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Caladrius Tightens Strategic Focus and Provides 2016 Revenue Guidance

On January 6, 2016 Caladrius Biosciences, Inc. ("Caladrius" or the "Company") (NASDAQ: CLBS), a cell therapy company combining an industry-leading development and manufacturing services provider with a therapeutic development pipeline, announces an increased focus of its strategic priorities and provides 2016 revenue guidance based on growth at its PCT subsidiary.

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Following a comprehensive review of the Company’s existing operations and development pipeline, as well as an updated assessment of current market dynamics, current and expected future competitive therapies, and the Company’s financial resources, Caladrius has decided to shift greater focus and resources to its growing cell therapy process development, optimization and manufacturing services business at its PCT subsidiary. The rapidly developing cell therapy industry, with several cell-based therapies approaching market approvals which are expected to generate additional demand for commercial manufacturing infrastructure, along with PCT’s continued trend of strong revenue growth, supports the Company’s commitment to focus on growth opportunities for PCT. The Company has also reconfirmed its commitment to pursue further development of its immune modulation platform, with a Phase 2 proof-of-concept clinical trial for a T regulatory cell therapy as the primary focus, while choosing to discontinue the current Phase 3 study of CLBS20 as monotherapy for metastatic melanoma.

"Moving forward, we strongly believe PCT represents a compelling opportunity for near- and long-term shareholder value creation and we intend to continue to invest resources in expanding that business, where we are already experiencing noteworthy year-over-year revenue growth," stated David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius.
"For nearly 17 years, PCT has been an integral partner to the regenerative medicine industry by leveraging its cell therapy-focused, bicoastal development and manufacturing infrastructure to support biotechnology and cell therapy companies," said Robert A. Preti, Ph.D., Chief Technology Officer of Caladrius and President of PCT. "We continue to focus on the design and implementation of sustainable, scalable, reliable and well-controlled manufacturing processes with optimized cost-of-goods as these are all critical success parameters in bringing new cell therapy and immunotherapy treatments to market. We are looking forward to further acceleration of these activities."
Dr. Mazzo continued, "The treatment paradigm in metastatic melanoma was transformed during the course of 2015 by the accelerating adoption of multiple immune checkpoint inhibitors used as monotherapy and in combination treatments. These new drugs have significantly improved outcomes in metastatic melanoma and therefore have altered the opportunity for a monotherapy such as CLBS20 in a landscape that is quickly converting to combination therapies. Therefore, we have concluded that, as designed, our current program in metastatic melanoma will not optimally leverage this asset and we will therefore discontinue the ongoing Phase 3 clinical study with CLBS20 as a monotherapy for the treatment of recurrent Stage III or Stage IV metastatic melanoma. As a result, we will reduce associated staff by approximately 40 employees at our Irvine, California facility. That said, we continue to believe in the potential of CLBS20 as a life-prolonging immunotherapeutic and will pursue licensing or partnership opportunities for its continued development as part of a combination therapy and in different oncology indications. The emphasis will be on collaborating with a company that will allow us to exploit the novel antigen presentation and T cell activation approach of CLBS20."

On the development front, Caladrius will focus its efforts on its T regulatory (Treg) cell therapy product candidate, CLBS03. CLBS03 is based on the Company’s novel immune modulation approach that seeks to restore immune balance by enhancing Treg cell number and function. The Company is planning to commence enrollment in a Phase 2 study for adolescents with recent-onset type 1 diabetes in the first quarter of 2016, in collaboration with Sanford Research, a non-profit research organization that supports an emerging translational research center focused on finding a cure for type 1 diabetes.

"The opportunity provided by polyclonal T cells in the treatment of autoimmune diseases is compelling and we are excited to be at the forefront of this technology’s development and to be working with recognized leaders in the field, such as Drs. Jeffrey Bluestone and Stephen Gitelman of the University of California, San Francisco and Dr. Kevan Herold of Yale University. We believe CLBS03 has the potential to be paradigm-changing in the treatment of recent-onset diabetes and, potentially, other autoimmune diseases. We look forward to initiating our Phase 2 clinical program in conjunction with Sanford Research in the first quarter of 2016," concluded Dr. Mazzo.

Financial Guidance
For 2015, Caladrius expects total revenue to be approximately $23 million, representing an increase of approximately 28% compared with 2014. For 2016, Caladrius expects total revenue to exceed $30 million, representing an increase of greater than 30% compared with the expected results for 2015. In order to accommodate this projected growth, Caladrius has budgeted to spend $6 million in capital improvements to increase PCT’s Allendale, NJ clean room capacity by 60%, and expects to complete the build-out in 2016. Caladrius also estimates that it will incur restructuring charges of approximately $1.0 million in connection with one-time employee termination costs, including severance and other benefits, in the first quarter of 2016. The Company estimates that the staff reduction will result in over $4 million in annualized compensation-related savings, and anticipates significant cost savings associated with terminating the CLBS20 study, which had been estimated to cost $35 million through its completion. In addition, with a narrowed focus on research and development initiatives, as well as a re-sizing of the Company’s general and administrative infrastructure, Caladrius expects to lower R&D, G&A and overall cash burn in 2016 compared to 2015. The Company also expects to incur significant non-cash intangible asset and goodwill impairment charges associated with the termination of the CLBS20 study and will assess the impact as of December 31, 2015 during its annual intangible asset impairment review process.

Aslan Pharma gets global rights for ASLAN003 compound

On January 6, 2016 Singapore-based biotech firm Aslan Pharmaceuticals (Nasdaq: ARRY) reported under a new agreement with partner Spanish pharma company Almirall (ALM: MC) it now has gained rights to develop and market its compound ASLAN003 (Press release, ASLAN Pharmaceuticals, JUN 6, 2016, View Source [SID:1234512875]).

Under the terms of the broadened deal, Almirall has granted Aslan global rights to develop and market ASLAN003 for all non-topical and non-dermatological indications including oncology.

Dr Mark McHale, chief operating officer of Aslan, said: "ASLAN003 is an exciting compound and the promising preclinical data we have observed in various tumor models suggest that it has the potential to become an important novel therapy in the emerging area of cancer metabolism with application to a range of different tumor types. This broadened agreement is a strong endorsement of the success of our collaborative work with Almirall to date and is reflective of both teams’ confidence in each other’s complementary expertise in the research and development of new medicines."

Almirall is eligible to receive development and regulatory based milestone payments and tiered-royalty payments on global sales of ASLAN003 upon successful commercialisation.

ASLAN003 is a small molecule inhibitor of DiHydroOrotate DeHydrogenase (DHODH), an enzyme which catalyses the key rate-limiting step in the synthesis of pyrimidines in mammalian cells, originally licensed to Aslan in 2012 as a treatment for rheumatoid arthritis.

SRI Biosciences and Stanford Cancer Institute Launch Drug Discovery Program

On January 6, 2016 The Stanford Cancer Institute (SCI) and SRI Biosciences, a division of the independent research center SRI International, reported that they are partnering up to enhance drug development efforts in response to a lack of innovative new treatments for cancer and other diseases (Press release, SRI International, JAN 6, 2016, View Source [SID:1234508782]).

According to PR Newswire, this collaboration, which is being called the SRI Biosciences-Stanford Drug Discovery and Development Program, brings together scientists from SRI and researchers and physicians from SCI to pursue the development of new compounds that can lead to cures.
The partnership builds on previous collaborations between the two institutions. For example, the development of Tirapazamine, an experimental cancer therapy, was made possible by the teamwork of SRI and SCI researchers.

Researchers and experts from both institutions see the program as the ideal support system to coordinate and catalyze diverse efforts to discover and refine novel substances that can be used for treatment. The program will provide access to necessary infrastructure that promotes understanding of disease mechanisms, drug discovery and drug development.

The Drug Discovery and Development Program will be coordinated by Sanjay V. Malhotra, associate professor of radiation oncology at Stanford, and Nathan Collins, executive director of the Pharmaceutical and Chemical Technologies Section in SRI Biosciences.

"The SCI-SRI Biosciences collaboration provides a fully integrated engine for taking ideas to the investigational new drug (IND) stage and beyond," Collins said. "Our focus is on developing ‘first-in-class’ drugs and delivering improved outcomes for patients."

Akebia Prices Public Offering of Common Stock

On January 6, 2016 Akebia Therapeutics, Inc. (NASDAQ:AKBA) reported the pricing of an underwritten public offering of 7,250,000 shares of common stock at a price to the public of $9.00 per share (Press release, Akebia , JAN 6, 2016, View Source [SID:1234508692]). In addition, Akebia has granted the underwriters a 30-day option to purchase up to an additional 1,087,500 shares of common stock in connection with the offering. All shares are being sold by Akebia. The net proceeds of the offering are expected to be approximately $61.0 million (or approximately $70.2 million if the underwriters exercise their option to purchase additional shares in full), after deducting underwriting discounts and commissions and other estimated offering expenses payable by Akebia. The offering is expected to close on January 12, 2016, subject to the satisfaction of customary closing conditions.

Morgan Stanley and UBS Investment Bank are acting as joint book-running managers for the offering. JMP Securities is acting as lead manager, and Needham & Company and Brean Capital are acting as co-managers for the offering.

Akebia intends to use the net proceeds from the offering to fund continued clinical development of vadadustat in patients with anemia secondary to chronic kidney disease (CKD), including to prepare, initiate and conduct its PRO2TECT Phase 3 program and to prepare and initiate its planned INNO2VATE Phase 3 program, to advance AKB-6899 through Phase 1 development in oncology, and the remainder for working capital and other general corporate purposes.

The shares of common stock described above are being offered by Akebia pursuant to its shelf registration statement on Form S-3 previously filed and declared effective by the Securities and Exchange Commission (SEC). The offering is being made only by means of a free writing prospectus, prospectus supplement and accompanying prospectus, which have been filed with the SEC. You may obtain these documents for free by visiting the SEC’s website at www.sec.gov. Copies of the free writing prospectus, prospectus supplement and accompanying prospectus relating to the offering may also be obtained, when available, from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014 or UBS Securities LLC, Attention: Prospectus Department, 1285 Avenue of the Americas, New York, NY 10019.

This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in the offering, nor shall there be any sale of these securities in any jurisdiction in which an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such jurisdiction.

Navidea Biopharmaceuticals Enrolls First Patient in Pediatric Trial of Lymphoseek®

On January 6, 2016 Navidea Biopharmaceuticals, Inc. (NYSE MKT: NAVB), reported that the first pediatric patient was enrolled in a clinical study comparing Lymphoseek (technetium Tc 99m tilmanocept) injection and vital blue dye (VBD) in a pediatric population of patients with melanoma, rhabdomyosarcoma, or other solid tumors (Press release, Navidea Biopharmaceuticals, JAN 6, 2016, View Source;p=RssLanding&cat=news&id=2126657 [SID:1234508674]). The study is designed to investigate how Lymphoseek compares with VBD in identifying lymph nodes as well as evaluate safety and tolerability in the pediatric population. Lymphoseek is designed for the precise identification of lymph nodes that drain from a primary tumor, which have the highest probability of harboring cancer and is approved for adult use only. Enrollment is currently planned at approximately six sites throughout the U.S. The first patient has been enrolled by the Nationwide Children’s Hospital in Columbus, OH.

"We are pleased to participate in this important clinical study of Lymphoseek in the pediatric patient population where no lymph node mapping agents have yet been approved," said Jennifer Aldrink, M.D., Assistant Professor of Clinical Surgery, The Ohio State University College of Medicine and Director of Surgical Oncology, Division of Pediatric Surgery at Nationwide Children’s Hospital in Columbus, OH. "Intraoperative Lymphatic Mapping (ILM) and Sentinel Lymph Node Biopsy (SLNB) are standards of care in adult and pediatric patients in several forms of cancer, and Lymphoseek is a new agent being used in many adult centers as an alternative to sulfur colloid formulation. Lymphoseek may have the potential to aid physicians in evaluating lymph nodes in children that are necessary for accurate disease staging and optimal post-surgical treatment."

"This study will provide further data on the overall clinical value of Lymphoseek, which has already shown to be a highly effective immunodiagnostic tool in adult patients. Medical literature supports the importance of lymph node evaluations in pediatric patients with rhabdomyosarcoma and melanomas noting that lymph node metastases are highly associated with poorer survival," said Michael Tomblyn, M.D., Chief Medical Officer of Navidea. "Until now, there have been few studies of ILM and SLNB in children. We look forward to the opportunity to evaluate Lymphoseek’s use in pediatric populations."

This study (NAV3-18) is a prospective, open-label, multicenter study comparing Lymphoseek and VBD as lymphoid tissue targeting agents in pediatric patients with melanoma, rhabdomyosarcoma, or other solid tumors who are undergoing lymph node mapping. Primary goals of this study are to evaluate safety and tolerability of Lymphoseek in this subject population and determine the concordance of in vivo detection rates of Lymphoseek and of VBD in tissue excised and histologically confirmed as lymph nodes. In addition, the study will measure other efficacy signals including assessment of the identified lymph node(s) to confirm: the presence/absence of tumor metastases; agent localization per tumor type; degree of localization (nodes per subject both intraoperatively and with preoperative SPECT/CT); reverse concordance parameters; change of subject stage based on histopathology and descriptive assessment on change in treatment plan; number of lymph nodes detected with Lymphoseek intraoperatively compared with preoperative SPECT/CT imaging.