On May 05, 2016 (GLOBE NEWSWIRE) — Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported financial results for the first quarter ended March 31, 2016 (Press release, Sunesis, MAY 5, 2016, View Source;p=RssLanding&cat=news&id=2165397 [SID:1234512014]). Loss from operations for the three months ended March 31, 2016 was $10.1 million. As of March 31, 2016, cash, cash equivalents and marketable securities totaled $40.0 million.

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"The first quarter of 2016 saw several important milestones, including, notably, the advancement of our unique BTK-inhibitor SNS-062 into the clinic in a Phase 1A study," said Daniel Swisher, Chief Executive Officer of Sunesis. "Resistance to available BTK-inhibitors is a growing concern, and SNS-062’s characteristics as a non-covalently binding inhibitor position it to address this emerging unmet need. Ongoing dose escalation in our Healthy Volunteer Study is proceeding well. We are actively planning for our Phase 1B/2 study in patients with B-cell malignancies to start later this year."

Mr. Swisher added: "We also continue to progress our efforts to bring vosaroxin to market in Europe as an important new treatment option for patients with relapsed/refractory AML. Our Marketing Authorization Application has now reached the 120-day comment and question time point. With continued regulatory advancement and strong outreach efforts underway, we aim to enter into a European collaboration later this year to support a market launch in 2017."

First Quarter 2016 and Recent Highlights

First Subject Dosed in Phase 1A Healthy Volunteer Study Evaluating Oral Non-Covalent BTK-inhibitor SNS-062. In March 2016, the first patient was dosed in a Phase 1A, randomized, double-blind, placebo-controlled dose-ranging study to investigate the safety, pharmacokinetics and pharmacodynamics of its oral, next-generation, non-covalently binding BTK-inhibitor, SNS-062, in healthy subjects. If a successful outcome is achieved in Phase 1A SNS-062 is expected to proceed to a Phase 1B/2 study in patients with B-cell malignancies later this year.

Secured $15 Million Venture Loan. In March 2016, Sunesis entered into a $15 million loan agreement with Bridge Bank, a division of Western Alliance Bank, and Solar Capital Ltd. The loan was used for the repayment of existing indebtedness and will be used for general corporate purposes.

First Patient Treated in Vanderbilt University-Sponsored Phase 2 VITAL Study of Vosaroxin in Combination with Infusional Cytarabine in Patients with Previously Untreated AML. In March 2016, the first patient was treated in an investigator-sponsored study of vosaroxin and cytarabine in adult patients with previously untreated acute myeloid leukemia (AML). The trial is being conducted at the Vanderbilt-Ingram Cancer Center at Vanderbilt University under the direction of Michael R. Savona, M.D., FACP, Associate Professor of Medicine and Director of Hematology Early Therapeutics Program, and Stephen A. Strickland, M.D., MSCI, Assistant Professor of Medicine.

Strengthened Executive Management Team and Board of Directors. In March 2016, Sunesis announced the appointment of Geoffrey Parker to the Sunesis Board of Directors. In February 2016, Sunesis announced the promotion of Deborah A. Thomas, Ph.D., to the role of Senior Vice President, Regulatory Affairs, Quality Assurance, and Non-Clinical Development.

Validation of Marketing Authorization Application by the European Medicines Agency for Vosaroxin in AML. At year-end 2015, the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for vosaroxin as a treatment for relapsed refractory acute myeloid leukemia (AML) in patients aged 60 years and older. Validation confirms that the submission is complete and initiates the Centralized Review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The MAA, if authorized, provides a marketing license valid in all 28 EU member states.

Financial Highlights

Cash, cash equivalents and marketable securities totaled $40.0 million as of March 31, 2016, as compared to $46.4 million as of December 31, 2015. The decrease of $6.4 million was primarily due to $10.7 million of net cash used in operating activities and $8.0 million of principal and final payments against notes payable, partially offset by $12.3 million raised from debt financing. An additional $2.5 million in net loan proceeds was received on April 1, 2016. This capital is expected to be sufficient to fund operations through the second quarter of 2017.

Revenue for the three months ended March 31, 2016 was $0.6 million, as compared to $0.9 million for the same period in 2015. The decrease between the periods was primarily due to the increase in estimated performance period through which the remaining balance of deferred revenue will be amortized.

Research and development expense was $6.2 million for the three months ended March 31, 2016 as compared to $4.5 million for the same period in 2015. The increase between the periods was primarily due to the increase of $1.3 million in consulting and other outside services costs and $0.4 million in clinical trials and medical affairs expenses.

General and administrative expense was $4.3 million for the three months ended March 31, 2016 as compared to $5.1 million for the same period in 2015. The decrease between the periods was primarily due to a $0.8 million decrease in personnel costs and professional services and commercial costs.

Interest expense was $0.3 million for the three months ended March 31, 2016, compared to $0.2 million for the same period in 2015.

Net other income was nil for the three months ended March 31, 2016 as compared to net other expense of $0.1 million for the same period in 2015. The amount for the period in 2015 was primarily comprised of non-cash credits or charges for the revaluation of warrants issued in the October 2010 underwritten offering.

Cash used in operating activities was $10.7 million for the three months ended March 31, 2016, including a $0.5 million milestone payment relating to the MAA filing, as compared to $11.6 million for the same period in 2015.

Sunesis reported loss from operations of $9.9 million for the three months ended March 31, 2016 as compared to $8.8 million for the same period in 2015. Net loss was $10.1 million for the three months ended March 31, 2016, as compared to $9.1 million for the same period in 2015.

Conference Call Information

Sunesis will host an update conference call today, May 5th at 11:00 a.m. Eastern Time. The call can be accessed by dialing (877) 771-6242 (U.S. and Canada) or (440) 996-5676 (international) and entering passcode 96476414. To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at www.sunesis.com. The webcast will be recorded and available for replay on the company’s website for two weeks.

About QINPREZO (vosaroxin)

QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed or refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.

The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.

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.