On November 6, 2015 Lixte Biotechnology Holdings, Inc. (OTCQB: LIXT) reported that in an ongoing Phase I trial its lead anti-cancer compound, LB-100, was associated with stabilization of a variety of advanced cancers that had been progressing despite extensive prior treatment (Filing, 8-K, Lixte Biotechnology, NOV 6, 2015, View Source [SID:1234508181]). The results were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference, Boston, on November 6. The authors were Vincent Chung, City of Hope, Duarte, CA; Donald Richards, Texas Oncology, Tyler, TX; Fadi Braiteh, Comprehensive Cancer Centers of Nevada, Las Vegas, NV; John S. Kovach, Lixte Biotechnology Holdings, Inc., East Setauket, NY; Aaron Scott Mansfield, Mayo Clinic, Rochester, MN.

A total of 21 patients received LB-100 for 3-consecutive days in 3-week cycles. Nine of these patients had stabilization of their disease without significant toxicity. One patient with pancreatic cancer received 14 cycles of LB-100 (42 weeks); one with thymoma, 9 cycles; one with testicular and one with carcinoid of the lung, 5 cycles; two with ovarian, 3 and 6 cycles; and one with NSCLC and one with duodenal cancer, 3 cycles. The average number of cycles received at the last four evaluable dose levels of LB-100 was 4.8 (14.4 weeks). In a study by Mansfield et al (2015 JCO 33[15] suppl:2567), the average number of cycles of a new drug in 51 NCI-sponsored Phase 1 trials conducted from 1994-2014 involving 1841 patients was 2.0 (6-8 weeks).

John S. Kovach M.D., founder and president of Lixte, said "Lixte interprets the results as showing that LB-100 has single agent activity in suppressing the growth of several types of cancer. What is highly encouraging is that stabilization of disease occurred in the absence of dose-limiting toxicity. In fact, most patients tolerated repeated doses without any ill-effects. As pre-clinical studies have shown that LB-100 potentiates the effectiveness of cytotoxic agents, we believe that LB-100 alone and in combination with standard anti-cancer drugs and/or radiation may offer new therapeutic options for a spectrum of neoplastic diseases."

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On November 6, 2015 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the presentation of three posters describing niraparib at the 2015 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), November 5-9, 2015, in Boston, Massachusetts (Press release, TESARO, NOV 6, 2015, View Source [SID:1234508096]). The posters describe the homologous recombination deficiency (HRD) analysis being performed on tumor samples from the ongoing Phase 3 NOVA trial of niraparib in patients with ovarian cancer, in addition to preclinical data demonstrating that niraparib crosses the blood brain barrier in animal models and highlighting potential genetic markers of resistance in animal models of small cell lung cancer.

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"We are executing a very comprehensive development program for niraparib, and believe that the emerging profile of this promising candidate warrants expansion to a variety of tumor types, as a monotherapy and in combination with immuno-oncology and other targeted agents," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "We are very optimistic about the potential of niraparib for patients with ovarian, breast and other cancers, and we look forward to initiating several new trials in the coming months."

Presentation Details:

Sunday, November 8, 2015, 12:30 PM to 3:30 PM
Homologous recombination deficiency of high grade serous ovarian tumors from the NOVA Phase III clinical study
Abstract: C53, Location: Session C, Hall C-D

Saturday, November 7, 2015, 12:30 PM to 3:30 PM
The PARP inhibitor, niraparib, crosses the blood brain barrier in rodents and is efficacious in a BRCA2-mutant intracranial tumor model
Abstract: B168, Location: Session B, Hall C-D

Saturday, November 7, 2015, 12:30 PM to 3:30 PM
A mouse avatar tumor maintenance study identified a subset of SCLC patient-derived tumor xenograft models sensitive to the PARP inhibitor niraparib
Abstract: B169, Location: Session B, Hall C-D

About Niraparib

Niraparib is an orally active and potent poly (ADP-ribose) polymerase, or PARP, inhibitor. A Phase 1/2 monotherapy study of niraparib has been completed in more than 100 patients with advanced solid tumors. Two Phase 3 trials are currently ongoing to evaluate a single oral dose of niraparib for patients with ovarian cancer and for patients with BRCA-positive breast cancer.

On November 6, 2015 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended September 30, 2015 (Press release, Stemline Therapeutics, NOV 6, 2015, View Source [SID:1234508095]).

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Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "During the third quarter, we made significant progress on many fronts as we continue to advance our clinical programs forward. We are actively enrolling patients, and opening additional sites, in the expansion stage of our ongoing SL-401 pivotal trial in blastic plasmacytoid dendritic cell neoplasm, BPDCN. We look forward to sharing clinical updates from both the lead-in and initial expansion stages of this trial at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) meeting this December, and on into next year."

Dr. Bergstein continued, "We remain focused on the BPDCN indication while continuing to pursue expansion opportunities for SL-401 in additional malignancies. We have single agent trials currently open in several indications and we anticipate combination studies coming on-line as well. We are also continuing to develop our other pipeline candidates, SL-701 and SL-801, and look forward to treating our first patient with SL-801, our novel XPO1 inhibitor, early next year. With a strong cash position and multiple ongoing programs advancing across a range of indications of unmet medical need, we remain focused on achieving our objective of building a leading commercial stage biopharmaceutical company."

Third Quarter 2015 Financial Results Review

Stemline ended the third quarter of 2015 with $104.0 million in cash, cash equivalents and investments, as compared to $58.6 million as of December 31, 2014. In the first quarter of 2015, the Company completed an equity offering raising $68.6 million in gross cash proceeds on the sale of 4.4 million common shares.

For the third quarter of 2015, Stemline had a net loss of $9.2 million, or $0.53 per share, compared with a net loss of $6.9 million, or $0.53 per share, for the same period in 2014.

Research and development expenses were $7.3 million for the third quarter of 2015, which reflects an increase of $2.3 million compared with $5.0 million for the third quarter of 2014. The higher expenses during the third quarter were primarily attributable to the ramp up of SL-401 and SL-701 clinical activities. Also, we incurred costs relating to SL-801 IND-enabling studies.

General and administrative expenses were $2.2 million for the third quarter of 2015, which reflects an increase of $0.2 million compared with $2.0 million for the third quarter of 2014. The higher costs were primarily attributable to an increase in non-cash stock based compensation expense relating to employees.

On November 6, 2015 PharmaMar (MSE:PHM), a world-leading biopharmaceutical company in the discovery and development of innovative marine-derived anticancer drugs, reported that its novel Antibody-Drug Conjugate (ADC) demonstrates strong anticancer activity in vitro and in vivo against tumors expressing HER2 derived from breast, gastric and ovarian cancers (Press release, PharmaMar, NOV 6, 2015, View Source [SID:1234508082]).
The novel ADC MI130004 consists of a new marine-derived tubulin inhibitor (PM050489) that is covalently bound to the HER2 antibody trastuzumab via a nonhydrolysable linker. The results have been presented today in a poster presentation during the Therapeutic Agents – Biological session at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference "Molecular Targets and Cancer Therapeutics" taking place in Boston, November 5-9.

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When tested in a panel of tumor cells expressing the receptor HER2 in their surface (HCC-194, SK-BR3 and BT-747) or tumor cells that do not express it (MCF-7 and MDA-MB-231), MI13004 demonstrated strong potency in those cells expressing the receptor. The authors found that the ADC MI130004 impairs tubulin polymerization, causing disorganization of the microtubule network leading to mitotic failures and halting cell division in cells expressing HER2, similar to the effect of the payload PM50489.

In the study, HER2-expresing tumor cells from breast, gastric and ovarian cancers were subcutaneously implanted into immunosuppressed mice to develop tumors up to certain volume (about 114 mm3). Upon treatment with MI130004 at different
doses once a week over a course of 5 weeks, mice treated with the highest dose showed substantial tumor shrinkage with complete tumor remissions in all the mice implanted with BT747 breast tumor cells that lasted up to 120 days. MI130004 was
shown to induce complete remissions in some of, but not all, the mice implanted with the breast tumor cells JIMT-1, the two gastric cancer cells Gastric-008 and N87 and the two ovarian cancer cells SK-OV-3 and A2780cis. Complete tumor remissions lasted even 385, 354 and 341 days in mice implanted with SK-OV-3, Gastric-008 and A2780cis, respectively.

The effect of MI130004 in all HER2-expressing tumors was confirmed 24h after treatment by showing mitotic aberrations in tumor cells indicating an anticancer activity mediated by microtubule inhibition and blockade of cell division.

Title: MI130004, a new ADC with a payload of marine origin shows outstanding activity against HER2-expressing tumors (Abstract A147). Session Start/End Time and location: Friday, Nov 06, 12:15 PM – 3:15 PM, session A, Hall C-D.

On November 6, 2015Mirati Therapeutics, Inc. ("Mirati") (NASDAQ: MRTX) reported financial results for the third quarter ended September 30, 2015 and provided an update on its drug development programs (Filing, 8-K, Mirati, NOV 6, 2015, View Source [SID:1234508079]).

"Over the last quarter we made substantial progress against our goals, including strengthening our balance sheet," said Charles M. Baum, M.D., Ph.D., president and CEO, Mirati. "In September, we completed a successful financing that enables us to advance all of our clinical development programs to the next stage."

Recent and Upcoming Pipeline Highlights

Operations:

· Completed a public offering of 2.25 million shares of common stock at $45.00 per share in September 2015, generating net proceeds of $94.9 million

MGCD265: Molecularly targeted kinase inhibitor

· World Conference on Lung Cancer 2015: Presented data demonstrating the favorable tolerability and clinical efficacy of MGCD265, with confirmed responses in non-small cell lung cancer (NSCLC) patients with MET and Axl gene amplification
· The poster can be found on the Company’s website at www.mirati.com
· Additional information about this clinical trial of MGCD265 is available at www.clinicaltrials.gov using identifier: NCT00697632

· The Company expects to initiate a single arm, open-label Phase 2 study in NSCLC patients, with driver alterations in MET, by the end of the year. MET alterations occur in up to 7% of NSCLC patients

MGCD516: Molecularly targeted kinase inhibitor

· European Cancer Congress (ECC) 2015: Presented interim clinical data from the ongoing Phase 1 dose escalation study of MGCD516 in patients with advanced solid tumors.
· Demonstrated that MGCD516 is well tolerated with a favorable pharmacokinetic profile
· The poster can be found on the Company’s website at www.mirati.com
· Additional information about this clinical trial of MGCD516 is available at www.clinicaltrials.gov using identifier: NCT02219711

· The Company has initiated the expansion cohorts in selected patients and expects preliminary data in 2016. The initial focus will be on NSCLC in patients with genetic driver mutations, including RET, Trk and DDR

Mocetinostat: Class I & IV HDAC inhibitor

· Announced an immuno-oncology clinical trial collaboration with MedImmune, the global biologic research and development arm of AstraZeneca, to evaluate the safety and efficacy of mocetinostat in combination with durvalumab, an investigational anti-PD-L1 immune checkpoint inhibitor. The initial Phase 2 study will be conducted in patients with NSCLC and is expected to begin in the first half of 2016

Third Quarter 2015 Financial Results

Cash, cash equivalents, and short-term investments were $138.0 million at September 30, 2015, compared to $29.3 million at December 31, 2014. In September 2015, the Company completed a public offering of 2.25 million shares of its common stock, generating net proceeds of $94.9 million. In February 2015, the Company completed a public offering of 2.6 million shares of its common stock, generating net proceeds of $48.4 million.

Research and development expenses for the third quarter of 2015 were $14.6 million, compared to $6.9 million for the same period in 2014. Research and development expenses for the nine months ended September 30, 2015 were $34.0 million, compared to $19.0 million for the same period in 2014. The increases in research and development expenses primarily reflect costs to advance the clinical development of its three oncology development programs, MGCD265, MGCD516 and mocetinostat. General and administrative expenses for the third quarter of 2015 were $4.2 million, compared to $3.7 million for the same period in 2014. General and administrative expenses for the nine months ended September 30, 2015 were $12.2 million, compared to $9.3 million for the same period in 2014. The increases in general and administrative expenses primarily reflect higher compensation costs including non-cash stock-based compensation expense.

Other income and expense, net, for the third quarter of 2015 was expense of less than $0.1 million compared to income of $1.9 million for the same period in 2014. Other income and expense, net, for the nine months ended September 30, 2015 was income of $0.1 million compared to expense of $4.6 million for the same period in 2014. Other income and expense, net, for the third quarter and nine months ended September 30, 2014 primarily reflects losses arising from the change in fair value of our warrant liability. During 2014, we amended the warrant agreements to allow for the warrants to be denominated in U.S. dollars. The amended warrants qualified for equity classification and were reclassified into stockholders’ equity.

Net loss for the third quarter of 2015 was $18.7 million, or $1.11 per share basic and diluted, compared to net loss of $8.6 million, or $0.64 per share basic and $0.72 per share diluted for the same period in 2014. Net loss for the nine months ended September 30, 2015 was $46.1 million, or, $2.86 per share basic and diluted, compared to net loss of $33.3 million, or $2.47 per share basic and diluted for the same period in 2014.

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