On February 27, 2019 ALX Oncology, a clinical-stage immuno-oncology company developing therapies to block the CD47 checkpoint mechanism, reported that preclinical data for its SIRPα antibody research program will be presented at the 2019 Keystone Symposia Conference (Keystone), Cancer Immunotherapy in Whistler, British Columbia and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, Georgia (Press release, ALX Oncology, FEB 27, 2019, View Source [SID1234533751]).

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CD47/SIRPα interaction is a key checkpoint mechanism exploited by cancer cells to escape immunological surveillance. While CD47 is widely expressed in human cells, SIRPα, the CD47 inhibitory receptor, is mainly expressed in myeloid cells and neurons. ALX148, a CD47 blocker with an inactive Fc domain, is generally well tolerated and demonstrates anti-cancer activity in combination with trastuzumab and pembrolizumab in patients with anti-HER2 and checkpoint inhibitor resistant/refractory disease (SITC 2018, P335). Another approach to block this interaction is to target SIRPα.

"Targeting the CD47/SIRPα pathway is an exciting new approach that shows promise in clinical trials. By targeting SIRPα, we can investigate the similarities and differences of an orthogonal strategy to inhibit this axis," said Hong Wan, Ph.D., ALX Oncology’s Chief Scientific Officer. "Our panel of proprietary high affinity monoclonal antibodies provides diverse epitope coverage across the extracellular surface of SIRPα. These antibodies are cross-reactive to human, monkey and rodent SIRPα variants, which enables robust clinical translation. Importantly, these antibodies bind to all human SIRPα variants, a critical attribute for a global patient population."

ALX Oncology’s SIRPα antibodies enhance the antitumor activity of immune checkpoint inhibitors, with reduction of metastases, eradication of tumors, and acquisition of memory immune response in tumor-bearing mice with intact immune systems. The cellular immune response in syngeneic models shows that these SIRPα antibodies enhance innate and adaptive anti-cancer immunity, providing a rationale for combination with other immunotherapies. In an exploratory toxicology study in monkeys, the selected SIRPα antibodies demonstrate a favorable pharmacokinetic, target occupancy and tolerability profile.

Together, these preclinical data provide a compelling rationale to advance the development of anti-SIRPα therapy for patients with cancer.

Keystone Presentation Information
Title: Discovery of monoclonal antibodies targeting myeloid checkpoint SIRPα to enhance anti-tumor immunity
Session: Poster session 3
Session Date: Wednesday, March 13, 2019
Location: Whistler Conference Centre
Poster Number: 3022

AACR Presentation Information
Title: Antibodies to SIRPα enhance innate and adaptive immune responses to promote anti-tumor activity
Session Category: Immunology
Session Title: Therapeutic Antibodies 1
Session Date and Time: Sunday, March 31, 2019 1:00 PM – 5:00 PM ET
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 23
Abstract Number: 562

On February 27, 2019 Helix BioPharma Corp. (TSX: HBP), (FSE: HBP) ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported that together with the Moffitt Cancer Center ("Moffitt"), a poster entitled "Improving survival in pancreatic cancer using Doxorubicin in combination with L-DOS47" will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2019 which is taking place on March 29 – April 3, 2019 at the Georgia World Congress Center, Atlanta, Georgia, USA.

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The presentation will describe the use of a preclinical pancreatic mouse model to study the effects of LDOS47 alone or in combination therapies. This model was generated by modifying a mouse pancreatic cancer cell line (Panc02) to express the human ceacam6 antigen that is recognized by L-DOS47. The model is designed to be used for monotherapy L-DOS47 efficacy studies and for combination studies with various agents, including immunotherapies. In addition, to therapeutic studies, the model may also be helpful in studying the effects of L-DOS47 in modifying tumor pH and the tumor immune environment. The presentation will describe the development of the cell line, generation of tumor grafts, and show responses to L-DOS47 in combination with doxorubicin treatments. The usefulness of the model, including difficulties of this model, will also be described. This work is part of an on-going collaboration with Moffitt and is being performed to support the clinical development of L-DOS47 and the DOS47 platform.

"I would like to thank Dr. Robert (Bob) Gillies and the Moffitt team on leading this work" said Heman Chao, Helix’s Chief Executive Officer. "Following on the excellent safety and tolerability results from the Phase I monotherapy lung cancer study in Poland and continuing good progress made with the U.S. Phase I LDOS47 combination study with pemetrexed and carboplatin, we are very excited to launch the pancreatic program and expand the clinical application of L-DOS47."

Presentation details are as follows:

Session Category: Experimental and Molecular Therapeutics
Session Title: Cellular Responses to Anticancer Agents 1: The Microenvironment and Metastasis
Session Date and Time: Monday April 1, 2019 from 8:00 AM-12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B.

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 MannKind Corporation (NASDAQ:MNKD) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Mannkind, FEB 26, 2019, View Source [SID1234533692]).

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Fourth Quarter Results

For the fourth quarter of 2018, total revenues were $16.0 million, reflecting Afrezza net revenue of $5.7 million and collaboration and services revenue of $10.3 million. Afrezza net revenue increased 28% on a GAAP basis compared to $4.5 million for the fourth quarter of 2017. In the fourth quarter of 2017, we recognized a $1.4 million change in estimate to Afrezza net revenue; when this adjustment is excluded, Afrezza net revenue increased 86% (non-GAAP) compared to the fourth quarter of 2017, primarily driven by higher product demand and a more favorable mix of cartridges. Collaborations and services revenue increased $10.2 million primarily attributable to the United Therapeutics licensing and research agreements.

Afrezza cost of goods sold (COGS) was $5.0 million for the fourth quarter of both 2018 and 2017. Afrezza COGS in the fourth quarter of 2018 reflected a one-time charge of $2.0 million related to an amendment fee associated with our insulin supply agreement, offset by lower inventory write-offs in 2018 of $0.8 million and $0.7 million lower spending associated with manufacturing absorption. Afrezza gross profit was $0.7 million for the fourth quarter, the first quarterly gross profit recognized from Afrezza sales. When the one-time charge of $2.0 million related to the amendment fee is excluded, Afrezza gross profit was $2.7 million (non-GAAP) for the fourth quarter.

Research and development (R&D) expenses for the fourth quarter of 2018 were $1.1 million compared to $3.5 million for the fourth quarter of 2017. The decrease of $2.4 million was primarily due to $0.8 million associated with the United Therapeutics research agreement, which was classified as a cost of collaborations and services revenue, and a decrease in spending of $0.8 million related to clinical trials.

Selling, general and administrative (SG&A) expenses were $18.0 million for the fourth quarter of 2018 compared to $23.3 million for the fourth quarter of 2017. The decrease of $5.3 million was primarily due to $5.0 million in selling expenses associated with our first direct-to-consumer television advertising campaign in the fourth quarter of 2017.

Interest expense on notes (facility financing obligation and senior convertible notes) was $0.6 million for the fourth quarter of 2018 compared to $2.1 million for the fourth quarter of 2017. The $1.5 million decrease was primarily due to a reduction in the debt principal balances.

The net loss for the fourth quarter of 2018 was $9.7 million, or $0.06 per share, compared to the $32.8 million net loss in the fourth quarter of 2017, or $0.28 per share. The lower net loss is mainly attributable to an increase in total revenues of $11.5 million and a decrease in total expenses of $9.6 million.

Full Year 2018 Results

For the full year ended December 31, 2018, total revenues were $27.9 million, reflecting Afrezza net revenue of $17.3 million and collaborations and service revenue of $10.6 million. Afrezza net revenue increased 88% compared to $9.2 million for the same period in 2017, primarily reflecting increased product demand and a more favorable mix of cartridges. Collaborations and services revenue increased $10.3 million primarily attributable to the United Therapeutics licensing and research agreements.

Afrezza COGS for the year ended December 31, 2018 was $19.4 million compared to $17.2 million for the year ended December 31, 2017. The increase of $2.2 million was primarily attributable to an increase in costs associated with increased Afrezza sales and a one-time charge of $2.0 million related to an amendment fee associated with our insulin supply agreement, offset by a decrease of $0.8 million in inventory write-offs.

R&D expenses for the year ended December 31, 2018 were $8.7 million compared to $14.1 million for the same period in 2017. This $5.4 million decrease was primarily attributable to lower clinical trials expenses of $2.2 million, a $1.7 million decrease in salary-related expenses and a $0.8 million decrease in research and development supply and services costs.

SG&A expenses were $79.7 million for the year ended December 31, 2018 compared to $75.0 million for the same period in 2017. The $4.7 million increase was primarily due to an increase of $2.9 million in headcount-related expenses associated with commercial operations, an increase in spending of $1.7 million in our human resources, accounting, corporate communications, and office support departments, a $1.4 million increase in medical affairs support, a $1.3 million increase in stock-based compensation expense, and a one-time $1.1 million expense to transition corporate support functions from Connecticut to our headquarters in California, which were partially offset by a decrease in selling expenses of $4.7 million associated with our 2017 direct-to-consumer television advertising campaign.

Interest expense on notes (facility financing obligation and senior convertible notes) was $5.1 million for the year ended December 31, 2018 compared to $9.5 million for the same period in 2017. The $4.4 million decrease was primarily due to a reduction in the debt principal balances.

The net loss for the year ended December 31, 2018 was $87.0 million, or $0.60 per share, compared to $117.3 million for the year ended December 31, 2017, or $1.13 per share. The lower net loss is mainly attributable to an increase in Afrezza net revenue of $8.1 million, an increase in collaboration revenue of $10.3 million and a decrease in total expenses of $15.5 million.

"Our fourth quarter and full year 2018 results showed excellent progress in executing against our Afrezza growth plan and recognized for the first time revenues associated with our license and collaboration agreement with United Therapeutics," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "The fourth quarter of last year was the first time we reported gross profit for Afrezza and we ended the year with a strong cash position thanks to the United Therapeutics deal and a public offering of common stock and warrants in December."

Cash and Cash Equivalents

Cash, cash equivalents and restricted cash at December 31, 2018 was $71.7 million compared to $48.4 million at December 31, 2017. The increase was primarily due to the net proceeds of $26.4 million from a second quarter registered direct offering of common stock and warrants and $37.5 million from a fourth quarter public offering of common stock and warrants, partially offset by the net cash used in operating activities of $37.7 million (inclusive of two payments from United Therapeutics totaling $55.0 million).

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

Conference Call

MannKind will host a conference call and presentation webcast to discuss these results today at 9:00 a.m. Eastern Time. To participate in the live call by telephone, please dial (888) 394-8218 or (323) 701-0225 and use the participant passcode: 7809405. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 7809405#. A replay will also be available on MannKind’s website for 14 days.

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.