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8-K – Current report

On August 10, 2015 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the second quarter ended June 30, 2015 and recent company developments (Filing, 8-K, Manhattan Pharmaceuticals, AUG 10, 2015, View Source [SID:1234507159]).

Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "The second quarter was a busy and exciting time for the Company, as the data presentations made during the quarter continue to reinforce our belief that the safety and efficacy profiles of TG-1101 and TGR-1202, alone or in combination together in our "1303" regimen, allow for safe and efficacious multiple drug regimens, which we firmly believe is the future of patient care in the treatment of B-cell malignancies. We remain focused on commencing additional combination registration trials in the coming months, and aggressively recruiting into our ongoing GENUINE Phase 3 clinical trial." Mr. Weiss continued, "From a financial perspective, with cash on hand of more than $125 million on a pro forma basis, we are well positioned to execute on our goals and bring the Company to substantial value creating milestones."

Recent Developments and Highlights

· Clinical data on the combination of TG-1101 and TGR-1202 was presented at the 51st American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held in Chicago, Illinois, as well as in poster presentations at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting held in Vienna, Austria and the International Congress on Malignant Lymphoma (ICML), held in Lugano, Switzerland

· Single agent clinical data for TGR-1202 was presented at the ASCO (Free ASCO Whitepaper) Annual Meeting, as well as in oral presentations at the EHA (Free EHA Whitepaper) and Lugano ICML meetings

· Clinical data on the triple combination of TG-1101, TGR-1202, and ibrutinib was presented in an oral presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting, and in an oral presentation at the Lugano ICML meeting

· Updated results from a Phase 2 clinical trial of TG-1101 in combination with ibrutinib in relapsed/refractory Chronic Lymphocytic Leukemia (CLL) was presented in an oral presentation at the Lugano ICML meeting

· Presently have over 120 sites open for the Company’s GENUINE Phase 3 Clinical Trial of TG-1101 in combination with ibrutinib in patients with High-Risk Chronic Lymphocytic Leukemia

Reaffirming 2015 Milestones

· Continue to aggressively recruit into the GENUINE Phase 3 Clinical Trial of TG-1101 in combination with ibrutinib

· Commence additional combination Phase 3 clinical trials, particularly for the Company’s proprietary "1303" combination of TG-1101 plus TGR-1202 in patients with Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin’s Lymphoma (NHL)

· Launch new triple therapy combination trials in addition to the currently enrolling Phase 1/2 trial of TG-1101 plus TGR-1202 plus ibrutinib

· Continue to push forward our preclinical development programs, including the IRAK4, anti-PD-L1, and anti-GITR programs, as well as opportunistically seek to identify drug candidates with complimentary mechanisms of action

· Commence clinical development program for the treatment of autoimmune diseases

· Present updated data on Phase 1 and 2 clinical trials at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, in December 2015, held in Orlando, Florida

Financial Results for the Second Quarter 2015

At June 30, 2015 the Company had cash, cash equivalents, investment securities, and interest receivable of $110.6 million, which includes approximately $51.2 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the year (approximately $42 million of which was previously disclosed in connection with our last quarterly update), as compared to December 31, 2014 when we had $78.9 million.

Pro-forma cash, cash equivalents, investment securities, and interest receivable as of June 30, 2015 are approximately $126.4 million, including $15.8 million of net proceeds from the utilization of the ATM sales facility during the third quarter of 2015.

Our consolidated net loss for the second quarter ended June 30, 2015, excluding non-cash items, was approximately $10.9 million, which included approximately $4.8 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and commercialization. The consolidated net loss for the second quarter ended June 30, 2015, inclusive of non-cash items, was $17.1 million, or $0.38 per diluted share, compared to a consolidated net loss of $12.0 during the comparable quarter in 2014, representing an increase in consolidated net loss of $5.1 million. The increase in consolidated net loss during the second quarter ended June 30, 2015 was primarily the result of other research and development expenses for TG-1101 and TGR-1202 increasing approximately $5.8 million and $1.3 million, respectively, over the comparable period in 2014. The increase in other research and development expenses related to TG-1101 was primarily the result of increased manufacturing and clinical trial expenses related to ongoing and planned future Phase 3 registration programs. These increases were partially offset by the $1.2 million of non-cash stock expense recorded in conjunction with the license to the IRAK4 inhibitors program during the quarter ended June 30, 2014 and a decrease of $1.5 million in non-cash compensation expense related to equity incentive grants over the comparable period in 2014.

Our consolidated net loss for the six months ended June 30, 2015, excluding non-cash items, was approximately $20.1 million, which included approximately $9.1 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and commercialization. The consolidated net loss for the six months ended June 30, 2015, inclusive of non-cash items, was $31.7 million, or $0.73 per diluted share, compared to a consolidated net loss of $19.5 million during the comparable period in 2014, representing an increase in consolidated net loss of $12.2 million. The increase in consolidated net loss during the six months ended June 30, 2015 was primarily the result of other research and development expenses for TG-1101 and TGR-1202 increasing approximately $9.8 million and $2.5 million, respectively, over the comparable period in 2014. The increase in other research and development expenses related to TG-1101 was primarily the result of increased manufacturing and clinical trial expenses related to ongoing and planned future Phase 3 registration programs.

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MabVax Therapeutics Holdings Provides Corporate Update and Reports Second Quarter 2015 Financial Results

On August 10, 2015 MabVax Therapeutics Holdings, Inc. (OTCQB: MBVX), a clinical-stage oncology drug development company, reported a corporate update and reported financial results for the quarter ended June 30, 2015 (Press release, MabVax, AUG 10, 2015, View Source [SID:1234507158]).

"We are reporting progress toward achieving our near-term drug development milestones and with our capital formation," stated MabVax’s President and Chief Executive Officer David Hansen. "We completed a $11.7 million equity financing led by OPKO Health, Inc., and its Chairman and Chief Executive Officer, biotech investor and entrepreneur Dr. Phillip Frost, which put MabVax in one of the best financial positions in our corporate history. We finished the second quarter with approximately $7.2 million in cash and equivalents, including the release on June 30, 2015, of $3.5 million of funds from the equity financing to the Company that were being held in escrow by OPKO Health, Inc. and Frost Gamma Investment Trust. We believe that our current cash position is sufficient to support our near-term drug discovery and development efforts."

"The encouraging results from our non-human primate safety study for HuMab 5B1 further support our plan to file two Investigational New Drug applications, or INDs, with the FDA by 2015 year-end and begin both clinical trials in the first quarter of 2016," he added. "The GMP manufacturing of our clinical supplies of HuMab 5B1-based product for both clinical trials is on track for delivery in the fourth quarter of this year."

MabVax plans to initiate two complementary Phase I clinical trials in the first quarter of 2016. One clinical trial is aimed at determining the safety and potential utility of HuMab 5B1 as a therapeutic agent in subjects with metastatic pancreatic cancer. The second clinical trial is aimed at demonstrating the utility of 89Zr-HuMab 5B1, the Company’s radio-labeled HuMab 5B1 antibody, as a next-generation PET imaging agent for the diagnosis, staging, and management of pancreatic cancer.

Second Quarter and Recent Highlights

Significant financing transaction with a leading investor – April 10, 2015 – closed on approximately $11.7 million in a private placement led by OPKO Health, Inc. and its Chairman and CEO Dr. Phillip Frost. On June 30, 2015, OPKO Health, Inc. and Frost Gamma Investment Trust investments in the aggregate of $3.5 million associated with the financing were released from escrow.

Encouraging results of non-human primate toxicology study – On May 5, 2015, announced results from non-GLP toxicology testing of HuMab 5B1 antibody completed by a leading independent contract research organization. These results detailed that the antibody, administered in either a single dose or repeated doses, produced no significant adverse findings even at the highest dosage levels tested. Non-human primates in this acute dose range finding study were challenged with multiple dose levels to assess drug pharmacokinetics, as well as with repeated doses of the antibody to identify any adverse toxicology signals. These studies were conducted in the most relevant animal models with material produced by the Company’s GMP manufacturing partner. The antibody as tested is representative of the clinical supply material scheduled for delivery later this year for the planned Phase I clinical trials.

Rockefeller University Collaboration – In July 2015, entered into a research collaboration agreement to supply Rockefeller University’s Laboratory of Molecular Genetics and Immunology with antibody material to explore the mechanism of action of constant region (Fc) variants of the HuMab 5B1 in the role of tumor clearance. The Company will supply additional research materials as requested by the university, which is evaluating ways to optimize the function.

Second Quarter 2015 Financial Results

Grant revenues totaled approximately $137,000.
Research and development expenses were $2.3 million.
General and administrative expenses totaled approximately $4.2 million.
Net loss for the quarter was $6.4 million, or $0.29 per share, on approximately 25.2 million shares of common stock outstanding.
Completed $11.7 million financing including the release of $3.5 million in funds held in escrow.
At June 30, 2015, cash and cash equivalents totaled $7.2 million.

About HuMab 5B1
In pre-clinical research, MabVax’s HuMab 5B1 antibody has demonstrated high specificity, affinity and lack of cross-reactivity with closely related antigens. The antibody has also shown potent cancer cell killing capacity and efficacy in animal models of pancreatic, colon and small cell lung cancer. When combined with a radio-label as a novel PET imaging agent, 89Zr-HuMab 5B1 has demonstrated high image resolution of tumors in established xenograft animal models, making it attractive as a potential companion diagnostic for the therapeutic product.

Kite Pharma Reports Second Quarter 2015 Financial Results

On August 10, 2015 Kite Pharma, Inc. (Kite) (Nasdaq:KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous T cell therapy (eACT) products for the treatment of cancer, reported financial results for the quarter ended June 30, 2015 (Press release, Kite Pharma, AUG 10, 2015, View Source [SID:1234507155]).

"This past quarter has been marked by a number of significant milestones. Most notably, we launched the first Company-sponsored clinical trial of our lead product candidate, KTE-C19," said Arie Belldegrun, M.D., FACS, Chairman, President and Chief Executive Officer. "We also entered into multiple collaborations, and expanded our manufacturing capabilities. In addition, we were very pleased to convene our first investor day on June 23rd, which allowed us to unveil new initiatives and highlight the progress of both our chimeric antigen receptor (CAR) and T cell receptor (TCR) programs."

Highlights of Developments in Second Quarter 2015

Commenced Kite’s Phase 1/2 clinical trial of KTE-C19, an anti-CD19 CAR T-cell therapy, for treatment of refractory, aggressive Non-Hodgkin’s Lymphoma (NHL).

Presented clinical biomarker results at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which demonstrated that conditioning chemotherapy was associated with a significant rise in homeostatic cytokines and chemokines, which could favor expansion, activation, and trafficking of CAR T cells.

Announced our first TCR product candidate, targeting HPV-16 E6, that we plan to advance to a company-sponsored clinical trial.
Entered into an exclusive worldwide collaboration with bluebird bio to advance second generation TCR products to treat HPV-associated cancers.

Partnered with The Leukemia & Lymphoma Society to enhance the development of KTE-C19 in refractory, aggressive NHL and to launch CAR T-cell therapy educational programs.

Completed construction of our clinical supply manufacturing facility in Santa Monica, California.
Began construction on our manufacturing facility in El Segundo, California to support our commercial plans.
Hosted our first Investor Day, which focused on our future research, clinical, and manufacturing plans.

Second Quarter 2015 Financial Results

Cash Position: As of June 30, 2015, Kite had $392.9 million in cash, cash equivalents, and marketable securities, compared to $367.0 million as of December 31, 2014.

Cash Burn: The net increase of $25.9 million in the first half of 2015 was primarily due to $26.7 million in proceeds from the underwriters’ exercise of the over-allotment option from the follow-on public offering and the $60.0 million upfront payment from the Amgen collaboration. This increase was partially offset by cash outflows related to the acquisition of TCF, license obligations, and funding of ongoing operations, including for the advancement of the KTE-C19 program.

Net Loss: GAAP net loss attributable to common stockholders was $20.9 million, or $0.48 per share, for the second quarter of 2015, compared to $17.9 million, or $2.27 per share, for the second quarter of 2014. Non-GAAP net loss attributable to common stockholders for the second quarter of 2015 was $11.5 million, or $0.26 per share. Non-GAAP net loss for the second quarter of 2015 excludes non-cash stock-based compensation expense of $9.4 million. Please see "Note Regarding Use of Non-GAAP Financial Measures" for a reconciliation of GAAP net loss to non-GAAP net loss.

Revenue: Collaboration revenue was $4.4 million for the second quarter of 2015 compared to $0 for the second quarter of 2014. The increase was primarily comprised of the amortization of deferred revenue related to the $60.0 million upfront payment received from Amgen in the first quarter of 2015.

Total Operating Expenses: Total GAAP operating expenses for the second quarter of 2015 were $26.4 million compared to $11.1 million for the second quarter of 2014.

R&D Expenses: GAAP research and development (R&D) expenses were $16.6 million for the second quarter of 2015, compared to $7.4 million for the second quarter of 2014. The increase of $9.2 million was primarily due to costs associated with the ongoing KTE-C19 Phase 1/2 clinical trial in DLBCL, preparing for the additional trials in acute lymphoblastic leukemia (ALL), mantle cell lymphoma (MCL), and chronic lymphocytic leukemia (CLL) later this year, as well as increased personnel expense, including non-cash stock-based compensation, and costs related to growing the Company’s operations in the US and EU.

G&A Expenses: GAAP general and administrative (G&A) expenses were $9.8 million for the second quarter of 2015, compared to $3.7 million for the second quarter of 2014. The increase of $6.1 million was primarily due to increased personnel expense, including non-cash stock-based compensation, and other professional expenses to support growing the Company’s operations, as well as license obligations.

2015 Financial Guidance: Kite’s guidance remains unchanged. Kite expects to burn between $100 million and $125 million in cash for the full year 2015, which includes both operating expenses and capital expenditures. This guidance does not include cash inflows or outflows for business development activities.

8-K – Current report

On August 10, 2015 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported financial results for the second quarter 2015 and commented on recent accomplishments and clinical development plans for selinexor, its lead product candidate (Filing, 8-K, Karyopharm, AUG 10, 2015, View Source [SID:1234507152]).

"Important data describing the clinical benefit of selinexor across multiple solid and hematologic malignancies was presented during the quarter, including single agent anti-tumor activity and durable disease control in patients with recurrent glioblastoma, advanced sarcomas, ovarian and endometrial cancers. We also presented survival data in patients with relapsed/refractory diffuse large B-cell lymphoma treated with selinexor, along with combination data of selinexor with chemotherapy in patients with heavily pretreated acute myeloid leukemia," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "In addition, we continue to execute against the selinexor clinical development plan with the initiation of a Phase 2 study in patients with quad-refractory multiple myeloma and make steady progress enrolling patients in our other on-going later phase clinical trials in acute myeloid leukemia, diffuse large B cell lymphoma and Richter’s transformation. Furthermore, we made some important changes to certain trials based on our growing experience with selinexor. Finally, we recently met with FDA and now have a path forward for a phase 2/3 study in liposarcoma. In the second half of 2016, we look forward to reporting preliminary top-line data from our later phase clinical trials in AML, DLBCL and Richter’s transformation, as well as data from the first 80 patients in our later phase clinical trial in multiple myeloma in the middle of 2016."

Conference Call Information:
To access the conference call, please dial (855) 437-4406 (US) or (484) 756-4292 (international) at least five minutes prior to the start time and refer to conference ID 98056569. A live audio webcast of the call will be available under "Events & Presentations" in the Investor section of Karyopharm’s website, View Source, approximately two hours after the event.

Scientific Presentations and Publications:

• Presented positive clinical data with single-agent, oral selinexor in on-going Phase 2 and Phase 1b clinical studies across multiple solid tumors at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting including anti-tumor activity and disease control in patients with recurrent glioblastoma, advanced sarcomas, heavily pre-treated gynecological cancers and across multiple malignancies in Asian patients, including:

• anti-tumor activity, including brain penetration at clinically relevant drug levels, with a 13% overall response rate (ORR) and a 38% disease control rate (DCR) in patients with recurrent glioblastoma in an ongoing Phase 2 clinical trial;

• durable activity, including longer progression free survival (PFS) than last prior regimen, in an ongoing Phase 1b clinical study in patients with advanced sarcomas, including liposarcoma;

Targeting Disease at the Nuclear Pore

• promising anti-tumor activity or disease control across ovarian, endometrial and cervical cancers with disease control rates (DCR) of up to 62% and several patients remaining on study for up to 12 months in an ongoing Phase 2 clinical trial in patients with heavily pre-treated, progressive gynecological cancers;

• anti-tumor activity across a variety of malignancies in a Phase 1 clinical trial evaluating the activity of selinexor in Asian patients with advanced malignancies.

• Presented clinical data with oral selinexor, both as single agent and in combination with chemotherapy, in a number of hematologic malignancies including diffuse large B-cell lymphoma (DLBCL) and acute myeloid leukemia (AML) at the 20th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) 2015 Annual Meeting, including:

• updated survival data from an ongoing Phase 1b clinical trial of single-agent selinexor in heavily pre-treated patients with DLBCL in which patients with a response to selinexor (N=12) demonstrated a median overall survival (OS) of greater than 10 months (median not reached) and PFS was 24 months, significantly longer than those without a response (N=27; OS 3.5 months, PFS 1.2 months);

• preliminary Phase 2 results from an ongoing clinical trial of selinexor in combination with chemotherapy (idarubicin/Ara-C) in 18 evaluable patients with relapsed or refractory AML demonstrated a 56% ORR, including nine patients with complete remission (CR/CRi) and one patient with a partial remission (PR).

• Presented clinical and preclinical data with single agent, oral selinexor at the 13th International Conference on Malignant Lymphoma (ICML) in DLBCL patients with MYC, BCL2 and/or BCL6 translocations, so called "Double Hit and Triple Hit" Lymphomas — areas of significant unmet medical need associated with poor prognosis and limited standard-of-care treatment options:

• In an ongoing Phase 1 clinical trial in 14 relapsed, refractory DLBCL patients with triple, double or single hit MYC, BCL2 and/or BCL6 translocations, selinexor demonstrated clinically meaningful activity with a 43% ORR (PR or better) including two CRs, four PRs and two additional patients achieving stable disease (SD).

• In preclinical models, selinexor demonstrated potency in double hit DLBCL cell lines in vitro and in an aggressive derived xenograft (PDX) model of triple hit DLBCL, with 84% tumor growth inhibition.
Regulatory and Intellectual Property Updates:

• Karyopharm met with the FDA in July and plans to initiate a Phase 2/3 clinical trial of selinexor versus placebo to treat liposarcoma in the second half of 2015. Accrual to Karyopharm’s Phase 1b clinical trial in sarcomas, including liposarcoma, is nearly complete.

• Granted U.S. patent for KPT-350, an oral SINE compound being developed for the treatment of inflammatory and autoimmune diseases. This patent, which will expire in 2033 absent any patent term extensions, covers the composition of matter for KPT-350, as well as certain other compositions and related methods.

• Granted U.S. patent covering method of treatment using certain SINE compounds, including selinexor and verdinexor. This patent will expire in 2032 absent any patent term extensions, and the covered methods of treatment include methods for treating viral infections, inflammatory disorders and cancer.

Targeting Disease at the Nuclear Pore

Clinical Development Plans:

• Karyopharm initiated a single-arm trial in multiple myeloma called STORM, for Selinexor Treatment of Refractory Myeloma, which will initially include 80 patients. If the data from the initial 80 patients is promising, the study may be expanded to potentially support accelerated approval. Preliminary top-line data from this study are anticipated in mid-2016.

• Karyopharm is actively enrolling patients in three later-stage clinical studies evaluating selinexor: one in older patients with relapsed/refractory AML (SOPRA study), the second in patients with relapsed/refractory DLBCL (SADAL study) and the third in patients with Richter’s transformation (SIRRT study). Preliminary top-line data from all three studies are anticipated in the fourth quarter of 2016.

• Following evaluation of over 1,000 patients treated with selinexor to date, Karyopharm has determined that the recommended phase 2 dose (RP2D) for patients with the majority solid tumors and selected hematologic malignancies is 60 mg fixed dose, twice weekly; the maximum tolerated dose is ~120 mg. The recommended dose in multiple myeloma is 80 mg selinexor + 20 mg of dexamethasone together, twice weekly. Doses of up to 100 mg twice weekly will continue to be evaluated in certain indications.

• In July 2015, Karyopharm amended the SOPRA study, a Phase 2 randomized clinical trial of single-agent, oral selinexor in older patients with acute myeloid leukemia, or AML, to reduce the dose from 55mg/m2 to a fixed dose of 60mg, which corresponds to approximately 35 mg/m2. Dosing will remain twice weekly. This change was implemented based on ongoing safety and tolerability evaluations in the SOPRA study, as well as maturing data from AML patients in the Phase 1 first-in-human clinical trial of selinexor. The SOPRA study uses a two-to-one randomization of AML patients to selinexor or physician’s choice and, therefore, approximately twice as many cases of sepsis would be expected on the selinexor arm compared with the physician’s choice arm. As of the end of July 2015, there have been eight reports of sepsis in seven patients receiving selinexor 55 mg/m2 on the SOPRA study, as compared with two reports of sepsis in two patients receiving physician’s choice on that study. Therefore, although the numbers are small, and sepsis is often observed in patients with AML, the incidence of sepsis appears to be higher in the patients receiving selinexor. In addition, as our data are maturing, an apparent increase in the incidence of sepsis in patients with relapsed or refractory AML receiving high doses of selinexor twice weekly was noted in Karyopharm’s Phase 1 clinical trial in hematologic malignancies. Importantly, doses of 60mg twice weekly do not appear to be associated with any increase in sepsis or other infection-related events in patients with hematologic malignancies or solid tumors. In addition, the majority of the patients with AML in the Phase 1 study who showed a response to selinexor treatment, including patients with complete remissions, received selinexor at doses of approximately 60mg or below. As a result of the change in dose, the SOPRA study will now have an interim assessment in mid-2016 with topline data expected in the fourth quarter of 2016.

• In July 2015, Karyopharm amended the protocol of SIRRT, a Phase 2 clinical study of single-agent, oral selinexor in patients with Richter’s transformation, an aggressive form of lymphoma, to include patients with newly diagnosed Richter’s transformation. There is no standard of care for patients with Richter’s transformation and these patients have an extremely poor prognosis. As a result of these factors, and in order to improve patient accrual, in consultation with key opinion leaders in the area, Karyopharm determined that there was a compelling rationale to amend the SIRRT protocol to include patients who had not yet received chemotherapy to treat Richter’s transformation. Karyopharm is now implementing the revised protocol across SIRRT study sites in the United States and Europe.

• Karyopharm expects to commence the STOMP ("Selinexor and Backbone Treatments of Multiple Myeloma Patients") study in the third quarter with support from Myeloma Canada. In this multi-arm clinical study, Karyopharm plans to evaluate the combination of selinexor and low dose dexamethasone with backbone therapies including bortezomib, pomalidomide or lenalidomide in patients with multiple myeloma. Selinexor and low dose dexamethasone is already being combined with Kyprolis in an Investigator Sponsored Trial, where promising preliminary data were presented at ASH (Free ASH Whitepaper) 2014.

Targeting Disease at the Nuclear Pore

• Karyopharm is currently conducting company-sponsored trials of single-agent selinexor in four solid tumor indications. At ASCO (Free ASCO Whitepaper) 2015, Karyopharm reported responses and disease control in patients with heavily pretreated gynecologic malignancies (SIGN study) and in recurrent glioblastoma multiforme (KING study); accrual to these studies is continuing. Karyopharm is also continuing to accrue patients to the SHIP study, a phase 2 study of selinexor in previously treated, hormone-refractory prostate cancer. The fourth phase 2 solid tumor study, the STARRS study, involves patients with relapsed or refractory squamous cell tumors. Enrollment to the head and neck cohort of this study has been completed and, due to very slow accrual in the lung and esophageal squamous carcinoma cohorts, Karyopharm is terminating further enrollment to these arms and finalizing the study. Additional trials with selinexor in combination with various chemotherapies are ongoing and may include patients with squamous cell carcinomas.

• In addition, a number of investigator-sponsored (ISTs) or company-sponsored clinical studies evaluating the potential of selinexor in combination with either chemotherapy or targeted agents are currently ongoing or planned.

Second Quarter June 30, 2015 Financial Results
Cash, cash equivalents and investments as of June 30, 2015, including restricted cash, totaled $256.0 million, compared to $285.3 million as of March 31, 2015.

For the quarter ended June 30, 2015, research and development expense was $27.0 million compared to $13.2 million for the quarter ended June 30, 2014. For the quarter ended June 30, 2015, general and administrative expense was $6.2 million compared to $3.3 million for the quarter ended June 30, 2014. The increase in research and development expenses 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 the costs of being a public company and an increase in stock-based compensation.
Karyopharm reported a net loss of $32.7 million, or $0.92 per share, for the quarter ended June 30, 2015, compared to a net loss of $16.4 million, or $0.55 per share, for the quarter ended June 30, 2014. Net loss includes stock-based compensation expense of $4.5 million and $3.9 million for the quarters ended June 30, 2015 and June 30, 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 2018, including moving the four later-stage clinical studies to their next data inflection points. Karyopharm expects to end 2015 with greater than $200 million in cash, cash equivalents and investments.
"Karyopharm continues to maintain a very strong balance sheet, with approximately $256M in cash as of the end of the second quarter of 2015," said Justin Renz, Executive Vice President, Chief Financial Officer & Treasurer. "As for financial guidance this year, we remain on track to end 2015 with greater than $200M in cash."

Intrexon Announces Second Quarter and First Half 2015 Financial Results

On August 10, 2015 Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported its second quarter and first half results for 2015 (Press release, Intrexon, AUG 10, 2015, View Source;p=RssLanding&cat=news&id=2078523 [SID:1234507150]).

Business Highlights and Recent Developments:

During the second quarter, Intrexon’s exclusive collaboration and license agreement with the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, to develop and commercialize chimeric antigen receptor T-cell (CAR-T) cancer therapies became effective, resulting in the receipt of $115 million, 50% of which is payable to ZIOPHARM Oncology, Inc., as an upfront fee in July 2015. Focused on the generation of leading-edge products that empower the immune system in a regulated manner, the collaboration’s first two CAR-T targets of interest have been selected and Intrexon has initiated research and development efforts on these programs;

Granted a special stock dividend of ZIOPHARM Oncology, Inc. (NASDAQ: ZIOP) shares owned by Intrexon to its shareholders valued at approximately $172 million at the time of distribution. Holders of Intrexon common stock received 0.162203 shares of ZIOPHARM common stock at $9.67 per share with respect to each outstanding share of Intrexon common stock they owned;
Announced a Cooperative Research and Development Agreement with the National Cancer Institute (NCI). The principal goal is to develop and evaluate improved adoptive cell transfer-based immunotherapies (ACT) using NCI proprietary methods for the identification of autologous peripheral blood lymphocytes with naturally occurring endogenous anti-tumor activity combined with the RheoSwitch Therapeutic System for introducing spatially and temporally controlled interleukin-12 (IL-12) expression in ACT/PBL/IL-12 for the treatment of patients with solid tumor malignancies;

Completed the acquisition of Okanagan Specialty Fruits, the pioneering agricultural company behind the Arctic apple, the world’s first non-browning apple without the use of any flavor-altering chemical or antioxidant additives;

Entered into a multi-year collaboration with an investment fund sponsored by Harvest Capital Strategies, LLC. The fund is dedicated to the inventions and discoveries of Intrexon and will have the exclusive rights of first-look and first negotiation for Intrexon’s investment proposals suitable for pursuit by a startup. The fund will be complementary to Intrexon’s ongoing programs and will not prohibit the Company’s ability to execute other collaborations and joint ventures;

In collaboration with Fibrocell Science, Inc. (NASDAQ: FCSC) announced submission of an Investigational New Drug Application to the U.S. Food and Drug Administration for FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a debilitating genetic skin disorder caused by a mutation in the gene encoding type VII collagen (COL7), a protein that forms anchoring fibrils which hold together the layers of skin. Additionally, the companies reported positive proof-of-concept data from in vivo pre-clinical studies for FCX-007. Fibrocell expects to initiate a Phase I/II clinical trial by year-end to evaluate the safety, mechanism of action, and efficacy of FCX-007;

Expanded relationship with Oragenics (NYSE MKT: OGEN) through a new Exclusive Channel Collaboration (ECC) to pursue development of biotherapeutics for oral mucositis and other diseases of the oral cavity, throat, and esophagus, including clinical advancement of ActoBiotics AG013 for the treatment of oral mucositis; and

In conjunction with Oragenics, announced selection of lead clinical candidate for the lantibiotics program and reported positive in vivo efficacy data in critical animal study on multiple compounds from Oragenics’ Mutacin 1140 platform. Lantibiotics are a class of antibiotics with a novel mechanism of action active against a broad spectrum of Gram-positive bacteria, including multi-drug resistant infectious bacteria, which could provide an important new tool in the fight against global bacterial antibiotic resistance.

Second Quarter Financial Highlights:

Total revenues of $44.9 million, an increase of 281% over the second quarter of 2014;
Net loss of $40.7 million attributable to Intrexon, or $(0.37) per basic share;
Excluding the special stock dividend of ZIOPHARM Oncology, Inc. shares, Pro Forma Net Income Attributable to Intrexon during the second quarter would have been of $0.9 million, or $0.01 per basic share;
Adjusted EBITDA of $54.4 million, or $0.50 per basic share; and
Cash consideration received for reimbursement of research and development services, Cost Recovery, covered 60% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries).

First Half Financial Highlights:

Total revenues of $78.7 million, an increase of 301% over the first half of 2014;
Net loss of $13.6 million attributable to Intrexon, or $(0.13) per basic share;
Excluding the special stock dividend of ZIOPHARM Oncology, Inc. shares, Pro Forma Net Income Attributable to Intrexon during the first half would have been $28.0 million, or $0.26 per basic share;
Adjusted EBITDA of $39.8 million, or $0.37 per basic share; and
Total consideration received for technology access fees and reimbursement of research and development services covered 187% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries).

"We continue satisfactorily to balance contemporary inputs to outputs while advancing a growing portfolio of programs that should provide significant and unburdened contribution to our bottom line," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "The scalability of our technology platforms and our organizational model are allowing us to grow our company rapidly across an ever diversifying array of great product candidates while we add important new talent to our team. That we can execute such an ambitious plan while also making acquisitions that provide positions of genuine industrial leadership is a testament to the great team that so zealously advances our mission to ‘power the bioindustrial revolution.’"

Second Quarter 2015 Financial Results Compared to Prior Year Period

Total revenues were $44.9 million for the quarter ended June 30, 2015 compared to $11.8 million for the quarter ended June 30, 2014, an increase of $33.1 million, or 281%. Product revenue includes $12.6 million from the sale of pregnant cows, live calves and the sale of livestock used in production. Service revenue totaling $11.6 million relates to the provision of in vitro fertilization and embryo transfer services performed. Collaboration revenues increased $5.4 million due to (i) the recognition of deferred revenue for upfront payments received from our license and collaboration agreement with the biopharmaceutical business of Merck KGaA, which became effective in May 2015, and from collaborations signed by us between July 1, 2014 and June 30, 2015, (ii) the recognition of research and development services performed by us pursuant to these new collaborations, and (iii) increased research and development services performed by us related to collaboration programs in effect prior to July 1, 2014 as a result of progression of current programs and the initiation of new programs with these collaborators.

Total operating expenses were $62.3 million for the quarter ended June 30, 2015 compared to $29.9 million for the quarter ended June 30, 2014, an increase of $32.4 million, or 108%. Research and development expenses were $20.4 million for the quarter ended June 30, 2015 compared to $14.4 million for the quarter ended June 30, 2014, an increase of $5.9 million, or 41%. Salaries, benefits and other personnel costs increased $2.5 million due to (i) increases in research and development headcount to support the new collaborations discussed above, and (ii) compensation expenses related to performance and retention incentives for research and development employees. Lab supplies and consultants increased $2.1 million due to the increased level of research and development services provided to our collaborators. Selling, general and administrative expenses were $23.7 million for the quarter ended June 30, 2015 compared to $15.4 million for the quarter ended June 30, 2014, an increase of $8.3 million, or 54%. Salaries, benefits and other personnel costs increased $5.5 million due to (i) the inclusion of selling, general and administrative employees of companies we have acquired since July 1, 2014, including Trans Ova and ActoGeniX, and (ii) compensation expenses related to performance and retention incentives for general and administrative employees. Depreciation and amortization increased $0.8 million primarily as a result of property and equipment and intangible assets acquired from Trans Ova. Total operating expenses for the quarter ended June 30, 2015 also include $18.2 million of products and services costs which primarily consist of employee compensation costs, livestock, feed, drug supplies and facility charges related to the production of such products and services.

Total other expense, net, was $21.0 million for the quarter ended June 30, 2015 compared to $33.8 million for the quarter ended June 30, 2014, a decrease of $12.8 million, or 38%. This decrease was primarily related to the changes in the value of our securities portfolio, including a realized gain of $81.4 million which resulted from the special stock dividend of all of our shares of ZIOPHARM to our shareholders in June 2015.

First Half 2015 Financial Results Compared to Prior Year Period

Total revenues were $78.7 million for the six months ended June 30, 2015 compared to $19.6 million for the six months ended June 30, 2014, an increase of $59.1 million, or 302%. Product revenue includes $20.1 million from the sale of pregnant cows, live calves and the sale of livestock used in production. Service revenue totaling $20.0 million relates to the provision of in vitro fertilization and embryo transfer services performed. Collaboration revenues increased $12.4 million due to (i) the recognition of deferred revenue for upfront payments received from our license and collaboration agreement with the biopharmaceutical business of Merck KGaA, which became effective in May 2015, collaborations signed by us between July 1, 2014 and June 30, 2015 and our collaboration with Intrexon Energy Partners, which was signed in March 2014, (ii) recognition of research and development services performed by us pursuant to these new collaborations, and (iii) increased research and development services performed by us for collaborations in effect prior to July 1, 2014 as a result of the progression of current programs and the initiation of new programs with these collaborators.

Total operating expenses were $183.3 million for the six months ended June 30, 2015 compared to $55.6 million for the six months ended June 30, 2014, an increase of $127.7 million, or 230%. Research and development expenses were $99.7 million for the six months ended June 30, 2015 compared to $26.5 million for the six months ended June 30, 2014, an increase of $73.2 million, or 276%. In January 2015, we issued 2,100,085 shares of our common stock valued at $59.6 million to the University of Texas MD Anderson Cancer Center, or MD Anderson, in exchange for an exclusive license to certain technologies owned by MD Anderson. Salaries, benefits and other personnel costs increased $5.9 million due to (i) increases in research and development headcount to support the new collaborations discussed above, and (ii) compensation expenses related to performance and retention incentives for research and development employees. Lab supplies and consultants expenses increased $4.0 million as a result of the increased level of research and development services provided to our collaborators. Selling, general and administrative expenses were $51.3 million for the six months ended June 30, 2015 compared to $29.0 million for the six months ended June 30, 2014, an increase of $22.3 million, or 77%. Salaries, benefits and other personnel costs increased $13.4 million due to (i) the inclusion of selling, general and administrative employees of companies we have acquired since July 1, 2014, including Trans Ova and ActoGeniX, and (ii) compensation expenses related to performance and retention incentives for general and administrative employees. Stock-based compensation expenses for the annual options granted to our non-employee directors, pursuant to our non-employee director compensation policy, increased $1.0 million due to a higher grant-date fair value in 2015 compared to 2014. Legal and professional expenses increased $2.7 million primarily due to costs associated with acquisitions, the license agreement with MD Anderson, the January 2015 public securities offering and other business development activity. Depreciation and amortization increased $1.6 million primarily as a result of property and equipment and intangible assets acquired from Trans Ova. Total operating expenses for the six months ended June 30, 2015 also include $32.1 million of products and services costs which primarily consist of employee compensation costs, livestock, feed, drug supplies and facility charges related to the production of such products and services.

Total other income, net, was $94.7 million for the six months ended June 30, 2015 compared to total other expense, net, of $11.8 million for the six months ended June 30, 2014, an increase of $106.5 million, or 903%. This increase was primarily related to the changes in the value of our securities portfolio, including a realized gain of $81.4 million which resulted from the special stock dividend of all of our shares of ZIOPHARM to our shareholders in June 2015.