On November 9, 2015 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, reported financial results for the three and nine months ended September 30, 2015 and provided a business update (Filing, 8-K, Galectin Therapeutics, NOV 9, 2015, View Source [SID:1234508135]). These results are included in the Company’s Quarterly Report on Form 10-Q, which has been filed with the U.S. Securities and Exchange Commission.

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Management commentary

"Galectin Therapeutics has never been in a better position advancing the development of our galectin-3 inhibitor in non-alcoholic steatohepatitis (NASH) and other therapeutic indications," said Jim C. Czirr, executive chairman of the company. "Our pipeline has progressed considerably during the third quarter and recent weeks, and we now have five clinical studies underway with our proprietary compound, GR-MD-02, in four different indications.

"We began a second Phase 2 study in NASH with advanced fibrosis, the NASH-FX study, during the quarter, and we continued to enroll patients in our Phase 2 study in NASH with cirrhosis, the NASH-CX study. We also began a pilot study to evaluate the potential efficacy of GR-MD-02 in patients with moderate-to-severe plaque psoriasis. More recently, after the close of the quarter we announced that the Providence Portland Medical Center submitted an Investigational New Drug (IND) application to study GR-MD-02 in combination with Keytruda in metastatic melanoma. This is in addition to its ongoing study of GR-MD-02 in combination with Yervoy in the same indication. To support this increased activity at Galectin, during the quarter we hired an industry veteran, Adam E. Allgood, Pharm.D., R.Ph. as executive director of clinical development," Mr. Czirr added.

Peter G. Traber, M.D., president, chief executive officer and chief medical officer, said, "NASH has been a topic of keen interest within the medical community as this disease appears to be overtaking hepatitis C as the number one cause of liver transplants, affecting up to 6 million people in the U.S. We are very excited about the potential of GR-MD-02 to treat the fibrosis and cirrhosis that can accompany NASH, and we are working hard to make efficacious treatment a reality.

"We are very pleased with the pace of our clinical studies and remain on track with our stated timelines," added Dr. Traber. "The NASH-CX trial, which if successful has potential to be a component of a pivotal study, began enrolling patients with NASH cirrhosis, with 156 patients in total to be enrolled. Patients are being evaluated with either 8 mg/kg of GR-MD-02, 2 mg/kg of GR-MD-02 or placebo, and are being randomized 1:1:1. The primary endpoint for this trial is change in hepatic venous pressure gradient (HVPG) compared with placebo, with secondary endpoints of fibrosis stage on biopsy as well as the percent of collagen on biopsy at one year of treatment. Additionally, the HVPG and liver biopsy measurements will be correlated with non-invasive measurements of liver fibrosis and function using FibroScan and 13C-methacetin breath test, respectively. We expect top-line data readout by the end of 2017.

Dr. Traber continued, "The NASH-FX trial in 30 patients with NASH with advanced fibrosis, but not cirrhosis, is proceeding as planned. This four-month treatment trial will evaluate the efficacy of 8 mg/kg of GR-MD-02 versus placebo, using non-invasive endpoints. LiverMultiScan, a multi-parametric nuclear magnetic resonance imaging method developed by Perspectum Diagnostics, will be used to measure the change in inflammation and fibrosis as a primary endpoint. The secondary goal is to compare the primary endpoint of LiverMultiScan with two secondary endpoints that are non-invasive measures of liver stiffness that correlate with fibrosis, magnetic resonance elastography and FibroScan. We expect data readout from the NASH-FX trial in the third quarter of 2016.

"We have also begun screening patients with moderate-to-severe plaque psoriasis in a proof-of-concept, open-label Phase 2a trial. In this trial 10 patients will be treated with 8mg/kg of GR-MD-02 every 2 weeks over 90 days. Research has shown that galectin-3 is increased in the skin of psoriasis patients, thus we have good support for the mechanism of action of our compound. In addition, as previously disclosed, one subject in our Phase 1 NASH study had an apparent remission of psoriasis. As such, we believe it is worthwhile to explore this indication further in this small study.

"We expect that the Portland Cancer Center will begin enrolling patients in its Phase 1b study of GR-MD-02 in combination with Keytruda within the coming weeks, following the filing of its IND in October. Keytruda is an immune checkpoint inhibitor indicated for patients whose melanoma had progressed after treatment with Yervoy or targeted therapy in melanomas that have a BRAF mutation. Preclinical data show that GR-MD-02 holds potential to increase the effectiveness of other therapies and represents a promising approach to enhance cancer immunotherapy. GR-MD-02 is also the subject of an ongoing study in combination with Yervoy being conducted by same investigators at the Portland Cancer Center. We are very pleased to be supplying our compound to the investigators," Dr. Traber concluded.

Additional information about the Company’s clinical development program and clinical trials is available on the CEO Perspectives website at View Source

Financial Results
For the three months ended September 30, 2015, the Company reported a net loss applicable to common stockholders of $6.2 million, or ($0.26) per share, compared with a net loss applicable to common stockholders of $3.9 million, or ($0.17) per share, for three months ended September 30, 2014. The increase in net loss applicable to common stockholders is largely due to higher research and development expenses related to the Company’s Phase 2 clinical program.
Research and development expense for the three months ended September 30, 2015 was $4.5 million, compared with $2.0 million for three months ended September 30, 2014. The increase is primarily due to increased costs related to the Company’s Phase 2 clinical program.

General and administrative expense for the three months ended September 30, 2015 was $1.4 million, compared with $1.5 million for the three months ended September 30, 2014. The primary reason for the decrease was related to a decrease in stock-based compensation expense in the three months ended September 30, 2015, compared with 2014.

For the nine months ended September 30, 2015, the Company reported a net loss applicable to common stockholders of $16.2 million, or ($0.69) per share, compared with a net loss applicable to common stockholders of $13.0 million, or ($0.60) per share, for the nine months ended September 30, 2014. The increase in net loss applicable to common stockholders is largely due to higher research and development expenses related to the Company’s Phase 2 clinical program.

Research and development expense for the nine months ended September 30, 2015 was $10.2 million, compared with $6.3 million in the prior year’s period. The higher research and development expense is related to increased expenses associated with the Company’s Phase 2 clinical studies.

General and administrative expense for the nine months ended September 30, 2015 was $5.2 million, compared with $5.4 million for the nine months ended September 30, 2014. The decline was due to lower stock-based compensation expense.
As of September 30, 2015, the Company had $21.3 million of non-restricted cash and cash equivalents available to fund future operations. The Company believes that its cash on hand as of September 30, 2015 is sufficient to fund currently planned operations and research and development activities through September 30, 2016.

On November 9, 2015 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), a biopharmaceutical company that applies systems biology to develop cancer treatments and diagnostics, reported its third quarter 2015 financial results (Press release, Merrimack, NOV 9, 2015, View Source [SID:1234508166]). Merrimack will host a live conference call and webcast today, Monday, November 9 at 4:30 p.m., Eastern time, to provide an update on Merrimack’s progress as well as a summary of these results.

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Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 69525411. A listen-only webcast of the call can be accessed in the Investors section of Merrimack’s website, investors.merrimack.com, and a replay of the call will be archived there for six weeks following the call.

Key Recent Events

Approval of ONIVYDE (irinotecan liposome injection) by the U.S. Food and Drug Administration on October 22, 2015;
Commercial launch of ONIVYDE in the United States on October 26, 2015;

Enrollment of first patient in a Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer; and

Achievement of $66.5 million in net milestone payments from collaborations in 2015, consistent with Merrimack’s previous financial guidance.

Upcoming Milestones

Merrimack anticipates the following clinical milestones:

Initiation of a Phase 3 clinical study of ONIVYDE in front-line HER2-negative gastric cancer in 2016;

Results from the Phase 2 clinical study of MM-121 in patients with heregulin-positive, locally advanced or metastatic non-small cell lung cancer in the second half of 2016;

Results from the Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer in the first half of 2017;

Results from HERMIONE, the Phase 2 clinical study of MM-302 in patients with HER2-positive metastatic breast cancer designed to support a potential Accelerated Approval application to the FDA, in 2017; and

Results from the Phase 2 clinical study of MM-141 in patients with front-line metastatic pancreatic cancer who have high serum levels of free IGF-1 in 2017.

Third Quarter 2015 Financial Results

Revenue for the third quarter of 2015 was $16.4 million compared with revenue of $28.0 million for the third quarter of 2014, a decrease of $11.6 million or 41%. This decrease was primarily due to the termination of Merrimack’s collaboration with Sanofi effective December 17, 2014 and was offset by increased revenue recognized related to Merrimack’s collaboration with Baxalta during the third quarter of 2015.

Research and development expenses for the third quarter of 2015 decreased $5.9 million over the corresponding quarter of the preceding year. This decrease was primarily due to $12.0 million of non-recurring ONIVYDE collaboration milestones that were expensed in the third quarter of 2014. This decrease was offset by increased spending on Merrimack’s remaining pipeline.

General and administrative expenses for the third quarter of 2015 increased $8.9 million over the corresponding quarter of the preceding year. This increase was primarily attributable to increased infrastructure and personnel expenses as Merrimack prepared for the commercialization of ONIVYDE.

Merrimack’s net loss for the third quarter of 2015 was $42.4 million, or basic and diluted net loss per share available to common stockholders of $0.38, as compared to a net loss for the third quarter of 2014 of $28.0 million, or basic and diluted net loss per share available to common stockholders of $0.27.

Financial Outlook

Merrimack expects to be able to fund operations into the second quarter of 2016 based on the following:

– unrestricted cash and cash equivalents and available-for-sale securities of $62.4 million as of September 30, 2015;
– $47.5 million of milestones from Baxalta anticipated in the fourth quarter of 2015 related to the first patient dosed in the Phase 2 clinical study of ONIVYDE in previously untreated front-line metastatic pancreatic cancer; and
– anticipated cost sharing reimbursements from Baxalta.

Additional funding for operations would also come from:

– anticipated receipt of $46.5 million of net milestones related to ONIVYDE in 2016 from Baxalta, after offsetting payments to PharmaEngine;

– sales of ONIVYDE; and

– up to $15.0 million in additional debt that is currently available at Merrimack’s option under its loan agreement with Hercules Technology Growth Capital.

Upcoming Investor Conferences

Merrimack will attend the following investor conferences this fall:

Credit Suisse 24th Annual Healthcare Conference on November 11 in Scottsdale, AZ;
Jefferies Autumn 2015 Global Healthcare Conference on November 18 in London;
Oppenheimer 26th Annual Healthcare Conference on December 8 in New York; and
Guggenheim 3rd Annual Boston Healthcare Conference on December 15 in Boston.
Live webcasts of the presentations at the Credit Suisse 24th Annual Healthcare Conference, Jefferies Autumn 2015 Global Healthcare Conference and Oppenheimer 26th Annual Healthcare Conference can be accessed by visiting the Investors section of Merrimack’s website at investors.merrimack.com. A replay of the webcasts will be archived there for two weeks following each presentation.

(Filing, 10-Q, Karyopharm, NOV 9, 2015, View Source [SID:1234508116])

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(Filing, 10-Q, Isis Pharmaceuticals, NOV 9, 2015, View Source [SID:1234508162])

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On November 9, 2015 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported company highlights and financial results for the third quarter ended September 30, 2015 (Press release, Ignyta, NOV 9, 2015, View Source [SID:1234508136]).

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"Since the beginning of the third quarter of 2015, we have continued to make significant progress toward our objective of becoming a leading precision oncology biotechnology company that can provide patients with a variety of compelling cancer treatment options," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "We continued to make strategic additions to our clinical pipeline that can help us eradicate residual disease in precisely defined patient populations through our acquisition from Lilly of exclusive worldwide rights to taladegib, a potent, orally bioavailable small molecule hedgehog/smoothened antagonist that has achieved compelling clinical proof of concept and a recommended Phase 2 dose in a Phase 1 dose escalation clinical trial. We also are grateful to Lilly for concurrently investing $30 million by acquiring 1.5 million shares of our common stock at $20 per share."

"In addition, we made strong progress executing development of our existing clinical-stage product candidates, including presenting promising Phase 1 data at leading conferences for entrectinib and RXDX-105, initiating our potentially registration-enabling STARTRK-2 Phase 2 clinical trial of entrectinib, and initiating our Phase 1/1b clinical trial of RXDX-107," continued Dr. Lim. "We expanded our team with incredibly talented people who, along with our existing team members, will help advance our multiple, complementary development programs for the benefit of cancer patients."

Company Highlights

Taladegib Exclusive License and Concurrent Stock Purchase by Lilly

In November 2015, Ignyta announced it had exclusively licensed from Eli Lilly and Company worldwide rights relating to Lilly’s taladegib oncology development program in exchange for an upfront payment of $2.0 million in cash and the issuance to Lilly of approximately 1.2 million shares of Ignyta’s common stock. Taladegib is a potent, orally bioavailable small molecule hedgehog/smoothened antagonist that has achieved compelling clinical proof of concept and a recommended Phase 2 dose in a Phase 1 dose escalation clinical trial. Ignyta also licensed exclusive worldwide rights to the topical formulation of taladegib, which is a late preclinical stage program being developed for the potential treatment of patients with superficial and nodular basal cell carcinoma.

Concurrently with the license, Ignyta entered into a stock purchase agreement with Lilly under which Lilly purchased a further 1.5 million shares of Ignyta common stock at a price of $20 per share in a private placement.

Presentation of RXDX-105 Clinical Data at ENA Conference

In November 2015, Ignyta announced interim results from the company’s ongoing Phase 1 clinical trial of RXDX-105, the company’s orally-available, small molecule multikinase inhibitor with potent activity against such key targets as RET and BRAF, which were presented at the 27th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics in Boston, Massachusetts.

The dose escalation clinical trial was designed to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D), as well as preliminary anti-cancer activity, of single agent RXDX-105 in patients with advanced or metastatic solid tumors that were not selected based on any molecular alteration.

As of the October 26, 2015, data cut-off for the presentation, the findings showed:

A total of 41 patients with a range of solid tumors were dosed in the clinical trial;
RXDX-105 was well tolerated to date:

The most frequent treatment-emergent adverse events were fatigue, vomiting, nausea, decreased appetite, constipation, diarrhea, hypertension and muscle spasms;

Three Grade 3 dose-limiting toxicities were observed: maculopapular rash, fatigue and diarrhea, each of which resolved upon study drug interruption;

There were no treatment-related serious adverse events. Two Grade 4 adverse events had occurred, consisting of intestinal obstruction and anemia, neither of which was considered to be treatment-related. No Grade 5 treatment-related adverse events or cumulative adverse events were observed;

The MTD and RP2D had not yet been determined;

Pharmacokinetic measurements showed increased exposure with increasing dose, with a half-life compatible with once-daily dosing. Dosing in the fed state appears to further increase exposure;

Exposure was reaching levels expected to be efficacious based on tumor growth inhibition in animal models of RET- and BRAF-driven tumors; and

Tumor regression was observed in six patients treated with 275 mg, including one confirmed partial response (40% reduction) in a patient with non-small cell lung cancer (NSCLC) with a KRAS G12C mutation. Two additional patients with thyroid cancer and squamous cell lung cancer exhibited reductions of 20% and 27%, respectively. In patients with tumor regression, there appears to be an exposure/response correlation.

Initiation of STARTRK-2 Phase 2 Clinical Trial of Entrectinib

In September 2015, Ignyta announced the initiation of its Phase 2 clinical trial of entrectinib, the company’s proprietary oral tyrosine kinase inhibitor targeting solid tumors that harbor activating alterations to NTRK1, NTRK2, NTRK3, ROS1 or ALK. This clinical trial is called STARTRK-2, the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases." The trial is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for gene rearrangements of the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types that harbor a rearrangement of one of the genes encoding any one of entrectinib’s five protein targets.

Presentation of Updated Interim Entrectinib Clinical Trial Results at European Cancer Conference

In September 2015, the company announced updated interim results of its Phase 1 clinical trials of entrectinib, which were presented in an oral presentation session at the 2015 European Cancer Congress (ECC 2015) in Vienna, Austria.

The clinical trials included the ALKA-372-001 study and the STARTRK-1 study. Both trials were designed to determine the MTD and/or RP2D, as well as preliminary anti-cancer activity, of single agent entrectinib in patients with solid tumors with the relevant molecular alterations: NTRK1 (encoding TrkA), ROS1 or ALK for ALKA-372-001 and NTRK1/2/3 (encoding TrkA/B/C), ROS1 or ALK for STARTRK-1.

As of the August 15, 2015, data cut-off for the presentation, the findings showed:

A total of 92 patients with a range of solid tumors were dosed across both clinical trials, with nine patients treated at or above the RP2D beyond six months and one patient beyond one year.
Entrectinib was well tolerated:

Across both studies, the most frequent (>10% incidence) treatment-related adverse events were fatigue, dysgeusia, paresthesia, nausea, and myalgia. Seven of these were Grade 3 in severity, consisting of fatigue (4 patients), cognitive impairment (2 patients), and diarrhea (1 patient). No Grade 4 treatment-related adverse events were observed;

Across both studies, there were only three treatment-related serious adverse events: Grade 3 cognitive impairment and Grade 3 myocarditis, both of which occurred above the RP2D, and Grade 2 fatigue. All events were reversible and resolved upon dose modification;

The fixed daily dose RP2D was determined to be 600 mg, taken orally once per day (QD);

18 patients across both clinical trials met the company’s expected Phase 2 eligibility criteria, which include:
Presence of an NTRK1/2/3, ROS1 or ALK gene rearrangement, as opposed to other types of molecular alterations (e.g., SNPs, amplifications, deletions);

ALK-inhibitor and/or ROS1-inhibitor naïve; and

Treatment at or above the RP2D;

The response rate in the 18 patients who met these criteria across both studies was 72% (13 responses out of 18 treated patients, as assessed by the clinical sites). Nine of these responders remained on study treatment with durable responses of up to 21 treatment cycles. An additional 3 patients remained on study with stable disease. The responses included:

3 responses out of 4 patients with an NTRK1, NTRK2 or NTRK3 gene rearrangement, including patients with NSCLC, colorectal cancer and salivary gland cancer, with one of the responding patients remaining on treatment at 6 months; a fourth patient with an astrocytoma remained on treatment after two months with stable disease;

6 responses, including one complete response, out of 8 patients with a ROS1 gene rearrangement, all of which were in NSCLC. All of the patients who responded remained on treatment, the longest at 21 months; and

4 responses out of 6 patients with an ALK gene rearrangement, including two NSCLC patients and two patients with other solid tumors; two of the 4 responders had subsequently progressed.

Entrectinib had demonstrated objective tumor response in the central nervous system (CNS), a frequent site of metastases and progression of advanced solid tumors.

Issuance of Patent Covering Composition of Matter of RXDX-107

In October 2015, Ignyta announced that the U.S. Patent and Trademark Office issued U.S. Patent No. 9,150,517, entitled "Bendamustine Derivatives and Methods of Using Same." This patent contains claims that cover the composition of matter of Ignyta’s product candidate RXDX-107, and pharmaceutical compositions comprising RXDX-107. RXDX-107 is the company’s new chemical entity, next generation chemotherapeutic comprising an alkyl ester of bendamustine encapsulated in human serum albumin (HSA) to form nanoparticles. The patent has an expiration date of 2033, which does not include any potential patent term extension.

Initiation of Phase 1/1b Clinical Trial of RXDX-107

In September 2015, the company announced the initiation of its Phase 1/1b clinical trial of RXDX-107. This multicenter, open-label, dose-escalation clinical trial is designed to determine the MTD, RP2D, tolerability, pharmacokinetics and preliminary clinical activity of RXDX-107 in adult patients with locally advanced or metastatic solid tumors.

Enhancement of Leadership Capacity

In September 2015, Ignyta announced that Igor Bilinsky, Ph.D., was appointed to the newly-created role of General Manager, Immuno-Oncology and Senior Vice President, Special Operations, and that Valerie Harding, Ph.D., was appointed to the newly-created role of Senior Vice President, Chemistry, Manufacturing, and Controls.

Third Quarter 2015 Financial Results

For the third quarter of 2015, net loss was $14.6 million, or $0.49 per share, compared with $10.7 million, or $0.55 per share, for the third quarter of 2014.

Ignyta did not record any revenue for the three months ended September 30, 2015 or September 30, 2014.

Research and development expenses for the third quarter of 2015 were $10.4 million, compared with $8.6 million for the third quarter of 2014. The increase was primarily due to an increase in activities relating to development of entrectinib and the company’s other product candidates, including the assets acquired from Teva Pharmaceutical Industries Ltd. in March 2015. The increase between periods was also due to personnel expenses related to hiring and engaging additional employees and consultants to help advance the company’s product candidates and facilities-related expenses as a result of the expansion of the company’s leased facilities space.

General and administrative expenses were $3.9 million for the third quarter of 2015, compared with $2.2 million for third quarter of 2014. The increase was primarily caused by increases in personnel costs and investor relations, audit, legal and intellectual property costs.

At September 30, 2015, the company had cash, cash equivalents and available-for-sale securities totaling $163.1 million and current and long-term debt of approximately $31.0 million. At December 31, 2014, the company had cash, cash equivalents and available-for-sale securities totaling $76.6 million and current and long-term debt of approximately $21.0 million.