PGEN - Precigen’s Overnight Cell Manufacturing Technology Could Offer Advanced AML Patients The Option Of Promising CAR-T Therapy
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Acute myeloidleukemia (AML) is among the mostcommon types of leukemia in adults. The term leukemia refersto cancers of the bone marrow and blood cells. Doctors refer to AML as“acute” because the condition is known to progress rapidly.
The five-year survivalrate for patients with AML is as low as 5%, leading to approximately11,000 deaths annually in the US. Each year sees 20,000newly diagnosed cases of AML and, after initial treatments,roughly 50% relapse with a very poor prognosis.
Precigen Inc. (NASDAQ: PGEN) , a biopharmaceutical company specializing in thedevelopment of novel gene and cell therapies, has developed aground-breaking technology that enables the treatment of AML usingtargeted CAR-T therapy administered overnight right at the facilitywhere a patient is being treated.
In some cancers, such as advanced AML, even aday’s delay in treatment could make a difference in survival.
The main treatment formost types of AML is currently chemotherapy, which has been themainstay of treatment for various types of cancers for decades.
But over the past several years, theU.S. Food and Drug Administration (FDA) has approved new categories ofcancer medicines – among them CAR-T – which are transforming thetreatment picture of certain cancers effectively, and in some caseshelping to stave off cancer for many years.
CAR-T Therapy Developed By Precigen Could Potentially Be APromising First In The Treatment Of AML
AML progresses very rapidly, making itessential to treat it as quickly as possible. Because of this,autologous CAR-T therapy, which usually takes 3-4 weeks to manufacturefor each patient, has not been successful in treating patients withadvanced AML.
Thecurrent process for making autologous CAR-T therapy involves multiple,time-consuming and costly steps that include drawing patient blood,transporting it to a central manufacturing facility, transfecting thepatient’s T cells with a lentivirus for antigen targeting, andexpanding the cells for weeks in the lab before shipping the therapyback for infusion into the patient. T cells, a key component of theimmune system, help the body fight infection and tumors.
In contrast,Precigen’s technology condenses this time-consuming process intoovernight manufacturing using the patient's own cells to make itsUltraCAR-T cells.
This is made possible by using the company’s patentedUltraPorator®, a custom manufacturing device, designed fordecentralized use to enable UltraCAR-T to be made and administered inany hospital where the patient is being treated.
Precigen is conducting aPhase 1b clinical trial of its PRGN-3006 UltraCAR-T in patients withadvanced AML who have failed multiple prior therapies.
The company released encouragingclinical data last year , raising hopes that PRGN-3006 couldprove to be a significant new treatment choice for relapsed orrefractory AML patients who have run out of other options.
Updated data presentedearlier this month at the 64th Annual Meeting and Exposition of theAmerican Society of Hematology showed that a single infusion ofUltraCAR-T cells following lymphodepletion demonstrated robustexpansion and persistence in blood and bone marrow and resulted in a27 percent objective response rate (ORR) is heavily pre-treated r/rAML patients.
David A. Sallman, MD, Assistant Member in the Department ofMalignant Hematology at the H. Lee Moffit Cancer Center and ResearchInstitute, and a lead investigator for the PRGN-3006 clinical trial,said a 27 percent ORR in this patient population, which has limitedtreatment options, is a significant result. He reported PRGN-3006 hasan excellent safety profile with no drug-limiting toxicities observedto date, and noted that a patient who received PRGN-3006 followingallo-HSCT has responded to treatment for more than 18 months.
The success of its clinical trials couldestablish the viability of reducing the processing time of autologousCAR-T therapy for AML from weeks to overnight – a time advantagethat could save lives at a significantly lower cost.
Combining The Best Of Autologous AndAllogenic CAR-T technology For Optimum Results?
There are two forms ofCAR-T (chimeric antigen receptor T cell) technology. One is autologous(custom-made for each patient with the patient's own cells), andthe other is allogenic (made with donor-sourced cells and packaged asan off-the-shelf product).
The current market for autologous CAR-T therapycomprises key players including Gilead Sciences, Inc. (NASDAQ:GILD), Bristol-Myers Squibb Co. (NYSE: BMY), Novartis AG (SWX: NOVN) and Johnson & Johnson (NYSE: JNJ).
Although autologousCAR-T has a record of good outcomes, its high cost and prolonged timeto manufacture (up to 28 days) have been seen as majordrawbacks.
Clinical trials of off-the-shelf allogenic CAR-T have been metwith limited success so far. The drawbacks of allogenic CAR-T includereduced durability of response and potential issues relating to therejection of the donated graft by the patient (graft vs host disease),which could prove to be treatment-limiting and outweigh the potentialcost and convenience benefits of this type of treatment.
Companies developingallogeneic CAR-T include Allogene Therapeutics Inc. (NASDAQ:ALLO), Crispr Therapeutics AG (NASDAQ: CRSP), EditasMedicine Inc. (NASDAQ: EDIT) and Caribou Biosciences Inc. (NASDAQ: CRBU) .
The Precigen approach seeks to combine the efficacyand safety of autologous CAR-T with the potentially lower cost andconvenience of allogenic CAR-T.
Precigen’s UltraCAR-T is a platform technology,potentially adaptable to the treatment of many different cancers, inboth liquid and solid tumors. Other UltraCAR-T candidates indevelopment include PRGN-3005 for recurrent platinum-resistantovarian, fallopian tube or primary peritoneal cancer, and PRGN-3007for treating a variety of cancers, including chronic lymphocyticleukemia, mantle cell leukemia, acute lymphocytic leukemia andtriple-negative breast cancer.
The company also reports that PRGN-3006 UltraCAR-Thas demonstrated a favorable safety profile to date with nodose-limiting toxicities or neurotoxicity. PRGN-3006 has also received orphandrug designation and fasttrack status from the FDA.
"We are very pleased to receive the FDA'sFast Track designation, which facilitates development and expeditesthe review process of drugs that address serious conditions and highunmet medical needs," says Helen Sabzevari, PhD, President and CEOof Precigen.
Dr.Sabzevari added, "AML is a rapidly progressing disease with a verypoor prognosis. The Fast Track designation will help facilitate thetimely development of this program and we look forward to workingclosely with the FDA to potentially bring this new and highlydifferentiated overnight UltraCAR-T therapy to patients."
Click herefor more information about Precigen, Inc. (PGEN)
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Photo by National Cancer Institute on Unsplash
Precigen: Advancing Medicinewith Precision™Precigen (Nasdaq: PGEN) is a dedicated discovery andclinical stage biopharmaceutical company advancing the next generationof gene and cell therapies using precision technology to target themost urgent and intractable diseases in our core therapeutic areas ofimmuno-oncology, autoimmune disorders, and infectious diseases. Ourtechnologiesenable us to find innovative solutions for affordablebiotherapeutics in a controlled manner. Precigen operates as aninnovation engine progressing a preclinical and clinical pipeline ofwell-differentiated therapies toward clinical proof-of-concept andcommercialization. For more information about Precigen, visitwww.precigen.com or follow us on Twitter @Precigen, LinkedIn orYouTube.
This post contains sponsored advertisingcontent. This content is for informational purposes only and is notintended to be investing advice.
Some of the statementsmade in this press release are forward-looking statements. Theseforward-looking statements are based upon the Company's currentexpectations and projections about future events and generally relateto plans, objectives, and expectations for the development of theCompany's business, including the timing and progress ofpreclinical studies, clinical trials, discovery programs and relatedmilestones, the promise of the Company's portfolio of therapies,and in particular its CAR-T and AdenoVerse therapies. Althoughmanagement believes that the plans and objectives reflected in orsuggested by these forward-looking statements are reasonable, allforward-looking statements involve risks and uncertainties, includingthe possibility that the timeline for the Company's clinicaltrials might be impacted by the COVID-19 pandemic, and actual futureresults may be materially different from the plans, objectives andexpectations expressed in this press release. The Company has noobligation to provide any updates to these forward-looking statementseven if its expectations change. All forward-looking statements areexpressly qualified in their entirety by this cautionary statement.For further information on potential risks and uncertainties, andother important factors, any of which could cause the Company'sactual results to differ from those contained in the forward-lookingstatements, see the section entitled "Risk Factors" in theCompany's most recent Annual Report on Form 10-K and subsequentreports filed with the Securities and ExchangeCommission.
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