In this SNN Q&A, StockNewsNow.com's Editor-in-Chief, Robert Kraft, spoke with David J. Mazzo, PhD, Chief Executive Officer and Director of Caladrius Biosciences, Inc. (NASDAQ: CLBS) to discuss the company, direction of the Caladrius since its name change from NeoStem, current indications, and more…
SNN: Please provide an overview of Caladrius Biosciences, Inc.
DM: Caladrius is among the first of a new breed of immunotherapy companies with proven expertise and unique experience in cell process optimization, development and manufacturing. We combine a leading cell therapy service provider (our subsidiary, PCT) with a development pipeline including a late-stage clinical program based on proprietary platform technology for immuno-oncology, as well as additional platform technologies for immunomodulation and ischemic repair. This integrated approach benefits patients and the cell therapy industry as whole by facilitating the bringing of significant life-improving medical treatments to market.
SNN: The company was formerly known as NeoStem. Why the name change? Has the direction of the company changed with name and new CEO?
DM: The name change reflected our company’s integrated approach to cell therapy, to create a foundation for better market understanding of our position in the field. The company has evolved over the years and we are now working on more than just stem cells. We work with a broad range of therapeutic cells including dendritic cells/cancer initiating cells, T regulatory cells, and bone marrow-derived CD34 cells in disease areas including oncology, autoimmune disorders and ischemic repair.
In Roman mythology, the Caladrius is a bird that visits the ill and is said to be able to absorb patients’ illnesses and fly away, dispersing the illness and healing both itself and the patient. In that same spirit of recovery, Caladrius Biosciences is committed to bringing life-improving therapies to market, driving the evolution of the cell therapy industry, and generating industry-leading growth through our significant operational scale, our innovation and our executional excellence.
Since I became CEO in January of 2015, Caladrius has achieved a number of significant milestones. We’ve launched our Phase 3 trial—called the Intus study—for the treatment of stage III recurring or stage IV metastatic melanoma, and received a landmark $17.7 million grant from the California Institute for Regenerative Medicine to help fund the trial. We’ve entered into a collaboration agreement with Sanford Research to develop our T regulatory cell therapy product candidate, CLBS03, for the treatment of adolescents with recent-onset type 1 diabetes (T1D). Our external manufacturing partner subsidiary, PCT, has signed on new clients and expanded relationships with others. We’ve seen a great deal of progress in the company in only the past 10 months, and I am confident that even more progress will come in the months and years ahead.
SNN: What indications is Caladrius currently targeting with your platform technology for “immuno-oncology”?
DM: Our most advanced therapeutic candidate is CLBS20, an investigative treatment for stage III recurrent or stage IV metastatic melanoma. Enrollment for the Phase III trial is taking place now at sites across the country.
Melanoma is the most lethal form of skin cancer. Patients who have progressed to stage IV melanoma have a cancer that has metastasized—or spread—to distant sites in the body such as the lymph nodes, lungs, liver or brain. As a result, advanced melanoma is exceedingly difficult to treat, with a five-year survival rate of approximately 15%.
Treatments for stage IV melanoma are typically directed at slowing the growth of the cancer and prolonging survival. Current treatment options include radiation, chemotherapy, surgical resection, immunotherapy, or a combination approach. Unfortunately, current therapies have had limited impact on long-term survival for most patients. Even with the newer targeted therapies (checkpoint inhibitors and specific enzyme inhibitors), half of patients with metastatic melanoma still die within 13 to 17 months from the start of such treatment. Administering certain anti-melanoma drugs at higher doses or in combinations may be more effective, but often results in more severe side effects.
The product candidate has been granted both Fast Track and Orphan Drug designations from the FDA, and its Phase 3 study protocol is the subject of a Special Protocol Assessment with the FDA. Additionally, the European Medicines Agency has classified CLBS20 as an Advanced Therapeutic Medicinal Product. The safety profile of the candidate shows minimal toxicities, to date, in its two completed Phase 2 trials.
The immune-oncology technology platform is potentially applicable across multiple solid tumor cancers, including ovarian, lung and colon cancers, hepatocellular carcinoma, renal cell carcinoma and glioblastoma multiforme.
SNN: On the development side, how does the company’s platform differ from other melanoma therapies?
DM: This patient-specific cancer immunotherapy is intended to help the immune system target cancer- or tumor-initiating cells (commonly referred to as ‘cancer stem cells’), which are thought to rapidly proliferate cancer cells, fueling tumor growth, and ultimately spreading the disease throughout the body.
CLBS20 uses the patient’s own immune cells and tumor-initiating cells to create a therapeutic vaccine. CLBS20 is one of a few therapies in development targeting these cancer or tumor-initiating cells; other therapies, which target different tumor cells, may treat existing cancer but may not be as likely to prevent tumor recurrence.
The new class of approved melanoma therapies, called checkpoint inhibitors, operate by taking the brakes off of an existing, but suppressed, immune response in the body. CLBS20 potentially creates a new immune response where none exists, and could be synergistic with those checkpoint inhibitors.
Because of the method of CLBS20’s formulation, the immune cells used are loaded with a full range of patient-specific tumor-associated antigens, rather than certain predetermined antigens. This allows the immune system to “see” the target (tumor-initiating cells in this case) more clearly.
SNN: What is the patient/market size for these indications?
DM: There are 20,000 estimated new cases of, and an estimated 10,000 deaths from, metastatic melanoma each year in the United States.
The US market size for melanoma is about $1 billion. For the combination of the other indications I mentioned previously (ovarian, lung and colon cancers, hepatocellular carcinoma, renal cell carcinoma and glioblastoma multiforme), there exists a multibillion-dollar lifecycle opportunity.
SNN: Can you update our audience with regard to the company’s clinical development? What else is in the pipeline?
DM: In addition to our investigational melanoma therapy, in September of this year we entered into a collaboration agreement with Sanford Research to develop our T regulatory cell therapy product candidate, CLBS03, for the treatment of adolescents with recent-onset type 1 diabetes (T1D). The initial focus of the collaboration will be the execution of a clinical trial to evaluate the safety and efficacy of CLBS03 in adolescents. The Phase 2 study (The Sanford Project: Trex Study) has an open and active IND in place and subject enrollment is expected to begin as early as the first quarter of 2016.
CLBS03 is a personalized medicine consisting of each patients’ own regulatory T cells that have been expanded in number and functionally enhanced by a proprietary method developed by our manufacturing subsidiary, PCT.
In addition, Caladrius is actively seeking partnerships to allow for clinical trial execution in Japan (under the country’s new regenerative medicine law) for CLBS12, our experimental therapy for critical limb ischemia, and seeking partnership opportunities in other indications for our platform technologies.
SNN: PCT, LLC, a subsidiary of Caladrius Biosciences – can you describe this side of the business and the synergies with the company’s core business strategy?
DM: Our wholly-owned subsidiary, PCT, is Caladrius Biosciences’ Center of Excellence for process development, engineering and manufacturing of cell therapies. We are providing these services for an impressive and growing list of established cell therapy leaders. We see a significant revenue upside potential as client development-stage projects advance to commercial products.
With proven expertise and more than 16 years of experience, PCT supports the advancement of its clients’ life-changing medical treatments by mitigating the risks of product development and providing efficient, cost-effective, custom manufacturing solutions to advance clinical development programs towards commercialization and beyond.
Specifically, PCT’s areas of expertise include:
- cGMP infrastructure, includingc GMP manufacturing, technology transfer, GTP processing, and logistics
- Innovation, including engineering, consulting, instrumentation, and manufacturing development
To date, PCT has worked with more than 120 clients and more than 20,000 products.
SNN: What is your background?
DM: I come to Caladrius with more than 30 years’ experience in the pharmaceutical industry, amassing international experience in the development of pharmaceutical products. Prior to joining Caladrius Biosciences, I held a number of executive positions in emerging (e.g., Regado Biosciences and Chugai Pharma USA) and big pharma companies (e.g., Schering-Plough, Hoechst Marion Roussel and Rhone-Poulenc Rorer) and played a key role in the development and commercialization of new medicines for the treatment of allergy and inflammatory disorders, oncology and endocrinology, cardiovascular disease, infectious disease and women’s health disorders I also have more than 10 years’ experience serving as an independent Director of public companies, including being a director of Avanir Pharmaceuticals from 2005 until its sale to Otsuka in 2015. During that same period, I have been and continue to be a director and, since 2006, the Chairman of the board of directors of pSivida, Inc.
I earned my BA in the Honors Program (Interdisciplinary Humanities) and my BS in Chemistry from Villanova University. I later received an MS in chemistry and a PhD degree in analytical chemistry from the University of Massachusetts, Amherst, and was a Research Fellow at the Ecole Polytechnique Federale in Lausanne, Switzerland.
For more information about Caladrius Biosciences, Inc., please visit: www.Caladrius.com
Caladrius Biosciences, Inc. had final approval of the content of this article and is wholly responsible for the validity of the statements contained in the article. Opinions expressed are the opinions of the Company and not of StockNewsNow.com or its officers, directors or employees. The interview and the information contained therein does not constitute investment advice by Stock New Now or any of its affiliates. Each reader is encouraged to consult with his or her own advisors before making any investment decisions, which decisions are the sole responsibility of the reader. Our full user terms and conditions, including legal disclaimers, can be found at HERE
© 2017 Stock News Now
Supported by Superior Web Solutions