On January 20, 2021 Nicox SA (Euronext Paris: FR0013018124, COX), an international ophthalmology company, reported a business update and financial highlights for Q4 2020 for Nicox SA and its subsidiaries (the "Nicox Group"), as well as key expected value-inflection milestones in 2021 (Press release, NicOx, JAN 20, 2021, View Source [SID1234574120]).

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Key Expected Milestones
NCX 470 first Phase 3 trial, Mont Blanc: Nicox’s lead clinical product candidate, NCX 470 is a novel nitric oxide (NO) donating prostaglandin analog. Mont Blanc is a 3-month safety and efficacy trial evaluating NCX 470 ophthalmic solution, 0.1%, against latanoprost ophthalmic solution, 0.005%, for lowering of intraocular pressure (IOP) in patients with open-angle glaucoma or ocular hypertension. Top-line results are now prudently expected in H1 2022, instead of Q4 2021, given probable delays in recruitment due to COVID-19.
NCX 4251 Phase 2b trial, Mississippi: NCX 4251 is a novel patented ophthalmic suspension of fluticasone propionate nanocrystals. Mississippi is evaluating once-daily dosing NCX 4251 0.1% versus placebo for the treatment of acute exacerbations of blepharitis. Top-line results are currently expected in Q4 2021.
We expect to enter into additional agreements for ZERVIATETM (cetirizine ophthalmic solution), 0.24%, further enlarging the licensed territories and increasing potential future revenue.
Fourth Quarter 2020 and Recent Operational Highlights
Innovative pipeline

The second Phase 3 trial of NCX 470, Denali, for the lowering of IOP in patients with open-angle glaucoma or ocular hypertension, was initiated in the U.S. on November 9, 2020. Denali is a 3-month trial evaluating the safety and efficacy of NCX 470 ophthalmic solution, 0.1% versus latanoprost ophthalmic solution, 0.005% and will also include a long-term safety extension. The trial is financed jointly and in equal parts by Nicox and its Chinese partner Ocumension. Top-line results are currently expected in Q4 2022.
The Phase 2b trial of NCX 4251, Mississippi, for the treatment of acute exacerbations of blepharitis was initiated in the U.S. on December 14, 2020. Top-line results are currently expected in Q4 2021.
A Phase 3 trial of ZERVIATETM (cetirizine ophthalmic solution), 0.24%, for the treatment of ocular itching associated with allergic conjunctivitis, conducted and financed by Nicox’s Chinese partner Ocumension, was initiated in China in December 2020. This trial will support the submission of a Chinese New Drug Application.
NCX 1728 was selected as the first development candidate in a new class of agents for IOP lowering where NO-mediated effects are enhanced by concomitant action of phosphodiesterase-5 (PDE5) inhibition within the same molecule. Further optimization of the ophthalmic formulations of NCX 1728 will continue prior to initiating formal pre-Investigational New Drug (IND) tests required for the filing of an IND application.
Commercial products

The total number of prescriptions1 for VYZULTA (latanoprostene bunod ophthalmic solution), 0.024%, in the U.S. increased by 29% in the fourth quarter of 2020 compared to the fourth quarter of 2019 and by 16% compared to the third quarter of 2020 despite the challenging situation due to the COVID-19 pandemic. Along with the U.S., Canada and Argentina, VYZULTA has also been launched by Nicox’s global partner Bausch + Lomb in Mexico and was recently approved in Colombia.
ZERVIATETM (cetirizine ophthalmic solution), 0.24%, U.S. prescriptions2 increased by 56% in the fourth quarter of 2020 over the third quarter of 2020. ZERVIATE has been commercialized in the U.S. since March 2020 by Nicox’s U.S. partner Eyevance Pharmaceuticals, which was acquired in September 2020 by Santen Holdings U.S. Inc., a wholly owned subsidiary of Santen Pharmaceutical Co., Ltd of Japan, for $225 million.
Corporate

The Company completed a €15 million private placement with investors including long-term shareholder HBM Healthcare Investments alongside specialist institutional investors in the U.S. and Europe.
The European Patent Office granted a formulation patent for NCX 470, extending the European exclusivity to 2039. The equivalent U.S. patent has already been granted, and NCX 470 is also covered by granted composition of matter patents.
Fera Pharmaceuticals, Nicox’s partner for naproxcinod, will evaluate naproxcinod as a potential adjuvant treatment for patients with COVID-19 infection. Fera plans to initiate pre-clinical proof-of-concept studies in models of COVID-19 infection in early 2021.
Ora and Nicox have agreed to terminate their license agreement for the development of NCX 4280 targeting lid swelling, or morning eye congestion. All rights to NCX 4280 will return to Nicox and there are no current plans to continue the development.
We continue to closely watch the spread and impact of the COVID-19 pandemic and we will provide an update of any delays.

Fourth Quarter 2020 Financial Highlights
As of December 31, 2020, the Nicox Group had cash and cash equivalents of €47.8 million as compared with €28.0 million at December 31, 2019 and €42.2 million at September 30, 2020. Net revenue3 for the fourth quarter of 2020 was €5.8 million (consisting of €0.3 million of royalty payments and €5.5 million of license payments recognized from €14.0 million paid by Ocumension in March 2020 and initially recorded as prepaid income pursuant to accounting principles). Net revenue3 for the fourth quarter of 2019 was €0.6 million and consisted entirely of royalty payments. Net revenue3 for the full year 2020 was €8.9 million (€2.4 million in net royalties, €6.5 million in license payments), compared to €6.9 million (€2.1 million in net royalties, €4.8 million in license payments) for the full year 2019.

As of December 31, 2020, the Nicox Group had financial debt of €18.4 million in the form of a bond financing agreement with Kreos Capital signed in January 2019 and a €2 million credit agreement with Société Générale and LCL, guaranteed by the French State, and granted in August 2020 in the context of the COVID-19 pandemic.

Only the figure related to the cash position of the Nicox Group as of December 31, 2019 is audited; all other figures of this press release are non-audited.
Notes

Bloomberg data, comparing the period of the weeks ending October 2, 2020 to January 1, 2021 with the periods of the weeks ending July 3, 2020 to September 25, 2020 and October 4, 2019 to December 27, 2019
Bloomberg data, comparing the period of the weeks ending October 2, 2020 to January 1, 2021 with the period of the weeks ending July 3, 2020 to September 25, 2020
Net revenue consists of revenue from collaborations less royalty payments, which corresponds to Net profit in the consolidated statements of profit or loss

On January 20, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported that due to demand, the underwriter has agreed to increase the size of the previously announced offering and purchase on a firm commitment basis 12,500,000 American Depositary Shares (ADSs) of the Company, at a price to the public of $2.40 per ADS, less underwriting discounts and commissions (Press release, BioLineRx, JAN 20, 2021, View Source [SID1234574112]). Each ADS represents fifteen ordinary shares, par value NIS 0.10 per share, of the Company. The closing of the offering is expected to occur on or about January 22, 2021, subject to satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the sole book-running manager for the offering.

The Company also has granted to the underwriter a 30-day option to purchase up to additional 1,875,000 ADSs at the public offering price, less underwriting discounts and commissions.

The gross proceeds to BioLineRx, before deducting underwriting discounts and commissions and offering expenses and assuming no exercise of the underwriter’s option to purchase additional ADSs, are expected to be approximately $30 million. The Company intends to use the net proceeds from this offering for general corporate purposes, which may include but are not limited to working capital and funding clinical trials.

The securities described above are being offered by BioLineRx pursuant to a "shelf" registration statement on Form F-3 (File No. 333-251857) previously filed with the Securities and Exchange Commission (the "SEC") on December 31, 2020 and declared effective by the SEC on January 11, 2021. The offering of the securities is being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the securities being offered have been filed with the SEC and are available on the SEC’s website at View Source." target="_blank" title="View Source." rel="nofollow">View Source A final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and, upon filing, may be obtained on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

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

On January, 19, 2021 miRagen Therapeutics, Inc. (NASDAQ: MGEN), a development-stage biotechnology company, reported its name change to Viridian Therapeutics, Inc. ("Viridian") (Press release, Viridian Therapeutics, JAN 19, 2021, View Source [SID1234639899]). Beginning tomorrow, Viridian will trade on NASDAQ under the ticker symbol "VRDN" and its common stock will trade under a new CUSIP number, 92790C104.

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The Company also announced today the appointment of Jonathan Violin, Ph.D., M.B.A. as President and Chief Executive Officer (CEO) and member of the Board of Directors. Dr. Violin, who previously served as Viridian’s President and Chief Operating Officer (COO), succeeds Lee Rauch as CEO and member of the Board of Directors. Ms. Rauch will remain as strategic advisor for the Company.

In addition, Viridian appointed internationally recognized neuro-ophthalmologist, Barrett Katz M.D ., M.B.A., as Chief Medical Officer (CMO). Dr. Katz comes to Viridian from BridgeBio Pharma, Inc. where he developed therapeutics to treat orphan eye diseases.

"The leadership team changes and Viridian Therapeutics name reflect the continuing evolution of the company and our patient-centric model of innovation," said Dr. Violin. "We’re leveraging proven biology and technology to efficiently allocate research and development resources, while addressing strategic gaps related to access, delivery, quality of life, and efficacy. We are thrilled to attract someone with Dr. Katz’s depth of expertise in serving patients and leading scientific and clinical programs."

During his tenure at BridgeBio, Dr. Katz held leadership positions in two subsidiaries, as President and CMO of Retinagenix and CEO of Fortify Therapeutics. Prior to BridgeBio, he was CMO at GenSight Biologics where he oversaw early- and late-stage clinical programs. He held the Francis DeJur Chair of Ophthalmology at the Montefiore Medical Center and Albert Einstein College of Medicine in New York , where he also served as Professor of Ophthalmology, Neurology and Neurosurgery, as well as the Executive Director of the Office of Clinical Trials . He previously served as CEO of Danube Pharmaceuticals , CMO of Fovea Pharmaceuticals and VP for Medical Affairs and Strategy at Eyetech. Dr. Katz received an M.D. from Case-Western Reserve University School of Medicine , an M.B.A. from the University of Rochester’s Simon School of Business , and an A.B. from Colgate University .

Prior to co-founding privately held Viridian Therapeutics, Dr. Violin had founded and served as CEO of two virtual drug discovery companies, Quellis Biosciences and Dianthus Therapeutics, and co-founded and held several executive positions at Trevena, Inc. He holds a Ph.D. in biomedical sciences from the University of California San Diego School of Medicine , an M.B.A. from the Fuqua School of Business at Duke University , and a B.S. from Duke University .

Viridian is developing multiple product candidates to treat patients who suffer from thyroid eye disease (TED), a debilitating orphan disease that can cause bulging eyes, or proptosis, as well as double vision and potential blindness. TED significantly impacts quality of life, imposing a high physical and mental burden on patients. There is currently one Food and Drug Administration (FDA)-approved treatment for TED, an intravenously administered monoclonal antibody that targets the insulin-like growth factor-1 receptor (IGF-1R).

"Patients with TED have limited treatment options," said Dr. Katz . "Viridian has a clear and compelling strategy to better serve these patients. I am delighted to help build upon the Company’s recent momentum and eager to design and implement robust clinical programs for our lead product candidates."

Viridian’s most advanced product candidate is VRDN-001, an intravenously administered anti-IGF-1R monoclonal antibody which, the Company expects to proceed directly to a phase 2 trial, pending feedback from the FDA. In October, the Company obtained exclusive worldwide rights from ImmunoGen, Inc. to develop and commercialize VRDN-001 for all non-oncology indications that do not use radiopharmaceuticals, including the treatment of TED.

VRDN-002 is the Company’s second-generation product candidate, incorporating half-life extension technology, and is intended for subcutaneous administration. Viridian holds exclusive rights to develop and commercialize antibody therapeutics targeting IGF-1R using the Xtend TM half-life extension technology developed and owned by Xencor, Inc.

In the second half of 2021, Viridian expects to file Investigational New Drug (IND) applications for both VRDN-001 and VRDN-002.

On January 19, 2021 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company dedicated to improving life with its wholly proprietary ARCUS genome editing platform, reported that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for PBCAR19B, a next-generation, stealth cell, CD19 product candidate for patients with relapsed/refractory (R/R) Non-Hodgkin Lymphoma (NHL) (Press release, Precision Biosciences, JAN 19, 2021, View Source [SID1234576682]).

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"We are pleased to receive IND clearance for PBCAR19B, which has shown in preclinical studies, to delay both T cell and natural killer cell-mediated allogeneic rejection," said Matt Kane, Chief Executive Officer of Precision BioSciences. "We believe that the ability to reduce rejection by both cell types holds potential for improved persistence of allogeneic CAR T cells and that, by bringing PBCAR19B into the clinic while continuing to develop our lead allogenic candidate PBCAR0191, we have two opportunities to achieve our goal of producing deep and durable clinical responses."

The Phase 1 study will be a non-randomized, open-label, single-dose, dose-escalation and dose-expansion study deigned to evaluate the safety and clinical activity of PBCAR19B at increasing flat dose levels (2.7 x 108 – 8.1 x 108 CAR T cells) in patients with R/R NHL. The primary objective of the study is to identify the maximum tolerated dose and any dose-limiting toxicities.

About PBCAR19B
PBCAR19B is a next-generation, stealth cell candidate for patients with CD19-positive malignancies such as relapsed/refractory Non-Hodgkin Lymphoma. PBCAR19B is designed to improve the persistence of allogeneic CAR T cells following infusion by preventing rejection by T cells and natural killer (NK) cells. In addition to the CAR gene, the stealth vector carries a short hairpin RNA (shRNA) that suppresses expression of beta-2 microglobulin (B2M), a component of Class 1 major histocompatibility complex (MHC) molecules found on the cell surface. Reducing or knocking down Class 1 expression on allogeneic CAR T cells has been shown to reduce CAR T cell killing by cytotoxic T cells. The stealth vector also carries a HLA-E gene intended to prevent rejection of CAR T cells by NK cells that can be stimulated as a result of reduced MHC molecule expression on the cell surface.

On January 19, 2021 BridgeBio Pharma, Inc. ("BridgeBio") (Nasdaq: BBIO) and Eidos Therapeutics, Inc. ("Eidos") (Nasdaq: EIDX) reported that the stockholders of each of BridgeBio and Eidos voted to approve all proposals related to BridgeBio’s acquisition of all of the outstanding shares of Eidos common stock that BridgeBio does not already own (Press release, BridgeBio, JAN 19, 2021, View Source [SID1234576237]). The merger is expected to close on or about January 26, 2021, subject to the satisfaction or waiver of other conditions to closing.

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At a special meeting of Eidos stockholders held virtually today, Eidos stockholders approved the adoption of the previously disclosed merger agreement with BridgeBio and each of the other proposals related to BridgeBio’s proposed acquisition of Eidos.

At a special meeting of BridgeBio stockholders held virtually today, BridgeBio stockholders approved the proposal to issue common stock to Eidos stockholders in connection with the proposed acquisition.

"We are ready and eager to welcome Eidos back into BridgeBio’s ecosystem of scientific innovation," said Neil Kumar, Ph.D., founder and CEO of BridgeBio and CEO of Eidos. "We believe this merger will allow us to fully unlock the potential of acoramidis, the investigational therapy Eidos is developing to target transthyretin (TTR) amyloidosis (ATTR), creating value for patients and investors. I’d like to thank BridgeBio and Eidos stockholders for their support and their overwhelming approval of this transaction."

Holders of Eidos common stock may elect to receive, for each share of Eidos common stock issued and outstanding immediately prior to the effective time of the merger (the "Effective Time") that is not owned by BridgeBio or any of its subsidiaries and that is not a restricted share award, either (1) 1.85 shares of common stock of BridgeBio (the "Stock Consideration") or (2) $73.26 in cash (the "Cash Consideration" and, together with the Stock Consideration, the "Merger Consideration"), subject to proration. The Cash Consideration will be prorated as necessary to ensure that the aggregate amount of cash consideration payable in the merger is no greater than $175 million. Any Eidos stockholders who do not make a proper election by 5:00 p.m., New York City time, on January 21, 2021 will be deemed to have elected to receive the Stock Consideration for their shares of Eidos common stock.