On March 7, 2016 The European Medicines Agency (EMA) launched its new PRIME (PRIority MEdicines) scheme to strengthen support to medicines that target an unmet medical need (Press release, EMA, MAR 7, 2016, View Source [SID:1234509429]). The scheme focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options. These medicines are considered priority medicines within the European Union (EU). Schedule your 30 min Free 1stOncology Demo! Through PRIME, EMA offers early, proactive and enhanced support to medicine developers to optimise the generation of robust data on a medicine’s benefits and risks and enable accelerated assessment of medicine applications. This will help patients to benefit as early as possible from therapies that may significantly improve their quality of life.
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By engaging with medicine developers early, PRIME aims to strengthen clinical trial designs to facilitate the generation of high quality data for the evaluation of an application for marketing authorisation. Early dialogue and scientific advice also ensure that patients participate in trials that are likely to provide the necessary data for an application for marketing authorisation, and help to make best use of limited resources.
"The launch of PRIME is a major step forward for patients and their families that have long been hoping for earlier access to safe treatments for their unmet medical needs, such as rare cancers, Alzheimer’s disease and other dementias," says Vytenis Andriukaitis, EU Commissioner for Health and Food Safety. "Through enhanced scientific support this scheme could also help, for example, to accelerate the development and authorisation of new classes of antibiotics or their alternatives in an era of increasing antimicrobial resistance." The Commissioner also highlights that PRIME optimises the use of the current regulatory framework that can contribute to the European Commission’s priorities in terms of boosting innovation, jobs, growth and competitiveness.
"Our goal is to foster better planning of medicine development to help companies generate the high quality data we need to assess quality, safety and efficacy of medicines," explains Professor Guido Rasi, EMA’s Executive Director. "Patients with no or insufficient treatments could then benefit from scientific progress and cutting edge medicines as soon as possible."
PRIME builds on the existing regulatory framework and available tools such as scientific advice and accelerated assessment. This means that a PRIME medicine is expected to benefit from accelerated assessment at the time of an application for marketing authorisation.
"We want to ensure that breakthroughs in medicines reach patients quicker," says Dr Tomas Salmonson, Chair of the Committee for Medicinal Products for Human Use (CHMP). "By strengthening collaboration between the scientific committees, and by gaining and sharing knowledge on the medicine throughout the development, we will not only accelerate patients’ access but also ensure an efficient use of available resources."
To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on early clinical data. Once a candidate medicine has been selected for PRIME, the Agency:
appoints a rapporteur from EMA’s CHMP or from the Committee on Advanced Therapies (CAT) in the case of an advanced therapy, to provide continuous support and help to build knowledge ahead of a marketing authorisation application;
organises a kick-off meeting with the CHMP/CAT rapporteur and a multidisciplinary group of experts from relevant EMA scientific committees and working parties, and provides guidance on the overall development plan and regulatory strategy;
assigns a dedicated EMA contact point;
provides scientific advice at key development milestones, involving additional stakeholders such as health technology assessment bodies to facilitate patients’ quicker access to the new medicine;
confirms potential for accelerated assessment at the time of an application for marketing authorisation.
While PRIME is open to all companies on the basis of preliminary clinical evidence, micro-, small- and medium-sized enterprises (SMEs) and applicants from the academic sector can apply earlier on the basis of compelling non-clinical data and tolerability data from initial clinical trials. They may also request fee waivers for scientific advice. Since SMEs and academia often lack experience with the regulatory framework, they can benefit in particular from earlier scientific and regulatory advice.
Strengthened regulatory toolkit for medicines addressing unmet needs
EMA has released guidance documents on PRIME as well as a comprehensive overview of the EU early access regulatory tools, i.e. accelerated assessment, conditional marketing authorisation and compassionate use. Revised guidelines on the implementation of accelerated assessment and conditional marketing authorisation are also published today. All these tools are reserved for medicines addressing major public health needs. The revised guidelines provide more detailed information based on past experience. They encourage early dialogue between the various stakeholders which is crucial to optimise use of these tools. Although PRIME is specifically designed to promote accelerated assessment, it will also help to make best use of other EU early access tools and initiatives, which can be combined whenever a medicine fulfils the respective criteria.
PRIME was developed in consultation with the Agency’s scientific committees, the European Commission and its expert group on Safe and Timely Access to Medicines for PatientsExternal link icon (STAMP) as well as the European medicines regulatory network. This network of national competent authorities and its many experts who conduct the scientific evaluations is key to the success of the new scheme.
The main principles of PRIME were released for a two-month public consultation in 2015 and the comments received were taken into account in the final version.
Year: 2016
Argos Therapeutics Announces Financing of Up to $60 Million
On March 07, 2016 Argos Therapeutics, Inc. (Nasdaq:ARGS) ("Argos"), an immuno-oncology company focused on the development and commercialization of truly individualized immunotherapies for the treatment of cancer based on the Arcelis technology platform, reported that it has entered into a securities purchase agreement for the sale of up to $60 million of Argos common stock and warrants in a private placement financing (Press release, Argos Therapeutics, MAR 7, 2016, View Source [SID:1234509400]). Argos expects that this financing will fund operations into the second quarter of 2017, when the Company expects to have final data from its pivotal phase 3 ADAPT trial of AGS-003 (the "ADAPT Study").
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The financing will take place in up to three tranches. Under the securities purchase agreement, at the initial closing, which is expected to occur on or about March 9, 2016, Argos will sell and the investors will purchase, for a total purchase price of $19,882,915, a total of 3,652,430 shares of common stock and warrants to purchase a total of 2,739,323 shares of common stock (0.75 shares of common stock for each share of common stock purchased), based on a purchase price per share of common stock and accompanying warrant equal to $5.44375. At the second closing, Argos has agreed to sell and the investors have agreed to purchase, for an additional purchase price of $29,824,520, a total of 5,478,672 shares of common stock and warrants to purchase a total of 4,109,005 shares of common stock at the same price and on the same terms as the first tranche. The second closing is conditioned on the Independent Data Monitoring Committee (the "IDMC") for the ADAPT Study at or following the IDMC’s next regular meeting following the initial closing (currently scheduled for June 2016) (the "First IDMC Meeting") recommending that Argos continue the ADAPT Study or discontinue the ADAPT Study based on favorable efficacy data. The warrants to be issued in each closing will have an exercise price of $5.35 per share and expire five years from the date of issuance. Participants in the financing include Pharmstandard International S.A., Forargos B.V., Tianyi Lummy International Holdings Group Ltd., China BioPharma Capital I, L.P., TVM V Life Science Ventures GmbH & Co. KG and Wasatch Funds Trust.
Under the securities purchase agreement, Pharmstandard has also agreed that, at Argos’s option following the satisfaction of certain conditions, including the IDMC having made a recommendation at or following its next regular meeting after the First IDMC Meeting (currently anticipated to be held in November or December 2016), that the Company continue the ADAPT Study or discontinue the ADAPT Study based on favorable efficacy data, and the Company’s cash position at such time, it shall purchase at the third closing up to $10,292,563 of shares of common stock (without warrants) at a price per share to be determined pursuant to an agreed upon formulation. The dollar amount committed to be purchased by Pharmstandard at the third closing is subject to reduction on a dollar-for-dollar basis for certain cash amounts raised by Argos after the initial closing through equity or debt financings or collaborations. All three closings will be subject to the satisfaction of certain customary closing conditions.
Argos expects that the proceeds from the initial closing will enable it to fund the company’s ongoing expenses into the third quarter of 2016, and that the proceeds from all three closings, if such closings occur, will enable it to fund the company’s ongoing expenses into the second quarter of 2017 when it expects final data from its pivotal phase 3 ADAPT trial of AGS-003.
The securities sold in this private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws, and accordingly may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Argos has agreed to file registration statements with the Securities and Exchange Commission ("SEC") registering the resale of the shares of common stock issued in this private placement and the shares of common stock issuable upon the exercise of the warrants issued in the private placement.
This release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.
Oxford BioMedica Announces Expanded Collaboration and Licence with Immune Design
On March 7, 2016 Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE: OXB), a leading gene and cell therapy group, reported a new and expanded collaboration with Immune Design Corp. ("Immune Design"), a clinical-stage immunotherapy company focused in oncology, as well as a non-exclusive, royalty-bearing, intellectual property licence to Immune Design (Press release, Oxford BioMedica, MAR 7, 2016, View Source [SID:1234509399]). Schedule your 30 min Free 1stOncology Demo! The licence involves the use of lentiviral vector-based products for the in vivo treatment or prevention of cancer. The collaboration builds on Immune Design’s earlier process development collaboration with the Group, signed in 2012, which covered the development of analytic assays. Financial and other terms have not been disclosed.
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John Dawson, Chief Executive Officer of Oxford BioMedica, commented: "We are delighted to sign an IP licence and an expanded collaboration agreement with Immune Design. Oxford BioMedica has world-leading capabilities in lentiviral vector process development and manufacture and these agreements with Immune Design are further evidence of our attractiveness as a partner to other companies and academic institutions working with lentiviral vector based products. I expect that during 2016 we will be able to make further announcements of similar agreements with other parties"
Celgene Notified of ANDA Filing for ABRAXANE®
On March 7, 2016 Celgene Corporation (NASDAQ: CELG, CELGZ) reported that it has received a Paragraph IV Notice Letter advising that Actavis LLC submitted an Abbreviated New Drug Application (ANDA) to the U.S. Food and Drug Administration (FDA) seeking authorization from the FDA to manufacture and market a generic version of ABRAXANE (paclitaxel protein-bound particles for injectable suspension) (albumin bound) 100 mg/vial in the United States (Press release, Celgene, MAR 7, 2016, View Source [SID:1234509390]). Schedule your 30 min Free 1stOncology Demo! The Notice Letter contains Paragraph IV certifications against certain patents relating to ABRAXANE. Celgene is assessing the notice. Celgene intends to vigorously defend its extensive intellectual property rights relating to ABRAXANE.
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About ABRAXANE
In the U.S., ABRAXANE for Injectable Suspension (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) is indicated for the treatment of breast cancer after failure of combination chemotherapy for metastatic disease or relapse within six months of adjuvant chemotherapy. Prior therapy should have included an anthracycline unless clinically contraindicated. ABRAXANE is indicated for the first-line treatment of locally advanced or metastatic non-small cell lung cancer, in combination with carboplatin, in patients who are not candidates for curative surgery or radiation therapy. ABRAXANE is also indicated for the first-line treatment of metastatic adenocarcinoma of the pancreas in combination with gemcitabine.
Molecular Partners Reveals Programs in Immuno-Oncology
On March 07, 2016 Molecular Partners AG (ticker: MOLN), a clinical-stage biopharmaceutical company that is developing a powerful new class of therapies known as DARPins, reported additional details about its immuno-oncology pipeline, including two early-stage multi-DARPin programs (Press release, Molecular Partners, MAR 7, 2016, View Source [SID:1234509389]). The first program targets the validated immune checkpoint PD-1 as well as VEGF-A, aiming to enhance PD-1 efficacy. The second program is designed to potently activate T-cells in the tumor without activating circulating T-cells, thus circumventing systemic toxicities. Schedule your 30 min Free 1stOncology Demo! Christian Zahnd, Chief Executive Officer of Molecular Partners, will present additional details about these discovery programs at the 36th Annual Cowen Health Care Conference in Boston, Mass. on Wed., March 9 at 10:40 a.m. Eastern Time.
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"The DARPin platform is ideally suited to develop highly differentiated immuno-oncology therapies with the potential to overcome the limitations of first-generation approaches," said Dr. Michael Stumpp, Chief Scientific Officer at Molecular Partners. "We are excited to contribute to the rapidly evolving field of immuno-oncology as we build our proprietary oncology pipeline. Our early-stage programs evaluate exciting new principles that were previously out of reach: enhanced immune checkpoint blockers and locally activated agonists."
Immuno-oncology is a revolutionary approach to anti-cancer treatment that redirects the body’s immune system to fight cancer cells. Investigators are studying many different ways to modulate immune checkpoints used by the body to regulate the immune system. While many of these clinical studies are yielding promising results, novel approaches are needed.