Akebia has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Akebia, 2017, NOV 9, 2016, View Source [SID1234521574]).

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On November 9, 2016 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the three months ended September 30, 2016, and provided an overview of recent corporate developments and upcoming milestones for its research and development programs (Press release, CytRx, NOV 9, 2016, View Source;p=RssLanding&cat=news&id=2220972 [SID1234516514]).

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"This month we expect to report additional data from our pivotal, global Phase 3 clinical trial of aldoxorubicin in patients with second-line soft tissue sarcomas (STS)," said Steven A. Kriegsman, CytRx’s Chairman and CEO. "We then plan to schedule a pre-NDA meeting with the FDA. Additionally, we recently completed enrollment in the aldoxorubicin Phase 2b clinical trial in second-line small cell lung cancer in September. We now estimate that top-line results will be available in the first half of 2017 as the number of progression events has not yet been reached."

Third Quarter 2016 and Recent Developments

Presented Positive Aldoxorubicin Combination Clinical Trial Results at ESMO (Free ESMO Whitepaper) 2016. On October 10, 2016, CytRx presented a poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress featuring interim clinical data from its on-going clinical trial of aldoxorubicin in combination with ifosfamide and mesna in patients with soft tissue sarcomas. Of 36 evaluable patients, 13 of 36 (36%) achieved a partial response of the target lesion by RECIST 1.1 criteria, 22 of 36 (61%) had stable disease, and one patient had progressive disease. Median progression-free survival has not yet been reached. While the combination did have certain toxicities, none were treatment-limiting.

Completed enrollment in its Global Phase 2b Clinical Trial in Second-Line Small Cell Lung Cancer. On September 6, 2016, CytRx announced that it completed enrollment in its global, randomized Phase 2b clinical trial comparing aldoxorubicin to topotecan in 135 patients with small cell lung cancer (SCLC) who have progressed or relapsed to prior chemotherapy. The primary endpoint is progression-free survival. The number of progression events has not yet been reached, and CytRx currently expects to announce top-line data in the first half of 2017.

Strengthened the Balance Sheet with an Equity Financing. On July 20, 2016, CytRx completed a public offering of common stock and one-year warrants for total net proceeds of aproximately $18.3 million. If exercised in full, the warrants would provide up to an additional $20 million in capital.

Reported Initial Analysis of its Pivotal, Global Phase 3 Aldoxorubicin Trial in STS. On July 11, 2016, CytRx reported interim results from its global, randomized, Phase 3 clinical trial of aldoxorubicin compared to investigator’s choice therapy in patients with relapsed or refractory STS. The Company also previously announced that it expects to report additional data from the trial in the fourth quarter. Following the subsequent analysis, CytRx plans to schedule a pre-NDA meeting with the FDA to seek marketing approval. In addition, patients in the Phase 3 clinical trial continue to be followed for overall survival, a secondary endpoint of the trial.

Capital Conservation. During the third quarter, CytRx embarked on a plan to reduce spending until additional results from the aldoxorubicin Phase 3 STS clinical trial are available and the Company meets with the FDA. This included reducing headcount, stopping pre-commercialization activities for aldoxorubicin and suspending further development of DK049.

Pipeline Generation Activities. CytRx’s expanded its drug discovery efforts to create a pipeline of oncology candidates utilizing the Company’s LADRTM technology to attach ultra-high potency drugs to albumin (10-1000 times more potent than traditional chemotherapies limited to antibodies only) to target tumors.

Upcoming Milestones

Present interim data from the on-going Phase 1b/2 clinical trial of aldoxorubicin in combination with ifosfamide/mesna at the Connective Tissue Oncology Society (CTOS) Annual Meeting being held on November 9-12, 2016, in Lisbon, Portugal.
Announce additional data from CytRx’s pivotal, global Phase 3 clinical trial of aldoxorubicin in patients with second-line STS in November 2016.
Report top-line results from the global Phase 2b clinical trial evaluating aldoxorubicin versus topotecan in patients with second-line SCLC in the first half of 2017.
Schedule and hold a pre-NDA meeting with the FDA regarding aldoxorubicin as a treatment for patients with relapsed or refractory advanced STS.
Third Quarter 2016 Financial Results

CytRx reported cash and cash equivalents of $58.9 million as of September 30, 2016.

Net loss for the quarter ended September 30, 2016 was $12.2 million, or $0.13 per share, compared with a net loss of $7.1 million, or $0.11 per share, for the quarter ended September 30, 2015. During the third quarter of 2016, CytRx recognized a non-cash gain on warrant derivative liability of $0.2 million, compared to a non-cash gain of $3.5 million for the three-month period ended September 30, 2015.

Research and development (R&D) expenses were $8.9 million for the third quarter of 2016, and included development expenses of $6.7 million for the aldoxorubicin program. R&D expenses were $8.5 million for the third quarter of 2015.

General and administrative (G&A) expenses were $2.8 million for the third quarter of 2016, compared to $2.2 million for the third quarter of 2015. G&A expenses for the third quarter 2016 included non-cash employee stock-compensation expense of $0.6 million, compared to $0.5 million for the same period in 2015.

On November 9, 2016 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported financial results for the quarter ended September 30, 2016 (Press release, Inovio, NOV 9, 2016, View Source [SID1234516542]). The following financial results provide a year-over-year comparison of the third quarter in 2016 and 2015. Total revenue was $12.5 million compared to $24.2 million. Total operating expenses were $32.7 million compared to $20.5 million. The net loss attributable to common stockholders was $20.8 million, or $0.28 per share for the third quarter 2016, compared to net income of $5.6 million, or $0.08 per share in the third quarter of 2015.

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Revenue

The decrease in revenue for the comparable periods was primarily due to $15.0 million of revenue recognized in the third quarter 2015 from the up-front payment received from our partnership agreement with MedImmune. Accounting recognition of the remainder of the $27.5 million upfront payment was deferred and will be triggered by future events. The net income achieved during the third quarter 2015 was attributable to the increase in revenue and may not repeat in future quarters.

Operating Expenses

Research and development expenses were $27.0 million compared to $16.1 million for the third quarter ending 2016 and 2015 respectively. The increase was primarily related to increased investment in our product development programs – notably the DARPA funded Ebola program and clinical trial preparations for the initiation of the VGX-3100 phase III study. General and administrative expenses were $5.8 million compared to $4.4 million.

Capital Resources

As of September 30, 2016, cash and cash equivalents and short-term investments were $119.7 million compared with $163.0 million as of December 31, 2015. There were 74.0 million shares outstanding and 81.8 million fully diluted.

During the three months ended September 30, 2016, the Company sold 448,848 shares of common stock under its ATM common stock sales agreement for net proceeds of $4.2 million, with an average price of $9.45 per share.

Inovio’s balance sheet and statement of operations are provided below. Form 10-Q providing the complete 2016 Third quarter financial report can be found at: View Source

Corporate Update

Clinical Development

The U.S. Food and Drug Administration (FDA) requested additional information regarding Inovio’s submission for its proposed phase III clinical program for VGX-3100, placing the program on clinical hold. The study had not yet been initiated and has not enrolled or dosed subjects. In its initial communication the FDA requested additional data to support Inovio’s shelf-life claim for the single-use disposable array of the newly designed and manufactured CELLECTRA 5PSP immunotherapy delivery device. Inovio expects to receive a formal letter in November, which may request other information, and estimates the start of the phase III clinical program will be delayed until the first half of 2017, pending resolution of the FDA’s request. This clinical hold does not affect other Inovio clinical programs.
Initiated a phase I Zika DNA vaccine trial in Puerto Rico to test for safety, immune responses and initial evidence of efficacy. The placebo-controlled double-blind trial will assess differences in Zika infection rates in 160 healthy participants given either placebo or vaccine as part of an exploratory endpoint. This is the second human Zika vaccine trial initiated by Inovio. All 40 subjects for the first clinical study have been fully enrolled and dosed.
Expanded phase I Ebola vaccine trial by fully enrolling an additional 125 subjects in a second stage after generating positive initial safety and immune response data in the first set of 75 healthy volunteers. The study will assess immune response characteristics generated with fewer intradermal administrations, lower doses, and with and without its DNA-based IL-12 immune activator.
Corporate Development

Inovio incorporated a 100%-owned subsidiary, GENEOS Therapeutics, Inc., to develop and commercialize neo-antigen based personalized cancer therapies. While Inovio pursues the unique potential of its SynCon immunotherapy design to break tolerance and create cancer products targeting universal tumor specific antigens, GENEOS will exclusively focus on leveraging Inovio’s potent DNA immunotherapy technology platform to advance the emerging field of patient-specific neo-antigen therapies. Inovio’s clinically validated DNA based platform is well suited for advancing individualized therapies due to its rapid product design and manufacturing benefits, ability to combine multiple neo-antigens into formulations, and generation of potent killer T cell responses that are needed to drive clinical efficacy. GENEOS plans to independently raise capital and build a team to execute this complementary business model. Inovio will continue its focus on advancing its universal antigen-specific cancer immunotherapy portfolio, including INO-3112 (with Medimmune), INO-5150, INO-1400, and INO-5401, as well as its pre-cancer (VGX-3100) and infectious disease products.
Licensed a veterinary vaccine for foot and mouth disease (FMD) to Plumbline Life Sciences, an animal health company headquartered in South Korea. Plumbline will fund all development activities for this FMD vaccine and pay Inovio milestone payments as well as royalties on potential product sales.
Inovio expanded its leadership team with the appointment of multiple individuals to lead the functions of business development, biologic and device manufacturing, regulatory, and oncology clinical development.

On November 9, 2016 MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported results for its first quarter ended September 30, 2016 (Press release, MEI Pharma, NOV 9, 2016, View Source [SID1234516663]). The Company also highlighted recent progress with its pipeline of drug candidates and previewed upcoming data presentations.

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"The past quarter was one of unprecedented achievement for the company, highlighted by the receipt of Breakthrough Therapy Designation and a global strategic partnership for Pracinostat," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "Now we approach the end of the calendar year in a position of significant strength, armed with a fully funded Phase III program, a pipeline of drug candidates advancing in the clinic and a healthy cash position that affords us the ability to not only execute our clinical development plan, but also to enhance our pipeline with the right opportunity."

Pipeline Update

Strategic partnership for development and commercialization of Pracinostat. In August 2016, the Company entered into an exclusive licensing, development and commercialization agreement with Helsinn Healthcare, SA, a Swiss pharmaceutical corporation, for Pracinostat, an investigational agent currently being evaluated in clinical studies for acute myeloid leukemia (AML) and other potential indications. Under the terms of the agreement, Helsinn is granted a worldwide exclusive license to Pracinostat and is responsible for funding its global development and commercialization. As compensation, MEI Pharma will receive near-term payments of $20 million, including a $15 million upfront payment and a $5 million payment upon the earlier of (i) dosing of the first patient in the upcoming Phase III study of Pracinostat in newly diagnosed AML patients unfit to receive induction therapy, or (ii) March 1, 2017. In addition, MEI Pharma will be eligible to receive up to $444 million in potential regulatory and sales-based milestones, along with royalty payments on the net sales of Pracinostat. In a related transaction, Helsinn made a $5 million equity investment in MEI Pharma.
Dose-optimization study of Pracinostat plus azacitidine in high-risk MDS. As part of the license, development and commercialization agreement, the Company will work with Helsinn to determine an optimal dosing regimen of Pracinostat in combination with azacitidine for the treatment of high and very high risk myelodysplastic syndrome (MDS). Based on its clinical experience with the combination, the Company believes that an optimized dose may improve tolerability in this population, which in turn could result in increased efficacy of the combination vs. azacitidine alone. MEI Pharma will be responsible for the conduct of the study and Helsinn will share the cost. The study is anticipated to commence in the first half of calendar year 2017.
Breakthrough Therapy Designation from U.S. Food and Drug Administration (FDA). In August 2016, the Company announced that the FDA granted Breakthrough Therapy Designation for Pracinostat in combination with azacitidine for the treatment of patients with newly diagnosed AML who are ≥75 years of age or unfit for intensive chemotherapy. According to the FDA, Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. In addition, the Company announced that agreement has been reached with the FDA on the proposed Phase III AML study design.
Phase Ib study of PI3K delta inhibitor ME-401 open for enrollment. In September 2016, the Company’s Phase 1b clinical study of ME-401 in patients with relapsed refractory chronic lymphocytic leukemia/small lymphocytic lymphoma or follicular lymphoma was opened for enrollment. ME-401 is a potent and highly selective oral PI3K delta inhibitor with the potential for a wide therapeutic window that may lead to safer treatment options, including combination treatment options, for patients with lymphomas. This study will allow the Company to study the safety and tolerability of ME-401 over time as it seeks to identify an optimal dose for future Phase 2 studies. Interim data from the study is expected by the middle of calendar year 2017.
Investigator-sponsored study of mitochondrial inhibitor ME-344 open for enrollment. In August 2016, an investigator-sponsored study of ME-344 in combination with the vascular endothelial growth factor (VEGF) inhibitor bevacizumab (marketed as Avastin) in patients with HER2-negative breast cancer was opened for enrollment. The study is being conducted in collaboration with the Spanish National Cancer Research Centre in Madrid. Pre-clinical data from the collaboration showed substantially enhanced anti-tumor activity of ME-344 in cancer cells when combined with VEGF inhibitors due to a disruption of both mitochondrial and glycolytic metabolism. Data from the investigator-sponsored study are expected by the end of calendar year 2017.
Upcoming Data Presentations

Long-term survival and response data from Phase II study of Pracinostat in AML. Data from a multi-center Phase II clinical study of Pracinostat and azacitidine in older patients with AML who are not eligible for induction chemotherapy have been selected for oral presentation at the upcoming ASH (Free ASH Whitepaper) Annual Meeting in San Diego on December 3, 2016. The presentation by principal investigator Dr. Guillermo Garcia-Manero, MD Anderson Cancer Center, is expected to highlight the durable responses and long-term survival benefit achieved in this investigational study, which appeared to compare favorably to azacitidine alone in a recent international Phase III study (AZA-AML-001). Pracinostat is an investigational agent and is not approved for use in the U.S.
Exposure data from clinical study of ME-401 supporting wide therapeutic window. Additional data from a first-in-human clinical study of ME-401 in healthy volunteers will be presented at the American Association of Pharmaceutical Scientists (AAPS) Annual Meeting in Denver on November 15, 2016. The presentation will highlight formulation selection and development for ME-401, including exposure margins based on clinical data and preclinical toxicity supporting the potential for an improved therapeutic window from repeated dosing compared to the approved PI3K delta inhibitor idelalisib (marketed as Zydelig).
Financial Highlights

As of September 30, 2016, MEI Pharma had $58.9 million in cash, cash equivalents and short-term investments, which the Company believes will be sufficient to fund operations through at least calendar year 2017.
Net loss was $4.3 million, or $0.12 per share, for the three months ended September 30, 2016, compared to $5.7 million, or $0.17 per share, for the previous quarter, and $4.6 million, or $0.13 per share for the same period in 2015.
Research and development expenses were $1.6 million for the three months ended September 30, 2016, compared to $2.8 million for the same period in 2015. The decrease was primarily due to a reduction in clinical trial expenses for Pracinostat and ME-344.
General and administrative expenses were $2.7 million for the three months ended September 30, 2016, compared to $1.8 million for the same period in 2015. The increase was primarily due to professional service costs associated with the Helsinn license agreement.

On November 9, 2016 Eagle Pharmaceuticals, Inc. ("Eagle" or "the Company") (Nasdaq:EGRX) reported its financial results for the three- and nine-months ended September 30, 2016 (Press release, Eagle Pharmaceuticals, NOV 9, 2016, View Source [SID1234516517]). Highlights of and subsequent to the third quarter of 2016 include:

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Business Highlights:

Bendeka total market share rose to 88%, as of November 6, 2016;
The Centers for Medicare & Medicaid Services (CMS) established a unique J-Code (J9034) for Bendeka effective January 1, 2017;
Positive initial results of animal study exploring use of Ryanodex in MDMA (Ecstasy) induced hyperthermia conducted at the National Institutes of Health (NIH);
Extended our licensing agreement with Teva Pharmaceutical Industries Ltd. (Teva) to include certain territories outside the US and Canada; and
Repurchased $18.0 million of Eagle common stock during the third quarter for a total of $32.0 million since commencing the Share Repurchase Program on August 9, 2016.
Financial Highlights:

Total revenue was $37.8 million during the third quarter of 2016 compared to $5.7 million for the three months ended September 30, 2015;
Product sales increased to $7.8 million during the third quarter of 2016 compared to $3.3 million for the prior year period;
Royalty income increased to $26.2 million during the third quarter of 2016 compared to $2.4 million for the prior year period;
License and other income was $3.8 million during the third quarter of 2016;
Sales of Ryanodex grew 92% to $2.5 million during the third quarter of 2016 compared to $1.3 million for the prior year period;
Net income was $12.0 million, or $0.77 per basic and $0.73 per diluted share, compared to a net loss of $10.2 million, or $(0.65) per basic and diluted share, for the three months ended September 30, 2016 and 2015, respectively; and,
Cash and cash equivalents were $59.3 million and accounts receivable were $47.1 million as of September 30, 2016.
"We had another strong quarter delivering results for our shareholders, reflecting our ability to execute Eagle’s strategy to develop and commercialize improved formulations that enhance patients’ lives. Bendeka market share continues to ramp up and is approaching our joint goal with Teva of 90%. We believe the CMS final ruling to establish a unique J-Code for Bendeka will not only aid further adoption, but also provide greater access for patients and facilitate reimbursement. We remain confident in our ability to drive continued growth in our bendamustine franchise well beyond 2019," stated Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals.

"We are also particularly excited about the multiple opportunities within our pipeline to drive substantial value beyond 2020. We are very encouraged by early results of clinical testing to explore the potential of Ryanodex for the treatment of both exertional heat stroke and MDMA (Ecstasy) induced hyperthermia. If approved, both indications would address the needs of very significant new patient populations for whom no pharmaceutical treatment options are currently available. Our development work on additional product candidates continues, providing us with a robust pipeline from which to create long term value," added Tarriff.

"We remain focused on optimizing the deployment of our capital on behalf of shareholders and continue to evaluate opportunities to do so. As part of that effort, since August 9, we purchased a total of $32 million through our stock buyback program and are pleased with our solid financial position," concluded Tarriff.

Third Quarter 2016 Financial Results

Total revenue for the three months ended September 30, 2016 was $37.8 million, as compared to $5.7 million for the three months ended September 30, 2015. A summary of total revenue is outlined below:

Three Months Ended
September 30,

Increase
2016 2015
(in thousands)
Product sales $ 7,837 $ 3,314 $ 4,523
Royalty income 26,246 2,422 23,824
License and other income 3,750 3,750
Total revenue $ 37,833 $ 5,736 $ 32,097

Product sales increased $4.5 million to $7.8 million driven by due to increases in Bendeka, Non-Alcohol Docetaxel Injection, Argatroban, and Ryanodex net product sales of Ryanodex, offset by a decrease in net product sales of diclofenac-misoprostol. Royalty income increased $23.8 million to $26.2 million, as a result of the launch of Bendeka in January 2016.

Cost of revenue increased by $6.7 million to $10.4 million in the three months ended September 30, 2016 from $3.8 million in the three months ended September 30, 2015. This $6.7 million net increase resulted from $0.8 million in cost of revenue for Non-Alcohol Docetaxel Injection, an increase of $0.8 million in the cost of Argatroban, and an increase of $5.3 million related to the cost of Bendeka product sales: $2.4 million of product sales, $2.4 million of royalties and $0.5 million of other expense, and a decrease of $0.2 million in cost of revenue for Ryanodex.

Research and development expenses decreased by $3.7 million to $3.2 million in the three months ended September 30, 2016, compared to $6.9 million in the prior year quarter. The decrease resulted from certain cost reimbursements from one of our suppliers, non-recurrence of project spending for Ryanodex (dantrolene sodium) for exertional heatstroke related to the completion of the clinical treatment portion of the safety and efficacy study, and lower project spending for pemetrexed, offset by the increase in project spending for bivalirudin, and other projects, and higher salary and other personnel-related expenses due to increased headcount.

SG&A expenses increased $6.4 million to $11.9 million in the third quarter of 2016 compared to $5.5 million in the three months ended September 30, 2015. Personnel-related expenses accounted for the bulk of the $6.4 million increase and were due to overall expansion of the business.

Net income for the third quarter was $12.0 million, or $0.77 per basic share and $0.73 per diluted share, compared to a net loss of $10.2 million, or ($0.65) per basic and diluted share in the three months ended September 30, 2015, as a result of the factors discussed above.

Liquidity

As of September 30, 2016, the Company had $59.3 million in cash and cash equivalents; $47.1 million in receivables, with approximately $30.9 million due from Teva; and no debt.

Events Subsequent to the End of the Third Quarter

The Centers for Medicare & Medicaid Services (CMS) established a unique, product-specific billing code, or J-code (J9034), for Bendeka (bendamustine hydrochloride) Injection. The J-code will become effective on January 1, 2017.

The new J-code provides reimbursement coding clarity to outpatient facilities and physicians that administer Bendeka, facilitating access for patients and Medicare, Medicaid and commercial insurance reimbursement.

The J-Code decision triggered a $40 million milestone payment from Teva and an increase in Eagle’s royalty from 20% to 25% of net sales of Bendeka.