Month: May 2017

ImmunoGen Announces Results from Mirvetuximab Soravtansine Phase 1 First-in-Human Dose-Escalation Trial Published in Cancer

On May 1, 2017 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the results from a Phase 1 dose-escalation study evaluating mirvetuximab soravtansine (IMGN853) in patients with folate receptor alpha (FRα)-positive solid tumors were published in the journal Cancer (Press release, ImmunoGen, MAY 1, 2017, View Source [SID1234518773]). The previously disclosed data demonstrated encouraging clinical activity and a manageable safety profile for mirvetuximab soravtansine (IMGN853), and informed the dose that was used in Phase 1 expansion cohorts and the ongoing Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer.

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"These Phase 1 results have played an important role in determining the appropriate dose for mirvetuximab soravtansine in the recently initiated Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer," said Kathleen Moore, M.D., Associate Professor, Department of Obstetrics and Gynecology at the Stephenson Cancer Center at the University of Oklahoma. "The combination of these data and the recent data published in the Journal of Clinical Oncology further support the dose that has been chosen and patients who have been selected for FORWARD I."

The open-label, Phase 1 dose-escalation study treated a total of 44 patients with recurrent ovarian (52%) or endometrial cancer (25%), along with renal cell carcinoma and non-small cell lung cancer (11% and 9%, respectively). Patients received mirvetuximab soravtansine on day 1 of a 21-day cycle (Q3W dosing) with cycles repeated until dose-limited toxicity or progression, concluding the recommended dose for future trials is 6.0 mg/kg of mirvetuximab (based on adjusted ideal body weight) dosed once every three weeks. On the basis of the study findings, and additional data that demonstrated the importance of FRα expression levels for optimal mirvetuximab sorvatansine activity, the Company designed the Phase 3 FORWARD I trial utilizing this dose in patients with platinum-resistant ovarian cancer, along with a Phase 1b trial evaluating mirvetuximab in combination with standard-of-care chemotherapy and targeted agents.1

Mirvetuximab soravtansine exhibited a manageable safety profile and encouraging preliminary clinical activity. Adverse events (AEs) were generally mild with the majority being grade 1 or grade 2 (least severe grades). The most commonly observed AEs were fatigue, blurred vision and diarrhea.1

The publication, "Phase I dose-escalation study of mirvetuximab soravtansine (IMGN853), a folate receptor alpha-targeting antibody-drug conjugate, in patients with solid tumors," is available on the Cancer website.

About Mirvetuximab Soravtansine

Mirvetuximab soravtansine (IMGN853) is the first FRα-targeting ADC. It uses a FRα-binding antibody to target the ADC specifically to FRα-expressing cancer cells and a potent anti-tumor agent, DM4, to kill the targeted cancer cells.

Mirvetuximab soravtansine is ImmunoGen’s lead program and is in Phase 3 testing as a single agent for the treatment of platinum-resistant ovarian cancer. The candidate is also being assessed in combination regimens for both platinum-resistant and platinum-sensitive disease in Phase 1b/2 FORWARD II trial.

About Ovarian Cancer and FRα

In 2016, approximately 22,300 new cases of ovarian cancer will be diagnosed in the U.S. and more than 14,200 women will die from the disease.2 ImmunoGen estimates that 60% of ovarian cancer cases have medium or high FRα expression.

Standard first-line therapy for ovarian cancer is a platinum-based regimen. Once the cancer becomes platinum-resistant, treatment options include single-agent cytotoxic therapies such as pegylated liposomal doxorubicin, paclitaxel, or topotecan.

Trieza Therapeutics

Trieza Therapeutics is a new company developing immunomodulatory oncolytic viruses for the treatment of cancer (Company Web Page, MPM Capital, MAY 1, 2017, View Source [SID1234518761]). Founded as a spin-out from Potenza Therapeutics, Trieza has a portfolio containing multiple pre-clinical candidates.

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SignalRx Discloses its Novel Immuno-Oncology Program Approach at the 12th Annual Drug Discovery Chemistry 2017 Meeting

On May 1, 2017 SignalRx Pharmaceuticals Inc., a clinical-stage company developing novel small-molecules therapeutics to inhibit key orthogonal and synergistic oncotargets for the treatment of cancer, reported the presentation last week of its novel approach to immune-oncology treatments (Press release, SignalRx, MAY 1, 2017, http://www.ireachcontent.com/news-releases/signalrx-discloses-its-novel-immuno-oncology-program-approach-at-the-12th-annual-drug-discovery-chemistry-2017-meeting-620879733.html [SID1234527322]). The presentation by Donald L. Durden, MD, PhD, senior scientific advisor for SignalRx, occurred at 10:30 am on Thursday April 27th, 2017, at the Drug Discovery Chemistry 2017 meeting in San Diego, CA.

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The presentation was entitled"Dual PI3K/BRD4 inhibitor (SF2523) for immuno-oncology to suppress MYC and the MQ immunosuppressive environment". SignalRx’s program provides a novel approach to immune-oncology and is summarized below:

SF2523 is the only known nM potent dual PI3K/BET bromodomain inhibitor; this provides maximal suppression of the oncogenic transcription factor MYC via enhanced MYC degradation (PI3K inhibition) coupled with decreased production of MYC by inhibiting BRD4 which inhibits transcription of MYC.
MYC has been shown by others to regulate the antitumor immune response through immune-oncology checkpoint receptors CD47 and PD-L1; we have demonstrated that SF2523 reduces the expression of PD-L1 in hepatocellular carcinoma cells, likely through the suppression of MYC.
SF2523 inhibits PI3K isoforms shown by others to regulate the immune suppression found in the tumor microenvironment
SF2523 blocks the transition of M1 macrophages to the immune-suppressing M2 macrophage stage which is expected to augment antitumor immunity
SF2523 is the only dual PI3K/BRD4 inhibitor with demonstrated in vivo antitumor, antimetastatic and immune activating activity.
SignalRx is also seeking partnering opportunities to accelerate the development of its programs and advance novel anticancer therapeutics into first-in-man clinical trials based on the promising profile and mode of action of its inhibitors. Since these are single molecules with a single PK/PD and toxicity profile, there is a great opportunity to streamline their development alone and in combination therapies.

Aeterna Zentaris Announces that ZoptEC Phase 3 Clinical Study of Zoptrex™ Did Not Achieve its Primary Endpoint

On May 1, 2017 Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX:AEZS) (the "Company") reported that the ZoptEC Phase 3 clinical study of Zoptrex (zoptarelin doxorubicin) in women with locally advanced, recurrent or metastatic endometrial cancer did not achieve its primary endpoint of demonstrating a statistically significant increase in the median period of overall survival of patients treated with Zoptrex as compared to patients treated with doxorubicin (Press release, AEterna Zentaris, MAY 1, 2017, View Source [SID1234518774]).

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Dr. Richard Sachse, the Company’s Chief Scientific Officer, stated, "The median overall survival period for patients treated with Zoptrex was 10.9 months compared to 10.8 months for patients treated with doxorubicin. This is not a statistically significant, clinically meaningful increase in overall survival and thus the ZoptEC Phase 3 clinical study did not meet its primary endpoint. In addition, Zoptrex generally performed no better than the comparator drug with respect to the secondary efficacy endpoints. For example, the median period of progression-free survival of the patients in the Zoptrex arm of the study was identical to that for patients in the doxorubicin arm. Finally, there was no meaningful difference between the two arms with respect to safety; the number of patients with cardiac disorders was similar – eight in the Zoptrex arm and nine in the doxorubicin arm. Therefore, the results of the study are not supportive to pursue regulatory approval."

David A. Dodd, the President and Chief Executive Officer of the Company, stated, "We are very disappointed with the outcome of the ZoptEC Phase 3 clinical study. Based on this outcome, we do not anticipate conducting clinical trials of Zoptrex with respect to any other indications. I would like to thank my colleagues within Aeterna Zentaris and our external team of clinical investigators for their dedication to and contributions on this project." Commenting on the Company’s plans, Mr. Dodd continued, "Our focus has now shifted entirely to filing our new drug application for Macrilen and, if the product is approved, to its commercial launch as soon as possible. We will also optimize our resources to be consistent with our focus on Macrilen-related efforts. We continue to believe in the potential that Macrilen provides for us to become a focused specialty pharmaceutical company. Our intention is to submit the Macrilen NDA in the third quarter of 2017 and, if the product receives FDA approval, to commercially launch the product in the first quarter of 2018."

LION BIOTECHNOLOGIES REPORTS FIRST QUARTER 2017 FINANCIAL RESULTS

On May 1, 2017 Lion Biotechnologies, Inc. (NASDAQ: LBIO), a biotechnology company developing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology, reported its first quarter 2017 financial results and provided a corporate update (Press release, Lion Biotechnologies, MAY 1, 2017, View Source [SID1234518772]).

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"We started 2017 with a clear focus on execution toward expanding our manufacturing capacity, our clinical program and initiation of engagement with health authorities. During the first quarter of 2017, we increased our manufacturing capacity and are now focused on expansion of our clinical program as well as access to clinical data through collaborations. I am very excited about our recently announced collaboration with MD Anderson, which allows us access to rare patient populations as well as a new method of manufacturing TIL using the MD Anderson process. Lion now has strategic relationships with leading academic and governmental institutions in the U.S. researching TIL technology including the NCI, MD Anderson, and the H. Lee Moffitt Cancer Center as well as one of a leading institution in Europe, the Karolinska Institute, providing Lion the ability to pursue a much broader clinical program than would otherwise be possible as a standalone company," said Dr. Maria Fardis, PhD, MBA, Chief Executive Officer of Lion Biotechnologies. "With this new collaboration in place, by year-end we may have partner-sponsored trials in pancreatic, glioblastoma, ovarian cancer, various sarcomas and melanoma combination trials with three of the approved checkpoint inhibitors in addition to the three ongoing Lion-sponsored TIL clinical studies in metastatic melanoma, head and neck and cervical cancers. This gives us a robust pipeline based on our TIL technology."

First Quarter 2017 and Recent Highlights

Partnerships:

Multi-year strategic alliance with MD Anderson Cancer Center: Lion entered a multi-year strategic alliance agreement involving multi-arm clinical trials to evaluate the safety and efficacy of TIL therapy in ovarian cancer, various sarcomas and pancreatic cancer. In addition, Lion and MD Anderson will conduct preclinical research exploring the expansion of TIL in other rare tumor types.
Manufacturing services agreement with PharmaCell enables expansion of clinical studies in Europe: Lion has entered in to a new, three-year Manufacturing Services Agreement (MSA) with PharmaCell B.V. (PharmaCell), a contract manufacturing services company based in the Netherlands, to manufacture the Company’s autologous cell therapy products. PharmaCell will manufacture TIL products for Lion in its clinical and commercial facility in Geleen, the Netherlands.
Clinical Trial Progress:

Enrollment in LN-144 Phase 2 melanoma study continues: Lion has initiated enrolling patients into the second cohort of its LN-144 program. This cohort utilizes the generation 2 manufacturing process. This process reduces the time from excision to infusion from approximately six weeks to approximately three and half weeks.
Two new Phase 2 studies initiated: The Company has now initiated two Phase 2 trials for LN-145 for the treatment of head and neck and cervical cancers. Clinical trial sites are open for both studies and they are actively screening for patients.
Regulatory:

Global conduct of clinical trials: The Company is engaging health authorities outside the U.S. to gain clarity around conduct of clinical trials outside of US.
Publications:

Publication of new translational data in the journal Science: Lion highlighted a publication in the journal Science that provided new translational data from a clinical trial of TIL therapy for the treatment of advanced metastatic cervical cancer conducted at the Surgery Branch of the National Cancer Institute (NCI). This trial has been supported in part by Lion under a Cooperative Research and Development Agreement (CRADA) with Dr. Steven Rosenberg, Chief of the Surgery Branch, NCI, National Institutes of Health.
First Quarter 2017 Financial Results

As of March 31, 2017, the Company held $147.2 million in cash and cash equivalents and short-term investments, compared to $166.5 million as of December 31, 2016.

GAAP and Non-GAAP net loss

GAAP net loss for the quarter ended March 31, 2017 was $20.7 million, or ($0.33) per share, compared to GAAP net loss of $6.9 million or ($0.14) per share for the quarter ended March 31, 2016.

Non-GAAP net loss, which excludes amounts related to stock-based compensation, for the quarter ended March 31, 2017 was $17.4 million, or ($0.28) per share, compared to non-GAAP net loss of $5.1 million, or ($0.10) per share for the quarter ended March 31, 2016. The non-GAAP net loss for the quarter ended March 31, 2017 excludes $3.3 million of non-cash stock-based compensation.

The Company believes that it is important for investors to understand these non-cash charges as they materially impact the net loss and loss per share calculations. See "Use of Non-GAAP Financial Measures" below for a description of the Company’s Non-GAAP Financial Measures. Reconciliation between certain GAAP and Non-GAAP measures is provided at the end of this press release.

GAAP and Non-GAAP expenses

GAAP research and development (R&D) expenses were $16.6 million for the quarter ended March 31, 2017, an increase of $12.4 million compared to the quarter ended March 31, 2016. The increase in R&D expense is due to increased spending on clinical activities and expansion of manufacturing capabilities. This level of spending is consistent with cell therapy companies at Lion’s stage of clinical development. In addition, R&D-associated stock option expenses were $1.4 million for the three months ended March 31, 2017. Non-GAAP R&D expenses were $15.2 million for the quarter ended March 31, 2017, an increase of $11.6 million, compared to $3.6 million for the quarter ended March 31, 2016.

GAAP general and administrative (G&A) expenses were $4.3 million, an increase of $1.4 million compared to the quarter ended March 31, 2016. The increase in G&A expense is primarily due to the increase in headcount and legal and outside services. Non-GAAP G&A expenses were $2.4 million, which excludes amounts related to stock-based compensation of $1.9 million, for the quarter ended March 31, 2017 an increase of $0.8 million, compared to $1.6 million for the quarter ended March 31, 2016.

Reconciliation between certain GAAP and Non-GAAP measures is provided at the end of this press release.

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses. These measures are not in accordance with, or an alternative to, generally accepted accounting principles, or GAAP, and may be different from non-GAAP financial measures used by other companies. The item included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release relates to the non-cash stock-based compensation expense which may fluctuate from period to period based on factors including the timing and accounting of grants for stock options and changes in the Company’s stock price which impacts the fair value of options granted. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding various financial and business trends relating to the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of Lion’s ongoing operating performance. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating operational performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. To the extent this release contains historical or future non-GAAP financial measures, the Company has also provided corresponding GAAP financial measures for comparative purposes. Reconciliation between certain GAAP and non-GAAP measures is provided at the end of this press release.