Lilly Drug Fails in Breast Cancer Trial, shares fall; Verastem Begins Phase 2 Trial of Defatinib in Lung Cancer Print E-mail
By Staff and Wire Reports   
Thursday, 26 September 2013 19:24
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 26, 2013. Eli Lilly and Co (NYS:LLY) said it will not seek a breast cancer approval for one of its most closely watched experimental drugs after it failed to significantly delay disease progression and showed no survival benefit in a late-stage trial.

The drug, ramucirumab, has been successful in gastric cancer trials and is also being tested for colon, liver and lung cancers, with key late stage data on those three expected next year. But Lilly shares fell more than 3 percent after it released the disappointing breast cancer results.

"While Street expectations were low for ramucirumab in breast, they were not quite this low," ISI Group analyst Mark Schoenebaum wrote in a note, saying the drug was expected to delay worsening of breast cancer but not extend survival.

"We believe there is approximately $500-$600 million currently in Street models for breast cancer. This will now need to come out of models," Schoenebaum said.

The company needs new medicines to offset steep revenue declines from drugs facing generic competition. It's top seller, the antidepressant Cymbalta, which had $1.5 billion (935 million pounds) in sales in the second quarter, loses U.S. patent protection later this year.

The company is counting on ramucirumab - which it gained with its $6.5 billion acquisition of ImClone Systems Inc in 2008 - and some promising diabetes treatments to help spur future growth.

The trial of patients with advanced breast cancer compared ramucirumab plus the chemotherapy drug docetaxel with docetaxel alone. A delay in disease progression seen with the Lilly drug was not statistically significant and no survival benefit was demonstrated, the company said.

A separate Phase III trial, testing ramucirumab in advanced gastric cancer, met its main goal by extending patients' overall survival. It was the second stomach cancer trial that showed the drug improved survival. The company said it will provide detailed data from that study some time next year.

Lilly said it expects to complete its submission seeking U.S. approval for gastric cancer by the end of this year. Given the unmet need in that cancer, it could qualify for an expedited priority review, potentially cutting its U.S. approval timeline.

Eli Lilly shares were down $1.83, or 3.5 percent, at $50.78 in afternoon trading on the New York Stock Exchange.



==================



Verastem, Inc. (NASDAQ: VSTM)
, focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced the initiation of a Phase 2 trial of defactinib (VS-6063), a potent inhibitor of focal adhesion kinase (FAK), in KRAS-mutated non-small cell lung cancer (NSCLC).

“We currently do not have any effective targeted treatment options for patients with KRAS-mutated non-small cell lung cancer (NSCLC) which represents about 30% of the NSCLC population,” said Dr. David Gerber, Associate Professor of Internal Medicine, Lung Cancer Specialist at the University of Texas Southwestern Medical Center and Study Chairman of the defactinib clinical trial. “Research by Dr. Scaglioni here at UTSW has demonstrated that inhibition of Focal Adhesion Kinase in KRAS-mutated NSCLC with a secondary mutation in either p16 or p53, extends survival in xenograft models of this disease.”

Dr. Scaglioni's group at UTSW published a paper in 2013 in Cancer Discovery titled “RHOA-FAK is a Required Signaling Axis for the Maintenance of KRAS-Driven Lung Adenocarcinoma” that describes the critical role of FAK signaling in NSCLC that has both a KRAS-mutation and accompanying secondary mutation in the p16/ARF/INK4a locus (p16) or p53. As reported in the paper, genetic silencing or deletion of FAK replicated the effects of treatment with Verastem's first generation small molecule inhibitor of FAK, VS-6062, in both in vitro and xenograft models of the disease.

"The reported rates vary, but we believe approximately 10-15% of NSCLC tumors have both the KRAS mutation and an accompanying secondary mutation in p16 or p53 leading to silencing of those tumor suppressor genes, which appears to be a prerequisite for the activity of FAK inhibitors in KRAS-mutated NSCLC models,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Patients with KRAS mutated tumors derive limited benefit from chemotherapy. This study is designed to elucidate the role of the secondary mutation in response to FAK inhibitor treatment and hopefully make a meaningful impact on the clinical response to therapy in a subset of patients with difficult to treat NSCLC.”

The Phase 2 study is designed to assess the effect of defactinib on Progression Free Survival (PFS), Overall Response Rate (ORR) and Overall Survival (OS). There will be 4 study arms, including patients with a KRAS-mutation, or a KRAS-mutation with accompanying secondary mutations in p16, p53 or both p16 and p53. The trial is being conducted at 8-10 sites in the US and is expected to enroll up to 44 patients (11 per arm) in the first stage of the Simon two-stage trial design. A Simon two-stage trial design incorporates a single, pre-planned interim analysis to determine which enrollment arms are exhibiting a favorable response to treatment and allows for enrollment of an additional 12 patients with the same tumor mutation profile per arm.

“With the initiation of this study we now have a total of five clinical studies underway,” said Robert Forrester, Verastem President and Chief Executive Officer. “In addition to the NSCLC trial, we recently initiated the registration-directed COMMAND study in mesothelioma, a Phase 1 trial of defactinib in Japan and continue enrollment in the Phase 1b combination study of defactinib and paclitaxel for patients with ovarian cancer. In parallel to the development of defactinib, we have an ongoing Phase 1 study of VS-4718 in advanced solid tumors. We will continue to execute on our development plans and remain committed to bringing novel therapies targeting cancer stem cells to patients with few to no adequate treatment options.”



Also Thursday:



Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR)
, an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat debilitating and often fatal rare diseases, announced today that the company will participate in the Leerink Swann Rare Disease Roundtable in New York City.

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO)
, today announced the addition of Paul J. Clancy, executive vice president and chief financial officer at Biogen Idec, to the company’s board of directors.

Agenus Inc. (NASDAQ: AGEN)
today announced that it is has entered into a non-exclusive license agreement with VaxLogic, LLC, a subsidiary of PharmLogic LLC, for the use of QS-21 Stimulon®1 adjuvant in the development of select addiction, allergy and respiratory disease vaccine candidates.

The Alkaline Water Company Inc. (OTCBB: WTER)
, developers of an innovative, state of the art, proprietary electrolysis beverage process, packaged and sold under the trade name Alkaline88, a premier alkaline drinking water for balanced lifestyles, has announced its flagship Alkaline88 product will be available through internet retailer Amazon.com.

Baxano Surgical, Inc. (Nasdaq:BAXS)
, a medical device company focused on designing, developing and marketing minimally invasive products to treat degenerative conditions of the spine affecting the lumbar region, today announced regulatory approvals and distribution agreements for certain of its products in Mexico, Colombia and Turkey.

Bioheart, Inc. (OTCQB: BHRT)
, a biotechnology company focused on the discovery, development and, subject to regulatory approval, commercialization of autologous cell therapies for the treatment of chronic and acute heart damage, announced today preliminary data from its phase I ANGEL Trial.

Bravo Enterprises Ltd. (OTC:BB-OGNG)
announces that the Company has launched a two year leasing program for the home and office air-to-water machines known as the AIRMAX 3000, with a buyout option.

Chimerix, Inc. (Nasdaq:CMRX)
, a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced the publication of results from its Phase 2 Study CMX001-201 evaluating brincidofovir (CMX001) for the prevention of cytomegalovirus (CMV) infection in hematopoietic cell transplant (HCT) recipients.

CollabRx, Inc. (Nasdaq:CLRX)
, a data analytics company focused on informing clinical decision making in molecular medicine, announces that Thomas R. Mika, Chief Executive Officer, will present at the LD MICRO Invitational on Wednesday, October 2, 2013 at 1:00 p.m. PST / 4:00 p.m. EST.

Cryoport, Inc. (OTCBB: CYRX)
and OCASA, Inc. have entered into a master services agreement to provide global cold chain logistics solutions for life science and biotech commodities requiring cryogenic temperatures.

Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY)
announced today that new data from its Aubagio® (teriflunomide) and Lemtrada™ (alemtuzumab) clinical development programs will be presented during the 29th Congress of the European Committee for Research and Treatment in Multiple Sclerosis (ECTRIMS) and the 18th Annual Conference of Rehabilitation in MS (RIMS) to be held in Copenhagen, Denmark, October 2-5.

Hyperion Therapeutics (Nasdaq:HPTX)
announced today that the company is scheduled to present at the Leerink Swann Rare Disease Day on Wednesday, October 2 at 9:20 a.m. ET.

ImmuDyne, Inc. (OTCQB: IMMD)
announced today that it has added inventory for the launch of Its new dietary supplement lines for sale; iNR Wellness™, iNR Wellness MD™, and iNR Strength™, with a retail value of over $1.25 million.

MRI Interventions, Inc. (OTCQB: MRIC)
announced today that its ClearPoint® Neuro Intervention System for real-time MRI-guided navigation in minimally-invasive neurosurgery procedures has been utilized at Cincinnati Children's Hospital to perform deep brain stimulation (DBS) surgery for treatment of pediatric dystonia while the patient rested under general anesthesia.

Neuralstem, Inc. (NYSE MKT: CUR)
announced that President and CEO, Richard Garr, will present at the Stem Cells & Regenerative Medicine Congress, on Monday, September 30 at 12:00 PM at the Hyatt Regency in Cambridge, MA .

Nymox Pharmaceutical Corporation (Nasdaq:NYMX)
announced today that enrollment in the company's NX03-0040 US Phase 2 clinical trial of NX-1207 for localized prostate cancer is nearing completion with over 80% patients now enrolled.

OxySure Systems, Inc. (OTCQB: OXYS)
a medical technology company that has pioneered an FDA-approved solution to produce medically pure oxygen from dry, inert powders for emergency and short duration use - announced today that it will be exhibiting at the National Safety Council's (NSC) 2013 Congress & Exposition, the world's largest annual "must attend" event for safety, health and environmental professionals taking place in Chicago, IL from September 30 - October 2, 2013.

Raptor Pharmaceutical Corp. (Nasdaq:RPTP)
today announced management will present at the Leerink Swann Rare Disease Roundtable in New York on October 2, 2013 at 8:40 a.m. EDT/5:40 a.m. PDT.

Response Biomedical Corp. (TSX:RBM) (OTCBB:RPBIF)
is pleased to announce the brokered and non-brokered private placement of Subscription Receipts for aggregate gross proceeds of up to $4,000,000.

Retrophin, Inc. (OTCQB: RTRX)
today announced that it has elected Cornelius (“Neal”) E. Golding to its Board of Directors, effective October 1, 2013.

The Willis-Ekbom Disease (WED) Foundation and XenoPort, Inc. (Nasdaq: XNPT) announced today that they have launched a new “Patient Odyssey” survey, aimed at measuring the challenges of long-term disease management, quality of life (QoL) impact and emotional burden associated with restless legs syndrome/Willis-Ekbom Disease (RLS/WED) among patients, as well as the impact of the disease on relationships with spouses or partners. NYS:LLY) said it will not seek a breast cancer approval for one of its most closely watched experimental drugs after it failed to significantly delay disease progression and showed no survival benefit in a late-stage trial. The drug, ramucirumab, has been successful in gastric cancer trials and is also being tested for colon, liver and lung cancers, with key late stage data on those three expected next year. But Lilly shares fell more than 3 percent after it released the disappointing breast cancer results. "While Street expectations were low for ramucirumab in breast, they were not quite this low," ISI Group analyst Mark Schoenebaum wrote in a note, saying the drug was expected to delay worsening of breast cancer but not extend survival. "We believe there is approximately $500-$600 million currently in Street models for breast cancer. This will now need to come out of models," Schoenebaum said. The company needs new medicines to offset steep revenue declines from drugs facing generic competition. It's top seller, the antidepressant Cymbalta, which had $1.5 billion (935 million pounds) in sales in the second quarter, loses U.S. patent protection later this year. The company is counting on ramucirumab - which it gained with its $6.5 billion acquisition of ImClone Systems Inc in 2008 - and some promising diabetes treatments to help spur future growth. The trial of patients with advanced breast cancer compared ramucirumab plus the chemotherapy drug docetaxel with docetaxel alone. A delay in disease progression seen with the Lilly drug was not statistically significant and no survival benefit was demonstrated, the company said. A separate Phase III trial, testing ramucirumab in advanced gastric cancer, met its main goal by extending patients' overall survival. It was the second stomach cancer trial that showed the drug improved survival. The company said it will provide detailed data from that study some time next year. Lilly said it expects to complete its submission seeking U.S. approval for gastric cancer by the end of this year. Given the unmet need in that cancer, it could qualify for an expedited priority review, potentially cutting its U.S. approval timeline. Eli Lilly shares were down $1.83, or 3.5 percent, at $50.78 in afternoon trading on the New York Stock Exchange. ================== Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, today announced the initiation of a Phase 2 trial of defactinib (VS-6063), a potent inhibitor of focal adhesion kinase (FAK), in KRAS-mutated non-small cell lung cancer (NSCLC). “We currently do not have any effective targeted treatment options for patients with KRAS-mutated non-small cell lung cancer (NSCLC) which represents about 30% of the NSCLC population,” said Dr. David Gerber, Associate Professor of Internal Medicine, Lung Cancer Specialist at the University of Texas Southwestern Medical Center and Study Chairman of the defactinib clinical trial. “Research by Dr. Scaglioni here at UTSW has demonstrated that inhibition of Focal Adhesion Kinase in KRAS-mutated NSCLC with a secondary mutation in either p16 or p53, extends survival in xenograft models of this disease.” Dr. Scaglioni's group at UTSW published a paper in 2013 in Cancer Discovery titled “RHOA-FAK is a Required Signaling Axis for the Maintenance of KRAS-Driven Lung Adenocarcinoma” that describes the critical role of FAK signaling in NSCLC that has both a KRAS-mutation and accompanying secondary mutation in the p16/ARF/INK4a locus (p16) or p53. As reported in the paper, genetic silencing or deletion of FAK replicated the effects of treatment with Verastem's first generation small molecule inhibitor of FAK, VS-6062, in both in vitro and xenograft models of the disease. "The reported rates vary, but we believe approximately 10-15% of NSCLC tumors have both the KRAS mutation and an accompanying secondary mutation in p16 or p53 leading to silencing of those tumor suppressor genes, which appears to be a prerequisite for the activity of FAK inhibitors in KRAS-mutated NSCLC models,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Patients with KRAS mutated tumors derive limited benefit from chemotherapy. This study is designed to elucidate the role of the secondary mutation in response to FAK inhibitor treatment and hopefully make a meaningful impact on the clinical response to therapy in a subset of patients with difficult to treat NSCLC.” The Phase 2 study is designed to assess the effect of defactinib on Progression Free Survival (PFS), Overall Response Rate (ORR) and Overall Survival (OS). There will be 4 study arms, including patients with a KRAS-mutation, or a KRAS-mutation with accompanying secondary mutations in p16, p53 or both p16 and p53. The trial is being conducted at 8-10 sites in the US and is expected to enroll up to 44 patients (11 per arm) in the first stage of the Simon two-stage trial design. A Simon two-stage trial design incorporates a single, pre-planned interim analysis to determine which enrollment arms are exhibiting a favorable response to treatment and allows for enrollment of an additional 12 patients with the same tumor mutation profile per arm. “With the initiation of this study we now have a total of five clinical studies underway,” said Robert Forrester, Verastem President and Chief Executive Officer. “In addition to the NSCLC trial, we recently initiated the registration-directed COMMAND study in mesothelioma, a Phase 1 trial of defactinib in Japan and continue enrollment in the Phase 1b combination study of defactinib and paclitaxel for patients with ovarian cancer. In parallel to the development of defactinib, we have an ongoing Phase 1 study of VS-4718 in advanced solid tumors. We will continue to execute on our development plans and remain committed to bringing novel therapies targeting cancer stem cells to patients with few to no adequate treatment options.” Also Thursday: Aegerion Pharmaceuticals, Inc. (Nasdaq:AEGR), an emerging biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat debilitating and often fatal rare diseases, announced today that the company will participate in the Leerink Swann Rare Disease Roundtable in New York City. Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), today announced the addition of Paul J. Clancy, executive vice president and chief financial officer at Biogen Idec, to the company’s board of directors. Agenus Inc. (NASDAQ: AGEN) today announced that it is has entered into a non-exclusive license agreement with VaxLogic, LLC, a subsidiary of PharmLogic LLC, for the use of QS-21 Stimulon®1 adjuvant in the development of select addiction, allergy and respiratory disease vaccine candidates. The Alkaline Water Company Inc. (OTCBB: WTER) ("Alkaline" or the "Company"), developers of an innovative, state of the art, proprietary electrolysis beverage process, packaged and sold under the trade name Alkaline88, a premier alkaline drinking water for balanced lifestyles, has announced its flagship Alkaline88 product will be available through internet retailer Amazon.com. Baxano Surgical, Inc. (Nasdaq:BAXS), a medical device company focused on designing, developing and marketing minimally invasive products to treat degenerative conditions of the spine affecting the lumbar region, today announced regulatory approvals and distribution agreements for certain of its products in Mexico, Colombia and Turkey. Bioheart, Inc. (OTCQB: BHRT), a biotechnology company focused on the discovery, development and, subject to regulatory approval, commercialization of autologous cell therapies for the treatment of chronic and acute heart damage, announced today preliminary data from its phase I ANGEL Trial. Bravo Enterprises Ltd. (OTC:BB-OGNG) announces that the Company has launched a two year leasing program for the home and office air-to-water machines known as the AIRMAX 3000, with a buyout option. Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced the publication of results from its Phase 2 Study CMX001-201 evaluating brincidofovir (CMX001) for the prevention of cytomegalovirus (CMV) infection in hematopoietic cell transplant (HCT) recipients. CollabRx, Inc. (Nasdaq:CLRX), a data analytics company focused on informing clinical decision making in molecular medicine, announces that Thomas R. Mika, Chief Executive Officer, will present at the LD MICRO Invitational on Wednesday, October 2, 2013 at 1:00 p.m. PST / 4:00 p.m. EST. Cryoport, Inc. (OTCBB: CYRX) and OCASA, Inc. have entered into a master services agreement to provide global cold chain logistics solutions for life science and biotech commodities requiring cryogenic temperatures. Genzyme, a Sanofi company (EURONEXT: SAN and NYSE: SNY) announced today that new data from its Aubagio® (teriflunomide) and Lemtrada™ (alemtuzumab) clinical development programs will be presented during the 29th Congress of the European Committee for Research and Treatment in Multiple Sclerosis (ECTRIMS) and the 18th Annual Conference of Rehabilitation in MS (RIMS) to be held in Copenhagen, Denmark, October 2-5. Hyperion Therapeutics (Nasdaq:HPTX) announced today that the company is scheduled to present at the Leerink Swann Rare Disease Day on Wednesday, October 2 at 9:20 a.m. ET. ImmuDyne, Inc. (OTCQB: IMMD) announced today that it has added inventory for the launch of Its new dietary supplement lines for sale; iNR Wellness™, iNR Wellness MD™, and iNR Strength™, with a retail value of over $1.25 million. MRI Interventions, Inc. (OTCQB: MRIC) announced today that its ClearPoint® Neuro Intervention System for real-time MRI-guided navigation in minimally-invasive neurosurgery procedures has been utilized at Cincinnati Children's Hospital to perform deep brain stimulation (DBS) surgery for treatment of pediatric dystonia while the patient rested under general anesthesia. Neuralstem, Inc. (NYSE MKT: CUR) announced that President and CEO, Richard Garr, will present at the Stem Cells & Regenerative Medicine Congress, on Monday, September 30 at 12:00 PM at the Hyatt Regency in Cambridge, MA . Nymox Pharmaceutical Corporation (Nasdaq:NYMX) announced today that enrollment in the company's NX03-0040 US Phase 2 clinical trial of NX-1207 for localized prostate cancer is nearing completion with over 80% patients now enrolled. OxySure Systems, Inc. (OTCQB: OXYS) a medical technology company that has pioneered an FDA-approved solution to produce medically pure oxygen from dry, inert powders for emergency and short duration use - announced today that it will be exhibiting at the National Safety Council's (NSC) 2013 Congress & Exposition, the world's largest annual "must attend" event for safety, health and environmental professionals taking place in Chicago, IL from September 30 - October 2, 2013. Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced management will present at the Leerink Swann Rare Disease Roundtable in New York on October 2, 2013 at 8:40 a.m. EDT/5:40 a.m. PDT. Response Biomedical Corp. (TSX:RBM) (OTCBB:RPBIF) is pleased to announce the brokered and non-brokered private placement of Subscription Receipts for aggregate gross proceeds of up to $4,000,000. Retrophin, Inc. (OTCQB: RTRX) today announced that it has elected Cornelius (“Neal”) E. Golding to its Board of Directors, effective October 1, 2013. The Willis-Ekbom Disease (WED) Foundation and XenoPort, Inc. (Nasdaq: XNPT) announced today that they have launched a new “Patient Odyssey” survey, aimed at measuring the challenges of long-term disease management, quality of life (QoL) impact and emotional burden associated with restless legs syndrome/Willis-Ekbom Disease (RLS/WED) among patients, as well as the impact of the disease on relationships with spouses or partners.



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