FDA Grants Regenerative Medication Superior Remedy (RMAT) designation to Autolus’ CAR T cell remedy, obe-cel, for the remedy of grownup B-ALL

Autolus Limited

Autolus Restricted

LONDON, April 25, 2022 (GLOBE NEWSWIRE) — Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical firm creating next-generation programmed T cell therapies, right this moment introduced that the U.S. Meals and Drug Administration (FDA) has granted Regenerative Medication Superior Remedy (RMAT) designation to its lead gene remedy obecabatagene autoleucel (obe-cel), a CD19-directed autologous chimeric antigen receptor (CAR) T remedy that’s being investigated within the ongoing FELIX Part 2 examine of grownup relapsed / refractory B-Acute Lymphocytic Leukemia (ALL).

The FDA grants RMAT designation to drug candidates in recognition of the remedy’s potential to handle vital unmet medical wants in sufferers with severe or life-threatening situations. RMAT designation offers vital advantages within the drug growth course of, designed to facilitate and expedite growth and regulatory evaluation.

“RMAT designation is a crucial regulatory milestone for obe-cel and highlights its potential to handle the unmet medical want for grownup sufferers with relapsed and refractory B-ALL,” mentioned Dr. Christian Itin, Chief Govt Officer of Autolus. “RMAT designation from FDA, PRIME designation from EMA and ILAP designation from MHRA facilitate regulatory interactions with key well being authorities and helps our drive to convey this modern remedy to sufferers as shortly as attainable.”

obe-cel has beforehand been granted Priority Medicines (PRIME) designation by the European Medicines Company (EMA) and Progressive Licensing and Entry Pathway (ILAP) by the Medicines and Healthcare merchandise Regulatory Company (MHRA), United Kingdom.

About Autolus Therapeutics plc
Autolus is a clinical-stage biopharmaceutical firm creating next-generation, programmed T cell therapies for the remedy of most cancers. Utilizing a broad suite of proprietary and modular T cell programming applied sciences, the Firm is engineering exactly focused, managed and extremely energetic T cell therapies which can be designed to raised acknowledge most cancers cells, break down their protection mechanisms and eradicate these cells. Autolus has a pipeline of product candidates in growth for the remedy of hematological malignancies and strong tumors. For extra data, please go to www.autolus.com.

About obe-cel (AUTO1)
obe-cel is a CD19 CAR T cell investigational remedy designed to beat the restrictions in medical exercise and security in comparison with present CD19 CAR T cell therapies. Designed to have a quick goal binding off-rate to attenuate extreme activation of the programmed T cells, obe-cel might scale back toxicity and be much less vulnerable to T cell exhaustion, which might improve persistence and enhance the power of the programmed T cells to interact in serial killing of goal most cancers cells. In collaboration with Autolus’ tutorial companion, UCL, obe-cel is at the moment being evaluated in a Part 1 medical trials for B-NHL. Autolus has progressed obe-cel to the FELIX trial, a possible pivotal trial for grownup ALL.

About obe-cel FELIX medical trial
Autolus’ FELIX Part 1b/2 medical trial of obe-cel is enrolling grownup sufferers with relapsed / refractory B-precursor ALL. The trial had a Part 1b element previous to continuing to the one arm, Part 2 medical trial. The first endpoint is general response price, and the secondary endpoints embody length of response, MRD detrimental CR price and security. The trial is designed to enroll roughly 140 sufferers throughout 34 of the main tutorial and non-academic facilities in the US, United Kingdom and Europe. [NCT04404660]

Ahead-Trying Statements
This press launch incorporates forward-looking statements inside the which means of the “protected harbor” provisions of the Personal Securities Litigation Reform Act of 1995. Ahead-looking statements are statements that aren’t historic details, and in some instances will be recognized by phrases equivalent to “might,” “will,” “might,” “expects,” “plans,” “anticipates,” and “believes.” These statements embody, however aren’t restricted to, statements relating to Autolus’ growth of the obe-cel program; the longer term medical growth, efficacy, security and therapeutic potential of its product candidates, together with progress, expectations as to the reporting of knowledge, conduct and timing and potential future medical exercise and milestones; expectations relating to the initiation, design and reporting of knowledge from medical trials; expectations relating to regulatory approval course of for any product candidates; and the Firm’s anticipated money runway. Any forward-looking statements are primarily based on administration’s present views and assumptions and contain dangers and uncertainties that might trigger precise outcomes, efficiency, or occasions to vary materially from these expressed or implied in such statements. These dangers and uncertainties embody, however aren’t restricted to, the dangers that Autolus’ preclinical or medical applications don’t advance or end in authorised merchandise on a well timed or value efficient foundation or in any respect; the outcomes of early medical trials aren’t at all times being predictive of future outcomes; the associated fee, timing and outcomes of medical trials; that many product candidates don’t develop into authorised medication on a well timed or value efficient foundation or in any respect; the power to enroll sufferers in medical trials; attainable security and efficacy considerations; and the impression of the continued COVID-19 pandemic on Autolus’ enterprise. For a dialogue of different dangers and uncertainties, and different vital components, any of which might trigger Autolus’ precise outcomes to vary from these contained within the forward-looking statements, see the part titled “Threat Components” in Autolus’ Annual Report on Type 20-F filed with the Securities and Trade Fee on March 10, 2022, in addition to discussions of potential dangers, uncertainties, and different vital components in Autolus’ subsequent filings with the Securities and Trade Fee. All data on this press launch is as of the date of the discharge, and Autolus undertakes no obligation to publicly replace any forward-looking assertion, whether or not because of new data, future occasions, or in any other case, besides as required by legislation.

Olivia Manser
+44 (0) 7780 471568
[email protected]

Julia Wilson
+44 (0) 7818 430877
[email protected]

Susan A. Noonan
S.A. Noonan Communications
[email protected]