Month: March 2016

8-K – Current report

On March 7, 2016 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the fourth quarter and year ended December 31, 2015 and provided recent company developments along with a business outlook for 2016 (Filing, 8-K, Manhattan Pharmaceuticals, MAR 7, 2016, View Source [SID:1234509393]).

Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "2015 was a transformational year for our Company as we launched our first registration study, the GENUINE Phase 3 study, and also obtained an SPA for our proprietary combination of TG-1101 and TGR-1202, the ‘1303’ regimen, enabling our UNITY-CLL trial for patients with front-line and previously treated CLL. During 2016 we will be focused on executing our ongoing Phase 3 clinical programs as well as expanding our ‘1303’ regimen into registration-directed trials for both diffuse large b-cell lymphoma and indolent lymphomas." Mr. Weiss continued, "We are also very excited to begin exploring the potential of our products in autoimmune disorders. Our IND has been cleared by the FDA Division of Neurology and we plan to launch our first Phase 2 trial in multiple sclerosis (MS) for TG-1101 in the next 30-60 days. We believe this Phase 2 study could support the commencement of a Phase 3 program next year. From a financial perspective, with more than $100 million in cash and investments we remain well positioned to execute on our business plan."

2015 Highlights

· Expanded our product portfolio through a global collaboration with Checkpoint Therapeutics to develop and commercialize anti-PDL1 and anti-GITR antibody research programs from Dana Farber Cancer Institute in hematologic malignancies
· Commenced enrollment into the GENUINE Phase 3 clinical trial, which is now open in over 150 sites throughout the US
· Presented the first data from the triple combination study of TG-1101 + TGR-1202 + ibrutinib showing not only that the combination was well tolerated, but produced a 100% ORR in patients with high-risk CLL/SLL and a 75% ORR in indolent NHL, which includes Follicular Lymphoma and Marginal Zone Lymphoma
· Obtained an SPA for the UNITY-CLL Phase 3 clinical trial of the Company’s proprietary combination of TG-1101 + TGR-1202 (aka "TG-1303")
· Launched a new Phase 1/2 triple therapy study of TG-1101 + TGR-1202 + the PD-1 checkpoint inhibitor pembrolizumab, the first clinical trial to evaluate the safety and efficacy of the triple combination of a PI3K delta inhibitor with an anti-CD20 mAb and an anti-PD-1 checkpoint inhibitor
· Announced key data updates at the major medical conferences throughout 2015

Key Objectives for 2016

· Aggressively recruit into the GENUINE Phase 3 clinical trial
· Aggressively enroll into the UNITY-CLL Phase 3 clinical trial
· Commence the UNITY- DLBCL Phase 2b clinical trial
· Initiate a Phase 2 clinical trial in Multiple Sclerosis (MS)
· Commence a registration trial for iNHL
· Present updated data on the Phase 1 and 2 clinical trials at major hematology/oncology conferences during 2016

Financial Results for the Fourth Quarter and Full Year 2015

At December 31, 2015, the Company had cash, cash equivalents, investment securities, and interest receivable of $102.4 million, as compared to $78.9 million at December 31, 2014.

Our consolidated net loss for the year ended December 31, 2015, excluding non-cash items, was approximately $47.3 million, including other research and development expenses of $43.4 million, of which approximately $23.4 million related to manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the year ended December 31, 2015, inclusive of non-cash items, was $63.0 million, or $1.38 per diluted share, compared to a consolidated net loss of $55.8 million for the year ended December 31, 2014. The increase in consolidated net loss during the year ended December 31, 2015 was primarily driven by increases in other research and development expenses for both TG-1101 and TGR-1202 as a result of manufacturing and clinical trial expenses related to ongoing and planned future Phase 3 registration programs as well as launch preparation activities. The increase in other research and development expenses was partially offset by expenses recorded during 2014 in conjunction with the Company’s licensing agreements for TGR-1202 and the IRAK4 inhibitors program, and a decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2014.

Our consolidated net loss for the fourth quarter ended December 31, 2015, excluding non-cash items, was approximately $14.8 million, including other research and development expenses of $13.7 million, of which approximately $7.4 million related to manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the fourth quarter ended December 31, 2015, inclusive of non-cash items, was $17.6 million, or $0.37 per diluted share, compared to a consolidated net loss of $18.8 million during the comparable quarter in 2014. The decrease in consolidated net loss during the fourth quarter ended December 31, 2015 was the result of a decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2014, partially offset by a modest increase in other research and development expenses for TGR-1202 as a result of clinical trial expenses related to ongoing and planned future Phase 3 registration programs.

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Launch of PRIME – Paving the way for promising medicines for patients

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).

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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.

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.

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]).

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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.

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]).

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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.

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.