On January 8, 2015 Pfizer reported that The U.S. Food and Drug Administration (FDA) has informed Pfizer that at this time there is no plan for an Oncologic Drugs Advisory Committee meeting for IBRANCE (palbociclib) (Press release Pfizer, JAN 8, 2015, View Source [SID:1234501294]). Pfizer continues to have an open and productive dialogue with the FDA as the application for IBRANCE advances. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is April 13, 2015. The Company reports that it has entered label discussions with the FDA and hopes to be able to bring IBRANCE to patients who need it as soon as possible.
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CytRx Announces Positive Interim Phase 2 Aldoxorubicin Results as a Treatment for HIV-Related Kaposi’s Sarcoma (KS)
On January 8, 2015 CytRx Corporation reported positive interim results from its ongoing Phase 2 clinical trial evaluating the safety and efficacy of aldoxorubicin for the treatment of Kaposi’s Sarcoma (KS) in HIV-infected patients (Press release CytRx, JAN 8, 2015, View Source [SID:1234501287]). The clinical results reported are from 9 patients, 6 of which carried the worst KS prognosis. Also, 4 of the 9 patients had received from 1 to 8 prior liposomal doxorubicin (Doxil) cycles. Efficacy results revealed that all 9 patients (100%) exhibited a decrease in skin lesions and in the number of cancer cells expressing the KS virus DNA. Of the 6 patients with lung tumors, 4 patients (66%) demonstrated either a partial or complete response, with no patients (0%) demonstrating progression. Data thus far show that aldoxorubicin can be detected in all tumor biopsies 24 hours following drug administration. Preliminary safety results showed only 2 patients (22%) experienced a grade 4 adverse event (transient neutropenia and anemia). Other adverse events were mild and most were unrelated to aldoxorubicin.
“The current standard-of-care for severe dermatological and systemic KS is Doxil, however, many patients experience significant toxicity, or exhibit minimal to no clinical response to this agent,” said Steven Kriegsman, Chairman and CEO of CytRx. “Aldoxorubicin has demonstrated effectiveness against a range of tumors in the phase 2 clinical trial, thus we are optimistic in regard to its potential as a treatment for KS. This data provides important insight into both the safety and efficacy of aldoxorubicin in this indication, and the lack of toxicity of our drug in these patients is very impressive. We look forward to further data using aldoxorubicin in KS as this study continues.”
Assuming a positive outcome of the ongoing Phase 2 clinical trial, CytRx plans to discuss with the FDA a potential pathway for the registration of aldoxorubicin for use in KS. The Company intends to submit the Phase 2 KS data for presentation at the 2015 ASCO (Free ASCO Whitepaper) annual meeting.
This open-label Phase 2 clinical trial is expected to enroll up to 30 patients, randomly assigned to three equally sized treatment arms which will receive aldoxorubicin at 50, 100 or 150 mg/m2 by 30-minute intravenous infusion. Because the KS patients in the study have compromised immune systems, the aldoxorubicin dosages administered in the trial are lower than those administered in the Company’s clinical testing of aldoxorubicin in patients with soft tissue sarcomas. Patients with advanced KS receive aldoxorubicin on day 1, then every 3 weeks until evidence of tumor progression, unacceptable toxicity or withdrawal of consent. The primary objectives of preliminary efficacy include evaluation of the size, number and nodularity of skin lesions, change in size and number of lung lesions and changes in the number of tumor cells that express the KS virus DNA (Human Herpes Virus 8). The Company is also evaluating the level of aldoxorubicin uptake into lesions. Safety is being assessed through monitoring of adverse events and the ability to remain on assigned treatment. The trial is being conducted at the Louisiana State University Health Sciences Center in New Orleans, LA. Possible additional sites are being considered to expedite enrollment.
KS is an orphan indication in the U.S.
Five-Year Collaboration Focused on CART and Hematopoetic Stem Cell Applications across a Range of Therapeutic Areas
On January 7, 2015 Intellia Therapeutics, a leader in the development of therapeutic products using CRISPR/Cas9 technology for gene editing and repair, reported a five-year research and development collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells (CARTs) and hematopoetic stem cells (HSCs) (Press release, Intellia Therapeutics, JAN 7, 2015, View Source [SID1234533042]). This collaboration comes only three months after Intellia was launched by Atlas Venture and Caribou Biosciences, providing an important validation of Intellia’s team and capabilities.
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CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications, including blood disorders, cancer and other genetic based diseases. It has been shown to be an efficient and precise method for gene editing across multiple cell and tissue types, making it an ideal platform for ex vivo applications, such as CART- and HSC-based therapies, as well as in vivo applications.
Under the terms of the agreement, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CARTs. Within HSCs, Novartis and Intellia will jointly advance multiple programs, and have agreed to a process for assigning development and ownership rights, which will enable Intellia to develop its own proprietary internal HSC pipeline.
In addition to increasing its equity holding in Intellia, Novartis is making an upfront payment, and providing technology access fees and funding for R&D programs during the five-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline. Intellia also reserves the right to pursue additional enabling partnerships in other areas of therapeutic interest.
"Our collaboration with Novartis is an important building block for Intellia that will greatly accelerate our effort to translate the promise of CRISPR/Cas9 into meaningful advances for 2 patients," said Nessan Bermingham, Ph.D., Chief Executive Officer and co-founder of Intellia. "CARTs and HSCs represent two of the most immediate opportunities for CRISPR therapeutic development, and Novartis, as a leader in this space, is the ideal partner with which to develop strong product pipelines in these areas."
Nuvilex Announces Name Change to PharmaCyte Biotech
On January 7, 2015 Nuvilex, Inc. (OTCQB:NVLX) reported that the Company has changed its name to PharmaCyte Biotech, Inc. Shares in PharmaCyte Biotech will trade under the new ticker symbol "PHCB" on the OTCQB electronic platform (Press release, PharmaCyte Biotech, JAN 7, 2015, View Source [SID:1234510563]). The new symbol is expected to become effective at the open of the market on January 8, 2014. The name change is part of the Company’s transformation process to operate solely as a pure biotechnology firm leveraging its Cell-in-a-Box technology, a proprietary cell encapsulation platform being utilized to develop "targeted" treatments for solid cancerous tumors and insulin dependent diabetes.
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"Over the past year, we’ve implemented an aggressive strategy to facilitate the advancement of the treatments we are developing for cancer and diabetes, with our Cell-in-a-Box technology at the core of these treatments. Our new name reflects the tremendous progress we’ve accomplished in terms of clinical development and signifies the structural completion of our transition to becoming a fully dedicated biotechnology company," said Kenneth L. Waggoner, Chief Executive Officer of PharmaCyte Biotech.
Some of the highlights in 2014 that have marked this transition include:
Receiving "orphan drug" designation from the U.S. Food and Drug Administration (FDA) for the Company’s pancreatic cancer treatment, with applications filed with the EMA and the TGA for the same status in Europe and Australia.
Development of a clinical protocol for a planned Phase 2b clinical trial with the goal of initiating the clinical trial in 2015.
A preclinical study being completed evaluating the effectiveness of the Company’s pancreatic cancer treatment on the accumulation of malignant ascites fluid often associated with the growth of abdominal cancers with positive results that have led to a follow-up study about to be launched.
The establishment of a world-wide Diabetes Consortium with a number of research agreements now in place with major universities and institutions that will permit the development of a break-through treatment for insulin dependent diabetes that combines Cell-in-a-Box with insulin-producing cells.
Progression at the University of Northern Colorado in the pursuit of treatments for brain and other forms of difficult to treat cancers that will combine cannabinoid or cannabinoid-like compounds with the Cell-in-a-Box technology.
"With exciting developments on the horizon for 2015, our work to develop treatments for both cancer and diabetes will move forward under our new name because it speaks to what we actually do here at PharmaCyte Biotech," concluded Mr. Waggoner.
Exelixis and Swedish Orphan Biovitrum AB (Sobi) Extend and Restructure Distribution Agreement for COMETRIQ® for Medullary Thyroid Cancer
On January 7, 2015 Exelixis reported that it has extended and restructured its agreement with Swedish Orphan Biovitrum AB (Sobi) to support the distribution and commercialization of COMETRIQ (cabozantinib) for progressive, unresectable, locally advanced or metastatic medullary thyroid cancer (MTC) in the European Union (EU), Switzerland, Norway, Russia, and Turkey (Press release Exelixis, JAN 7, 2015, View Source;p=RssLanding&cat=news&id=2004606 [SID:1234501288]). The agreement, which was established in February 2013 and due to expire on December 31, 2015, will now extend to December 31, 2019. Moreover, the payment structure of the partnership will transition from fixed fees paid by Exelixis to Sobi to support initial build out of COMETRIQ European commercial infrastructure to a sales margin-based approach. Exelixis continues to maintain commercial rights for all other potential cabozantinib oncology indications on a global basis.
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"Our amended agreement with Sobi will continue to allow Exelixis to provide COMETRIQ to MTC patients outside the United States," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "Over the course of this coming year, we anticipate reaching several key milestones for the company, including receipt of top-line data from the METEOR phase 3 trial of cabozantinib in metastatic renal cell carcinoma in the second quarter, and regulatory progress with our partnered compound cobimetinib for metastatic melanoma in both the United States and European Union."
"We are very pleased to extend the agreement with Exelixis and proud to support access to COMETRIQ in Europe. The evolution of our partnership with Exelixis will continue to bring significant value to patients," said Anders Edvell, Vice President and Head of Sobi Partner Products at Sobi.
Sobi exclusively markets, sells, and distributes COMETRIQ for its MTC indication in the covered territory of the European Union, Switzerland, Norway, Russia, and Turkey. In parts of the territory where COMETRIQ is not approved, Sobi administers a Named Patient Use program. Exelixis is responsible for regulatory approvals in the covered territory, and the company retains the ability to terminate the agreement at will at any time upon payment of certain pre-determined fees.