On February 26, 2019 Ophthotech Corporation (Nasdaq: OPHT) reported financial and operating results for the fourth quarter and full year ended December 31, 2018 and provided a business update (Press release, Ophthotech, FEB 26, 2019, View Source [SID1234533679]).

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"2018 was a significant year for transforming the Company by executing multiple business development transactions that resulted in the addition of four gene therapy research and development programs targeting orphan inherited retinal diseases and an age-related therapeutic development program to our retina portfolio," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "We welcomed Versant Ventures as a major shareholder of Ophthotech through the acquisition of Inception 4 and expanded our Board of Directors by adding three industry leaders. We look forward to topline data from our Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry age-related macular degeneration in the fourth quarter of 2019. We are committed to advancing and potentially expanding our pipeline of therapies for retinal diseases and creating value for our shareholders."

Gene Therapy Program Highlights of 2018
In 2018, the Company initiated multiple innovative gene therapy collaborations to discover and develop next-generation therapies for the treatment of inherited retinal diseases.

In June 2018, the Company entered into an exclusive global license agreement with the University of Florida Research Foundation (UFRF), Incorporated and the Trustees of the University of Pennsylvania (Penn) for rights to develop and commercialize a novel adeno-associated virus (AAV) gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic retinal disease. The novel AAV gene therapy construct is designed to knock down toxic rhodopsin and deliver a transgene for healthy rhodopsin via a single AAV vector in a mutation independent manner. The Company expects to initiate a Phase 1/2 clinical trial in RHO-adRP in 2020.

In August, proof-of-concept study results of this product candidate in a naturally occurring canine disease model were published in the prestigious journal Proceedings of the National Academy of Sciences of the USA. This publication entitled: "Mutation-independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV vector" was published by scientists at Penn and University of Florida.

In October 2018, the Company entered into its second series of gene therapy agreements with Penn and UFRF, including an exclusive option agreement for rights to negotiate to acquire an exclusive global license to develop and commercialize novel AAV gene therapy product candidates

for the treatment of diseases impacted by the bestrophin, or BEST1, gene. The Company expects to initiate a Phase 1/2 clinical trial in Best disease in 2021.

In February 2018, the Company entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center. The research seeks to utilize a minigene strategy to create novel AAV gene therapy product candidates for Leber Congenital Amaurosis type 10 due to CEP290 mutations (the most common type of LCA), and autosomal recessive Stargardt disease due to ABCA4 mutations, both of which are orphan inherited retinal diseases, and to evaluate different AAV gene delivery methods for application in the eye.

Therapeutic Program Highlights of 2018

Complement Factor C5 Inhibitor Program: Zimura

In October 2018, the Company completed patient enrollment for its ongoing randomized, double-masked, sham controlled, multi-center Phase 2b clinical trial of Zimura for the treatment of geographic atrophy secondary to dry AMD. The Company expects initial top-line data for this trial to be available in the fourth quarter of 2019.

Patient enrollment in the Phase 2b randomized, double-masked, sham-controlled, multi-center clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1) is completed and initial top-line data is expected to be available in in the second half of 2020.

Corporate Highlights of 2018

In October 2018, Ophthotech acquired Inception 4, Inc., a privately held company backed by Versant Ventures. Ophthotech gained worldwide development and commercialization rights to Inception 4’s small molecule inhibitors of HtrA1 (high temperature requirement A serine peptidase 1 protein). In addition, Versant Ventures agreed to help Ophthotech identify opportunities to expand the pipeline. Ophthotech obtained approximately $6.1 million in cash through its acquisition of Inception 4 and issued approximately 5.2 million shares to the shareholders of Inception 4. After giving effect to the transaction, funds affiliated with Versant Ventures own approximately 12.5% of the outstanding shares of Ophthotech’s common stock.

During 2018, the Company expanded its Board of Directors by adding leading industry experts: Adrienne L. Graves, Ph.D., former Chief Executive Officer of Santen, Inc., and Jane Pritchett Henderson, Chief Financial Officer of Turnstone Biologics and former Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics. Effective January 1, 2019, Calvin W. Roberts, M.D., Senior Vice President and Chief Medical Officer, Eye Care at Bausch Health Companies and Clinical Professor of Ophthalmology at Weill Medical College of Cornell University was elected to Ophthotech’s Board of Directors.

2019 Operational Update

As of December 31, 2018, the Company had $131.2 million in cash and cash equivalents.
The Company estimates its year end 2019 cash and cash equivalents will range between $80 million and $85 million based on its current 2019 business plan, including expansion of the Company’s gene therapy research and development programs, expansion of its HtrA1 development program, and the continuation of its clinical development programs for

Zimura (avacincaptad pegol sodium). This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

2018 Financial Highlights

Gain on Extinguishment of Royalty Purchase Liability: The Company recognized a gain on extinguishment of its royalty purchase liability of $125 million for the quarter and year ended December 31, 2018, due to the December 2018 termination of the Company’s royalty purchase and sale agreement with Novo Holdings A/S. The termination of the agreement relieved the Company of any obligation to pay Novo Holdings A/S future product royalties on sales of certain platelet derived growth factor (PDGF) antagonists and did not impact the Company’s cash balance.

Revenues: The Company did not have any collaboration revenue for the quarters ended December 31, 2018 and 2017 or during the year ended December 31, 2018, compared to $210 million for the year ended December 31, 2017. Collaboration revenue decreased for 2018 as compared to 2017 as the Company had recognized all deferred revenue associated with the completion of the Company’s licensing and commercialization agreement with Novartis Pharma AG during the third quarter of 2017 and had no collaboration revenue in 2018.

R&D Expenses: Research and development expenses were $16.1 million for the quarter ended December 31, 2018, compared to $7.9 million for the same period in 2017. Research and development expenses increased primarily due to $6.9 million in charges associated with the Company’s October 2018 acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs. For the year ended December 31, 2018, research and development expenses were $41.7 million compared to $66.3 million for 2017. Research and development expenses decreased primarily due to decreases in expenses related to the discontinuation of the Company’s Fovista Phase 3 clinical program and decreases in costs associated with the Company’s 2017 reduction in personnel program partially offset by charges associated with the Company’s October 2018 acquisition of Inception 4 and its HtrA1 inhibitor program and increases in costs associated with the Company’s ongoing Zimura and gene therapy development programs.

G&A Expenses: General and administrative expenses were $5.7 million for the quarter ended December 31, 2018, compared to $6.9 million for the same period in 2017. For the year ended December 31, 2018, general and administrative expenses were $23.6 million compared to $35.7 million for 2017. General and administrative expenses decreased primarily due to decreases in costs to support the Company’s operations and infrastructure and decreases in costs associated with its 2017 reduction in personnel program, which included facilities lease termination expenses incurred during the first quarter of 2017.

Net Income: The Company reported net income for the quarter ended December 31, 2018 of $104.1 million, or $2.62 per diluted share, compared to net loss of $9.5 million, or ($0.26) per diluted share, for the same period in 2017. For the year ended December 31, 2018, the Company reported net income of $63.1 million, or $1.70 per

diluted share, compared to net income of $114.2 million, or $3.17 per diluted share, for the same period in 2017.

Conference Call/Web Cast Information
Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 26, 2019 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 888-224-1005 (USA) or 323-994-2093 (International), passcode 6840989. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 6840989.

On February 26, 2019 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the year ended December 31, 2018, and provided a corporate update (Press release, Cellectar Biosciences, FEB 26, 2019, View Source [SID1234533678]).

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Fourth Quarter and Recent Corporate Highlights

·Announced additional positive top-line results from the relapse refractory multiple myeloma cohort in its ongoing Phase 2 clinical study of CLR 131, the company’s lead product candidate. In patients with an average of 5 prior lines of systemic therapy, CLR 131 achieved a 30% overall response rate in the first 10 evaluable patients. The company previously announced an overall response rate of 33% in patients with R/R diffuse large B-cell lymphoma (DLBCL) also receiving the single, 25mCi/m2 dose of CLR 131. All patients reported here were administered one, single 30-minute infusion of 25mCi/m2, which is approximately 60% less drug than fractionated dose currently being tested in the ongoing Phase 1 study.

·Initiated Cohort 6 of its ongoing Phase 1b study evaluating CLR 131 for the treatment of relapsed/refractory (R/R) multiple myeloma (MM).
oCohort 6 is evaluating up to four patients with each receiving two doses of 18.75 mCi/m2 of CLR 131 administered one week apart.
oThis fractionated dosing regimen will result in each patient being treated with an increase in average total exposure of approximately 60% over the Phase 2 efficacious dose of 25mCi/m2.
oThe fractionated dose administered in Cohort 5 indicated enhanced tolerability and safety compared with the single dose administered in Cohort 4 despite an 18% increase in the average dose. Additionally, patients in Cohort 5 experienced fewer adverse events.
oBased on these results and the DMC recommendation, Cellectar modified the single-dose regimen of its ongoing Phase 2 study of R/R hematologic malignancies to fractionated dosing and proceeded with Cohort 6 of the Phase 1 study.

·Granted the patent titled "Phospholipid Analogs as Diapeutic Agents and Methods of Use Thereof" by the Japanese Patent Office. The patent provides composition of matter and use protection for the company’s proprietary phospholipid ether (PLE) analogs and specifically the use of CLR 131 in breast, brain, leukemias, and a variety of other cancers

·Announced median overall survival (mOS) of 22 months in the single dose Cohorts 1-4 of the company’s ongoing Phase 1 clinical study evaluating CLR 131 for the treatment of relapsed/refractory (R/R) multiple myeloma (MM). All of these patients were heavily pretreated, averaging five prior lines of systemic therapy.

"We continue to make meaningful progress with the clinical development of CLR 131 and have now reported activity in three cohorts of our ongoing Phase 2 study in challenging relapse/refractory hematologic cancers: diffuse large B cell, Waldenstrom’s lymphoma and multiple myeloma. The recently announced 30% response rate in relapsed/refractory multiple myeloma coupled with the median overall survival of 22 months in patients receiving a single dose from the Phase 1 study is very encouraging," said James Caruso, president and CEO of Cellectar. "Going forward, we believe CLR 131, with a higher and more patient-friendly fractionated dosing regimen, has the potential to further improve upon its product profile and be an effective treatment in relapsed/refractory hematologic cancers. We look forward providing further updates and data for CLR 131 during 2019."

2018 Financial Highlights

Research and development expense for the year ended December 31, 2018 was $6.8 million, compared to $9.5 million for the year ended December 31, 2017. The overall decrease in research and development expense of 28% was due primarily to a decrease in accelerated depreciation expense due to the reassessed estimated useful life of the leasehold improvements and laboratory equipment in 2017. The reassessment of the useful lives for these assets was due to the company’s decision to close its manufacturing facility and outsource all of its manufacturing.

General and administrative expense for the year ended December 31, 2018 was $4.8 million, compared to $4.1 million for the year ended December 31, 2017. The increase of 17% for 2018 was primarily related to an increase of approximately $229,000 in purchased services related to accounting, investor relations and public company costs offset by a decrease in legal fees of approximately $157,000; and an increase of approximately $510,000 in personnel related costs.

The net loss attributable to year ended December 31, 2018 was ($15.5) million, or ($5.23) per share, respectively, compared with a net loss attributable to common stockholders for the year ended December 31, 2017 of ($15.0) million, or ($10.70) per share.

As of December 31, 2018, the company had cash, cash equivalents and restricted cash of $13.3 million compared to $10.1 million at December 31, 2017. The increase was largely attributable to cash received from financing activities of approximately $15.0 million, offset by cash used in operating activities of $11.4 million and cash used in investing activities of approximately $330,000. Consistent with prior guidance, the company believes its cash on hand is adequate to fund operations into the first quarter of 2020.

On February 26, 2019 Compugen Ltd. (NASDAQ: CGEN), a leader in predictive discovery and development of first-in-class therapeutics for cancer immunotherapy, reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Compugen, FEB 26, 2019, View Source [SID1234533677]).

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Compugen also announced a corporate restructuring to reduce costs by consolidating and streamlining R&D operations. Anticipated cost reductions are expected to extend the Company’s cash runway through mid-2020 to enable the planned expansion of the ongoing Phase 1 study for COM701, a first-in-class immunotherapy antibody targeting the PVRIG checkpoint, which continues to progress as planned. In addition, the Company will maintain investment in its proprietary computational discovery platform and will continue to advance its earlier stage immuno-oncology pipeline programs, which are the Company’s two long-term core value drivers.

"2018 was a pivotal year for Compugen in which two clinical trials were initiated for our computationally-discovered immuno-oncology programs, COM701 and BAY 1905254, and we partnered with Bristol-Myers Squibb and AstraZeneca," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "After reaching these important inflection points in our corporate development, the management team and Board undertook a strategic review of the Company’s operations and cost structure to ensure effective use of capital in support of the Company’s long-term objectives.

"This restructuring will allow us to focus the necessary resources to execute the COM701 study, including the initiation of combination studies with Opdivo. Of equal importance is the decision to consolidate and streamline our R&D infrastructure and continue investing in our two long-term core value drivers. These difficult decisions are necessary to effectively use our capital to ensure our future growth and the achievement of our strategic objectives. We will implement these organizational changes with the utmost respect for our employees, who I thank for their dedication and significant contributions made to our organization."

Corporate Restructuring
The restructuring includes:

Consolidation of R&D activities in one location (Israel). Positions and operations eliminated will also reduce overlapping R&D and G&A activities in Israel and the United States;

A 35% workforce reduction (approximately 35 employees), the majority of which is in R&D;

Certain preclinical activities will be outsourced to third-party service providers;

IND filing for COM902 in 2019 will proceed as planned. Initiation of the Phase 1 trial for this program will be dependent on the drug combination trials pursued under the collaboration with Bristol-Myers Squibb as well as available resources to support this trial;

Clinical development and business development activities will continue to operate in the United States; and

Compugen anticipates savings of up to $10 million on an annual basis. Cash expenditures for 2019 including one-time restructuring related costs, are expected to be in the range of $27 to $29 million, with the full effect of the savings reflected in 2020.

Recent Corporate Highlights

Published two peer-reviewed papers in Cancer Immunology Research demonstrating the role of PVRIG as a novel immune checkpoint target for cancer immunotherapy;

Awarded U.S. patent No. 10,124,061 from the U.S. Patent and Trademark Office for the method of using COM902, an anti-TIGIT monoclonal antibody, for activating T cells in cancer patients under the Cancer Moonshot program;

Signed a clinical trial collaboration with Bristol-Myers Squibb for a Phase 1 combination study of COM701 and Opdivo;

Received a $12 million investment from Bristol-Myers Squibb as part of the collaboration;

Initiated the COM701 monotherapy dose escalation Phase 1 clinical trial in advanced solid tumor patients; and

Bayer initiated a Phase 1 study for BAY 1905254, its first-in-class therapeutic antibody program targeting ILDR2, computationally-discovered by Compugen.

Financial Results
Revenues for the year ended December 31, 2018 were $17.8 million, compared with $0 in the comparable period of 2017. The revenues for 2018 reflect the upfront payment of $10 million from the license agreement with AstraZeneca and the $7.8 million milestone payment from Bayer AG in connection with the dosing of the first patient in the Phase 1 study of BAY 1905254.

R&D expenses for the fourth quarter and year ended December 31, 2018 were $7.5 million and $30.3 million, respectively, compared with $7.2 million and $28.6 million for the comparable periods in 2017. The increase reflects the expenses associated with the ongoing Phase 1 study of COM701.

Net loss for the fourth quarter of 2018 was $9.4 million, or $0.16 per diluted share, compared with a net loss of $9.3 million, or $0.18 per diluted share, in the comparable period of 2017. Net loss for the year ended December 31, 2018 was $22.6 million, or $0.41 per diluted share, compared with a net loss of $37.1 million, or $0.72 per diluted share, for the year ended December 31, 2017.

As of December 31, 2018, cash, cash related accounts, short-term and long-term bank deposits totaled $45.7 million, compared with $30.4 million at December 31, 2017. The Company has no debt.

Based on current cash and anticipated savings resulting from restructuring activities, the Company expects its cash runway to extend through mid-2020, not including potential cash inflow from existing or new business development arrangements.

Conference Call and Webcast Information
Compugen will hold a conference call to discuss its fourth quarter and full year 2018 results today, February 26, 2019, at 8:30 a.m. ET. To access the live conference call by telephone, please dial 1-888-407-2553 from the U.S., or +972-3-918-0610 internationally. The conference call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

On February 26, 2019 Cell Medica reported that it has been awarded an $8.7 million research grant from the Cancer Prevention and Research Institute of Texas (CPRIT) (Press release, Cell Medica, FEB 26, 2019, View Source [SID1234533676]). The grant will support preclinical and clinical development of the company’s off-the-shelf chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies to treat hematological and solid tumors.

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The CPRIT grant will support development programs being conducted in collaboration with Baylor College of Medicine (BCM) that are designed to address the limitations of the current first-generation autologous CAR-T cell therapies. The aim is to deliver an off-the-shelf approach, with simplified manufacturing, that can serve larger patient numbers, and which allows treatment closer to the time of patient presentation. The program currently includes four therapies in early-stage development.

CMD-502 is Cell Medica’s most advanced off-the-shelf therapy and a first-in-human study is expected to start in the second half of 2019. Called the ANCHOR study, it will be a dose escalation evaluation of CMD-502 in adults with relapsed or refractory (R/R) diffuse large B cell lymphoma (DLBCL), acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL).

CPRIT awarded a $15.3 million grant to Cell Medica in 2012, to support the establishment of operations in Houston, Texas and fund earlier development programs. Through the current grant, CPRIT-funded research will be conducted at both BCM and Cell Medica’s Houston facility.

Chris Nowers, CEO of Cell Medica, commented: "CPRIT was instrumental in enabling us to establish our US operations in Texas, so we are delighted to extend that collaboration through a further grant. This funding will accelerate development of our off-the-shelf CAR-NKT pipeline and, given CPRIT’s deep and broad review, also brings a strong independent validation of our platform and approach."

Dr. Carlos Ramos, Associate Professor, Center for Cell and Gene Therapy, at BCM and principal investigator in Phase 1 ANCHOR study, added: "It has been a great pleasure working with the multi-disciplinary team at Cell Medica in the development of its versatile CAR-NKT platform. Off-the-shelf CAR-NKT cell therapy has the potential to become a better and simpler approach to CAR therapy for patients with hematological and solid tumors.

"Although existing autologous CAR-T cell therapies have demonstrated impressive response rates, the patient-specific manufacturing process is technically challenging, costly, and time-consuming, and comes with complex logistics and substantial treatment delays. The unique properties of NKT cells bring the potential to solve these challenges."

On February 26, 2019 Kleo Pharmaceuticals Inc., a unique immuno-oncology company developing next-generation bispecific compounds designed to emulate or enhance the activity of biologics, reported that its Chief Executive Officer Douglas J. Manion, M.D., FRCP(C), will present at the 16th Annual BIO Asia International Conference, to be held March 5-6, 2019, at the Grand Hyatt Tokyo in Tokyo, Japan (Press release, Kleo Pharmaceuticals, FEB 26, 2019, View Source [SID1234533675]).

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Dr. Manion will present an overview of Kleo’s development strategy for its pipeline of small molecule and synthetic peptide compounds that function similar to highly complex biologics, while offering multiple potential advantages. The overview will also outline Kleo’s key value drivers including the three proprietary technology platforms that drive in-house drug development and partnership opportunities, the multiple in-house and collaborative channels through which it is building its immuno-oncology pipeline, and its discovery collaboration with PeptiDream.

Kleo is well-funded to achieve multiple preclinical and early clinical milestones following the November 2018 closing of an oversubscribed $21 million Series B financing to advance its first clinical candidates through IND-enabling studies and into the clinic in 2020.

Details of Kleo’s presentation are as follows:

Event: 16th Annual BIO Asia International Conference

Date: Tuesday, March 6th, 2019

Time: 8:45 AM JST

Location: Grand Hyatt Tokyo, Drawing Room, 2nd floor