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Nicox First Quarter 2019 Business Update and Financial Highlights

On April 18, 2019 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported Q1 2019 operational highlights, revenue and cash position for Nicox and its subsidiaries (the "Nicox Group"), as well as key upcoming milestones (Press release, NicOx, APR 18, 2019, View Source [SID1234535194]).

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Michele Garufi, Chairman and Chief Executive Officer of Nicox, said, "With two exciting programs in advanced clinical development and two products approved in the U.S. we are continuing apace to fully leverage our scientific, clinical, and commercial assets. Enrollment has now reached over 85% in the NCX 470 glaucoma clinical trial, and both this and the NCX 4251 blepharitis trial should generate topline results in Q4 of this year. We expect our recurrent revenues to ramp up as Bausch + Lomb progresses the international rollout of VYZULTA, and with the launch by Eyevance of ZERVIATE in the U.S. In addition, our ongoing discussions for ZERVIATE outside of the U.S. could result in agreements in the near future with further upfront, milestone and royalty payments."

Key Upcoming Milestones
NCX 470 Phase 2 results: Top-line data from the NCX 470 Phase 2 clinical trial for the lowering of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension expected in Q4 of this year.
NCX 4251 Phase 2 results: Phase 2 clinical trial in patients with acute exacerbations of blepharitis ongoing with top-line data expected in Q4 of this year.

ZERVIATE U.S. launch: Commercial launch of ZERVIATE (cetirizine ophthalmic solution), 0.24% in the U.S. planned by our partner Eyevance Pharmaceuticals for summer of this year. Nicox eligible for up to $3 million of a potential future milestone payment from Eyevance related to certain regulatory and near-term manufacturing objectives, which are expected to be received prior to the U.S. launch.

ZERVIATE ex-US partnering: Multiple discussions ongoing for potential new licensing agreements in significant markets.
Nicox’s ophthalmology programs to be presented at key scientific conferences including the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
First Quarter 2019 and Recent Operational Highlights
The total number of prescriptions for VYZULTA in the U.S. in the first quarter of 2019 increased by 18% compared to Q4 20181 and by 360% compared to the first quarter 20181.
On March 19, 2019, we announced the initiation of a Phase 2 clinical trial evaluating NCX 4251, our novel patented ophthalmic suspension of fluticasone propionate nanocrystals, being developed as the first targeted topical treatment of the eyelid margin for patients with acute exacerbations of blepharitis. We expect to report top-line results from this Phase 2 trial in the fourth quarter of 2019.
On March 15, 2019, we announced that we had we entered into an exclusive license agreement with Ocumension Therapeutics for the development and commercialization of Nicox’s product ZERVIATE (cetirizine ophthalmic solution), 0.24% for the treatment of allergic conjunctivitis for the Chinese market.
On January 25, 2019, we announced that we had entered into a bond financing for up to €20 million from Kreos Capital. The financing is structured as three tranches of which only the first tranche of €8 million has been drawn down. The exercise of the two other tranches is at Nicox’s sole discretion.
On January 8, 2019 we announced that we had reached the 50% patient enrollment threshold of our multicenter, U.S. Phase 2 clinical trial evaluating our lead product candidate, NCX 470, ahead of schedule. NCX 470 is a novel, second-generation nitric oxide (NO)-donating prostaglandin analog for the lowering of IOP in patients with open-angle glaucoma or ocular hypertension that has demonstrated 2 to 3 mmHg superior IOP lowering vs. the U.S. market leader LUMIGAN in head-to-head preclinical evaluations.
Also in January, our global partner, Bausch + Lomb, a leading global eye health company and wholly owned subsidiary of Bausch Health Companies, Inc., received approval in Canada of VYZULTA (latanoprostene bunod ophthalmic solution), 0.024%. VYZULTA is indicated for the lowering of IOP in patients with open-angle glaucoma or ocular hypertension.
First Quarter 2019 Financial Highlights
As of March 31, 2019, the Nicox Group had cash and cash equivalents of €23.5 million as compared with €22.0 million at December 30, 2018. Net revenue2 for the first quarter of 2019 was €0.430 million versus €0.075 million in the first quarter of 2018.

Moleculin Receives FDA Approval of Fast Track Designation for Annamycin

On April 18, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported that the U.S. Food and Drug Administration ("FDA") has approved its request for Fast Track Designation for its drug, Annamycin, for the treatment of relapsed or refractory acute myeloid leukemia ("AML") (Press release, Moleculin, APR 18, 2019, View Source [SID1234535192]).

"We are thrilled that Annamycin has been granted Fast Track Designation," commented Walter Klemp, Moleculin’s Chairman and CEO. "Not only does this make us eligible for accelerated approval and priority review, but it serves as an important validation of the significant unmet need we are trying to address. Currently, Annamycin is in separate Phase I/II trials in the U.S. and Europe for the treatment of AML and the Company has recently announced positive interim top line data."

A drug that receives Fast Track designation is eligible for some or all of the following:

More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

Actinium Pharmaceuticals, Inc. Announces Pricing of $16.5 Million Public Offering of Common Stock and Warrants

On April 18, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported the pricing of an underwritten public offering of 42,860,000 shares of its common stock at a combined public offering price of $0.385 per share of common stock (Press release, Actinium Pharmaceuticals, APR 18, 2019, View Source [SID1234535191]). The Company also announced the pricing of warrants to purchase up to 42,860,000 shares of common stock at an exercise price of $0.50 per share and with a term of 5 years commencing on the date of issuance. The offering is expected to close on or about April 23, 2019, subject to the satisfaction of customary closing conditions. The gross proceeds to Actinium from this offering are expected to be $16.5 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Actinium. Actinium intends to use the net proceeds from the sale of the common stock and warrants to fund its ongoing pivotal, Phase 3 SIERRA trial for its lead product candidate Iomab-B and progress Phase 1 trials from its refocused CD33 program to the proof of concept stage. Actinium will also use the proceeds to support its antibody warhead enabling technology platform, including its Iomab-ACT program, research and development and general working capital needs.

William Blair & Company, L.L.C. is acting as sole bookrunner for the offering.

The shares and warrants are being offered pursuant to an effective shelf registration statement (including a prospectus) on Form S-3 (File No. 333-216748) filed with the U.S. Securities Exchange Commission (the "SEC"). A preliminary prospectus supplement, dated April 17, 2019 and accompanying prospectus, dated October 24, 2017, relating to the offering have been filed with the SEC. To obtain a copy of the preliminary prospectus supplement, dated April 17, 2019, and the final prospectus supplement (when available) for this offering, please contact William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, or by calling (800) 621-0687, or emailing [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities, in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction.

Acorda First Quarter 2019 Update: Webcast/Conference Call Scheduled for May 2, 2019

On April 18, 2019 Acorda Therapeutics, Inc. (NASDAQ: ACOR) reported that it will host a conference call and webcast to report its first quarter 2019 update and financial results on Thursday, May 2 at 8:30 a.m. ET (Press release, Acorda Therapeutics, APR 18, 2019, View Source [SID1234535190]).

To participate in the conference call, please dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 4783943. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on May 2, 2019 until 11:59 p.m. ET on June 2, 2019. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 4783943. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

Inovio Publishes Cancer Killing Data of Its Transformative DNA-encoded Bi-specific T Cell Engagers (dBiTEs™) in a Peer-reviewed Journal

On April 18, 2019 Inovio Pharmaceuticals, Inc. (NASDAQ: INO) reported the company’s novel DNA-encoded Bi-specific T Cell Engagers (dBiTEs) generated potent anti-tumor activities and cleared established tumors in preclinical studies (Press release, Inovio, APR 18, 2019, View Source [SID1234535189]). Inovio’s dBiTE results were published in a JCI Insight article entitled, "DNA-encoded bi-specific T cell engagers and antibodies present long-term antitumor activity," by Inovio and its collaborators at The Wistar Institute. JCI Insight is a peer-reviewed journal published by the American Society for Clinical Investigation dedicated to well-executed preclinical and clinical research studies.

For this study, Inovio developed a novel dBiTE targeting the HER2 molecule which was tested in therapeutic models for the treatment of ovarian and breast cancers. Importantly, just a single dose of Inovio’s HER2 dBiTE resulted in high levels of corresponding BiTE in mice for up to four months, exceeding what is typically displayed with the currently approved BiTE’s short half-life of only a few hours. The HER2 dBiTE treatment effectively killed HER2-expressing tumor cells resulting in a near-complete tumor clearance.

Dr. J. Joseph Kim, Inovio’s President and CEO, said, "Inovio’s dBiTEs represent game-changing advancements in immuno-oncology. Leveraging Inovio’s in vivo synthetic nucleic expression platform, we have shown that just one dose of Inovio’s dBiTE could generate corresponding BiTEs at high levels for several months, demonstrating a dramatic advantage over conventional BiTEs. Our dBiTE technology could be utilized to overcome the greatest shortcomings of traditional BiTEs, particularly its incredibly short half-life. Based on these promising preclinical results, we plan to rapidly advance our first dBiTE candidate into clinical testing, as well as develop more cancer tumor-targeting dBiTEs as partnering candidates."

BiTEs are a class of artificial bi-specific monoclonal antibodies that has the potential to transform the immunotherapy landscape for cancer. They direct a host’s immune system, more specifically the T cells’ cytotoxic activity, against cancer cells. In layman’s terms, BiTEs are like a double-sided tape that binds to a tumor and to a cancer-killing T cell. One domain of the BiTE binds to the targeted tumor (like HER2 or CD19 expressing cells) while the other engages the immune system by binding directly to CD3 molecules on T cells. This double-binding activity drives T cell activation directly at the tumor resulting in a killing function and tumor destruction.

The biggest drawback of conventional protein-based BiTEs is their short half-life. The BiTEs have a half-life of only two hours, which requires patients to undergo continuous intravenous infusion for several weeks to maintain therapeutic levels, making treatment adherence more difficult and resulting in high levels of infusion-associated adverse events. In addition, just like other traditional monoclonal antibodies, conventional BiTEs are also manufactured in bioreactors, typically requiring costly large-scale manufacturing facility development and laborious production as well as having to deal with improper product folding and stability. They must also be kept and distributed frozen at all times. These difficulties collectively have limited the development and commercialization of conventional BiTEs as only one licensed product is currently on the market (BLINCYTO (blinatumomab)).

Inovio’s dBiTE is a new transformative application of Inovio’s dMAb platform. The dBiTEs share many advantages of Inovio’s dMAbs as they both are composed of engineered DNA sequences which encode antibody fragments. When administered by Inovio’s CELLECTRA delivery device, the patient’s own cells become the factory to manufacture functional BiTES encoded by the delivered dBiTE sequences. Inovio’s dBiTEs are developed with novel syntheic nucleics design using plasmid vectors and unique formulations allowing for rapidity of development, long-term product stability at refrigeration, ease of validated and scalable manufacturing and deployability.

About Inovio’s dBiTE program

Inovio’s dBiTEs are able to target the cytotoxic T cells to tumors by engaging proteins expressed in the tumor surface. The current preclinical models have shown proof that DNA technologies are in an advantageous position to launch a more ambitious BiTE program. The tumor-binding domain can be modified to engage multiple targets, of which preclinical data targeting HER2 and CD19 has been presented. Of these, the CD19dBiTE can be used to target B cell cancers and the HER2dBiTE can be used to treat advanced breast, ovarian, gastric, esophageal and endometrioid cancers.