On May 10, 2016 Genmab reported its Interim Report for the First Quarter of 2016 (Press release, Genmab, MAY 10, 2016, View Source [SID:1234512173]).

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Net Sales of DARZALEX (daratumumab) by Janssen for the first quarter of 2016 were USD 101.9 million, resulting in royalty income of DKK 83 million
Announced positive interim result in Phase III Castor study of daratumumab in relapsed or refractory multiple myeloma
Announced studies of daratumumab in combination with atezolizumab in a solid tumor and multiple myeloma
Achieved USD 5 million milestone for progress in the Phase II study of daratumumab in non-Hodgkin’s lymphoma (NHL) under collaboration with Janssen

Announced updated development plans for ofatumumab in autoimmune indications
U.S. Food and Drug Administration (FDA) Approval of Arzerra (ofatumumab) as extended treatment for recurrent or progressive chronic lymphocytic leukemia (CLL)
U.S. and EU regulatory submissions for ofatumumab in combination with fludarabine and cyclophosphamide for relapsed CLL
"The first quarter of 2016 saw continued rapid progress in the development of daratumumab with Janssen: We reported positive interim data in the Phase III Castor study of daratumumab in combination with bortezomib and dexamethasone, achieved the second milestone in the Phase II NHL study, and announced the first study to combine daratumumab with Roche’s anti-PDL1 antibody atezolizumab, in a solid tumor and multiple myeloma. We also started off the year with a number of achievements under our Arzerra collaboration with Novartis. Arzerra was approved in the U.S. as extended treatment for recurrent or progressive CLL and regulatory submissions for ofatumumab in combination with fludarabine and cyclophosamide in relapsed CLL were submitted in the U.S. and Europe. Furthermore, we announced that development of the subcutaneous formulation of ofatumumab in autoimmune indications will be focused on relapsing multiple sclerosis, with large Phase III studies run by Novartis expected to start later this year," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Financial Performance First Quarter
Revenue was DKK 170 million in the first quarter of 2016 compared to DKK 107 million in the first quarter of 2015. The increase of DKK 63 million, or 59%, was mainly driven by higher royalty and milestone revenue under our daratumumab collaboration with Janssen.

Operating expenses were DKK 154 million in the first quarter of 2016 compared to DKK 110 million in the first quarter of 2015. The increase of DKK 44 million, or 40%, was due to the additional investment in our pipeline of products, including the advancement of tisotumab vedotin, HuMax-AXL-ADC, HexaBody-DR5/DR5, DuoBody-CD3xCD20, and our other pre-clinical programs.

Operating income was DKK 16 million in the first quarter of 2016 compared to DKK 173 million in the first quarter of 2015. The decrease of DKK 157 million was driven by the one-time reversal of the ofatumumab funding liability of DKK 176 million in 2015 combined with increased operating expenses, which were partly offset by higher revenue.

On March 31, 2016, Genmab had a cash position of DKK 3,491 million, similar to the cash position of DKK 3,493 million at December 31, 2015.

Business Progress First Quarter to Present
Daratumumab
March: Announced that the Phase III Castor study (MMY3004) of daratumumab in combination with bortezomib and dexamethasone versus bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma met the primary endpoint of improving progression free survival (PFS) in a planned interim analysis (p<0.0001). Janssen will engage in a dialogue with the health authorities about the potential for these data to serve as the basis for a regulatory submission.

March: Announced that daratumumab will be investigated in Phase Ib clinical studies in combination with atezolizumab, an anti-PD-L1 antibody, in a solid tumor and multiple myeloma. The studies will be conducted under a collaboration agreement between Janssen Biotech, Inc. (Janssen) and Genentech, a member of the Roche Group.

March: Achieved the second milestone in the ongoing Phase II study of daratumumab in NHL, triggering a USD 5 million payment from Janssen.

Ofatumumab
March: Announced that supplemental regulatory applications for the use of Arzerra in combination with fludarabine and cyclophosphamide for the treatment of patients with relapsed CLL were submitted in the U.S. and EU by Novartis.

March: Announced an update on development plans for ofatumumab in autoimmune indications focusing on relapsing multiple sclerosis following the transfer of the rights to ofatumumab in this disease area from GlaxoSmithKline (GSK) to Novartis at the end of 2015. Phase III studies of the subcutaneous formulation of ofatumumab in relapsing multiple sclerosis are expected to be initiated by Novartis during the second half of 2016. The Phase III study of the subcutaneous formulation of ofatumumab in pemphigus vulgaris, which was started by GSK, will be discontinued.

January: The U.S. FDA approved a supplemental Biologics License Application (sBLA) for the use of Arzerra for extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive CLL.

Subsequent Events
April: Reported additional data from the Phase III Castor study of daratumumab in combination with bortezomib and dexamethasone versus bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma. The study met the primary endpoint of improving PFS; Hazard Ratio (HR) = 0.39, p<0.0001. The median PFS for patients treated with daratumumab has not been reached, compared to median PFS of 7.2 months for patients who did not receive daratumumab. Data from this study was accepted for oral presentation in a Plenary Session at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

April: Announced that MorphoSys filed a complaint at the U.S. District Court of Delaware against Genmab and Genmab’s collaboration partner Janssen, for patent infringement under U.S. patent no. 8,263,746 based on activities relating to the manufacture, use and sale of DARZALEX in the United States. Genmab and Janssen disagree with the allegations made by MorphoSys in its complaint for patent infringement related to CD38 antibodies and intend to vigorously contest those allegations.
April: The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the granting of a conditional marketing authorization for DARZALEX intended for the treatment of relapsed and refractory multiple myeloma. The recommendation is for the use of DARZALEX as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor (PI) and an immunomodulatory agent and who have demonstrated disease progression on the last therapy.

Outlook
Genmab is maintaining its 2016 revised financial guidance published on April 20, 2016.

On may 10, 2016 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2016 second quarter ended March 31, 2016 (Press release, Arrowhead Research Corporation, MAY 10, 2016, View Source [SID:1234512232]). The company is hosting a conference call at 4:30 p.m. EDT to discuss results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and enter Conference ID 5054757.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 404-537-3406 and enter Conference ID 5054757.

Fiscal 2016 Second Quarter and Recent Company Highlights

Corporate Events

Completed corporate name change to Arrowhead Pharmaceuticals, Inc. to better reflect the company’s stage of development and launched a new website at ArrowheadPharma.com with an updated corporate identity
ARC-520

Began dosing patients in three Phase 2b studies: the MONARCH study, 2007 long-term extension, and 2001 open-label extension
Presented promising ARC-520 hepatitis B data at The International Liver Congress 2016, including the following key findings:
ARC-520 and entecavir produced rapid HBV DNA suppression with all hepatitis B e-antigen (HBeAg) positive, treatment naïve patients achieving serum HBV DNA reductions of up to 5.5 log (99.9997%), and all HBeAg negative, treatment naïve patients achieving reductions that put them below the limit of quantitation
ARC-520 effectively inhibited HBV cccDNA-derived mRNA with observed viral protein reduction in HBV patients of up to 2.0 log (99%) after a single dose
ARC-520 had a long duration of effect with HBsAg still reduced by 83% after 2 months and 75% after 3 months, which is the final time point of the study, after a single dose
Based on HBsAg epitope profile analysis, poster authors and Arrowhead collaborators had previously identified a predictive hepatitis B surface-antigen (HBsAg) Clearance Profile associated with HBsAg clearance in antiviral therapy cohorts
There was a significant association between the development of an HBsAg Clearance Profile and ARC-520 therapy in HBV patients
Complexed HBsAg antibodies (anti-HBs) were developed and detected in HBV patients treated with ARC-520, which may represent a recovery of the immune system response
After monthly administration of 6-11 doses of ARC-520 in chimpanzees chronically infected with HBV, the ARC-520 target site sequences remained virtually unchanged, indicating that no drug resistance developed during the treatment period
ARC-521

Filed for regulatory clearance to begin a Phase 1/2 first-in-human study to assess single and multiple-doses of ARC-521 in healthy volunteers and HBV patients
ARC-AAT

Received Orphan Drug Designation by the European Medicines Agency
Platform and Early Pipeline

Presented promising new preclinical data the 2016 American Academy of Allergy, Asthma & Immunology Annual Meeting suggesting that ARC-F12, an RNAi therapeutic that inhibits the production of Factor XII (F12), has the potential to treat hereditary angioedema and to prevent thrombosis
Presented data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 (AACR16), showing that ARC-HIF2 inhibited tumor growth and promoted tumor cell death and structural degeneration in two different renal cell carcinoma tumor bearing mouse models
These data also show that important advancements are being made to Arrowhead’s Dynamic Polyconjugate (DPC) delivery platform to include extra-hepatic targeting capabilities
Presented data on ARC-LPA, a preclinical development program targeting lipoprotein (a), or Lp(a), for the treatment of cardiovascular disease, at the Arteriosclerosis, Thrombosis and Vascular Biology | Peripheral Vascular Disease (ATVB|PVD) 2016 Scientific Sessions
These data show that ARC-LPA and Arrowhead’s new delivery vehicles designed for subcutaneous administration can induce deep target gene knockdown with long duration of effect that may enable monthly, bi-monthly, or even less frequent administration
Selected Fiscal 2016 Second Quarter Financial Results

ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED CONDENSED FINANCIAL INFORMATION (unaudited)

Three Months Ended Six Months Ended
March 31, March 31,
OPERATING SUMMARY
2016 2015 2016 2015

REVENUE $ 43,750 $ 43,750 $ 87,500 $ 214,500
OPERATING EXPENSES
Research and development 10,020,826 11,640,794 20,359,659 29,387,524
Acquired in-process research and development - 10,142,786 - 10,142,786
Salaries and payroll-related costs 4,248,693 3,541,652 8,168,579 6,692,268
General and administrative expenses 3,818,335 1,696,623 5,769,944 3,782,826
Stock-based compensation 2,416,839 2,205,079 4,797,182 4,219,935
Depreciation and amortization 803,912 449,559 1,598,261 739,598
TOTAL OPERATING EXPENSES 21,308,605 29,676,493 40,693,625 54,964,937
OPERATING LOSS (21,264,855 ) (29,632,743 ) (40,606,125 ) (54,750,437 )
OTHER INCOME/(EXPENSE), PROVISION FOR INCOME TAXES 448,995 948,750 525,851 3,488,743
NET LOSS $ (20,815,860 ) $ (28,683,993 ) $ (40,080,274 ) $ (51,261,694 )

EARNINGS PER SHARE (BASIC AND DILUTED): $ (0.35 ) $ (0.51 ) $ (0.67 ) $ (0.93 )
WEIGHTED AVERAGE SHARES OUTSTANDING 59,779,128 55,719,923 59,663,270 55,200,512

FINANCIAL POSITION SUMMARY
March 31, September 30,
2016 2015
CASH AND CASH EQUIVALENTS 50,300,847 81,214,354
SHORT-TERM INVESTMENTS 11,160,442 17,539,902
TOTAL CASH RESOURCES (CASH, CASH EQUIVALENTS AND INVESTMENTS) 61,461,289 98,754,256
OTHER ASSETS 33,146,182 33,513,658
TOTAL ASSETS 94,607,471 132,267,914
TOTAL LIABILITIES 20,681,234 22,646,280
TOTAL STOCKHOLDERS’ EQUITY 73,926,237 109,621,634
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY 94,607,471 132,267,914

SHARES OUTSTANDING 59,960,711 59,544,677
PROFORMA SHARES OUTSTANDING (INCLUDING CONVERSION OF PREFERRED SHARES)

On May 10, 2016 Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported its first quarter results for 2016 (Press release, Intrexon, MAY 10, 2016, View Source [SID:1234512269]).

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Intrexon Corporation logo.
Business Highlights and Recent Developments:

National Health Surveillance Agency of Brazil (Anvisa) announced that Oxitec, a wholly-owned subsidiary of Intrexon, would receive a special temporary registration to deploy its genetically engineered mosquito, OX513A, known as ‘Friendly Aedes aegypti’, throughout the country;
The World Health Organization’s Vector Control Advisory Group issued a positive recommendation for planned pilot deployment of Oxitec’s self-limiting mosquito (OX513A) under operational conditions. Additionally the Pan American Health Organization has offered to provide technical support for pilot studies of OX513A as part of its response to the Zika epidemic;
The U.S. Food and Drug Administration (FDA) Center for Veterinary Medicine published a preliminary finding of no significant impact (FONSI) on Oxitec’s self-limiting OX513A Ae. aegypti mosquito for an investigational trial in the Florida Keys;
Announced the Cayman Islands Mosquito Research and Control Unit (MRCU) plans to utilize Oxitec’s solution for suppression of wild populations of Ae. aegypti to help reclaim the island from this disease-carrying pest. The program follows a successful trial of Oxitec’s mosquito in the Cayman Islands that reduced Ae. aegypti by 96%;
Acquired the business of EnviroFlight LLC and formed a joint venture with Darling Ingredients Inc. (NYSE: DAR), the world’s largest publicly traded developer and producer of sustainable natural ingredients from bio-nutrients. Through collaboration between Intrexon and Darling, EnviroFlight’s scalable approach utilizing black soldier fly larvae will be used in the generation of high-nutrition, low environmental impact animal and fish feed as well as fertilizer products;
Announced the pilot plant for Intrexon’s proprietary gas-to-liquids bioconversion platform is operational. The plant is dedicated to the production of isobutanol, a drop-in fuel with numerous advantages over other clean burning gasoline blendstocks;
With collaborator Fibrocell Science, Inc. (NASDAQ: FCSC) announced allowance from the U.S. FDA to initiate a Phase I/II clinical trial for FCX-007 in adults. FCX-007 is Fibrocell’s lead gene-therapy product candidate developed in collaboration with Intrexon for the treatment of the orphan indication of recessive dystrophic epidermolysis bullosa. Additionally Fibrocell received orphan drug designation from the FDA for FCX-013, its second gene-therapy product candidate developed in conjunction with Intrexon, for the treatment of localized scleroderma;
Collaborator ZIOPHARM Oncology (NASD: ZIOP) announced the first patient was treated in the dose escalation portion of the Phase I study of Ad-RTS-hIL-12 for advanced glioma, and additionally the first patient was enrolled in the Phase I study of second generation non-viral CD-19-specific CAR T-cell therapy for advanced lymphoid malignancies;
Announced the formation of Intrexon T1D Partners, LLC, a joint venture to develop ActoBiotics based antigen-specific immunotherapy to treat type 1 diabetes (T1D) in humans. The objective of the program is to create an easy-to-take pill for people afflicted with T1D to halt autoimmune-induced damage, both for early stage patients before they become insulin dependent and also for certain advanced stage patients to potentially prevent the requirement for insulin to be administered externally;
Entered into ECCs with two startups backed by the Harvest Intrexon Enterprise Fund, sponsored by Harvest Capital Strategies, LLC. Through the proprietary technologies of Intrexon, these companies will pursue new approaches for unmet needs in human health: Relieve Genetics, Inc. will focus on a breakthrough, non-opioid gene therapy approach for neuropathic pain and Exotech Bio, Inc. will utilize a novel exosome-based platform for delivering therapeutic RNA to treat select cancer indications; and
Announced the formation of Intrexon Crop Protection (ICP), a wholly-owned subsidiary dedicated to the biological control of agricultural pests and diseases. Through the utilization of Oxitec’s diverse self-limiting gene platform for insect control as well as the ActoBiotics system for the expression of targeted biologicals, ICP will precisely target single pest species thereby avoiding off-target effects of conventional pesticide applications on the broader ecosystem.
First Quarter Financial Highlights:

Total revenues of $43.4 million, an increase of 28% over the first quarter of 2015;
Net loss of $64.4 million attributable to Intrexon, or $(0.55) per basic share, including non-cash charges of $50.6 million;
Adjusted EBITDA of $1.9 million, or $0.02 per basic share;
Cash consideration received for reimbursement of research and development services covered 56% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries);
Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 92% of consolidated cash operating expenses; and
Cash, cash equivalents, and short-term and long-term investments totaled $336.0 million, and the value of equity securities totaled $61.3 million at March 31, 2016.
In addition, Intrexon’s Board of Directors has authorized management to prepare a plan to distribute to the shareholders of Intrexon certain shares of the Intrexon Crop Protection subsidiary. Any dividend will be subject to various conditions including the registration of the subsidiary pursuant to the Securities Exchange Act of 1934.

"Your company is capably executing its plan to accomplish all of its goals for 2016 – financially, programmatically and through its partnering – and I am very proud of the fine work being done by our team," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon. "Our principal internal business objective, as always, is the maximization of the value of our growing, diversified portfolio of commercial and financial interests across many products and industries. We seek to obtain this through minimal net expenditure of our company’s cash and this is for two reasons – to maximize our ultimate cash-on-cash return and to allow the cash flows resulting from the products that we have enabled to flow to our bottom line on a relatively unburdened basis. We made significant progress in this category during the first quarter and believe that we shall continue to extend our performance trajectory throughout the year and beyond.

"Programmatic progress – and this is where the vast majority of our 750 employees are engaged every day – is largely tracking to our expectations, and we look forward to detailing some of this during our upcoming conference call and to the announcement of further developments throughout the balance of the year. Our progress in partnering, especially around some of our mature assets, keeps much of our executive team very busy, and we shall update you on this as well, but we would be remiss if we did not here especially acknowledge a new partner, the Cayman Islands and its exemplary government under the leadership of Premier Hon. Alden McLaughlin, MBE, JP, MLA, which is the first government in the world to develop a comprehensive program across a territory to fight the Aedes aegypti mosquito, the principal disease vector for Zika, dengue and chikungunya."

Mr. Kirk concluded, "Finally, under the heading of stewardship, our board of directors and management recognize a fundamental desire to seek, identify and implement means of rewarding our shareholders that go beyond ‘merely’ building a great business. As was the case with last year’s $172M dividend of equity securities in ZIOPHARM Oncology, we recognize an opportunity for our shareholders to participate directly in the ownership of Intrexon Crop Protection ("ICP") which we believe will confer significant and direct value to our shareholders beyond that which could be realized in the event that ICP had remained a wholly owned subsidiary of Intrexon. You should expect further information on this development in the near future."

First Quarter 2016 Financial Results Compared to Prior Year Period

Total revenues were $43.4 million for the quarter ended March 31, 2016 compared to $33.8 million for the quarter ended March 31, 2015, an increase of $9.6 million, or 28%. Collaboration and licensing revenues increased $9.3 million over the quarter ended March 31, 2015 due to (i) the recognition of deferred revenue for upfront payments received from the Company’s license and collaboration agreements with the biopharmaceutical business of Merck KGaA, which became effective in May 2015, and from other collaborations signed by Intrexon between April 1, 2015 and March 31, 2016; and (ii) increased research and development services for these collaborations and for the progression of programs or the addition of new programs with previously existing collaborators. Product revenues were $8.6 million for the quarter ended March 31, 2016 compared to $8.9 million for the quarter ended March 31, 2015, a decrease of $0.3 million, or 4%. The decrease primarily relates to a decrease in the quantities of livestock previously used in production and live calves sold due to lower customer demand for these products. Gross margin on product revenues declined for the same period primarily due to a decline in the average sales prices of livestock previously used in production. Service revenues were $10.7 million for the quarter ended March 31, 2016 compared to $10.0 million for the quarter ended March 31, 2015, an increase of $0.7 million, or 7%. The increase relates to an increase in the number of in vitro fertilization cycles performed due to higher customer demand.

Total operating expenses were $84.0 million for the quarter ended March 31, 2016 compared to $121.0 million for the quarter ended March 31, 2015, a decrease of $37.0 million, or 31%. Research and development expenses declined $53.4 million, or 67%, due primarily to the inclusion in 2015 of a $59.6 million payment in common stock for an exclusive license to certain technologies owned by the University of Texas MD Anderson Cancer Center (MD Anderson). This decrease was partially offset by increases in (i) salaries, benefits and other personnel costs for research and development employees (ii) lab supplies and consultant expenses, and (iii) depreciation and amortization. Salaries, benefits and other personnel costs increased $2.3 million due to (i) an increase in research and development headcount to support new and expanded collaborations and (ii) a full quarter of costs for research and development employees assumed in the Company’s various 2015 acquisitions. Lab supplies and consultant expenses increased $2.8 million as a result of (i) the progression into the preclinical phase with certain of Intrexon’s collaborators; (ii) the increased level of research and development services provided to the Company’s collaborators; and (iii) a full quarter of costs incurred as a result of the Company’s various 2015 acquisitions. Depreciation and amortization increased $1.9 million primarily as a result of (i) incurring a full quarter of depreciation and amortization on property and equipment and intangible assets acquired in the Company’s 2015 acquisitions, and (ii) amortization related to AquaBounty’s intangible assets upon regulatory approval in November 2015. Selling, general and administrative (SG&A) expenses increased $15.3 million, or 55%, over the first quarter of 2015. Salaries, benefits and other personnel costs for SG&A employees increased $6.0 million due to (i) increased headcount to support Intrexon’s corporate operations and increased stock compensation expense due to higher grant date fair values for stock options granted; (ii) a full quarter of stock compensation expense for a company-wide option grant to employees in March 2015 and (iii) a full quarter of salaries, benefits and other personnel costs for employees assumed in the Company’s 2015 acquisitions. Legal and professional expenses increased $3.7 million primarily due to (i) noncash expenses due pursuant to Intrexon’s services agreement with Third Security, LLC which the Company entered into in November 2015; (ii) legal fees for trial and post-trial activities for the Company’s litigation with XY, LLC; (iii) expenses incurred to support domestic and international government affairs for regulatory approvals necessary to commercialize the Company’s products and services; (iv) incremental costs incurred to support the ongoing operations of the Company’s 2015 acquisitions; and (v) other business development activities. For the quarter ended March 31, 2016, the Company also recorded $4.2 million in litigation settlement expenses arising from the final court order in the Company’s trial with XY, LLC.

Total other income (expense), net, was $(21.4) million for the quarter ended March 31, 2016 compared to $115.7 million for the quarter ended March 31, 2015, a decrease of $137.1 million, or 119%. This decrease was attributable to market changes in Intrexon’s current equity securities portfolio; in 2016, this portfolio no longer included shares of ZIOPHARM Oncology, Inc. since the Company distributed such shares, including all realized gains thereon, to the Company’s shareholders as a dividend in June 2015.

On May 10, 2016 GTx, Inc. (Nasdaq: GTXI) reported financial results for the first quarter ended March 31, 2016, and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAY 10, 2016, View Source;p=RssLanding&cat=news&id=2166792 [SID:1234512174]). The Company is currently enrolling patients in three clinical trials: two trials evaluating enobosarm as a potential treatment for women with advanced breast cancer and another assessing enobosarm as a potential treatment for stress urinary incontinence in postmenopausal women.

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"During the first quarter, we executed on several ongoing initiatives that we expect will make 2016 a year of considerable progress," said Dr. Robert J. Wills, Executive Chairman of GTx. "We have continued to make progress in the enrollment of our two advanced breast cancer clinical trials of enobosarm, with preliminary data from the first stage of each study expected by the end of 2016. Also, we are excited to be collaborating with doctors on a clinical trial that is the first clinical study to use a SARM to treat stress urinary incontinence in postmenopausal women and expect data from this study late in 2016."

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product candidate, a selective androgen receptor modulator (SARM), is being developed as a targeted treatment for two advanced breast cancer indications: (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical trials, the primary efficacy endpoint will be clinical benefit, which is defined as a complete response, partial response or stable disease.

ER+/AR+ breast cancer: We currently expect to complete enrollment in the first stage of the open-label, Phase 2 clinical trial of enobosarm in women with metastatic or locally advanced ER+/AR+ breast cancer in the second quarter of 2016, to allow us to determine during the fourth quarter of this year whether there is sufficient safety and efficacy data to warrant proceeding with the second stage of the clinical study. While the first stage of the trial will evaluate 18 patients for each of the two dosing arms, 9 mg and 18 mg of enobosarm, the trial is designed to enroll up to 118 patients to obtain data from 44 evaluable patients in each study arm (a total of 88 evaluable patients) to assess the primary efficacy objective of clinical benefit response following 24 weeks of treatment.
AR+ TNBC: We currently expect to complete enrollment in the first stage of the open-label, proof-of-concept Phase 2 clinical trial of 18 mg of enobosarm in women with advanced AR+ TNBC in the third quarter of 2016, to allow us to determine by the end of 2016 whether there is sufficient safety and efficacy data to warrant proceeding with the second stage of the clinical study. While the first stage will include 21 evaluable patients, the trial is designed to enroll up to 55 patients in total in order to obtain data from 41 evaluable patients to assess the primary efficacy objective of clinical benefit response following 16 weeks of treatment.
SARMs in Non-Oncologic Indications: The Company is exploring SARMs as potential treatments for both stress urinary incontinence (SUI) and Duchenne muscular dystrophy (DMD), a rare disease characterized by progressive muscle degeneration and weakness.

SUI: We are currently enrolling patients in a Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm to treat up to 35 postmenopausal women with SUI, the first clinical trial to evaluate a SARM for SUI. Top-line data from the Phase 2 clinical trial is anticipated by the end of 2016.
DMD: The Company’s preclinical studies have continued to confirm beneficial effects from SARMs in mice genetically altered to simulate DMD, compared to control groups. The Company continues to advance its preclinical initiatives while pursuing a strategic collaboration with potential biopharma partners experienced in orphan drug development.
SARDs in Prostate Cancer: Our Selective Androgen Receptor Degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), including those who do not respond or are resistant to currently approved therapies. The Company believes that its SARD compounds will degrade multiple forms of the androgen receptor, including AR splice variants, such as AR-V7.

CRPC: Several lead SARD compounds are currently being evaluated in preclinical studies to select the best SARD compounds for continued development, as well as to develop data necessary to initiate first in human clinical trials in 2017.
First Quarter 2016 Financial Results

As of March 31, 2016, cash and short-term investments were $24.3 million compared to $29.3 million at December 31, 2015.
Research and development expenses for the quarter ended March 31, 2016 were $4.0 million compared to $2.9 million for the same period of 2015.
General and administrative expenses were $2.1 million for both the quarter ended March 31, 2016 and March 31, 2015.
The Company recognized a non-cash gain of $8.2 million and $2.6 million for the quarter ended March 31, 2016 and 2015, respectively, due to the change in fair value of the Company’s warrant liability. During the first quarter of 2016, the Company recorded a non-cash reclassification of this warrant liability to stockholders’ equity due to the modification of these warrants. No adjustments to the fair value of these warrants will be made in the future.
Net income for the quarter ended March 31, 2016 was $2.1 million compared to a net loss of $2.4 million for the same period in 2015. Net income for the quarter ended March 31, 2016 included the non-cash gain of $8.2 million related the revaluation of our warrant liability. The net loss for the quarter ended March 31, 2015 included a non-cash gain of $2.6 million related to the change in the fair value of the Company’s warrant liability.
GTx had approximately 141.7 million shares of common stock outstanding as of March 31, 2016. Additionally, there remain warrants outstanding to purchase approximately 64.3 million shares of GTx common stock at an exercise price of $0.85 per share.

On May 10, 2016 CEL-SCI Corporation (NYSE MKT: CVM) reported financial results for the quarter ended March 31, 2016 (Press release, Cel-Sci, MAY 10, 2016, View Source [SID:1234512270]).

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Key corporate and clinical developments during second quarter fiscal year 2016 include:

Enrolled an additional 186 patients in the global pivotal Phase 3 head and neck cancer trial during the first six months of FY 2016, a 32% increase in enrollment compared to the first six months of FY 2015.
Enrolled a monthly record of 41 patients in April 2016, following the end of Q2.
A total of 797 patients have been enrolled in the study as of April 30, 2016.
The Company’s CEO and CSO provided informative updates in multiple interviews, including Fox Business News.
Continued patient enrollment in the Phase 1 trial of Multikine* in HIV/HPV co-infected men and women with peri-anal warts at San Diego Naval Medical Center and University of California, San Francisco (UCSF).
Received significant litigation funding for arbitration lawsuit against former CRO that led to a narrowing of G&A expense and operating loss in Q2 FY 2016.
CEL-SCI reported an operating loss of ($6,273,603) for the quarter ended March 31, 2016 versus an operating loss of ($7,759,343) for the quarter ended March 31, 2015. The operating loss for the six months ended March 31, 2016 was ($12,056,735) versus ($17,755,084) during the six months ended March 31, 2015. The decrease in operating loss in the first half of fiscal 2016 was mostly attributable to a decrease in general and administrative expenses of approximately $5,803,000, as compared to the first half of fiscal 2015. A major component of the decrease is the approximate $3,254,000 gain on the derecognition of legal fees recognized pursuant to an agreement with Lake Whillans for funding litigation expenses in the Company’s arbitration against its former CRO. Additionally, during the first half of fiscal 2015, there was approximately $2,726,000 in employee compensation costs related to the issuance of shareholder approved shares of restricted stock released upon meeting predetermined milestones. Research and development expenses remained relatively consistent and decreased by $176,000 during the six months ended March 31, 2016.

CEL-SCI’s net loss available to common shareholders for the quarter ended March 31, 2016 was ($8,844,855) or ($0.07) per basic and diluted share, versus ($12,556,236) or ($0.17) per basic and diluted share during the quarter ended March 31, 2015. The net loss available to common shareholders for the six months ended March 31, 2016 was ($6,503,042) or ($0.06) per basic and diluted share, versus ($20,401,554) or ($0.27) per basic and diluted share during the same six months ended March 31, 2015. The decrease in net loss for the three and six month periods of 2016 as compared to the same periods in 2015 was primarily attributable to the decrease in operating loss and reduced loss and unrealized gain, respectively, on the fair value of warrants as a result of the change in the stock price between reporting periods.

About Multikine (Leukocyte Interleukin, Injection)

Multikine is an investigational immunotherapeutic agent that is being tested in an open-label, randomized, controlled, global pivotal Phase 3 clinical trial as a potential first-line treatment for advanced primary (not yet treated) squamous cell carcinoma of the head and neck. Multikine is designed to be a different type of therapy in the fight against cancer: one that appears to have the potential to work with the body’s natural immune system in the fight against tumors.