Exemplar Genetics to Present on Novel Research Models for Oncology at the World Preclinical Congress

On June 15, 2016 Exemplar Genetics reported its President, John Swart, Ph.D., will present today at the 15th Annual World Preclinical Congress during the Preclinical Models in Oncology session (Press release, Exemplar Genetics, JUN 15, 2016, View Source [SID:1234513374]). Dr. Swart’s presentation titled ‘Genetically Engineered Miniswine Models of Cancer’ will provide an overview of Exemplar’s novel ExeGen TP53R167H and ExeGen KRASG12D/+/TP53R167H/+ miniswine research models developed for oncology to help bridge the pre-clinical gap from small animal models to humans.

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!

Exemplar Genetics Logo
Dr. Swart commented, "We believe the development of these new genetically engineered miniature swine models will resolve inadequacies with traditional rodent models of cancer, which while valuable in certain aspects of research, have been inefficient at translating to human clinical trials."

As described in the Journal of Clinical Investigation publication, "Development and translational imaging of a TP53 porcine tumorigenesis model", current cancer research models are informative yet fail to accurately recapitulate human disease, and pre-clinical results utilizing these models often have poor translational value to the clinic. In particular, the lack of a large-animal model that accurately replicates human cancer pathologies has been a significant barrier to the development of effective diagnostics, as well as surgical and therapeutic interventions.

Exemplar’s genetically engineered ExeGen TP53R167H model expresses a mutation in the gene that encodes p53 (TP53), which is orthologous to one commonly found in humans. It is estimated p53 function is compromised in the vast majority of human tumors, through either TP53 gene mutation or alterations targeting the numerous regulators of p53 signaling. Studies with the TP53R167H model demonstrated tumor formation and characteristic chromosomal instability similar to what is seen in humans with mutant p53 alleles. Additionally, Exemplar has developed the ExeGen KRASG12D/+/TP53R167H/+ model which contains a conditional KRAS mutation on the background of TP53-targeted pigs that should allow for the inducement of human-like tumors in a tissue specific manner.

In April 2016, the U.S. Food & Drug Administration (FDA) exercised enforcement discretion in regard to Exemplar’s ExeGen low-density lipoprotein receptor (LDLR) miniswine clearing it for commercial use as a research model. As the first genetically engineered miniswine model reviewed and cleared by the FDA, this powerful investigational platform is available to researchers and drug developers helping forge a more reliable, consistent path from pre-clinical testing through human studies. Exemplar continues to work closely with the FDA to make each of the miniswine research models in its broad, extensive pipeline for use in the evaluation of several human health conditions including rare diseases, cancer, cystic fibrosis, neuromuscular/neurodegenerative disorders, and cardiovascular disease, available to researchers working on solutions for these devastating diseases.

BTG completes the acquisition of Galil Medical

On June 15, 2016 BTG plc (LSE: BTG), the specialist healthcare company, reported that it has completed the acquisition of Galil Medical, a leading provider of cryoablation products for the treatment of kidney and other cancers (Press release, BTG, JUN 15, 2016, View Source [SID:1234513373]).

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!

BTG announced on 6 May 2016 that it had entered into an agreement to acquire Galil.

Aegerion Pharmaceuticals and QLT Inc. Agree to Strategic Merger

On June 15, 2016 Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) ("Aegerion") and QLT Inc. (NASDAQ:QLTI) (TSX:QLT) ("QLT") reported that they have entered into a definitive merger agreement under which Aegerion will be merged with a wholly owned indirect subsidiary of QLT (Press release, QLT, JUN 15, 2016, View Source;p=RssLanding&cat=news&id=2177689 [SID:1234513357]). Upon completion of the proposed merger, each outstanding share of Aegerion common stock will be exchanged for 1.0256 shares of QLT common stock. QLT plans to change its name upon the closing of the proposed transaction to Novelion Therapeutics Inc. ("Novelion") and its common shares will trade on the NASDAQ Global Select Market and the Toronto Stock Exchange.

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!

A broad-based investor syndicate comprised of both new investors and existing shareholders of both companies (collectively, the "Investors") has committed to invest approximately $22 million in QLT and to vote in favor of the proposed transaction. This investment would be funded immediately prior to the transaction close and is expected to provide Novelion with additional capital to support future operations and the potential opportunity for targeted business development initiatives. Assuming completion of the proposed merger by the end of the third quarter of 2016, Novelion is expected to have an unrestricted cash balance of over $100 million.

The proposed transaction, which has been approved by the Boards of Directors of both companies, is expected to close late in the third quarter or during the fourth quarter of 2016, subject to approval by shareholders representing a majority of the outstanding common stock of each of QLT and Aegerion as well as other closing conditions.

Strategic Merger Designed to Transform Both Companies and Create Significant Value for Shareholders

The proposed merger is expected to create a strong, rare disease-focused global biopharmaceutical company with a diversified portfolio consisting of Aegerion’s two commercially branded products, Juxtapid (lomitapide) capsules and Myalept (metreleptin), and QLT’s QLT091001 ("Zuretinol Acetate" or "Zuretinol"), a Phase 3-ready Ultra-Orphan Fast Track and Orphan Drug designated asset being developed for the treatment of Inherited Retinal Disease caused by underlying mutations in RPE65 or LRAT genes ("IRD"), which indication comprises Leber Congenital Amaurosis ("LCA") and Retinitis Pigmentosa ("RP").

Aegerion’s Chief Executive Officer, Mary Szela, who will serve as Chief Executive Officer of Novelion following the close of the transaction, said: "We believe QLT’s clinical development team, and meaningful cash position, and Aegerion’s commercialization expertise will help unlock significant value in QLT’s Zuretinol asset and enable Novelion to pursue important milestones across a commercial and late-stage portfolio, including potential regulatory approval in Japan for Juxtapid and potential regulatory filings in Europe for Myalept. I believe that this proposed merger represents a fresh start and an opportunity to create significant value, and I look forward to driving our programs forward."

Dr. Geoffrey F. Cox, Ph.D., QLT’s Interim Chief Executive Officer, said: "Given the resource requirements for QLT to build out a global commercial infrastructure designed to most effectively maximize the value of our promising ultra-orphan Zuretinol asset, the QLT board determined that it would be advantageous to join forces with a strategic partner possessing the relevant orphan product infrastructure. We believe Aegerion and QLT are ideal complements, and we are confident that this transaction achieves virtually all of the principal goals and objectives of our strategic review process. In addition, QLT is expected to benefit from greater scale and diversification, as well as from a more liquid stock."

QLT’s Promising Ultra-Orphan Pipeline Product (Zuretinol acetate)

QLT is developing a synthetic retinoid product candidate (Zuretinol acetate) for the potential treatment of Inherited Retinal Disease caused by underlying mutations in RPE65 or LRAT genes (IRD) that prevent adequate functioning of the retinoid cycle, which indication comprises LCA and RP. LCA and RP are two forms of severe IRD resulting in progressive vision loss starting in childhood and leading to inevitable blindness.

There are currently no U.S. Food and Drug Administration ("FDA") or European Medicines Agency ("EMA") approved pharmacologic treatments for either LCA or RP, underscoring the significant unmet medical need. Zuretinol acetate is expected to advance to Phase 3 clinical trials in the third quarter of 2016 and has Orphan Drug designation from both FDA and EMA, as well as FDA Fast Track designation. As an oral product, Zuretinol acetate is being studied for its potential to treat the retinas of both eyes simultaneously. If approved, QLT believes it is possible to achieve first-line positioning in the treatment of IRD.

Private Placement

In combination with the proposed merger transaction, a broad-based investor syndicate will subscribe to purchase approximately $22 million in shares of QLT common stock for a purchase price of $1.76 per share is subject to the satisfaction or waiver of the conditions to closing the merger. These commitments would be funded immediately prior to merger closing to provide Novelion additional capital to support future operations and the potential opportunity for targeted business development initiatives.

The investor syndicate includes new investors, including Deerfield, and a broad group of existing Aegerion and QLT shareholders, including Armistice Capital, Broadfin Capital, Healthcare Value Capital, JW Asset Management, K2 & Associates Investment Management, Sarissa Capital, Tiger Legatus Capital Management, and others.

The subscription by the Investors to purchase shares of QLT common stock is subject to the satisfaction or waiver of the conditions to closing the merger. Each of the Investors has also agreed to vote its shares in QLT and Aegerion in favor of the transaction.

Corporate Governance

Following the close of the transaction, Novelion is expected to have its principal headquarters in Vancouver, British Columbia, where QLT is currently located, with business operations in Cambridge, Massachusetts.

The Board of Directors of Novelion will be comprised of ten members, including four QLT designees, four Aegerion designees and two shareholder representatives, one from Broadfin Capital and the other from Sarissa Capital. For a period of time that expires shortly after Novelion’s 2017 annual shareholder meeting, Sarissa Capital also has the right to designate an additional director to the Novelion Board.

Transaction Details

Under the terms of the merger agreement, Aegerion will become a wholly-owned indirect subsidiary of QLT, and each existing share of Aegerion common stock will be converted into the right to receive 1.0256 common shares of Novelion. As a result of the structure of this transaction, a repayment obligation with respect to Aegerion’s outstanding convertible notes will not be triggered.

The exchange ratio for the transaction is subject to certain adjustments if Aegerion’s previously disclosed securities class action litigation and Department of Justice ("DOJ") and Securities and Exchange Commission ("SEC") investigations are resolved prior to closing for amounts in excess of negotiated thresholds. In the event the class action litigation or DOJ and SEC investigations are not settled prior to closing, and in order to mitigate the risk of certain losses from these outstanding matters after transaction close, QLT will enter into a warrant agreement pursuant to which warrants will be issued to QLT shareholders and the Investors that would be exercisable for additional Novelion shares if the class action litigation or DOJ and SEC investigations are subsequently resolved for amounts in excess of negotiated thresholds.

Following completion of the proposed merger, QLT shareholders, including the Investors, who are investing in QLT immediately prior to closing, are expected to own approximately 67% and current Aegerion shareholders will own approximately 33% of Novelion’s common shares.

Concurrent with signing, Aegerion and QLT have entered into a loan agreement under which QLT has agreed to loan Aegerion up to $15 million for working capital. Aegerion will borrow $3 million in connection with execution of the Merger Agreement and may borrow up to $3 million per month in subsequent months, subject to certain conditions, if and to the extent such amounts are necessary in order for Aegerion to maintain an unrestricted cash balance of $25 million.

In addition to shareholder approval, the merger is subject to stock exchange approvals and other closing conditions, including, among others, regulatory approval and completion of a specified minimum of the private placement in QLT by the Investors.

Greenhill & Co., LLC is acting as the exclusive financial advisor to QLT and Weil, Gotshal & Manges LLP is serving as QLT’s legal counsel. J.P. Morgan is acting as the exclusive financial advisor to Aegerion and Ropes & Gray LLP is serving as Aegerion’s legal counsel.

Delcath Announces Acceptance Of Abstract For Presentation At 6th European Post-Chicago Melanoma/Skin Cancer Meeting

On June 15, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported that data from a large single hospital experience conducted at Southampton University Hospital in the United Kingdom has been accepted for oral presentation at the 6th European Post-Chicago Melanoma/Skin Cancer Meeting to be held in Munich, Germany at the Leonardo Royal Hotel from June 30 – July 1, 2016 (Press release, Delcath Systems, JUN 15, 2016, View Source;p=RssLanding&cat=news&id=2177653 [SID:1234513355]).

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!

The abstract authored by I. Karydis, et al. is titled, "Chemosaturation via percutaneous hepatic perfusion – an update on a single centre experience of treating metastatic uveal melanoma." Additional information about the Post-Chicago scientific program can be found here and the complete abstract will be available for download approximately 10 days prior to the congress.

Rgenix Announces $33 Million Series B Financing to Develop First-in-Class Cancer Therapeutics

On June 15, 2016 Rgenix, a cancer therapeutics company developing first-in-class drugs targeting novel cancer pathways, reported a $33 million Series B financing led by Novo A/S and Sofinnova Partners, with participation from existing investors including Partnership Fund for New York City, Alexandria Venture Investments, and Conegliano Ventures LP (Press release, Rgenix, JUN 15, 2016, View Source [SID1234523088]). The financing will support clinical development of Rgenix’s lead drug candidates, RGX-104 and RGX-202, as well as further development of its therapeutics pipeline.

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!

"We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics"

Tweet this
"We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics," said Masoud Tavazoie, M.D. Ph.D., Chief Executive Officer and co-founder of Rgenix. "This financing validates the potential of our lead immunotherapy RGX-104, which will be entering clinical trials this fall, and also demonstrates the strength of our discovery platform, developed in the laboratory of Rgenix co-founder Dr. Sohail Tavazoie at The Rockefeller University. The funding will enable our team to deliver an innovative therapy to cancer patients while simultaneously pushing forward our pipeline of other novel drug candidates."

Antoine Papiernik, Managing Partner at Sofinnova Partners, commented: "We are very excited to back such a high quality team at Rgenix. We also believe that RGX-104 could revolutionize treatment to cancer patients that today lack effective therapies."

Despite recent advances in cancer therapy, most patients will eventually succumb to their disease due to drug resistance and immune evasion. RGX-104 is a small molecule that reverses immune evasion and drug resistance by targeting immunosuppressive cells in the tumor microenvironment via a novel pathway, resulting in strong anti-tumor activity in several drug-resistant cancer types in pre-clinical models, both as a single agent and in combination with approved immunotherapies such as PD-1 inhibitors. The target of RGX-104 was discovered using Rgenix’s miRNA platform that has yielded several new cancer targets across multiple prevalent cancer types.

"We are excited to support Rgenix’s novel approach to treating cancers of high unmet need," said Nilesh Kumar, Senior Principal of Novo Ventures*. "Rgenix has an exciting platform founded on strong science from The Rockefeller University; the lead program is a first in class opportunity addressing a key mechanism in tumor immunosuppression in various cancer types."

In connection with the financing, Nilesh Kumar of Novo Ventures and Antoine Papiernik of Sofinnova Partners will join the Rgenix Board of Directors together with existing members, including Executive Chairman Eric Rowinsky, M.D., Masoud Tavazoie, M.D. Ph.D., Nancy Chang, Ph.D., and Saeed Tavazoie, Ph.D.