1stOncology™: Cancer Intelligence Service – Page 16939 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Moderna Launches Caperna LLC, Its Fourth Venture, to Develop Personalized Cancer Vaccines

On October 22, 2015 Moderna Therapeutics, a pioneer in the development of messenger RNA (mRNA) Therapeutics, reported the launch of Caperna LLC, the fourth Moderna venture, which will focus exclusively on the advancement of personalized cancer vaccines.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

Caperna will apply Moderna’s mRNA vaccine technology to the field of cancer vaccines, building on advances in recent years in cancer immunotherapy, including the availability of checkpoint inhibitors and the ability to rapidly determine individual patient mutations. Caperna will develop personalized cancer vaccines that encode a patient’s specific neoepitopes, utilizing Moderna’s unique infrastructure to manufacture within weeks small batches of vaccines tailored to each patient.

Substantial efforts at Moderna’s infectious disease venture Valera have demonstrated preclinical efficacy of Moderna’s mRNA-based vaccines in multiple viral disease models, which has led to the nomination of several viral vaccine development candidates. These advancements have spurred the formation of Caperna and the pursuit of vaccine development for the treatment of cancer.

Tal Zaks, M.D., Ph.D., Chief Medical Officer of Moderna, who was formerly Senior Vice President and Head of Global Oncology at Sanofi, will serve as Interim President of Caperna. Nicholas Valiante, Ph.D., who recently joined Moderna from Novartis Vaccines where he was Global Head of Immunology & Immunotherapy Research, will serve as Vice President and Head of Personalized Vaccine Sciences.

"With such a potent vaccine platform and a manufacturing process that lends itself directly to rapid production of patient-specific therapies, we believe Moderna’s mRNA approach will offer distinct advantages in the development of new cancer therapies," said Dr. Zaks. "We expect our ability to specifically activate and direct the immune system will synergize with checkpoint inhibitor therapies like PD-1 antibodies."

"What is perhaps most exciting about our approach and our core protein expression platform is its potential to leverage a broad spectrum of leading-edge drug modalities," said Stéphane Bancel, Chief Executive Officer of Moderna. "Caperna is an important example of Moderna’s unique ability to rapidly translate our ever-evolving understanding of mRNA into new therapeutic opportunities that have the potential to impact and change numerous lives. Caperna will be backed by Moderna’s $840 million of cash on hand."

Onkaido, Moderna’s initial oncology venture, remains focused on the development of mRNA-based therapeutics for oncology in areas outside of the personalized cancer vaccine effort at Caperna.

FDA approves new treatment for advanced pancreatic cancer

On October 22, 2015 The U.S. Food and Drug Administration reported it approved Onivyde (irinotecan liposome injection), in combination with fluorouracil and leucovorin, to treat patients with advanced (metastatic) pancreatic cancer who have been previously treated with gemcitabine-based chemotherapy (Press release, , OCT 22, 2015, View Source [SID:1234507767]).
According to the National Cancer Institute, there will be 48,960 new cases of pancreatic cancer diagnosed in the U.S. in 2015, and nearly the same number of deaths caused by the disease (40,560). Pancreatic cancer can be difficult to diagnose early and treatment options are limited, especially when the disease has spread to other parts of the body (metastatic disease) and surgery to remove the tumor is not possible.

"Many FDA staff who review drug applications are clinicians as well, so it’s especially rewarding when we are able to expedite access to new treatments for patients with unmet needs," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "By using the Priority Review designation for the application for Onivyde, patients will have earlier access to a drug that helps extend survival."

The FDA granted Priority Review and orphan drug designations for Onivyde. Priority review status is granted to applications for drugs that, if approved, would be a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan drug designation provides incentives such as tax credits, user fee waivers, and eligibility for orphan drug exclusivity to assist and encourage the development of drugs for rare diseases.

The effectiveness of Onivyde was demonstrated in a three-arm, randomized, open label study of 417 patients with metastatic pancreatic adenocarcinoma whose cancer had grown after receiving the chemotherapeutic drug gemcitabine or a gemcitabine-based therapy. The study was designed to determine whether patients receiving Onivyde plus fluorouracil/leucovorin or Onivyde alone lived longer than those receiving fluorouracil/leucovorin. Patients treated with Onivyde plus fluorouracil/leucovorin lived an average of 6.1 months, compared to 4.2 months for those treated with only fluorouracil/leucovorin. There was no survival improvement for those who received only Onivyde compared to those who received fluorouracil/leucovorin.

In addition, patients receiving Onivyde plus fluorouracil/leucovorin had a delay in the amount of time to tumor growth compared to those who received fluorouracil/leucovorin. The average time for those receiving Onivyde plus fluorouracil/leucovorin was 3.1 months compared to 1.5 months for those receiving fluorouracil/leucovorin.

The safety of Onivyde was evaluated in 398 patients who received either Onivyde with fluorouracil/leucovorin, Onivyde alone or fluorouracil/leucovorin. The most common side effects of treatment with Onivyde included diarrhea, fatigue, vomiting, nausea, decreased appetite, inflammation in the mouth (stomatitis) and fever (pyrexia). Onivyde was also found to result in low counts of infection-fighting cells (lymphopenia and neutropenia). Death due to sepsis following neutropenia has been reported in patients treated with Onivyde.

The labeling for Onivyde includes a boxed warning to alert health care professionals about the risks of severe neutropenia and diarrhea. Onivyde is not approved for use as a single agent for the treatment of patients with metastatic pancreatic cancer.

Onivyde is marketed by Merrimack Pharmaceuticals Inc. of Cambridge, Massachusetts.

Knowledge-Based Software Can Significantly Expedite Creation of High-Quality Radiotherapy Treatment Plans for Precise Tumor Targeting

On October 22, 2015 Varian Medical Systems reported that studies presented earlier this week at the 2015 annual meeting of the American Society for Radiation Oncology (ASTRO) confirmed that knowledge-based treatment planning software can dramatically improve the speed and quality of cancer care (Press release, Varian Medical Systems, OCT 22, 2015, View Source [SID:1234507766]). One award-winning study by a team using RapidPlan software from Varian Medical Systems (NYSE: VAR) showed that radiotherapy treatment planning for cervical cancer can be done in minutes rather than hours, with superior quality.

In a presentation that was named among the "Best of ASTRO" and won the Basic/ Translational Science Abstract Award in the physics category, Nan Li, PhD, postdoctoral fellow at the University of California, San Diego (UCSD), and her team1 reported on their use of a RapidPlan model that was based on a refined sample of 86 previously-treated cervical cancer cases. They found that treatment planning time for intensity-modulated radiation therapy took an average of 6.85 minutes.[1] According to Kevin Moore, PhD, senior author on the study, UCSD dosimetrists estimate that manual GYN planning would require anywhere from 2-6 hours of optimization. "The use of knowledge-based planning represents a considerable time savings and reduced personnel costs," he said.

RapidPlan also improved plan quality compared to conventionally generated plans by minimizing the impact on normal surrounding tissues. "With both dramatic efficiency gains and improved normal tissue sparing, the final automated planning module was validated as both a clinical trial quality control system and a valuable tool for high-quality clinical planning in cervical cancer," observed Li.

Moore and his physician colleagues from UCSD also described work where stereotactic radiosurgery (SRS) treatment plans created using automated knowledge-based planning algorithms that they developed were set against manually-created clinical plans in a blinded comparison study.[2]

"In a clear majority of the cases, automated SRS planning demonstrated superior or equivalent plan quality to existing manual planning processes," Moore said. "Further refinement of algorithms to balance the complex clinical tradeoffs for high-priority organs-at-risk . . . will likely improve this technique further."

Researchers from Duke University evaluated a "rapid learning approach" in which clinicians "train" the RapidPlan tool by establishing a base knowledge model and continuously evaluate and update this knowledge model using subsequent cases. In their research on pelvic cancer cases, Jackie Wu, PhD, professor of radiation oncology, and her colleagues compared the RapidPlan rapid learning approach to the batch training method: knowledge modeling based on a static set of training cases.[3]

"The rapid learning approach is able to learn knowledge models for multiple cancer types in the pelvic region with comparable accuracy to the batch training method and with improved efficiency," Dr. Wu said. "This approach will facilitate the implementation of the knowledge based radiation therapy planning in clinics."

Knowledge-based planning enables clinicians to extract information from past clinical experience and use it to generate mathematical models that expedite the creation of new treatment plans. The software helps the planner quickly generate a new treatment plan that achieves the physician’s tumor coverage and normal tissue sparing goals, greatly reducing the need for time-consuming, manual trial-and-error processes while still optimizing quality.

"Varian’s RapidPlan software is a very robust knowledge-based planning solution that was developed to help clinical teams enhance quality, consistency, and efficiency in radiotherapy treatment planning," said Kolleen Kennedy, president of Varian Oncology Systems. "We are gratified to see research being conducted and presented, affirming the value of this tool for enhancing access to quality cancer treatment around the world. With the publication in the Lancet, last month, of a special report on the need to expand global access to radiotherapy, Varian is pleased to be offering tools like RapidPlan designed to improve utilization of radiotherapy around the world." [4]

OPKO Health Expands Portfolio of Tests for Inherited Forms of Cancer

On October 22, 2015 OPKO Health, Inc. (NYSE:OPK) reported that its GeneDx business unit has expanded its inherited cancer panel offerings and genetic tests to include four additional genes (Press release, Opko Health, OCT 22, 2015, View Source [SID:1234507759]).

The four genes are POLD1, POLE, SCG5/GREM1, and SMARCA4. Three of the genes, POLD1, POLE and SCG5/GREM1, are associated with colon polyposis, colon cancer and endometrial cancer; while SMARCA4 is associated with an increased risk of a rare form of ovarian cancer. With the addition of these genes to the inherited cancer testing portfolio, it is possible to more effectively identify patients at risk. Once abnormalities are identified, patients can work with their healthcare providers to understand their specific cancer risk and what they can do to manage that risk.

"The addition of these four genes to the genetic tests now available from GeneDx can help in the diagnosis of rare syndromes associated with increased risk of colorectal, endometrial, ovarian and other cancers," said Marc. D. Grodman, M.D., CEO of BioReference Laboratories Inc., a subsidiary of OPKO.

The new gene panels and tests are available now for physicians to order from the GeneDx website. For more information, please visit www.genedx.com or email us at [email protected].

Roche delivers strong sales growth in the first nine months of 2015

On October 22, 2015 Roche reported strong sales growth in the first nine months of 2015 (Press release, Hoffmann-La Roche , OCT 22, 2015, View Source [SID:1234507757]).

Group sales increased 6% at constant exchange rates1, 2% in Swiss francs

Pharmaceuticals Division sales up 6%, driven by strong growth of HER2-positive breast cancer medicines, as well as Avastin and Esbriet

Diagnostics Division sales increased 6%, driven by Professional Diagnostics, Molecular Diagnostics and Tissue Diagnostics

Full-year outlook raised

Positive phase III data presented on ocrelizumab, the first medicine for relapsing and for primary progressive forms of multiple sclerosis (MS)

Launch of cobas EGFR Mutation Test v2, the first liquid biopsy PCR test from Roche

For the seventh year running, Roche ranked most sustainable healthcare company in the Dow Jones Sustainability Indices (DJSI)

Commenting on the Group’s first nine months, Roche CEO Severin Schwan said: "With sales continuing to grow strongly, we are raising our outlook for the full year. I am very pleased about the positive newsflow coming from our product pipeline. This includes data on our cancer immunotherapy medicine atezolizumab in bladder and lung cancer and, in particular, strong data on ocrelizumab in both relapsing and primary progressive forms of MS. These medicines have the potential to make a big difference to people living with these terrible diseases."

Group demonstrated continued strong growth

Sales of the Roche Group increased 6% to CHF 35.5 billion in the first nine months. Growth was driven by all regions in the Pharmaceuticals Division and Professional Diagnostics’ sales.

The Swiss franc strengthened considerably against the euro during the first nine months of 2015, whilst weakening against the US dollar. The Japanese yen continued to weaken against the Swiss franc, as did Latin American and most European currencies. Overall, there was a negative currency impact of four percentage points on sales growth.

The DJSI again recognised Roche as the most sustainable company in the healthcare industry for the seventh consecutive year. Roche scored highest in the management of environmental and social topics, and with more than 100 new business partnerships established last year, DJSI also noted Roche’s strong culture of collaboration, broad approach to innovation, and commitment to furthering patient access to healthcare.

HER2 franchise, Avastin and Esbriet driving growth in the Pharmaceuticals Division

In Pharmaceuticals, the oncology and immunology products drove the division’s sales for the first nine months. Sales of the HER2-positive breast cancer medicines, Herceptin, Perjeta and Kadcyla, grew 19%. The outlook for this franchise was further strengthened in July after the European Commission approved Perjeta combination therapy for use before surgery. Avastin (+9%) and MabThera/Rituxan (+5%) also recorded continued strong growth.

In immunology, Actemra/RoActemra (+22%), which is used mainly to treat rheumatoid arthritis, and Xolair (+25%), which is now used in the treatment of chronic hives as well as asthma, grew again significantly. Sales of anti-viral medicine Valcyte and oral chemotherapy Xeloda declined as these medicines are no longer patent protected. Sales of hepatitis medicine Pegasys and eye treatment Lucentis dropped as a result of increased competition.

Strong demand for Esbriet, a treatment for idiopathic pulmonary fibrosis (IPF), a fatal lung disease, continued throughout the third quarter and sales totalled CHF 386 million in the first nine months. In September, additional data were presented from a pooled analysis of three phase III studies that suggested a reduction in treatment-emergent risk of death for IPF patients on Esbriet for more than two years.2 In the same month, Esbriet received approval in Switzerland.

Recently, key milestones were achieved for Roche’s Cotellic (cobimetinib) for use in combination with Zelboraf in advanced melanoma. The company announced final phase III data showing a significant increase in overall survival, and in September, the Committee for Medicinal Products for Human Use (CHMP) recommended EU approval for Cotellic plus Zelboraf for the treatment of patients with BRAF V600 mutation-positive metastatic melanoma. This combination therapy was approved in Switzerland in August and a US FDA decision is anticipated by the end of the year.

Laboratory business main growth contributor in Diagnostics Division
Sales of the Diagnostics Division increased 6%, driven primarily by immunodiagnostic products from the Professional Diagnostics business area (+7%). The Molecular and Tissue Diagnostics business areas also performed well, with sales up 10% and 12%, respectively. Sales in Diabetes Care declined 3% due to ongoing challenging market conditions.

With four instruments and four test approvals and launches this year, the Diagnostics Division further broadened its industry leading product portfolio. Roche recently launched the cobas EGFR Mutation Test v2 that utilises plasma and/or tumour tissue for non-small cell lung cancer diagnosis and treatment monitoring.

Significant clinical data released at key medical meetings
At this year’s European Cancer Congress (ECC), results were presented from several clinical studies that are supporting regulatory discussions on alectinib, atezolizumab and Cotellic, plus data on earlier-stage cancer immunotherapies. Roche is studying more than 20 medicines in cancer immunotherapy, eight of which are in clinical trials.

At the congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), Roche presented positive phase III data for ocrelizumab in people with relapsing forms of MS and in primary progressive MS. Ocrelizumab is the first medicine to show a clinically meaningful impact on the progression of disability in people with primary progressive MS (PPMS) in a pivotal phase III trial. In this study, the proportion of patients with adverse events and serious adverse events was similar in the ocrelizumab and placebo groups. In two studies in people with the most common form of the disease, relapsing MS, ocrelizumab was superior to high-dose interferon beta-1a in reducing key markers of MS activity over the two-year controlled treatment period: annualised relapse rate, disability progression, and areas of MS-related inflammation and brain injury. The proportion of patients with adverse events and serious adverse events was similar in the ocrelizumab and interferon beta-1a groups. Roche will submit these data to global health authorities in 2016 to seek approval of ocrelizumab for relapsing forms of MS and PPMS.

In September, the US FDA granted Breakthrough Therapy Designation for ACE910 to prevent bleeding episodes in hemophilia A patients aged 12 and older. This is now the ninth Breakthrough Therapy Designation granted for a Roche medicine.

Raised outlook for 2015
Based on the strong performance recorded in the first nine months of 2015, Roche now expects sales growth in the mid-single digit range, at constant exchange rates. Core earnings per share are targeted to grow ahead of sales at constant exchange rates3. Roche expects to further increase its dividend in Swiss francs.