On August 8, 2017 Kite Pharma, Inc. (Nasdaq:KITE), a leading cell therapy company, reported second quarter 2017 financial results and provided a corporate update for the period ended June 30, 2017 (Press release, Kite Pharma, AUG 8, 2017, View Source [SID1234520184]).

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"We’ve continued to make significant progress on key clinical and commercial milestones in the last six months alone," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "With the anticipated events on the horizon for the remainder of 2017, the potential for CAR-T to become one of the most powerful anti-cancer agents for certain patients may finally be realized."

Second Quarter 2017 Financial Results

Revenue was $10.1 million for the second quarter of 2017.
Research and development expenses were $70.9 million for the second quarter of 2017, including $13.1 million of non-cash stock-based compensation expense.
General and administrative expenses were $41.1 million for the second quarter of 2017, including $12.1 million of non-cash stock-based compensation expense.
Net loss was $109.8 million, or $1.94 per share, for the second quarter of 2017.
Non-GAAP net loss for the second quarter of 2017 was $84.7 million, or $1.50 per share, excluding non-cash stock-based compensation expense of $25.2 million.
As of June 30, 2017, Kite had $781.1 million in cash, cash equivalents, and marketable securities.
Recent Highlights

Axicabtagene Ciloleucel (axi-cel) Regulatory and Clinical Development

The submission of axi-cel to the U.S. Food and Drug Administration (FDA) remains under review with a PDUFA Action Date of November 29, 2017.
Submitted a Marketing Authorization Application to the European Medicines Agency (EMA) for axi-cel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for autologous stem cell transplant. This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA.
Patients are now being treated in ZUMA-5, the Phase 2 trial of axi-cel in indolent B-Cell Non-Hodgkin lymphoma.
Patients in the European Union (EU) are now being treated with axi-cel. Kite is currently enrolling adult patients with relapsed/refractory DLBCL, PMBCL and TFL in certain EU medical centers.
A publication from researchers at the National Cancer Institute reported complete remissions up to 56+ months in patients with chemorefractory aggressive non-Hodgkin’s lymphoma (NHL) after receiving anti-CD19 CAR T-cells in a clinical trial.
KTE-C19 Development

At the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, Kite reported 73 percent minimum residual disease (MRD) negative complete remission rate in an updated analysis of the Phase 1 ZUMA-3 trial of KTE-C19 in adults with high burden relapsed/refractory acute lymphoblastic leukemia (ALL). Adverse events included cytokine release syndrome and neurologic events, and were generally reversible.
CAR-T Pipeline

Submitted an investigational new drug (IND) application for KITE-585, a CAR-T therapy candidate that targets BCMA expressed in multiple myeloma.
TCR Pipeline

Opened a Phase 1 clinical trial of KITE-718, a T-cell receptor (TCR) cell therapy candidate that targets MAGE A3/A6 antigen expressed in solid tumors, including non-small cell lung cancer, bladder cancer and head and neck cancer.
Axi-cel Commercial Preparation

Completed recruitment and training of cell therapy account managers to support customer service and logistical coordination.
Conducted test runs of technical operations for ordering, scheduling, processing and shipment of cell therapy product at key major medical institutions in preparation for potential approval and launch.
Intellectual Property

Favorable outcome at the United States Patent and Trademark Office (USPTO) in an ex parte reexamination of Kite’s seminal Eshhar ‘465 CAR-T patent (U.S. Patent No. 7,741,465) that confirmed the patentability of its amended claims. The Eshhar ‘465 patent term continues to June 2027, not including certain potential extensions.
Second Half 2017 Milestones

Axi-cel

Commercial launch of axi-cel in the United States, if approved.
One year follow-up data from ZUMA-1 study of axi-cel in patients with aggressive NHL.
Preliminary data from ZUMA-6 combination study of axi-cel and atezolizumab (PD-L1 checkpoint inhibitor) in refractory DLBCL.
KTE-C19

Preliminary follow-up Phase 1 data from ZUMA-3 and ZUMA-4 studies of pediatric and adult ALL, respectively.
Advance ZUMA-3 and ZUMA-4 studies into Phase 2.
KITE-585

Initiate Phase 1 KITE-585 trial in multiple myeloma.

Vical has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On August 8, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the second quarter ended June 30, 2017, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, AUG 8, 2017, View Source;p=RssLanding&cat=news&id=2292700 [SID1234520145]).

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"Since I joined the Company six months ago, we have conducted a thorough review of our strategy and operations, and are very optimistic about the opportunities before us," said Rick Fair, Bellicum’s President & Chief Executive Officer. "We continue to be encouraged by the results from our ongoing BPX-501 pediatric studies and our progress toward a filing in Europe. We have adjusted our plans for U.S. registrational trials to enable an efficient path to seeking approvals for the greatest areas of unmet need. Lastly, we continue to be excited about the clinical progress of our CAR T and TCR product candidates, and the application of our molecular switch platform for future pipeline expansion."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-501
Adjunct T-cell therapy incorporating the CaspaCIDe safety switch, administered after a haploidentical hematopoietic stem cell transplant (haplo-HSCT), to improve outcomes and reduce mortality

Data Update Suggests BPX-501 Improves Outcomes of Haploidentical Stem Cell Transplants
During the Presidential Symposium of the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June, Bellicum reported data from 98 pediatric patients within the BP-004 trial which showed rapid immune recovery, a low incidence of transplant-related mortality, a reduction in viral infections and a low rate of Graft versus Host Disease (GvHD) that was manageable with either standard treatments or rimiducid. The data suggest BPX-501 could improve outcomes of haploidentical stem cell transplants, providing an option for the many patients who could benefit from a life-saving transplant but lack a matched donor.

Positive Clinical Results of BPX-501 in Pediatric Leukemias
Also at EHA (Free EHA Whitepaper), Bellicum reported data from the BP-004 trial in a cohort of 47 pediatric patients with acute leukemias who lack a matched donor. The data showed rapid immune reconstitution and low rates of relapse and mortality, suggesting that BPX-501 may offer benefits in combination with HSCT in acute leukemia patients.

European BP-004 Pivotal Clinical Trial Progressing
Enrollment in the pivotal EU BP-004 trial remains on track for completion by the end of 2017. Bellicum expects to initiate an observational trial in pediatric patients receiving transplants from matched unrelated donors (MUD) without BPX-501 in the third quarter. Outcomes from these trials are expected to be the basis for filings of European Marketing Authorization Applications for BPX-501 and rimiducid. The Company expects to report top-line results of these studies in the second half of 2018, with MAA filings planned for 2019.

Company Clarifies U.S. Clinical Development Strategy
Bellicum is finalizing plans for the design of registrational trials of BPX-501 in the U.S. The Company’s current plans include conducting a controlled clinical trial in adult patients with acute myeloid leukemia (AML), which it expects to fund in part through its $16.9 million Product Development Award from the Cancer Prevention and Research Institute of Texas ("CPRIT"). In the pediatric non-malignant setting, Bellicum is designing a registrational trial to evaluate BPX-501 in a distinct subset of orphan inherited blood disorders.
BPX-601

Phase 1 BPX-601 Clinical Trial Continues
BPX-601 is Bellicum’s novel GoCAR-T product candidate, which is designed with its proprietary iMC activation switch to allow control over the level of stimulation and proliferation of the modified T cells. Enrollment and treatment is ongoing in Bellicum’s Phase 1 trial in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA).
BPX-701

Phase 1 BPX-701 Clinical Trial Continues
BPX-701 is a high affinity TCR product candidate designed with the CaspaCIDe safety switch, enabling the elimination or reduction of the engineered cells in the event of severe toxicities. Dosing has been initiated in the Company’s Phase 1 clinical trial in patients with refractory or relapsed AML and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
CORPORATE UPDATE

Addition of Chief Business Officer to Expand Partnership Opportunities
Greg Naeve, Ph.D., an accomplished product strategy and business development executive, is joining Bellicum’s leadership team in August 2017 from Pfizer, where he led efforts to identify and implement multiple strategic partnerships and translational science collaborations across Pfizer Worldwide R&D, including CAR T alliances with Cellectis and Servier.
PRECLINICAL RESEARCH

In April, Bellicum reported positive preclinical data at AACR (Free AACR Whitepaper) on its novel dual-switch technology incorporated into CAR T and TCR constructs, an approach offering the possibility of both activating cells to enhance efficacy and eliminating them to manage toxicity. Bellicum is working to incorporate its dual-switch technology into future CAR T and TCR product candidates.

The Company continues to work with academic collaborators to explore the applicability of CaspaCIDe in CD19 CARs, the first of which is expected to enter the clinic in the second half of this year in patients with B-cell malignancies.
SECOND QUARTER AND SIX MONTHS ENDED JUNE 30, 2017 FINANCIAL RESULTS

Bellicum reported a net loss of $24.5 million for the second quarter of 2017 and $46.4 million for the six months ended June 30, 2017, compared to a net loss of $16.5 million and $31.6 million for the comparable periods of 2016. The results included non-cash, share-based compensation charges of $3.2 million and $6.6 million for the second quarter and six months ended June 30, 2017 and $3.1 million and $6.2 million for the comparable periods in 2016.

As of June 30, 2017, cash, restricted cash and investments totaled $139.0 million. Based on current operating plans, Bellicum continues to expect to end 2017 with approximately $85 to $95 million in cash and investments, and that current cash resources will be sufficient to meet operating requirements through 2018.

Research and development expenses were $18.0 million and $33.3 million, for the three and six months ended June 30, 2017, respectively, compared to $12.0 million and $22.9 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to an increase in clinical development and manufacturing costs due to increased enrollment in trials, principally BPX-501, and increased personnel expenses, overhead charges and manufacturing facility start-up costs.

General and administrative expenses were $5.5 million and $11.4 million for the three and six months ended June 30, 2017, respectively, compared to $4.2 million and $8.5 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to the Company’s overall growth, including an increase in personnel related costs, principally due to hiring additional employees and severance costs, higher facility costs and increased legal, accounting and travel expenses.

At June 30, 2017, Bellicum had 33,193,229 shares of common stock outstanding.

On August 8, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported financial results for the quarter ended June 30, 2017 (Press release, Loxo Oncology, AUG 8, 2017, View Source [SID1234520187]). Loxo Oncology will not be conducting a conference call in conjunction with this earnings release.

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"I am very proud of the Loxo Oncology team for delivering a great second quarter," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "The larotrectinib (LOXO-101) presentations at ASCO (Free ASCO Whitepaper) and the simultaneous LOXO-195 publication in Cancer Discovery illustrate what can be accomplished for patients when a focused management team brings together the disparate disciplines of medicinal chemistry, comprehensive tumor testing and clinical-regulatory execution. In addition, the LOXO-292 IND moves the company into an exciting second target and third clinical-stage program. We look forward to the second half of 2017, when we plan to prepare the larotrectinib NDA submission, advance our TRK fusion diagnostics strategy with third party collaborators, and advance LOXO-292 efficiently through a dose escalation. We hope to be able to share initial clinical data from the LOXO-292 program by the end of the year."

Recent Highlights

Larotrectinib Adult and Pediatric Interim Clinical Data Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: In June, interim clinical data from three ongoing larotrectinib trials were reported in two oral presentations at ASCO (Free ASCO Whitepaper). The data demonstrated a 76 percent confirmed objective response rate (ORR) in 50 patients for whom follow-up was sufficiently long to include a confirmatory scan. Responses were observed across 17 unique tumor types harboring TRK fusions. The ASCO (Free ASCO Whitepaper) presentation included adult and pediatric patients with Response Evaluation Criteria In Solid Tumors (RECIST)-evaluable TRK fusion cancers, with an April 14, 2017 data cut-off. See the presented data here.
Larotrectinib Regulatory Updates: In May, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to larotrectinib for the "treatment of solid tumors with NTRK-fusion proteins." Loxo Oncology remains on track to submit a New Drug Application (NDA) for larotrectinib to the FDA in late 2017 or early 2018. The primary analysis for the NDA will rely upon central, independent radiology review. The company plans to announce these data, which will also include additional patient follow-up from the data set presented at ASCO (Free ASCO Whitepaper), before the end of 2017.
LOXO-195: In May, the FDA cleared the IND application for LOXO-195, Loxo Oncology’s next-generation TRK inhibitor. On June 3, 2017, a research brief was published in Cancer Discovery outlining the preclinical rationale for LOXO-195 and clinical proof-of-concept data from the first two patients treated. The Phase 1 trial is currently open for enrollment.
LOXO-292: In May, the first patient was enrolled in the Phase 1 clinical trial of LOXO-292, a highly selective RET inhibitor. This first-in-human, global, multi-center Phase 1 trial is evaluating LOXO-292 as a single agent in patients with advanced solid tumors.
LOXO-305: In July, Loxo Oncology announced the acquisition of the Redx Pharma Plc BTK inhibitor program, including lead candidate LOXO-305 (formerly RXC005). Under the terms of the agreement, Loxo Oncology has made a $40 million payment to Redx Pharma Plc. Loxo Oncology is not subject to milestone or royalty obligations. Lead candidate LOXO-305 was designed to reversibly bind BTK and preserve activity in the presence of the C481S acquired resistance mutation. Additionally, it was designed to avoid off-target kinases that have complicated the development of both covalent and reversible BTK inhibitors. LOXO-305 is expected to enter clinical development in 2018.
Equity Financing: In June, Loxo Oncology announced the closing of an underwritten public offering of 3,622,500 shares of common stock at a public offering price of $72.00 per share, which included the exercise in full by the underwriters of their option to purchase 472,500 additional shares of common stock. Gross proceeds to Loxo Oncology from this offering were approximately $260.8 million.
Second Quarter 2017 Financial Results

As of June 30, 2017, Loxo Oncology had aggregate cash, cash equivalents and investments of $467.6 million, compared to $141.8 million as of December 31, 2016.

Research and development expenses were $24.4 million for the second quarter of 2017 compared to $12.3 million for the second quarter of 2016. This increase was primarily due to expanded larotrectinib development activities including clinical costs and costs related to the companion diagnostics agreement with Roche, as well as additional development expenses related to LOXO-292 and increases in employment costs primarily due to increased headcount. Loxo Oncology also recognized research and development-related stock-based compensation expense of $3.5 million during the second quarter of 2017 compared to $0.2 million for the second quarter of 2016.

Research and development expenses were $44.6 million for the six months ended June 30, 2017, compared to $20.7 million for the six months ended June 30, 2016. This increase was primarily due to expanded larotrectinib development activities including clinical costs and costs related to the companion diagnostics agreement with Roche, as well as additional development expenses related to LOXO-292 and LOXO-195 and increases in employment costs primarily due to increased headcount. Loxo Oncology also recognized research and development-related stock-based compensation expense of $5.9 million during the six months ended June 30, 2017, compared to $0.5 million for the six months ended June 30, 2016.

General and administrative expenses were $6.5 million for the second quarter of 2017 compared to $3.8 million for the second quarter of 2016. The increase was primarily due to increases in preparation activities for the potential commercialization of larotrectinib, headcount and employment costs and general and administrative professional fees. Loxo Oncology also recognized general and administrative-related stock-based compensation expense of $2.0 million during the second quarter 2017 compared to $1.1 million for the second quarter of 2016.

General and administrative expenses were $11.3 million for the six months ended June 30, 2017, compared to $7.2 million for the six months ended June 30, 2016. The increase was primarily due to increases in preparation activities for the potential commercialization of larotrectinib, headcount and employment costs and general and administrative professional fees. Loxo Oncology also recognized general and administrative-related stock-based compensation expense of $3.5 million during the six months ended June 30, 2017, compared to $2.1 million for the six months ended June 30, 2016.

Net loss was $30.4 million and $54.9 million for the three and six months ended June 30, 2017, respectively, compared to $15.9 million and $27.5 million for the three and six months ended June 30, 2016, respectively.

On August 8, 2017 Cascadian Therapeutics, Inc. (NASDAQ:CASC), a clinical-stage biopharmaceutical company, reported financial results for the second quarter ended June 30, 2017, and provided an update on tucatinib, an investigational oral, small molecule kinase inhibitor that is highly selective for HER2 and the Company’s lead product in development (Press release, Cascadian Therapeutics, AUG 8, 2017, View Source [SID1234520146]).

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"During the second quarter, we were pleased to receive confirmation from the European Medicines Agency (EMA) that HER2CLIMB, if positive, could serve as a single registrational trial for submission to the European regulators for potential marketing approval, and that tucatinib was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of breast cancer patients with brain metastases," said Scott Myers, President and CEO of Cascadian Therapeutics. "We are now enrolling patients in HER2CLIMB on three continents. We are pleased with site activations and patient enrollment, which are currently ahead of our projections in North America."

Second Quarter and Recent Highlights

In July 2017, the Company announced that it received confirmation from the EMA that positive results from its ongoing pivotal trial of tucatinib, known as HER2CLIMB, could serve as a single registrational trial for submission of a Marketing Authorization Application to the EMA and potential marketing approval. The Company had received similar confirmation from the FDA in 2016.

In June 2017, the Company announced that tucatinib was granted orphan drug designation by the FDA for the treatment of breast cancer patients with brain metastases.
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $125.4 million as of June 30, 2017, compared to $62.8 million at December 31, 2016. The increase was primarily due to the result of net proceeds of $88.0 million from the Company’s January 2017 financing, less cash used in operations of $24.8 million.

Net loss attributable to common stockholders for the three months ended June 30, 2017 was $14.7 million, or $0.30 per share, compared with a net loss attributable to common stockholders of $25.1 million, or $1.57 per share, for the comparable period in 2016. The $10.4 million decrease in net loss attributable to common stockholders for the quarter was primarily due to the non-cash intangible asset impairment charge of $19.7 million offset by a $6.9 million tax benefit related to the reversal of the deferred tax liability. Both amounts were recorded in connection with the termination of the STC.UNM license agreement in 2016. The decrease was offset by an increase in research and development expenses of $5.1 million primarily due to greater activity related to the development of the Company’s product candidates.

Net loss attributable to common stockholders for the six months ended June 30, 2017 was $27.1 million, or $0.60 per share, compared to a net loss attributable to common stockholders of $38.0 million, or $2.39 per share, for the same period in 2016. The $10.9 million decrease in net loss attributable to common stockholders for the six months ended June 30, 2017 was primarily due to the non-cash intangible asset impairment charge of $19.7 million offset by a $6.9 million tax benefit related to the reversal of the deferred tax liability. Both of these amounts were recorded in connection with the termination of the STC.UNM license agreement in 2016. In addition, the decrease was due to lower general and administrative expenses of $4.5 million primarily due to compensation-related expenses in connection with management changes in the first quarter of 2016 and lower non-cash expense from the deemed dividend related to the beneficial conversion feature on convertible preferred stock. The decrease in net loss attributable to common stockholders were partially offset by increases in research and development expenses of $7.4 million due to greater activity related to the development of the Company’s product candidates.

2017 Financial Outlook

Cascadian Therapeutics expects operating expenses in 2017 to be slightly higher than in 2016, primarily due to an increase in activities related to the ongoing worldwide HER2CLIMB pivotal trial. Cash used in operations for 2017 is expected to be approximately $50.0 million to $54.0 million.

Cascadian Therapeutics believes the above financial guidance to be correct as of the date provided and is providing the guidance as a convenience to investors and assumes no obligation to update it.