On May 11, 2017 Peloton Therapeutics, Inc., a drug discovery and development company focused on advancing novel small molecule cancer therapies, reported initiation of patient dosing in a Phase 2 study of PT2385, the Company’s investigational first-in-class small molecule drug targeting hypoxia-inducible factor 2α (HIF-2α), for patients with von Hippel-Lindau (VHL) disease-associated kidney cancer (Press release, Peloton Therapeutics, MAY 11, 2017, View Source [SID1234519055]). The primary objective of the study is to assess the overall response rate (ORR) of VHL disease-associated clear cell renal cell carcinoma (ccRCC) tumors in untreated VHL patients who received PT2385. The study is being conducted in collaboration with the National Cancer Institute (NCI).

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"There is a significant need for new treatment options for VHL disease, a rare disease with serious and life-long consequences for patients, and for which there are no approved systemic therapies," said John A. Josey, Ph.D., Peloton’s Chief Executive Officer. "The current standard of care for patients with VHL disease-associated kidney cancer is surgery, which commonly does not result in a cure for these patients."
At the recent ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium, W. Marston Linehan, M.D., Chief of the Urologic Oncology Branch of the NCI, had noted "We are getting ready to start a trial of a drug targeting the HIF-2 pathway with the Peloton PT2385 drug, which we are very encouraged about."

PT2385 is a selective, orally active agent that blocks HIF-2α with potent anti-cancer activity in preclinical models of ccRCC. This open-label Phase 2 study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of PT2385 in patients with VHL disease who have at least one measurable VHL disease-associated ccRCC tumor (as defined by RECIST 1.1). PT2385 will be administered orally and treatment will be continuous unless there is disease progression. Changes in VHL disease-associated non-ccRCC lesions will also be evaluated.

"Patients with VHL disease-associated kidney cancer look forward to having the opportunity to participate in this first-ever study of a drug in patients with VHL disease that targets the immediate downstream effect of the VHL mutation," said Ilene Sussman of the VHL Alliance, a patient advocacy group for individuals with VHL disease. "If the drug is shown to be effective, it may reduce the number or frequency of surgeries needed. Overall, having an oral medication that could halt or reverse the progression of this disease would greatly benefit patients."
Further information on the clinical trial of PT2385 in VHL disease-associated kidney cancer can be found on clinicaltrials.gov (Study identifier: NCT03108066).

About VHL Disease
Von Hippel-Lindau disease is a hereditary cancer syndrome caused by a germline mutation in or deletion of the VHL gene, and patients are at risk for developing tumors and fluid-filled sacs (cysts) in a number of organs. Renal cell carcinoma occurs in about 70 percent of individuals with VHL disease and is the leading cause of mortality. Approximately 6,000 people have VHL disease in the U.S.

On May 11, 2017 TESARO Inc. (NASDAQ:TSRO) and Clinigen Group plc’s (AIM:CLIN) (‘Clinigen’) Idis Managed Access division reported that they have partnered to launch a Managed Access Program (also known as an Early Access Program) in Europe for the investigational PARP 1/2 inhibitor, niraparib, for patients with recurrent ovarian cancer (Press release, TESARO, MAY 11, 2017, View Source [SID1234519052]).

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Niraparib is currently an investigational agent in Europe and as such has not been granted approval by the European Commission. The niraparib marketing authorization application is under review by the European Medicines Agency.

Niraparib was recently approved by the United States (U.S.) Food and Drug Administration under the brand name ZEJULA for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.

Approximately 65,000 women are diagnosed with ovarian cancer in Europe every year. Ovarian cancer is the fifth-most frequent cause of cancer death among women. Despite high initial response rates to platinum-based chemotherapy, 85% of women with advanced ovarian cancer will see a recurrence of the disease after first line treatment. The efficacy of chemotherapy also diminishes over time.

Steve Glass, Chief Commercial Officer, North America and Europe for Clinigen said, "Following the successful delivery of the niraparib Managed Access Program in the US, we are pleased to be partnering with TESARO once again, providing eligible women in Europe the opportunity to gain access to this important investigational therapy."

Martin Huber, M.D., Senior Vice President, Chief Medical Officer for TESARO said, "We are proud to partner with Clinigen on this important Managed Access Program for women bravely facing ovarian cancer. The team at Clinigen has proven to be a partner of choice for TESARO as we look to address the needs of the ovarian cancer community."

About Niraparib
In Europe, niraparib is an investigational oral, once-daily poly (ADP-ribose) polymerase (PARP) 1/2 inhibitor for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. In preclinical studies, niraparib concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect.

On May 11, 2017 Atossa Genetics, Inc. (NASDAQ: ATOS) reported First Quarter ended March 31, 2017 financial results and provided an update on recent company developments (Press release, Atossa Genetics, MAY 11, 2017, View Source [SID1234519037]).

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Steve Quay, President and CEO, commented, "We are encouraged by our positive progress in advancing the endoxifen program with our ongoing Phase 1 endoxifen study and pleased that our fulvestrant microcatheter study is proceeding at Montefiore Medical Center. We look forward to completing our endoxifen Phase 1 study in the next quarter and commencing a Phase 2 study in the second half of 2017."

Recent Corporate Developments

Atossa’s important recent developments include the following:

May 2017 – Atossa received second positive safety decision in Phase 1 topical endoxifen study.
May 2017 – Institutional Review Board approved continuation of fulvestrant microcatheter Phase 2 study at Montefiore Medical Center.
April 2017 – Atossa received positive interim review from independent safety committee in Phase 1 topical endoxifen study.
April 2017 – Atossa enrolled first cohort of eight subjects in endoxifen study.
March 2017 – Atossa raised approximately $4.4 million in gross proceeds in a public offering.
March 2017 – Atossa opened its endoxifen Phase 1 clinical study.
Atossa plans to commence a Phase 2 clinical study of endoxifen in the second half of 2017.

Q1 2017 Financial Results

We are in the research and development phase and do not generate revenue.

Operating expenses: Total operating expenses were approximately $1.7 million for the three months ended March 31, 2017, consisting of general and administrative (G&A) expenses of approximately $1.1 million and R&D expenses of approximately $544,000. Operating expenses for the three months ended March 31, 2017 decreased approximately $641,000, or 27.5%, from approximately $2.3 million for the three months ended March 31, 2016, which consisted of G&A expenses of approximately $2.2 million, and R&D expenses of approximately $150,000.

The Company recorded a net loss of $1.7 million, for the three months ended March 31, 2017, as compared to a net loss of $2.3 million for the three months ended March 31, 2016.

On May 11, 2017 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended March 31, 2017 and reported on corporate developments. Unless specified otherwise, all amounts are in Canadian dollars (Press release, Aptose Biosciences, MAY 11, 2017, View Source [SID1234519036]).

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The net loss for the quarter ended March 31, 2017 was $4.4 million ($0.25 per share) compared with $5.1 million ($0.42 per share) in the quarter ended March 31, 2016. Total cash and cash equivalents and investments as of March 31, 2017 were $12.0 million (or $9.0 million US dollars) which, based on information currently available, provides the Company with sufficient resources to fund research and development and operations into Q2 2018.

"We, along with some of the nation’s leading hematology researchers, continue to generate compelling data on CG’806, an oral first-in-class pan-FLT3/BTK inhibitor that we plan to develop for patients with FLT3-driven acute myeloid leukemia and certain B-cell malignancies," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "Preclinical data presented at AACR (Free AACR Whitepaper) this past week demonstrated the ability of CG’806 to potently inhibit all mutant forms of FLT3 tested and to completely eradicate tumors in AML xenograft models in the absence of toxicities. Though early, we believe these data begin to position CG’806 as a best-in-class pan-FLT3 inhibitor for the treatment of AML. In addition, CG’806 is a potent non-covalent inhibitor of the wild type and C481S mutant forms of BTK, and we plan to develop CG806 in parallel for patients with B cell malignancies resistant and intolerant to covalent BTK inhibitors. We are working towards advancing this molecule into clinical trials within a year."

Corporate Highlights
In January 2017, Aptose announced the prioritization of its resources toward the development of CG’806, an oral preclinical compound being developed for patients with FLT3-driven acute myeloid leukemia (AML) and certain BTK-driven B-cell malignancies.

CG’806 was the subject of two poster presentations at the 2017 AACR (Free AACR Whitepaper) Hematologic Malignancies meeting held in Boston this past week (May 6-9).

° The first poster included data from studies conducted at The University of Texas MD Anderson Cancer Center, in which CG’806 demonstrated superior potency relative to competitive agents, against hematologic malignancy cell lines driven by various WT or mutant forms of FLT3. In addition, once daily oral dosing of CG’806 in a murine model achieved sustained micromolar plasma concentration over a 24 hour period, and was accompanied by complete elimination of AML FLT3-ITD tumors in the absence of toxicity.

° The second poster included highlighted studies conducted at Oregon Health & Science University (OHSU) and through a collaboration with the Beat AML Initiative, in which CG’806 demonstrated the ability to potently kill primary malignant cells in samples from patients with various hematologic malignancies including AML, CLL and others.

Separately, Aptose has begun formal studies on APTO-253, a phase 1 stage compound for AML, in an effort to define the root cause of recent manufacturing setbacks related to the intravenous formulation, and to restore the molecule to a state supporting clinical development and potential partnering. APTO-253, which effectively inhibits expression of the c-Myc oncogene, is a potential treatment for AML.
Financial Results
Our net loss for the three months ended March 31, 2017 was $4.4 million ($0.25 per share) compared with $5.1 million ($0.42 per share) during the three months ended March 31, 2016.
The decrease in the net loss during the three months ended March 31, 2017 compared with the three months ended March 31, 2016 is primarily related to savings from cancelling the LALS/Moffitt collaboration, lower stock-based compensation, and offset by development activities related to the CG’806 development program which started in the second half of 2016.
We utilized cash of $3.5 million in our operating activities in the three months ended March 31, 2017 compared with $4.5 million in the three months ended March 31, 2016. The decrease in cash used in operating activities in the current period is due mostly to increased accounts payable and accrual balances during the three months ended March 31, 2017.
Research and Development
Research and development expenses totaled $2.3 million in the three months ended March 31, 2017 compared with $2.3 million in the three months ended March 31, 2016. Research and development costs consist of the following:
Three months ended
(in thousands) March 31,
2017 March 31,
2016

Program costs – APTO-253 S 1,102 $ 1,040
Program costs – CG’806 540 -
Program costs – LALS/Moffitt - 485
Salaries 566 722
Stock-based compensation 68 56
Depreciation of equipment 19 12
$ 2,295 $ 2,315

Expenditures for the three months ended March 31, 2017 were comparable to the expenses incurred in the three months ended March 31, 2016. Higher program costs associated with the Company’s CG’806 program were offset by lower costs associated related to the cancellation of the LALS/Moffitt collaboration. Lower salaries expense was primarily related to severance payments made in the three months ended March 31, 2016 due to a reduction in Research & Development FTE.
General and Administrative
General and administrative expenses totaled $2.1 million in the three months ended March 31, 2017, compared to $2.6 million in the three months ended March 31, 2016. General and administrative costs consist of the following:
Three months ended
(in thousands) March 31,
2017 March 31,
2016

General and administrative excluding salaries $ 942 $ 1,133
Salaries 1,135 975
Stock-based compensation 13 479
Depreciation of equipment 11 21
$ 2,101 $ 2,608

General and administrative expenses excluding salaries, decreased in the three months ended March 31, 2017, compared with the three months ended March 31, 2016, mostly the result of lower travel, consulting and rent costs in the current year related to cost containment initiatives taken in the prior fiscal year. Salary charges in the three months ended March 31, 2017, increased slightly in comparison with the three months ended March 31, 2016, due to severance payments made in the current period that will result in savings in the following fiscal quarters.
Stock-based compensation decreased in the three months ended March 31, 2017, compared with the three months ended March 31, 2016, due to large forfeitures in the current period and also due to grants in prior periods having a greater fair value than the grants issued in the three months ended March 31, 2017, and therefore contributing to higher stock-based compensation in the prior year period.
Finance Expense
Three months ended
March 31,
2017 March 31,
2016
Foreign exchange loss - 196
$ - $ 196

Foreign exchange loss in the three months ended March 31, 2016, is the result of a decrease in the value of US dollar denominated cash and cash equivalents balances during the period due to an appreciation of the Canadian dollar compared to the US dollar. During this period the Company’s functional currency was the Canadian dollar.
Finance Income
Three months ended
March 31,
2017 March 31,
2016
Interest income $ 11 $ 47
Foreign exchange gain 30 –
$ 41 $ 47

Interest income represents interest earned on our cash and cash equivalent and investment balances. Foreign exchange gains in the three months ended March 31, 2017, are the result of an appreciation of the Canadian dollar compared to the US dollar. During this period the Company’s functional currency was the US dollar.
Effective January 1, 2017, the Company changed its functional currency to US dollars given the prevalence of US dollar denominated activities over time. The Company’s historic source of financing, with the exception of the recent at-the-market equity facility, has been in Canadian dollars and the Company still has a majority of its shareholders in Canada. For this reason the Company has chosen to keep the presentation currency as Canadian.

Aptose Biosciences Inc.
Condensed Consolidated Interim Statements of Loss and Comprehensive Loss
(unaudited)

Three Three
months ended months ended
(amounts in 000’s of Canadian Dollars except for per common share data) March 31, 2017 March 31, 2016
REVENUE $ - $ -

EXPENSES
Research and development 2,295 2,315
General and administrative 2,101 2,608
Operating expenses 4,396 4,923
Finance expense – 196
Finance income (41 ) (47 )
Net financing income (41 ) 149
Net loss for the period 4,355 5,072

Other comprehensive loss
Items that may subsequently be reclassified to earnings:
Foreign currency translation loss 123 -
Comprehensive loss for the period 4,478 5,072

Basic and diluted loss per common share $ 0.25 $ 0.42

The press release, the financial statements and the management’s discussion and analysis for the quarter ended March 31, 2017 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml

On May 11, 2017 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders, reported its financial results and business highlights for the first quarter ended March 31, 2017 (Press release, Cyclacel, MAY 11, 2017, View Source [SID1234519028]).

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The Company’s net loss applicable to common shareholders for the three months ended March 31, 2017 was $1.6 million, or $0.38 per share, compared to net loss applicable to common shareholders of $3.1 million, or $1.04 per share, for the first quarter of 2016. As of March 31, 2017, cash and cash equivalents totaled $12.7 million.

"Our development priorities going forward are our transcriptional regulation and DNA damage response clinical stage programs," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "In the Phase 1 study of our CYC065 CDK inhibitor in patients with solid tumors, we are close to establishing the recommended Phase 2 dose. Biomarker analysis shows evidence of target engagement and preliminary clinical activity. In parallel, we are reviewing with investigators study designs to test CYC065 in combination with approved agents in hematological indications. We are expanding our BRCA positive, sapacitabine and CDK inhibitor study to evaluate patients with ovarian and pancreatic cancers in addition to breast. We look forward to reporting our progress with these programs and the outcome of our final analysis of SEAMLESS data."

Quarter and Recent Highlights

Transcriptional regulation program: cyclin dependent kinase (CDK) inhibitor

· In the ongoing Phase 1, first-in-human trial of CYC065, a CDK2/9 inhibitor, in heavily pretreated patients with advanced solid tumors, we are expanding the sixth dose escalation level to a total of 9 patients with the objective of determining the recommended Phase 2 dose. Following analysis of biomarkers from patient specimens obtained at baseline and during CYC065 treatment, evidence of pharmacodynamic engagement has been observed, including a reduction in Mcl-1 expression. Preliminary evidence of clinical activity has been observed in two patients with MYC and CCNE (cyclin E) amplifications. These observations are consistent with the Company’s preclinical data and the drug’s mechanism of action.

· At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting, independent researchers from The University of Texas MD Anderson Cancer Center presented preclinical data demonstrating therapeutic potential of CYC065 as a targeted anti-cancer agent. The poster titled "The next generation CDK2/9 inhibitor CYC065 elicits marked antineoplastic effects in lung cancer by engaging anti-metastatic pathways" detailed data which show CYC065’s potential to cause anaphase catastrophe and inhibit migration and invasion of lung cancer cells including mutant KRAS, a key molecular features of such cancers.

þ 200 Connell Drive, Suite 1500, Berkeley Heights, NJ 07922, USA Tel +1 908 517 7330 Fax +1 866 271 3466
¨ 1 James Lindsay Place, Dundee, DD1 5JJ, UK Tel +44 1382 206 062 Fax +44 1382 206 067
www.cyclacel.com – [email protected]




· Preclinical data published in the Journal of National Cancer Institute demonstrated that CYC065 had prominent antitumor activity against lung cancer through anaphase catastrophe, a novel, mitosis-specific mechanism of action.

DNA damage response program

· The Phase 1 combination of sapacitabine and seliciclib CDK inhibitor is continuing enrollment in an extension study in an enriched population of approximately 20 patients with BRCA positive advanced breast cancer.

· A part 3 of this study has been designed with the goal of testing a revised dosing schedule in additional patients, including BRCA mutation positive, ovarian and pancreatic cancer patients.

SEAMLESS study in acute myeloid leukemia (AML)

· In February 2017, the Company reported that the study did not reach statistically significant superiority in overall survival (OS), although an improvement in complete remission rate was observed. In the stratified subgroup of patients with low baseline peripheral white blood cell count, comprising approximately two-thirds of the study’s population, an improvement in OS was observed for the experimental arm.

· The Company is currently analyzing stratified and exploratory subgroups to identify patients who are most likely to benefit from treatment with the experimental arm. Depending on this analysis, the Company may initiate discussions with European and U.S. regulators to determine a potential regulatory pathway.

PLK inhibitor CYC140

During the quarter, Cyclacel announced a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting. The poster, titled "The novel PLK1 inhibitor, CYC140: Identification of pharmacodynamic markers, sensitive target indications and potential combinations", detailed Cyclacel’s preclinical study to identify sensitive target indications including acute leukemia and esophageal cancer. The data demonstrated antitumor activity of CYC140 in preclinical xenograft models of acute leukemia and solid tumors, including esophageal cancer, with tumor growth delay, tumor regression and cures being observed. In addition, several pharmacodynamic markers were identified, and activity was demonstrated in a majority of malignant cell lines derived from AML, acute lymphoblastic leukemia (ALL) and esophageal cancer.

Financial highlights

As of March 31, 2017, cash and cash equivalents totaled $12.7 million, compared to $16.5 million as of December 31, 2016. The decrease of $3.8 million was due to net cash used in operating activities. Net proceeds of approximately $1.0 million were received in April 2017 from the sale of common stock through the Company’s at the market facility.

There were no revenues for the three months ended March 2017 compared to $0.1 million for the same period of the previous year. The revenue is related to previously awarded, UK government grants being recognized over the period to progress IND-directed preclinical development of CYC140, a novel, PLK-1 inhibitor, which was completed in November 2016.

Research and development expenses were $1.3 million compared to $2.5 million for the same period in 2016. The decrease was primarily due to reduced study and clinical supply costs associated with completion of the SEAMLESS study and 2016 expenditure related the development of CYC140.

General and administrative expenses were $1.4 million for each of the three months ended March 31, 2016 and 2017.

Other income, net for the three months ended March 31, 2017, was $0.8 million, compared to $0.2 million for the same period of the previous year. The increase is primarily related to income received under an Asset Purchase Agreement with Life Technologies Corporation, or LTC, (formerly Invitrogen Corporation), in respect of certain assets and intellectual property sold by the Company to LTC in December 2005.

The United Kingdom research & tax credit was $0.3 million for the three months ended March 31, 2017 compared to $0.5 million for the same period in 2016. The cash receipt for the 2016 tax credit of approximately $2.0 million is expected to be received in the second quarter of 2017.

After taking into account the expected $2.0 million cash receipt above and sales of common stock totaling $1.0 million from the at the market facility, pro forma cash at March 31, 2017 is approximately $15.7 million. The Company expects current pro forma cash to fund operations and ongoing programs to the end of 2018.

Net loss for the three months ended March 31, 2017 was $1.6 million compared to $3.0 million for the same period in 2016.