On May 10, 2016 Calithera Biosciences, Inc. (Nasdaq:CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2016 (Press release, Calithera Biosciences, MAY 10, 2016, View Source;p=RssLanding&cat=news&id=2167159 [SID:1234512234]). As of March 31, 2016, cash, cash equivalents and investments totaled $68.3 million.

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"During the first quarter, we continued to advance our development pipeline by recruiting patients into clinical studies of CB-839, and preparing for an Investigational New Drug (IND) application for CB-1158, as well as expanding our clinical leadership team," said Susan Molineaux, Ph.D., President and Chief Executive Officer of Calithera. "More recently, at the 2016 American Association of Cancer Research annual meeting, we and our collaborators presented four abstracts highlighting synergy studies of CB-839 and CB-1158. CB-839 is currently enrolling multiple combination cohorts in our Phase Ib clinical trials, for which updates are planned in June."

First Quarter 2016 and Recent Highlights

Enrollment continues across CB-839 Phase Ib combination cohorts. Data from the Phase I solid tumor trial has been accepted for presentation at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. Initial results of CB-839 in combination with paclitaxel in patients with triple negative breast cancer will be presented in a poster and discussion on June 5, 2016. Initial results of CB-839 alone and in combination with everolimus in patients will renal cell carcinoma will be presented in a poster on June 6, 2016.
CB-839 preclinical findings, including combinations with immuno-oncology therapies, presented at the American Association of Cancer Research. In April 2016, Calithera presented preclinical data for CB-839 in combination with immuno oncology-agents. The combination of CB-839 and anti-PD-L1 or anti-PD-1 substantially increased the number of tumor regressions seen in the CT26 syngeneic colon carcinoma model. Synergistic effects with CB-839 and anti-PD-L1 were also observed in a B16 melanoma model. Both of these agents are known to affect metabolism in the tumor microenvironment. Treatment with anti-PD-1 or PD-L1 increases glucose metabolism in T cells and CB-839 increases the amount of glutamine available for T cells because it inhibits the avid metabolism of glutamine by the tumor itself. T cells require adequate levels of these two key nutrients to expand and mount a strong anti-tumor response.
CB-1158 preclinical findings presented at the American Association of Cancer Research. CB-1158, a highly selective, orally bioavailable, small molecule inhibitor of human arginase with nanomolar potency, demonstrated single agent efficacy in animal models. Inhibition of tumor growth was accompanied by an increase in the local concentration of arginine, and the induction of multiple pro-inflammatory changes in the tumor microenvironment. CB-1158 increased CD8+ T-cell infiltrates in a lung tumor model. The addition of CB-1158 to anti-CTLA-4 and anti-PD-1 significantly inhibited tumor growth and reduced metastases in a mouse model that was resistant to dual checkpoint inhibitor therapy. CB-1158 was well tolerated as a single agent and in combination with checkpoint inhibitors in animal studies.
Key hire and promotion in clinical group. Calithera announced today the expansion of its clinical group with the hiring of Sam Whiting, M.D., Ph.D. as Vice President of Clinical Development. Most recently, Dr. Whiting served as Vice President of Research and Clinical Development at Gradalis, Inc. Previously Dr. Whiting was an assistant member at the Fred Hutchinson Cancer Research Center (FHCRC), and assistant professor at the University of Washington where he was clinical director of gastrointestinal oncology. Dr. Whiting received his M.D. and Ph.D. degrees and completed his internal medicine residency at the University of Washington, and medical oncology fellowship at the FHCRC. Keith Orford, M.D., Ph.D., who joined Calithera in January 2015, has been promoted to Senior Vice President of Clinical Development.
Selected First Quarter 2016 Financial Results

Research and development expenses were $7.1 million for the three months ended March 31, 2016, compared with $5.6 million for the same period in the prior year. The increase of $1.4 million was primarily attributed to increased development activities in Calithera’s arginase inhibitors program as it plans to file an IND in mid-2016, partially offset by a decrease of $0.4 million related to Calithera’s licensing arrangements.

General and administrative expenses were $2.6 million for the three months ended March 31, 2016, compared with $2.2 million for the same period in the prior year. The increase of $0.4 million was primarily due to higher employment related expenses, including stock-based compensation expense.

Net loss from operations for the three months ended March 31, 2016 was $9.6 million.

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 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the first quarter ended March 31, 2016 and recent company developments (Press release, TG Therapeutics, MAY 10, 2016, View Source [SID:1234512227]).

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Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "The first quarter was another productive one for TG with the issuance of long term patent protection for both TG-1101 and TGR-1202, the presentation of data at AACR (Free AACR Whitepaper) providing a scientific rationale for the observed safety differences seen with TGR-1202 in comparison to other PI3K delta inhibitors, and continued enrollment into our CLL Phase 3 trials, which remains our top priority for the year. More recently, we announced the commencement of our first Phase 2 study in Multiple Sclerosis, and plans to enter Phase 3 for MS next year." Mr. Weiss continued, "We have a long term vision to build best-in-class combination treatments across B-cell malignancies and our Phase 3 CLL trials are just the beginning as we look forward to announcing the opening of our UNITY-DLBCL program toward the end of this month and launching UNITY-iNHL before year-end. Our financial resources remain strong, leaving us well positioned to execute on our aggressive business plan."

Recent Developments and Highlights

Announced that a composition of matter patent had been issued in the U.S. for TGR-1202, the Company’s orally available PI3K delta inhibitor, providing patent protection through July 2033, exclusive of patent term extensions.
Announced that a composition of matter patent had been issued in the U.S. for TG-1101, providing patent protection through July 2029, exclusive of patent term extensions.
Presented pre-clinical data describing the differential regulation of human T-cells by TGR-1202 as opposed to other PI3K delta inhibitors in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016.
Recently announced the commencement of the Company’s first clinical trial of TG-1101 in Multiple Sclerosis.
Reaffirming 2016 Milestones

Continue to aggressively recruit into the GENUINE Phase 3 clinical trial of TG-1101 in combination with ibrutinib
Continue to aggressively enroll into the UNITY-CLL combination Phase 3 clinical trial of the Company’s proprietary combination of TG-1101 plus TGR-1202 (aka "TG-1303")
Commence the UNITY-DLBCL Phase 2b clinical trial
Enroll into the Phase 2 clinical trial in Multiple Sclerosis
Commence a registration trial for indolent NHL
Present updated data on the Phase 1 and 2 clinical trials at major hematology/oncology conferences during 2016
Financial Results for the First Quarter 2016

At March 31, 2016 the Company had cash, cash equivalents, investment securities, and interest receivable of $85.3 million, which we believe will be sufficient to fund our operations into the second quarter of 2018.

Our net loss for the first quarter ended March 31, 2016, excluding non-cash items, was approximately $12.1 million, which included approximately $4.3 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The net loss for the first quarter ended March 31, 2016, inclusive of non-cash items, was $13.8 million, or $0.28 per basic and diluted share, compared to a net loss of $14.6 million during the comparable quarter in 2015, or $0.35 per basic and diluted share. The decrease in net loss during the first quarter ended March 31, 2016 was the result of a decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2015, partially offset by an increase in other research and development expenses as a result of clinical trial expenses related to ongoing and planned future Phase 3 registration programs.

On May 10, 2016 Nicox S.A. (Euronext Paris: FR0013018124, COX), the international ophthalmic company, reported its first quarter 2016 revenues and cash position and provided an update on its activities (Press release, NicOx, MAY 10, 2016, View Source [SID:1234512225]).

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Michele Garufi, Chairman and Chief Executive Officer of Nicox, stated, "We have successfully started 2016 with our strongest quarter revenue to date, €3.5 million, reflecting more than 60% growth. We also achieved the important development milestone of the submission last month of the AC-170 NDA for allergic conjunctivitis, for which we expect to hear shortly from the FDA concerning the Priority Review status. In addition, the recent ARVO ophthalmology congress in Seattle featured presentations by both our partner, Bausch + Lomb, on latanoprostene bunod, and the Nicox teams on our NO-donating pipeline, raising significant interest in the scientific community around the results of our new research projects. We look forward to communicating the FDA’s feedback on the latanoprostene bunod and AC-170 NDAs in due course."

First-quarter financial highlights

The Group’s revenues in the first three months of 2016 totaled €3.5 million and consisted exclusively of European and International product sales. These compare to €2.1 million in the first quarter of 2015, a growth of over 60%. Nicox is currently evaluating a number of strategic options for its European commercial business for which discussions remain ongoing.

The Group had cash, cash equivalents and financial instruments of €20.8 million as of March 31, 2016. The cash burn in the first quarter 2016 included €3.1 million of non-recurrent spending related to submission of the AC170 New Drug Application (NDA) and additional work so support the AC-170 NDA review.

First-quarter and post-reporting period operational highlights

In January 2016, Nicox Ophthalmics, Inc. granted Ora Inc., the world’s leading ophthalmic clinical research and product development firm, exclusive worldwide rights for the development and commercialization of the OTC asset AC-120, an innovative drug-candidate for morning eyelid swelling ("puffy eyes").
Submission of AC-170 NDA to the U.S. Food and Drug Administration (FDA) on 18 April 2016, with a request for Priority Review, for the treatment of ocular itching associated with allergic conjunctivitis. This NDA has not yet been accepted for review by the FDA. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing, and to confirm if the Priority Review has been granted. AC-170 is a novel formulation of cetirizine, the active ingredient in Zyrtec1, which has been developed for the first time for topical application in the eye for the treatment of ocular itching associated with allergic conjunctivitis.
In May 2016, posters were presented by our partner Bausch + Lomb of clinical results for latanoprostene bunod and by Nicox on pipeline candidates NCX 667, NCX 1653, and NCX 4240 at the Association for Research in Vision and Ophthalmology (ARVO) 2016 Annual Meeting (see Press Release dated May 9, 2016).
Key Upcoming Milestones

Q2 2016: Decision by the FDA on the Priority Review status for AC-170
July 21, 2016: PDUFA date for latanoprostene bunod
September 22, 2016: First-half results

On May 10,2016 Puma Biotechnology, Inc. (NYSE: PBYI), a biopharmaceutical company, reported financial results for the first quarter ended March 31, 2016 (Press release, Puma Biotechnology, MAY 10, 2016, View Source [SID:1234512220]).

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Unless otherwise stated, all comparisons are for the first quarter 2016 compared to the first quarter 2015.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $71.0 million, or $2.19 per share, for the first quarter of 2016, compared to a net loss applicable to common stock of $52.5 million, or $1.66 per share, for the first quarter of 2015.

Non-GAAP adjusted net loss was $41.5 million, or $1.28 per share, for the first quarter of 2016, compared to non-GAAP adjusted net loss of $32.4 million, or $1.02 per share, for the first quarter of 2015. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release. The Company anticipates that non-GAAP net loss will decrease in subsequent quarters due to an expected reduction in clinical trial expenses and the completion of the regulatory filings for neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer in the United States and Europe.

Net cash used in operating activities for the first quarter of 2016 was $35.0 million. At March 31, 2016, Puma had cash and cash equivalents of $78.2 million and marketable securities of $103.0 million, compared to cash and cash equivalents of $31.6 million and marketable securities of $184.3 million at December 31, 2015. The Company anticipates that net cash used in operating activities will be lower in subsequent quarters due to a reduction in the expenses described above.

"To date in 2016, we have achieved a number of key milestones, including publication of the results of the Phase III ExteNET trial of neratinib in the extended adjuvant treatment of HER2-positive early stage breast cancer in The Lancet Oncology, the publication of neratinib in the front-line treatment of HER2-positive metastatic breast cancer (NEfERT-T Phase II trial) in JAMA Oncology in April, and numerous presentations on neratinib in HER2 non-amplified breast cancer that has a HER2 mutation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Our near term focus is on the filing of our regulatory submissions with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer, which we anticipate will occur in mid-2016 and the second quarter of 2016, respectively.

"We look forward to continuing our development of neratinib during 2016. We anticipate (i) submitting a New Drug Application (NDA) to the FDA in mid-2016 and submitting a Marketing Authorization Application (MAA) to the EMA during the second quarter of 2016 for neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer based on the positive ExteNET Phase III trial; (ii) reporting additional data from the Phase II trial of neratinib as an extended adjuvant treatment in HER2-positive early stage breast cancer using loperamide prophylaxis during the second quarter of 2016; (iii) reporting additional Phase II data from the FB-7 neoadjuvant HER2-positive breast cancer trial in the subgroup of patients who are MammaPrint High during the second quarter of 2016; (iv) reporting Phase II data from an investigator sponsored trial of neratinib in patients with HER2-negative breast cancer who have a HER2 mutation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2016; (v) reporting data from the Phase III trial of neratinib in third-line HER2-positive metastatic breast cancer patients in either the fourth quarter of 2016 or the first quarter of 2017; (vi) reporting data from the Phase II trial of neratinib in metastatic breast cancer patients with brain metastases during the fourth quarter of 2016; and (vii) reporting data from the Phase II trial of neratinib plus fulvestrant in patients with HER2 non-amplified breast cancer that has a HER2 mutation during the fourth quarter of 2016."

Operating Expenses

Operating expenses were $71.2 million for the first quarter of 2016, compared to $52.6 million for the first quarter of 2015.

General and Administrative Expenses:

General and administrative expenses were $11.0 million for the first quarter of 2016, compared to $7.9 million for the first quarter of 2015. The approximately $3.1 million increase resulted primarily from increases of approximately $1.2 million for stock-based compensation, $1.3 million for professional fees, $0.4 million for payroll and related costs, and $0.2 million for facility and equipment costs. These increases reflect overall corporate growth.

Research and Development Expenses:

Research and development (R&D) expenses were $60.2 million for the first quarter of 2016, compared to $44.7 million for the first quarter of 2015. The approximately $15.5 million increase resulted primarily from increases of approximately $8.2 million for stock-based compensation, $4.3 million for clinical trial expenses, $2.6 million for internal R&D and related expenses, and $0.4 million for consultants and contractors. We expect R&D expenses to decrease in subsequent quarters as we complete clinical trials and file for regulatory approvals for neratinib for the extended adjuvant treatment of HER2-positive early stage breast cancer in the United States and European Union.