designation for our product candidates or
to realize the expected benefits of these designations; our or our
collaborators’ ability to obtain and maintain regulatory approval
of our product candidates, and any related restrictions,
limitations and/or warnings in the label of an approved product
candidate; the rate and degree of market acceptance of any of our
product candidates; our ability to effectively manage the growth of
our operations; our ability to attract, retain, and motivate
executives and personnel; effects of system failures and security
breaches; insurance expenses and exposure to uninsured liabilities;
effects of tax rules; effects of the COVID-19 pandemic and variants
thereof, or any pandemic, epidemic, or outbreak of an infectious
disease; the success of our existing collaboration agreements, and
our ability to enter into new collaboration arrangements; our
current and future relationships with and reliance on third parties
including suppliers and manufacturers; our ability to obtain and
maintain intellectual property protection for our technology and
any of our product candidates; potential litigation relating to
infringement or misappropriation of intellectual property rights;
effects of natural and manmade disasters, public health emergencies
and other natural catastrophic events; effects of sustained
inflation, supply chain disruptions and major central bank policy
actions; market and economic conditions; risks related to ownership
of our common stock, including fluctuations in our stock price, and
other important factors discussed under the caption “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarterly period ended
March 31, 2023, as any such factors may be updated from time to
time in our other filings with the SEC, which are accessible on the
SEC’s website at www.sec.gov and the Investors page of our website
under SEC Filings at investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
1 Dose Level 4b = 500 × 106 (flat dose). FluCy750
lymphodepletion = fludarabine 30 mg/m2 × 3 days + cyclophosphamide
750 mg/m2 × 3 days.
2 Dose Level 2 = 540 × 106 (flat dose) with FluCy750
lymphodepletion (fludarabine 30 mg/m2 × 3 days + cyclophosphamide
750 mg/m2 × 3 days).
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230531005625/en/
Investor and Media Contact: Mei Burris Director, Investor
Relations and Finance Mei.Burris@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Precision BioSciences (NASDAQ:DTIL)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024