Caladrius Biosciences, Inc. (NASDAQ: CLBS) ("Caladrius"), a cell therapy company combining an industry-leading development and manufacturing services provider (through its subsidiary, PCT, LLC a Caladrius CompanyTM ("PCT")) with a select therapeutic development pipeline, announces financial results for the three months ended March 31, 2016.

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Financial and Business Highlights

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Achieved total revenues of $7.5 million for the first quarter of 2016, up 136% compared with $3.2 million in the first quarter of 2015 driven by higher Clinical Services revenue at PCT.
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Entered into a global collaboration and license agreement with Hitachi Chemical Co. America, Ltd. and Hitachi Chemical Co., Ltd. (collectively, "Hitachi Chemical"), selling a 19.9% equity stake in PCT for $19.4 million and licensing PCT’s cell therapy technology and know-how for certain Asian territories for $5.6 million and future royalties.
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Enrolled the first patient in The Sanford Project: T-Rex Study, a Phase 2 trial of CLBS03 (autologous expanded regulatory T cells, or Tregs) for the treatment of recent-onset type 1 diabetes (T1D) in adolescents.
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Received classification from the European Medicines Agency (EMA) of CLBS03 as an Advanced Therapeutic Medicinal Product (ATMP).
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Reached agreement with Japanese regulators on a Phase 2 development plan that could qualify for early conditional approval for CD34 cell therapy as a treatment for critical limb ischemia.

Management Commentary

"The significant revenue growth at PCT along with Hitachi Chemical’s implied valuation of our subsidiary further support our strategy to focus on growth opportunities in the emerging cell therapy manufacturing market," stated David J. Mazzo, PhD, Chief Executive Officer of Caladrius. "In addition to validating our expertise and know-how, the strategic partnership with Hitachi Chemical strengthens our financial position with $25 million in non-dilutive capital.

"We will continue to leverage the significant momentum in the regenerative medicine and cell therapy industries to grow our PCT business. We believe that the quality of PCT’s services, the increasing number of clinical trials planned and underway and the number of clinical programs nearing commercialization will provide a healthy platform for growth at PCT throughout 2016 and beyond.

"We are delighted that patients are being enrolled in the Sanford Project: T-Rex Study. Sanford Research, a leader of innovation and research in T1D, will provide and cover the costs of two initial clinical trial sites, which are expected to enroll most of if not all of the first cohort of subjects. After this first cohort has completed the three-month post-treatment visit, an interim safety analysis and early analysis of immunological biomarkers will be conducted. With positive results, more sites will be added to facilitate the timely enrollment of the second cohort of this important proof-of-concept study designed to show that CLBS03 can preserve pancreatic beta cell function and lower insulin use in adolescents with recent-onset T1D," concluded Dr. Mazzo.

First Quarter Financial Highlights

Total revenues for the first quarter of 2016 increased 136% to $7.5 million compared with $3.2 million for the first quarter of 2015. Gross margin on revenues was 17% in the first quarter of 2016, compared with gross margin of negative 6% in the first quarter of 2015.

Research and development (R&D) expenses for the first quarter of 2016 decreased 14% to $5.9 million compared with $6.8 million for the first quarter of 2015. The decrease was primarily related to lower costs subsequent to the discontinuation of Caladrius’ Intus Phase 3 clinical trial as well as decreased costs associated with our ischemic repair platform, compared to the prior year periods. These decreases were partially offset by an increase in expenses related to the initiation of The Sanford Project: T-Rex Phase 2 Study in type 1 diabetes, as well as one-time restructuring costs for severance and asset impairments.

Selling, general and administrative (SG&A) expenses decreased 42% to $6.5 million for the first quarter of 2016 compared with $11.1 million for the same period in 2015, which included expenses associated with executive management changes including one-time new hire compensation-related costs as well as separation-related costs. Equity-based compensation expenses were also significantly lower in the first quarter of 2016 compared to the prior year period.

The operating loss for the first quarter of 2016 was $11.1 million compared with an operating loss of $18.1 million for the first quarter of 2015, reflecting higher gross margin on sales, and lower R&D and SG&A expenses.

Total net loss for the first quarter of 2016 was $12.0 million, and $0.21 per share for Caladrius stockholders, compared with a net loss for the first quarter of 2015 of $19.2 million and $0.51 per share.

Balance Sheet and Cash Flow Highlights

As of March 31, 2016, Caladrius had cash and cash equivalents of $25.4 million. The cash and cash equivalents balance includes the receipt of $22.5 million from the Hitachi Chemical transaction and the payment of $7.0 million to Oxford Finance LLC for repayment of long-term debt, interest and fees. The net cash used in operating activities in the first quarter of 2016 was $8.0 million, compared with $14.2 million in the first quarter of 2015. During the current quarter, Caladrius also invested $1.0 million in capital expenditures primarily related to improvements to PCT’s Allendale, N.J. manufacturing facility.

2016 Financial Guidance

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Consolidated Revenues: exceed $30 million (greater than 30% increase compared with 2015) (guidance reaffirmed)

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Capital Improvements at PCT’s Allendale, NJ facility: ~$6 million in 2016 (guidance reaffirmed)

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CLBS03 Phase 2 Study Costs in 2016: $6 million to $7 million (guidance reaffirmed)

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Consolidated Annual Operating Cash Burn: $25 million to $28 million (new guidance)
(lower operating cash burn in the second half of 2016 than in the first half of the year)

On May 05, 2016 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported financial results for the quarter ended March 31, 2016 (Press release, Insmed, MAY 5, 2016, View Source [SID:1234512005]).

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Business Update

Global Phase 3 CONVERT study advancing. Patient enrollment continues to proceed on track in the company’s global phase 3 study of ARIKAYCE (liposomal amikacin for inhalation or LAI) in nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC) (CONVERT or INS-212 study). The CONVERT study is taking place in 16 countries and at more than 130 sites. The company continues to expect to achieve its enrollment objective in the second half of 2016.
EMA regulatory review of ARIKAYCE progressing. The company remains on track with previous guidance, having submitted its responses to the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) 180-day list of outstanding issues related to the company’s Marketing Authorization Application (MAA) for ARIKAYCE. Insmed expects to participate in an oral explanation meeting in the second quarter of 2016 and the CHMP to render an opinion on its MAA around the middle of 2016.

Data accepted for presentation at ATS 2016. Three ARIKAYCE-related abstracts and one treprostinil prodrug abstract have been accepted for presentation at the American Thoracic Society (ATS) 2016 International Conference taking place May 13-18 in San Francisco. The ATS presentations include (i) one-year follow-up data from the phase 2 112 study, (ii) lung distribution and retention data from a scintigraphy study in patients with NTM lung disease, (iii) a preclinical study of ARIKAYCE, and (iv) a preclinical study of a variety of treprostinil prodrugs.

Phase 1 clinical study of INS1009 submitted for presentation. Insmed has completed a phase 1 study of INS1009 and submitted the results for presentation at a future medical meeting. This first-in-human study of INS1009 was designed to determine the maximum-tolerated dose of a single dose of INS1009 and to characterize the pharmacokinetic profile of free treprostinil and INS1009 in healthy volunteers. INS1009 is one of the company’s nebulized treprostinil prodrugs, which may offer a differentiated product profile with therapeutic potential in rare pulmonary disorders such as pulmonary arterial hypertension (PAH), idiopathic pulmonary fibrosis (IPF), sarcoidosis, and severe refractory asthma.

Canadian patent strengthens global patent portfolio. The Canadian Intellectual Property Office issued patent no. 2,838,111, which covers pharmaceutical formulations that include mixtures of liposomal quinolone antibiotics, together with free, unencapsulated quinolone antibiotics, such as ciprofloxacin. The patent also covers the use of such formulations for the treatment of various pulmonary disorders, for example, in bronchiectasis patients. The Canadian patent complements ARIKAYCE’s global intellectual property estate. Counterpart patent applications to this Canadian patent are pending in other countries.

"2016 is off to a solid start with all of our clinical, regulatory, and commercial-readiness activities remaining on track with our previously stated timelines," said Will Lewis, president and chief executive officer of Insmed. "Our top corporate priority is our global phase 3 CONVERT study and we look forward to achieving our patient enrollment objective later this year. In parallel with our clinical activities, our team is advancing the regulatory process for ARIKAYCE in Europe. For INS1009, we completed the phase 1 study and submitted the results for presentation at an international respiratory congress in the third quarter. Lastly, our talented team of scientists remain focused on advancing a number of preclinical programs and identifying our next candidates for clinical development."

First Quarter Financial Results

For the first quarter of 2016, Insmed posted a net loss of $33.5 million, or $0.54 per share, compared with a net loss of $27.4 million, or $0.55 per share, for the first quarter of 2015.

Research and development expenses were $20.5 million for the first quarter of 2016, compared with $17.2 million for the first quarter of 2015. The increase was primarily due to the advancement of the company’s global phase 3 CONVERT study of ARIKAYCE in NTM lung disease.

General and administrative expenses for the first quarter of 2016 were $12.5 million, compared with $9.5 million for the first quarter of 2015. The increase was primarily related to pre-commercial activities in Europe, namely the buildout of the company’s infrastructure and NTM disease awareness activities, as well as an increase in headcount and related expenses.

Balance Sheet Highlights and Cash Guidance

As of March 31, 2016, Insmed had cash and cash equivalents of $253 million. Excluding depreciation and stock-based compensation expense, the company’s cash operating expenses for the quarter ended March 31, 2016 were $28 million. Insmed ended the first quarter of 2016 with $25 million in debt and working capital of $233 million.

The company is investing in the following activities in 2016: (i) clinical development of ARIKAYCE, (ii) regulatory and pre-commercial initiatives for ARIKAYCE, and (iii) preclinical and clinical activities for its earlier-stage pipeline. Insmed continues to expect its cash-based operating expenses for the first half of 2016 to be in the range of $58 to $68 million.

On May 5, 2016 Heron Therapeutics, Inc. (NASDAQ:HRTX), a biotechnology company focused on improving the lives of patients by developing best-in-class medicine that address major unmet medical needs, reported first quarter 2016 financial results and highlighted recent corporate progress (Press release, Heron Therapeutics, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165669 [SID:1234512002]).

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Recent Corporate Progress:
On April 18, 2016, Heron announced that the U.S. Food and Drug Administration (FDA) has indicated that there are no substantive deficiencies in the New Drug Application (NDA) for SUSTOL (granisetron) Injection, extended release, Heron’s lead product candidate for the prevention of chemotherapy-induced nausea and vomiting (CINV) in cancer patients, and has begun labeling discussions with the Company.

Heron has continued to implement a broad-based Phase 2 clinical program of HTX-011, its lead product candidate for the prevention of post-operative pain. In February 2016, Heron initiated a Phase 2 clinical trial of HTX-011 in patients undergoing abdominoplasty, and in April 2016, Heron initiated a Phase 2 clinical trial of HTX-011 in patients undergoing bunionectomy. In addition, the Company continues to enroll patients in an ongoing Phase 2 clinical trial in patients undergoing inguinal hernia repair.
"We continue to work closely with the FDA on our NDA for SUSTOL and look forward to bringing this important therapeutic to patients suffering from CINV," commented Barry D. Quart, Pharm.D., Chief Executive Officer of Heron Therapeutics. "We also continue to make important progress in our HTX-011 post-operative pain program, including the recent initiation of our fourth Phase 2 study of HTX-011. We look forward to reporting top-line data from our ongoing studies, beginning with data from our study in inguinal hernia repair, which we expect late in this quarter."

Results of Operations
As of March 31, 2016, Heron had approximately $100.4 million in cash, cash equivalents and short-term investments, compared to $131.2 million as of December 31, 2015. The net decrease in cash, cash equivalents and short-term investments was primarily due to net cash used in operating activities in the first quarter of 2016. Based on current operating plans and projections, Heron believes that its current working capital is sufficient to fund operations through 2016.

Heron’s net cash used for operating activities for the quarter ended March 31, 2016 was $32.4 million compared to net cash used for operating activities of $19.7 million for the same period in 2015.

Heron’s net loss for the quarter ended March 31, 2016 was $33.4 million, or $0.92 per share, compared to a net loss of $20.6 million, or $0.70 per share, for the same period in 2015.

The increase in net cash used for operating activities and net loss in the first quarter of 2016 as compared to the same period in 2015 were primarily due to costs incurred in preparation for the commercial launch of SUSTOL, as well as clinical and manufacturing costs related to our Phase 2 clinical studies for HTX-011 and costs associated with the development of HTX-019.

On May 05, 2016 Geron Corporation (Nasdaq:GERN) reported financial results for the first quarter ended March 31, 2016 and recent events (Press release, Geron, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165745 [SID:1234512001]).

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First Quarter 2016 Results

For the first quarter of 2016, the company reported operating revenues of $749,000 and operating expenses of $9.8 million compared to $537,000 and $10.0 million, respectively, for the comparable 2015 period. Revenues for the first quarter of 2016 and 2015 included royalty and license fee revenues under various non-imetelstat agreements. Operating expenses in the first quarter of 2015 included restructuring charges of $406,000 in connection with the company’s organizational resizing announced in March 2015. Net loss for the first quarter of 2016 was $8.8 million, or $0.06 per share, compared to $9.3 million, or $0.06 per share, for the comparable 2015 period. The company ended the first quarter of 2016 with $141.9 million in cash and investments.

Research and development expenses for the first quarter of 2016 and 2015 were $5.0 million for each period. Research and development expenses for 2016 primarily reflect the net result of higher costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen Biotech, Inc. (Janssen), partially offset by lower personnel related expenses as a result of the 2015 organizational resizing. The company expects research and development expenses to increase during the remainder of the year as the clinical development of imetelstat continues in collaboration with Janssen.

General and administrative expenses for the first quarter of 2016 were $4.8 million compared to $4.6 million for the comparable 2015 period. The increase in general and administrative expenses primarily reflects the net result of higher non-cash stock-based compensation expense, partially offset by lower personnel related costs due to the 2015 organizational resizing.

Interest and other income for the first quarter of 2016 was $256,000 compared to $149,000 for the comparable 2015 period. The increase in interest and other income in 2016 compared to 2015 primarily reflects higher yields on the company’s marketable securities portfolio.

Recent Company Events

Clinical Development by Janssen

In January 2016, the first patient was dosed in a Phase 2/3 clinical trial to evaluate imetelstat in patients with myelodysplastic syndromes (MDS). The trial, referred to as IMergeTM, will evaluate imetelstat in transfusion dependent patients with International Prognostic Scoring System (IPSS) Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA).

This is the second clinical study to be initiated under the terms of the exclusive worldwide imetelstat license and collaboration agreement between Geron and Janssen. Further information about the trial can be found at View Source

Presentations and Publications

Clinical data on imetelstat safety and efficacy from patients with a form of MDS known as refractory anemia with ring sideroblasts (MDS-RARS) enrolled as part of the Mayo Clinic Pilot Study were published online in the Blood Cancer Journal in March. The full text paper is available online at View Source

The data, previously presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2015, included nine patients enrolled in the study cohort, classified as having either IPSS intermediate-1 or intermediate-2 risk disease. Six of nine (66.7%) patients had prior treatment with ESAs. Three of the eight (37.5%) patients who were dependent on red blood cell transfusions at study entry became transfusion independent, defined as not requiring transfusions for at least eight weeks. The median duration of transfusion independence was 28 weeks (range: nine weeks to 37 weeks).

Two poster presentations by Janssen and academic collaborators describing non-clinical data on imetelstat were made at the 2016 annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in April:

Impact of hypomethylating agents on hTERT expression and synergistic effect in combination with imetelstat, a telomerase inhibitor, in AML cell lines (Abstract #2731)

Data presented described non-clinical results that treating acute myeloid leukemia (AML) cell lines with imetelstat enhanced the effects of agents currently used for the treatment of AML. These data extend the rationale from prior non-clinical studies for the potential use of imetelstat in hematologic myeloid malignancies, such as AML, including in combination with standard therapies.

Myelosuppression in patients treated with the telomerase inhibitor imetelstat is not mediated through activation of toll-like receptors (Abstract #2732)

Data presented described results from non-clinical studies that provide further evidence for potential on-target mechanisms of telomerase inhibition by imetelstat underlying the reduction in platelets observed in previously conducted imetelstat clinical trials.

On May 05, 2016 Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing T cell-directed vaccines and immunotherapies, reported corporate highlights and financial results for the first quarter ended March 31, 2016 (Press release, Genocea Biosciences, MAY 5, 2016, View Source [SID:1234511999]).

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"Our positive GEN-003 Phase 2 12-month efficacy data suggests that annual treatment with GEN-003 may offer genital herpes patients similar benefits to a full year of daily administration of oral antivirals. These data give us confidence in the potential of GEN-003 to become the cornerstone therapy for patients suffering from genital herpes," said Chip Clark, president and chief executive officer of Genocea. "For our recently initiated Phase 2b efficacy trial, we anticipate reporting virologic efficacy data in the third quarter of 2016, which we anticipate will confirm the efficacy of GEN-003 manufactured using improved processes at an increased scale. We expect clinical efficacy data versus placebo against potential Phase 3 endpoints at six months post dosing around the end of 2016 and we expect to conduct our end-of-Phase 2 meeting with the FDA in the first quarter of 2017. We also continue to advance our academic collaborations in the immuno-oncology field and expect to provide further updates in the coming months on our potential path to the clinic in 2017 for a neoantigen-based cancer vaccine."

Recent Business Highlights and Anticipated Milestones

GEN-003 – Immunotherapy for treatment of genital herpes in Phase 2 development. Greater than $1 billion potential revenue opportunity in U.S. alone

• Reported positive 12-month efficacy data from Phase 2 dose optimization trial
• Full data to be presented at an upcoming scientific meeting

In March 2016, Genocea announced positive 12-month efficacy data from its Phase 2 dose optimization trial evaluating GEN-003 for the treatment of genital herpes. GEN-003 demonstrated statistically significant reductions compared to baseline in the rate of viral shedding 12 months after dosing as well as sustained efficacy across secondary clinical endpoints, in each case across multiple dose groups. GEN-003 was safe and well tolerated by patients, with no serious adverse events reported related to the vaccine in the trial.

Multiple anticipated 2016 clinical milestones for GEN-003

• Phase 2b virologic efficacy data expected in third quarter of 2016
• Phase 2b 6-month clinical efficacy data expected in late 2016
• End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) expected in Q1 2017

In the third quarter of 2016, Genocea expects to report virologic efficacy data from a Phase 2b trial to confirm the efficacy of GEN-003 manufactured with Phase 3 processes at increased scale. Around the end of 2016, Genocea expects to report 6-month clinical efficacy versus placebo against the potential Phase 3 endpoint of the percentage of patients that are lesion free at 6-months after dosing. Positive data would provide an important step toward the end-of-Phase 2 meeting with the FDA, which is expected to occur in the first quarter of 2017.

This ongoing trial has enrolled approximately 135 subjects across the U.S. who have a history of recurrent genital herpes. Subjects will be randomized to one of three dose groups – placebo, 60µg per protein / 50µg of adjuvant, and 60µg per protein / 75µg of adjuvant – and will be monitored for 12 months.

Genocea also expects to commence a planned Phase 2b antiviral combination study in the second half of 2016. Clinical efficacy data from this trial is expected in the first half of 2017. If GEN-003 is additive to the effect of chronic suppressive oral anti-viral therapy, this would further strengthen GEN-003’s value proposition to patients and physicians.

First Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and investments as of March 31, 2016 were $95.7 million compared to $106.4 million as of December 31, 2015. Genocea expects that these funds will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2017.
Research and Development (R&D) Expenses: R&D expenses for the quarter ended March 31, 2016 decreased $1.2 million, to $7.3 million, from the same period in 2015. The decrease was driven by lower clinical costs due to the completion of the GEN-004 Phase 2a trial, which was ongoing in the first quarter of 2015, and the conduct of a smaller Phase 2 trial for GEN-003 in the first quarter of 2016 compared to the same period in 2015. GEN-003 manufacturing costs also decreased due to the timing of activities in support of clinical trial supply. These lower costs were partially offset by higher personnel and lab-related costs to advance Genocea’s pre-clinical product candidates and develop the ATLAS platform for immuno-oncology.
General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2016 were $3.9 million, compared to $3.4 million for the same period in 2015. The increase reflects higher personnel costs, consulting and professional fees, and depreciation expense, all of which support Genocea’s expanding R&D operations.
Refund of Research and Development Expense: A gain of $1.6 million for the quarter ended March 31, 2016 resulted from cash received pursuant to contractual obligations under a collaboration agreement with Isconova AB ("Isconova") (since acquired by Novavax, Inc.) to refund R&D expenses paid by Genocea to Isconova between 2009 and 2011 relating to the development of the Matrix-M2TM adjuvant technology.
Net Loss: Net loss was $9.8 million for the quarter ended March 31, 2016, compared to a net loss of $12.1 million for the same period in 2015.