Celularity’s Placental-Derived Allogeneic Cell Therapy Provides Clinically Meaningful Benefit and Durable Biological Effect in Patients with Moderate to Severe Crohn’s Disease in Phase 1, Phase 1b/2a and Phase 1b Studies
Legacy studies employing a single treatment course of two infusions seven days apart demonstrated greatest benefit in three early studies
Data support further investigation of novel genetically modified cell therapy in Crohn’s disease
Florham Park, NJ, – January 6, 2023 – Celularity Inc. (Nasdaq: CELU) (Celularity), a biotechnology company developing placental-derived allogeneic cell and biomaterial therapies, announced today that its re-analysis of long term follow-up data from three legacy Phase 1, Phase 1b/2a and Phase 1b studies of its legacy placental-derived mesenchymal-like adherent stromal cell (MLASC) therapy in moderate-to-severe Crohn’s disease patients showed clinically meaningful and durable benefit for up to two years in the limited number of patients studied and warrants further investigation. These studies also identified the optimal MLASC treatment schedule to be a single treatment course of two infusions seven days apart, with no further maintenance treatment required during the clinical study and follow-up period.
“We pioneered the development of our placental-derived allogenic therapy because we believe that our technology has broad application across a number of therapeutic areas with high unmet need,” said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. “We see promising signals like these Crohn’s disease data in several other therapeutic areas, which reinforces our confidence in potential therapeutic benefits of MLASCs as we advance the development of these therapeutic options for patients. These signals are guiding our investment decision to progress our novel genetically modified allogeneic placental-derived MLASC, APPL-001, in Crohn’s disease, where we believe this cell therapy candidate could make a significant difference.”
“These encouraging data showed clinically meaningful and durable response rates. For example, in a Phase 1 study, a clinical disease remission rate of 50 percent at two years as measured by the Crohn’s Disease Activity Index was observed following a single treatment course. Additionally, the clinical response rate at two years was 83 percent (5 of 6 patients). The therapy was generally well-tolerated with no dose-limiting toxicities observed,” said Sharmila Koppisetti, M.D., Senior VP, Non-oncology Clinical Affairs.
“The immune pathways involved in inflammation and fistula formation associated with Crohn’s disease are now well-characterized and we believe the broad activity of APPL-001 may allow us to target some of the key drivers of these processes. Despite therapeutic advances, Crohn’s disease remains an area of significant morbidity and requires therapies to target both the inflammatory processes and fistula formation. Crohn’s disease is representative of a number of autoimmune inflammatory diseases,” said Dr. Koppisetti.
About the Legacy Studies
This press release references the following studies:
- Safety and Tolerability of Human Placenta-Derived Cells in Treatment-resistant Crohn’s Disease: A Phase 1 Study. Mayer, et al. Inflammatory Bowel Disease Journal. Volume 19, Number 4, March-April 2013.
- Human Placenta-derived Cells for the Treatment of Moderate-to-severe Crohn’s Disease: A Phase 1b/2a Study. Melmed, G. Inflammatory Bowel Disease Journal. Volume 21, Number 8, August 2015.
In addition to these studies, the Company plans to submit the abstract, “Randomized, Double-Blind, Placebo-Controlled Dose-Escalation Study to Determine the Safety and Efficacy of Intra Venous Infusion of Human Placenta-Derived Cells for the Treatment of Crohn’s Disease,“ for presentation at an upcoming scientific meeting.
About Crohn’s Disease
Crohn’s disease is a type of inflammatory bowel disease (IBD). It causes swelling of the tissues (inflammation) in the digestive tract, which can lead to abdominal pain, severe diarrhea, fatigue, weight loss and malnutrition. Inflammation caused by Crohn’s disease can involve different areas of the digestive tract in different people, most commonly the small intestine. This inflammation often spreads into the deeper layers of the bowel.
Crohn’s disease can be both painful and debilitating, and sometimes may lead to life-threatening complications. There’s no known cure for Crohn’s disease, but therapies can greatly reduce its signs and symptoms and even bring about long-term remission and healing of inflammation. With treatment, many people with Crohn’s disease are able to function well. (Mayo Clinic, 2023)
Celularity Inc. (Nasdaq: CELU) headquartered in Florham Park, N.J., is a clinical stage biotechnology company leading the next evolution in cellular medicine by developing allogeneic cryopreserved off-the-shelf placental-derived cell therapies, including therapeutic programs using unmodified natural killer (NK) cells, genetically modified NK cells, T-cells engineered with a CAR (CAR T-cells), and mesenchymal-like adherent stromal cells (ASCs). These therapeutic programs target indications in cancer, infectious and degenerative diseases. In addition, Celularity develops and manufactures innovative biomaterials also derived from the postpartum placenta. Celularity believes that by harnessing the placenta’s unique biology and ready availability, it can develop therapeutic solutions that address significant unmet global needs for effective, accessible, and affordable therapies.
To learn more, visit celularity.com
Celularity Forward-Looking Statements
This press release includes “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995, as well as within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “can,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “intends,” “may,” “might,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “seek,” “should,” “strive,” “target,” “will,” “would” and the negative of terms like these or other comparable terminology, and other words or terms of similar meaning. The forward-looking statements in this press release include express and implied statements regarding progressing APPL-001 into clinical studies and its potential for Crohn’s disease and the benefits of Celularity’s approach to cellular medicine, among others. Many factors could cause actual results to differ materially from those described in these forward-looking statements, including but not limited to: the inherent risks in biotechnological development, including with respect to the development of novel cellular therapies, and the clinical trial and regulatory approval process; and risks associated with Celularity’s current liquidity, as well as developments relating to Celularity’s competitors and industry, along with those risk factors set forth under the caption “Risk Factors” in Celularity’s annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 31, 2022, as amended on July 15, 2022, and other filings with the SEC. These risks and uncertainties may be amplified by the current economic situation, including inflation, supply chain issues and overall economic uncertainty. If any of these risks materialize or underlying assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. There may be additional risks that Celularity does not presently know, or that Celularity currently believes are immaterial, that could also cause actual results to differ from those contained in the forward-looking statements. In addition, these forward-looking statements reflect Celularity’s current expectations, plans, or forecasts of future events and views as of the date of this communication. Subsequent events and developments could cause assessments to change. Accordingly, forward-looking statements should not be relied upon as representing Celularity’s views as of any subsequent date, and Celularity undertakes no obligation to update forward-looking statements to reflect events or circumstances after the date hereof, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws.