On March 14, 2016 Oncothyreon Inc. (NASDAQ:ONTY), a clinical-stage biopharmaceutical company dedicated to the development of therapeutic products that can improve the lives and outcomes of patients with cancer, reported a corporate update and reported financial results for the year and quarter ended December 31, 2015 (Press release, Oncothyreon, MAR 14, 2016, View Source [SID:1234509544]).

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"2015 was a significant year for Oncothyreon, with the company reporting encouraging data from two combination trials of ONT-380 demonstrating that this product candidate may be impactful on HER2-positive breast cancer patients, including those with brain metastases – a patient population in desperate need of new treatment options," said Christopher S. Henney, Chairman and interim CEO of Oncothyreon. "We believe our clinical results to date provided a strong foundation for us to advance ONT-380 into our recently initiated randomized, double-blind, placebo-controlled Phase 2 trial in combination with Herceptin and Xeloda, which includes enrolling patients with progressing central nervous system disease."

Corporate Update & Recent Highlights

Clinical Development:

ONT-380 Phase 2 Combination Trial Underway in Patients with HER2-Positive Breast Cancer. The randomized, double-blind, placebo control trial is evaluating ONT-380 in combination with Herceptin (trastuzumab) and Xeloda (capecitabine). ONT-380 is an oral, HER2-selective, central nervous system (CNS)-active tyrosine kinase inhibitor. The trial is targeted to enroll approximately 180 heavily pretreated patients with advanced HER2-positive breast cancer who present with or without brain metastases. Building on encouraging Phase 1b results, the primary and secondary endpoint objectives are designed to measure ONT-380’s contribution on both systemic and CNS disease, an area of unmet need for patients.

Data from Ongoing ONT-380 Phase 1b Combination Trials Show Objective, Durable Responses and Favorable Tolerability Profile. During 2015, data from two ongoing trials of ONT-380 were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the San Antonio Breast Cancer Symposium (SABCS).

ONT-380 in combination with Xeloda and/or Herceptin:
This Phase 1b trial enrolled patients with metastatic HER2-positive breast cancer with progression following prior treatment with Herceptin and Kadcyla (ado-trastuzumab emtansine or T-DM1). In addition to patients without a prior history of CNS disease, patients with untreated, asymptomatic CNS disease and patients with progressing CNS disease after prior local therapy were allowed to enroll in the trial and were followed for responses both systemically and in the CNS. Overall, ONT-380 in combination with Xeloda, Herceptin or both Xeloda and Herceptin has been well tolerated. As reported at ASCO (Free ASCO Whitepaper) 2015, in a total of 32 patients treated with these combinations, the majority of adverse events were Grade 1 or 2 in severity, with no reported Grade 3 diarrhea. As reported at SABCS 2015, the objective response rate across treatment groups was 42 percent and the CNS response rate was 33 percent, providing encouraging data to support moving forward with a follow-on Phase 2 study of ONT-380 in combination with Xeloda and Herceptin.

ONT-380 in combination with Kadcyla:
This Phase 1b trial enrolled patients with metastatic HER2-positive breast cancer with progression following prior treatment with Herceptin and a taxane. Patients may have received prior treatment with Perjeta (pertuzumab) and Tykerb (lapatinib). Patients with or without brain metastases were eligible. This trial has completed enrollment, but is ongoing with patients continuing to receive treatment. Clinical data from this trial were presented at SABCS 2014 and 2015, and at ASCO (Free ASCO Whitepaper) 2015. Overall, the combination of ONT-380 and Kadcyla was clinically well tolerated in 50 patients treated at the maximum tolerated dose of ONT-380, with the majority of adverse events either Grade 1 or Grade 2 in severity. Grade 3 diarrhea was reported in 4 percent of patients. Durable ( > 6 months) systemic and CNS responses and disease stabilization were seen, with an objective response rate of 41 percent and a CNS response rate of 33 percent.

Internal Research & Discovery Collaborations:

Research Collaboration Moving Chk1 Inhibitors Forward. During 2015, Oncothyreon continued preclinical activities to develop a small molecule against the checkpoint kinase 1 (Chk1) target in collaboration with Sentinel Oncology. IND-enabling studies are expected to begin in 2017.

Protocell Research Program Progressing. During 2015, Oncothyreon continued research on protocells, a novel nanoparticle platform technology that may enable the targeted delivery of a variety of therapeutic agents.

Leadership:

Expanded Leadership Team, CEO Search Underway. In January 2016, Oncothyreon announced the appointment of Christopher S. Henney, the company’s chairman, as interim CEO. The company’s comprehensive search for a new CEO is actively ongoing. Additionally, Oncothyreon appointed three new members to its Board of Directors: Steven P. James in March 2015 and Mark Lampert and Gwen Fyfe, M.D. in January 2016.

Full Year and Fourth Quarter 2015 Financial Highlights

Cash, cash equivalents and investments totaled $56.4 million as of December 31, 2015, compared to $63.7 million at December 31, 2014, a decrease of $7.3 million, or 11.5%. The decrease was primarily attributable to $28.9 million of cash used in operations during the year ended December 31, 2015, partially offset by the net proceeds of $22.4 million from the closing of concurrent but separate underwritten offerings of common stock and Series B convertible preferred stock in February 2015.

Research and development expenses for the fourth quarter of 2015 decreased by $19.1 million to $6.9 million from $26.0 million in the fourth quarter of 2014. Full year research and development expenses decreased by $18.4 million to $23.5 million in 2015 from $41.9 million in 2014. The fourth quarter and full year decrease in expense was primarily the result of the one-time upfront payment made to Array Biopharma in December 2014.

General and administrative expenses for the fourth quarter of 2015 increased by $0.4 million to $2.3 million, from $1.9 million in the fourth quarter of 2014. Full year 2015 general and administrative expenses were $9.3 million, an increase of $0.3 million from $9.0 million in 2014. This increase was primarily due to patent expenses related to our product candidates.

Net loss for the year ended December 31, 2015 was $32.6 million, or $0.34 per basic and diluted share, compared with a net loss of $50.0 million, or $0.64 per basic and diluted share, for the comparable period in 2014. Net loss for the three months ended December 31, 2015 was $9.1 million, or $0.10 per basic and diluted share, compared with a net loss of $27.6 million, or $0.30 per basic and diluted share, for the comparable period in 2014. The decrease in net loss for the year and quarter was primarily attributable to a $20.0 million upfront payment Oncothyreon made to Array BioPharma upon entering into an exclusive license agreement in December 2014. The decrease in net loss was partly offset by slightly higher general and administrative expenses and lower non-cash income from the change in the fair value of our warrant liability.

Financial Guidance

Oncothyreon believes the following financial guidance to be correct as of the date provided. Oncothyreon is providing this guidance as a convenience to investors and assumes no obligation to update it.

Oncothyreon currently expects operating expenses in 2016 to be higher than in 2015. This increase will primarily be related to expenditures associated with the Phase 2 trial of ONT-380. Oncothyreon currently expects cash used in operations in 2016 to be approximately $38.0 million to $40.0 million. With cash, cash equivalents and investments of $56.4 million as of December 31, 2015, Oncothyreon estimates that its cash, cash-equivalents and investments will be sufficient to fund operations for at least the next 12 months.

On March 14, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended December 31, 2015 (Press release, Stemline Therapeutics, MAR 14, 2016, View Source [SID:1234509512]).

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Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the fourth quarter, we made significant progress across all three of our development programs. Key achievements included 1) generating and presenting data from our ongoing Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) which we have designed to support potential registration, 2) treating our first patient in the second stage of our Phase 2 program of SL-701 in advanced brain cancer, and 3) the opening of our IND for SL-801 on schedule. At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December, we presented very strong data from our ongoing SL-401 trial in BPDCN, demonstrating a high overall response rate in BPDCN, with multiple complete responses. We are also very pleased with the pace of enrollment in BPDCN, and note that we are tracking in-line with our expectations. We look forward to sharing key clinical and regulatory updates on SL-401 throughout this year."

Dr. Bergstein continued, "We continue to evaluate SL-401 in several additional malignancies that overexpress IL-3R. Specifically, we have single agent trials enrolling AML patients with minimal residual disease and patients with advanced high-risk myeloproliferative neoplasms. We also expect our first clinical studies that combine SL-401 with other therapies will begin this year."

Dr. Bergstein concluded, "Our other pipeline candidates, SL-701 and SL-801, continue to advance. Our SL-701 trial is currently enrolling patients and we expect updates from this program in the second half of the year. The SL-801 IND is open, and we are on track to treat our first patient this quarter. With our strong cash position, we believe we have sufficient financial resources to achieve significant clinical and regulatory milestones this year and beyond."

Fourth Quarter 2015 Financial Results Review

Stemline ended the fourth quarter of 2015 with $97.5 million in cash, cash equivalents and investments, as compared to $104.0 million as of September 30, 2015, which reflects a cash burn of $6.5 million for the quarter. The company ended the fourth quarter of 2015 with 18.2 million shares outstanding.

For the fourth quarter of 2015, Stemline had a net loss of $10.2 million, or $0.58 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014. The net loss for full year 2015 was $37.2 million, or $2.15 per share, as compared with a net loss of $28.8 million, or $2.23 per share, in the prior year.

Research and development expenses were $7.9 million for the fourth quarter of 2015, which reflects an increase of $2.8 million, or 56%, compared with $5.1 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to the ramp up in our clinical trial activities for SL-401 and SL-801.

General and administrative expenses were $2.6 million for the fourth quarter of 2015, which reflects an increase of $0.6 million, or 30%, compared with $2.0 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to an increase in compensation expense relating to administrative employees.

On March 14, 2016 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the fourth quarter and full year 2015 and commented on recent accomplishments and clinical development plans for its pipeline of several SINE-based therapeutics including selinexor, its lead product candidate (Press release, Karyopharm, MAR 14, 2016, View Source [SID:1234509521]).

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"During 2015, we continued to expand our leadership position in the development of oral SINE-based oncology and non-oncology therapies with clinical advancement of selinexor across a number of hematologic and solid tumor indications and the presentation of encouraging data on our earlier-stage pipeline programs," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "Our broad and aggressive oncology clinical development program continues in 2016 with both company- and investigator-sponsored trials underway or planned to evaluate selinexor both as a single-agent and in combination with other existing and emerging oncology therapies. In addition, we made important progress with our non-oncology focused SINE compounds in a number of disease areas in which XPO1 is critically involved. We look forward to several potentially value-creating data readouts over the next 18 months."

Clinical Development Plans:
Selinexor in hematologic malignancies. Karyopharm is actively enrolling patients in several later phase clinical studies evaluating single-agent selinexor in hematologic malignancies, including patients with relapsed/refractory multiple myeloma (MM) (STORM study), older patients with relapsed/refractory acute myeloid leukemia (AML) (SOPRA study) and patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) (SADAL study). Karyopharm expects data from the first 80 patients in the STORM study to be available in mid-2016, at which point, it will evaluate whether to expand the study. Karyopharm also expects to provide an interim analysis update from the SOPRA study in late-2016 with top-line data expected in mid-2017. Based on discussions with the U.S. Food and Drug Administration (FDA), Karyopharm amended the protocol for the ongoing SADAL study to remove dexamethasone from the regimen and to evaluate selinexor as a single-agent in this patient population. The study will continue to compare 60 mg and 100mg twice weekly doses of selinexor with 100 patients per arm and at least 50% of patients with GCB-type DLBCL. Based on these amendments, the company expects to report top-line data from this study in early 2017.

Single-agent selinexor in solid tumors. Karyopharm is currently conducting company-sponsored trials of single-agent selinexor in three solid tumor indications including advanced unresectable dedifferentiated liposarcoma (SEAL study), heavily pretreated patients with gynecologic malignancies (SIGN study) and recurrent glioblastoma multiforme (KING study). Karyopharm is planning to present updated data from the SIGN and KING studies at an appropriate medical meeting in mid-2016. The Phase 2/3 SEAL study, evaluating single-agent oral selinexor versus placebo, was initiated earlier this year based on promising clinical data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2015 Annual Meeting in which durable stable disease and improvement in progression free survival compared to previous chemotherapies were observed. Top-line data from the Phase 2 portion of this study are expected in mid-2017.

Selinexor combinations. A number of investigator-sponsored and company-sponsored trials evaluating selinexor in combination with either chemotherapy or targeted agents in hematologic and solid tumor indications are currently ongoing or planned. In mid-2016, Karyopharm plans to initiate a Phase 2/3 study (SCORE study) evaluating the combination of selinexor, carfilzomib and dexamethasone in patients with refractory MM who were previously treated with a proteasome inhibitor and an immunomodulatory agent. In addition, Karyopharm expects to report top line data from the Phase 1 portion of a Phase 1b/2 study (STOMP study) evaluating selinexor in combination with commonly used treatments, including bortezomib, pomalidomide, or lenalidomide, for relapsed/refractory MM in late-2016.

KPT-8602, second generation SINE compound. In January 2016, Karyopharm initiated a Phase 1/2 study of oral KPT-8602, a novel, second generation, SINE compound, in patients with relapsed/refractory MM. Data from preclinical studies presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated single-agent anti-MM activity of KPT-8602 along with the potential for higher and/or more frequent dosing compared with selinexor. Top-line safety and tolerability data from this Phase 1/2 study are expected in late 2016.

KPT-9274, oral dual inhibitor of PAK4 and NAMPT. In mid-2016, Karyopharm plans to initiate clinical development in patients with heavily pretreated solid tumors or lymphoma for its oral dual inhibitor of PAK4 and NAMPT, KPT-9274.

Verdinexor (KPT-335). In May 2015, Karyopharm began clinical testing of verdinexor in a randomized, double-blind, placebo-controlled, dose-escalating Phase 1 clinical trial in healthy human volunteers in Australia. Verdinexor was found to be generally safe and well tolerated. Karyopharm is preparing to advance verdinexor for certain viral indications with an initial focus on influenza. Preclinical data provide strong support for other potential indications for verdinexor, including human immunodeficiency virus (HIV).
KPT-350. KPT-350 is an investigational new drug application-ready oral compound with a preclinical data package supporting potential efficacy in a number of neurological, autoimmune and inflammatory conditions. We plan to partner with a collaborator to undertake the clinical development and potential commercialization of KPT-350 in one or more mutually agreed indications.

Scientific Presentations and Publications:
Positive clinical data demonstrating the activity and tolerability of selinexor across multiple hematologic malignancies including MM and AML were presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting held in December 2015 in Orlando, Florida.

Updated data from an ongoing investigator-sponsored trial of selinexor in combination with carfilzomib and dexamethasone in refractory MM were presented by Andrzej Jakubowiak, MD, PhD, from The University of Chicago. The data demonstrated encouraging efficacy of selinexor in combination with carfilzomib and dexamethasone in heavily pretreated patients with highly refractory MM, including patients whose disease is refractory to previous carfilzomib-based combinations, suggesting the potential of this combination to overcome carfilzomib resistance. Based on these data, Karyopharm expects to initiate a Phase 2/3 clinical study (SCORE study) in mid-2016.

Karyopharm researchers and collaborators also presented preclinical data demonstrating the potential of selinexor to synergize with proteasome inhibitors to overcome drug resistance in MM.

Karyopharm presented data demonstrating the activity and tolerability of selinexor in combination with standard of care agents in the AML setting, including data from a Phase 2 trial investigating the efficacy and tolerability of selinexor in combination with arabinoside cytosine (Ara-C) and idarubicin in "fit" patients with relapsed and/or refractory AML. Karyopharm also presented data from a Phase 1 study determining the safety and efficacy of selinexor in combination with fludarabine and cytarabine in pediatric patients with relapsed and/or refractory acute leukemia.

Karyopharm researchers presented data establishing 60mg as the most appropriate selinexor dose for both efficacy and tolerability across many hematologic and solid cancers. These data support the selinexor first-in-human Phase 1 clinical trial solid tumor data published in Journal of Clinical Oncology in March 2016, which recommended a dose of 60mg given twice a week as the Phase 2 dose. This recommendation was based on superior patient tolerability, longer duration of therapy, and no demonstrable improvement in radiologic response or disease stabilization compared with higher doses.

Fourth Quarter and Year Ended December 31, 2015 Financial Results

Cash, cash equivalents and investments as of December 31, 2015, including restricted cash, totaled $210.0 million, compared to $214.8 million as of December 31, 2014.

For the year ended December 31, 2015, research and development expense was $97.7 million compared to $60.1 million for the year ended December 31, 2014. For the year ended December 31, 2015, general and administrative expense was $21.6 million compared to $15.9 million for the year ended December 31, 2014. The increase in research and development expense resulted primarily from the increase in expenses related to the continued clinical development of selinexor. The increase in general and administrative expense resulted primarily from an increase in personnel costs, including stock-based compensation expense.

Karyopharm reported a net loss of $118.2 million, or $3.32 per share, for the year ended December 31, 2015, compared to a net loss of $75.8 million, or $2.43 per share, for the year ended December 31, 2014. Net loss includes stock-based compensation expense of $17.1 million and $14.2 million for the years ended December 31, 2015 and 2014, respectively.

For the quarter ended December 31, 2015, research and development expense was $24.1 million compared to $20.0 million for the quarter ended December 31, 2014. For the quarter ended December 31, 2015, general and administrative expense was $5.3 million compared to $5.9 million for the quarter ended December 31, 2014. The increase in research and development expenses resulted primarily from the increase in expenses related to the continued clinical development of selinexor.

Karyopharm reported a net loss of $29.0 million, or $0.81 per share, for the quarter ended December 31, 2015, compared to a net loss of $25.9 million, or $0.79 per share, for the quarter ended December 31, 2014. Net loss includes stock-based compensation expense of $5.4 million and $4.6 million for the quarters ended December 31, 2015 and December 31, 2014, respectively.

Financial Outlook
Based on current operating plans, Karyopharm expects that its existing cash and cash equivalents will fund its research and development programs and operations into the middle of 2018, including advancing the four later-stage clinical studies to their next data inflection points. Karyopharm expects to end 2016 with at least $120 million in cash, cash equivalents and investments.

On March 14, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended December 31, 2015 (Filing, Q4/Annual, Stemline Therapeutics, 2015, MAR 14, 2016, View Source [SID:1234509551]).

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the fourth quarter, we made significant progress across all three of our development programs. Key achievements included 1) generating and presenting data from our ongoing Phase 2 trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) which we have designed to support potential registration, 2) treating our first patient in the second stage of our Phase 2 program of SL-701 in advanced brain cancer, and 3) the opening of our IND for SL-801 on schedule. At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December, we presented very strong data from our ongoing SL-401 trial in BPDCN, demonstrating a high overall response rate in BPDCN, with multiple complete responses. We are also very pleased with the pace of enrollment in BPDCN, and note that we are tracking in-line with our expectations. We look forward to sharing key clinical and regulatory updates on SL-401 throughout this year."

Dr. Bergstein continued, "We continue to evaluate SL-401 in several additional malignancies that overexpress IL-3R. Specifically, we have single agent trials enrolling AML patients with minimal residual disease and patients with advanced high-risk myeloproliferative neoplasms. We also expect our first clinical studies that combine SL-401 with other therapies will begin this year."

Dr. Bergstein concluded, "Our other pipeline candidates, SL-701 and SL-801, continue to advance. Our SL-701 trial is currently enrolling patients and we expect updates from this program in the second half of the year. The SL-801 IND is open, and we are on track to treat our first patient this quarter. With our strong cash position, we believe we have sufficient financial resources to achieve significant clinical and regulatory milestones this year and beyond."

Fourth Quarter 2015 Financial Results Review

Stemline ended the fourth quarter of 2015 with $97.5 million in cash, cash equivalents and investments, as compared to $104.0 million as of September 30, 2015, which reflects a cash burn of $6.5 million for the quarter. The company ended the fourth quarter of 2015 with 18.2 million shares outstanding.

For the fourth quarter of 2015, Stemline had a net loss of $10.2 million, or $0.58 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014. The net loss for full year 2015 was $37.2 million, or $2.15 per share, as compared with a net loss of $28.8 million, or $2.23 per share, in the prior year.

Research and development expenses were $7.9 million for the fourth quarter of 2015, which reflects an increase of $2.8 million, or 56%, compared with $5.1 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to the ramp up in our clinical trial activities for SL-401 and SL-801.

General and administrative expenses were $2.6 million for the fourth quarter of 2015, which reflects an increase of $0.6 million, or 30%, compared with $2.0 million for the fourth quarter of 2014. The increase in expenses during the current quarter was primarily attributable to an increase in compensation expense relating to administrative employees.

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On March 14, 2016 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2015, and provided an update on the Company’s recent progress (Press release, Bellicum Pharmaceuticals, MAR 14, 2016, View Source;p=RssLanding&cat=news&id=2148302 [SID:1234509523]).

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"In 2015, Bellicum made significant progress across all of our T-cell immunotherapy programs, reporting positive interim results with our lead product candidate BPX-501, and advancing our CAR T and TCR programs," said Tom Farrell, Bellicum’s President and Chief Executive Officer. "Data presented at ASH (Free ASH Whitepaper) from the BP-004 clinical trial with BPX-501 demonstrated disease-free outcomes in pediatric patients with genetic blood diseases. We believe that BPX-501, which was recently granted orphan drug designation by the FDA, could represent a vital treatment option for the many patients for whom a transplant is recognized as the preferred treatment, but who are not treated because they lack a perfect match donor."

Continued Mr. Farrell, "Building on this momentum, we look forward to achieving important milestones in 2016, with data updates from the BPX-501 program expected to be presented at several medical meetings. We also expect to launch Phase 1 clinical trials of three novel product candidates: BPX-701 and BPX-601 in mid-2016, and BPX-401 in the second half of 2016. The BPX-701 and BPX-601 programs were reviewed at the March 10th meeting of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC) and we were pleased with the engaging discussion and meeting outcomes."

2015 HIGHLIGHTS AND CURRENT UPDATES

DEVELOPMENT PROGRAMS

BPX-501

Adjunct T-cell therapy administered after allogeneic hematopoietic stem cell transplantation (HSCT), using genetically modified donor T cells incorporating our CaspaCIDe safety switch, is being evaluated in malignant and nonmalignant blood diseases.

Enrollment in Phase 1/2 BP-004 clinical trial continues at strong pace, with 63 pediatric patients enrolled in the E.U. and 12 patients enrolled in the U.S. to date.
Granted orphan drug designation by the FDA for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as "replacement T-cell therapy for the treatment of immunodeficiency and Graft versus Host Disease after allogeneic hematopoietic stem cell transplant."
Reported interim data from ongoing BP-004 trial, demonstrating disease-free outcomes in pediatric patients with genetic blood diseases who had undergone HSCT followed by BPX-501 donor T-cell replacement. Presented at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015, the data showed that treated patients achieved immune recovery significantly faster than historical control subjects not given BPX-501, as well as a significant reduction in time to hospital discharge (21 days sooner vs. historical controls) and reduced re-hospitalizations. In addition, none of the patients had developed chronic GvHD and no patient died of transplantation-related complications. Of the 39 patients treated (as of Nov. 30, 2015), 21 had genetic blood diseases, including Fanconi anemia, beta thalassemia, SCID and Wiskott-Aldrich Syndrome, and 18 had blood cancers.
DOTTI study clinical data published in BLOOD highlighted safety and effectiveness of CaspaCIDe-modified T-cell add-back. Results of a 12-patient investigator-sponsored trial conducted by Baylor College of Medicine demonstrated that the add-back led to improved immune reconstitution and infection control. The data also showed that GvHD can be rapidly controlled and resolved by administration of rimiducid, and that the productive anti-viral cells remain, repopulate and maintain immunity.
U.S. patent issued to Baylor College of Medicine for technology exclusively licensed to Bellicum. The patent, issued for methods of inducing selective apoptosis of cells, extends Bellicum’s proprietary rights to the use of its lead product candidate until at least 2031.
BPX-701

High affinity T cell receptor (TCR) product candidate, incorporating our CaspaCIDe safety switch, is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma (PRAME).

Bellicum continues to advance its next-generation, proprietary TCR product candidate targeting PRAME. The Company licensed the PRAME-specific TCR technology from Leiden University Medical Center in April 2015 and is preparing for the start of clinical trials for the initial planned indications of Refractory or Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS), with an additional clinical trial planned for metastatic uveal melanoma. Each of these are orphan indications in which PRAME is highly expressed and for which current treatment options are limited.
BPX-601

GoCAR-T product candidate, containing proprietary iMC (inducible MyD88/CD40) activation switch, is designed to treat solid tumors expressing prostate stem cell antigen (PSCA).

Continued to advance the Company’s first GoCAR-T product candidate toward the clinic. The planned indication for the initial Phase 1 study is non-resectable pancreatic cancer. Preclinical data reported at ASH (Free ASH Whitepaper) 2015 showed robust anti-tumor activity, and enhanced T-cell proliferation and persistence compared to traditional CAR T constructs.
Obtained an exclusive global license from Agensys for adoptive cell therapies targeting tumors expressing PSCA.
BPX-401

CIDeCAR product candidate, incorporating novel, proprietary MC costimulatory domain and CaspaCIDe safety switch, is designed to target blood cancers expressing CD19.

Presented preclinical data at ASH (Free ASH Whitepaper) 2015 highlighting the potent anti-tumor effects of BPX-401. The preclinical in vivo results showed that tumors can be eliminated quickly and safely with CIDeCAR cells. Notably, BPX-401 elicited dose-dependent elevation of cytokines, analogous to cytokine release syndrome, but cytokine levels were rapidly normalized upon administration of rimiducid, without loss of tumor control.
CORPORATE UPDATES

Committed to build-out of in-house U.S. manufacturing capabilities. The Company leased an additional 27,000 square feet at its current location and completed the design phase for the build-out of manufacturing space. The facility is designed to support the efficient manufacturing of our novel cellular immunotherapies for clinical trials and early commercial requirements.
Recently closed on a debt financing agreement with Hercules Capital to support the build-out of the Company’s U.S. manufacturing facilities. Under the loan terms, Bellicum can borrow up to $30 million, of which the final $10 million tranche is contingent upon achievement of specified milestones and approval by Hercules’ investment committee.
ANTICIPATED 2016 MILESTONES

BPX-501

Expect to provide an update on the nonmalignant patient cohort from the BP-004 clinical trial, as well as initial data for patients with blood cancers treated at the lead European clinical trial site, during the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) which takes place April 3 – 6, 2016.
Expect to meet with regulators in Europe and the U.S. in the second quarter of 2016, with the goal of defining the path to regulatory filing and approval initially for nonmalignant pediatric genetic diseases.
Anticipate presenting updated data from the BPX-501 program at ASH (Free ASH Whitepaper) in December 2016.
BPX-701

Expect to begin enrolling patients in a Phase 1 clinical trial for refractory or relapsed acute AML and MDS in mid-2016.
BPX-601

Expect to begin enrolling patients in a Phase 1 clinical trial in mid-2016 for non-resectable pancreatic cancer.
BPX-401

Expect to move a CIDeCAR product candidate directed to the CD19 antigen into the clinic in the second half of 2016.
Corporate

Expect to complete build-out of the U.S. cGMP viral vector and cellular therapy manufacturing facility by the end of 2016.
Expect to establish a European presence and initiate activities in anticipation of the potential commercialization of BPX-501 in future years.
Fourth Quarter and Full Year 2015 Financial Results

Cash Position and Guidance: Bellicum ended the year on December 31, 2015 with cash and investments totaling $150.4 million, compared to $191.6 million at December 31, 2014. Based on current operating plans and capital available under its loan facility, Bellicum expects to end 2016 with approximately $80 to $90 million in cash, cash equivalents and investments, and anticipates that current cash resources will be sufficient to meet operating requirements through 2017. The Company also expects that in 2016 it will invest approximately $25 to $30 million for capital projects to enable in-house U.S. manufacturing and to support the Company’s growth.

Grant Revenues were $34,000 and $282,000 for the fourth quarter and year ended December 31, 2015, respectively, and $14,000 and $1,780,000 during the comparable periods in 2014. The decrease in full year 2015 grant revenues was primarily due to the June 2014 expiration of the Company’s grant award from the Cancer Prevention and Research Institute of Texas.

R&D Expenses: Research and development expenses were $10.2 million and $33.6 million for the fourth quarter and year ended December 31, 2015, respectively, compared to $4.2 million and $12.1 million during the comparable periods in 2014. The higher expenses in the 2015 periods were primarily due to an increase in BPX-501 clinical and manufacturing costs as a result of increased patient enrollment in clinical trials, an increase in costs related to preclinical product candidates BPX-701, BPX-601 and BPX-401’s IND-enabling activities, and an increase in general research and development costs including personnel costs, and allocated overhead.

License fees were $3.0 million and $3.2 million for the fourth quarter and year ended December 31, 2015, respectively, compared to no license fees in 2014. The increase in fees was primarily due to a new license agreement with Agensys, an affiliate of Astellas, as consideration for rights granted to Bellicum under the agreement related to its BPX-601 product candidate, whereby Agensys was paid a non-refundable upfront fee of $3.0 million.

G&A Expenses: General and administrative expenses were $3.8 million and $12.7 million for the fourth quarter and year ended December 31, 2015, respectively, compared to $2.0 million and $4.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were primarily due to overall growth and public company related costs, including an increase in personnel, legal and accounting expenses and costs related to facilities, insurance and travel.

Net Loss: Bellicum reported a net loss of $16.8 million for the fourth quarter of 2015 and $48.5 million for the year ended December 31, 2015, compared to a net loss of $74.3 million and $84.0 million for the comparable periods in 2014. The net loss amounts for the 2014 periods included a charge of $43.2 million incurred in the fourth quarter of 2014 in conjunction with the ARIAD license restructure transaction and a non-cash accounting charge of $24.4 million recorded for the change in fair value of warrants that were exercised in conjunction with Bellicum’s December 2014 initial public offering, of which $23.2 million was a fourth quarter expense. The results also included non-cash, share-based compensation charges of $2.5 million and $8.4 million for the fourth quarter and year ended December 31, 2015, respectively, and $0.7 million and $0.9 million for the comparable periods in 2014.

Shares Outstanding: At December 31, 2015, Bellicum had 26,931,881 shares of common stock outstanding.