On March 1, 2021 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) for the treatment of eosinophil and mast cell-related diseases, reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a business update (Press release, Allakos, MAR 1, 2021, View Source [SID1234575828]).

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!

2020 Accomplishments

Announced positive results from our prospective prevalence study showing that 45% (181/405) of symptomatic patients biopsied with chronic unexplained gastrointestinal (GI) symptoms or functional gastrointestinal disorders (FGIDs) such as irritable bowel syndrome (IBS) and functional dyspepsia (FD) met the histologic criteria for eosinophilic gastritis (EG) and/or eosinophilic duodenitis (EoD). The results suggest that EG and/or EoD are significantly underdiagnosed among these patients. Millions of patients in the U.S. are under the care of a gastroenterologist and suffer from chronic unexplained gastrointestinal symptoms or FGIDs. These results provide evidence that prevalence of EG and EoD is significantly higher than reported in the literature.
Announced positive safety, pharmacokinetic and pharmacodynamic results from a randomized, double-blind, placebo-controlled Phase 1 study of subcutaneous (SC) lirentelimab in healthy volunteers. Bioavailability of SC lirentelimab was 63% and SC lirentelimab resulted in extended eosinophil suppression at all dose levels tested. At dose levels of 3.0 and 5.0 mg/kg and with the fixed dose of 300 mg, SC lirentelimab resulted in eosinophil suppression in all subjects through Day 85. The pharmacokinetic and pharmacodynamic results suggest that SC lirentelimab may be given monthly or potentially less frequently. SC lirentelimab was well tolerated, and there were no serious adverse events, no injection site reactions and no infusion-related reactions with SC lirentelimab.
Published results from a Phase 2 study of lirentelimab in patients with EG and/or EoD (ENIGMA) in the New England Journal of Medicine.
Announced positive interim results from an open-label long term extension study of ENIGMA. The results were accepted for oral presentation and presented virtually at the Digestive Disease Week (DDW) Annual Meeting.
Closed an underwritten public offering, issuing 3,506,098 shares of common stock at an offering price of $82.00 per share. Aggregate net proceeds received from the offering were approximately $271.7 million, after deducting underwriting discounts and commissions.
Upcoming 2021 Milestones

Topline data from a randomized, double-blind, placebo-controlled Phase 3 study of lirentelimab in patients with EG and/or EoD expected in the fourth quarter of 2021.
Topline data from a randomized, double-blind, placebo-controlled Phase 2/3 study of lirentelimab in patients with eosinophilic esophagitis (EoE) expected in the fourth quarter of 2021.
Initiation of a randomized, double-blind, placebo-controlled Phase 3 study of lirentelimab in patients with EoD expected in the second quarter of 2021.
Initiation of a randomized, double-blind, placebo-controlled Phase 2/3 study of SC lirentelimab in patients with EG and/or EoD expected in the second half of 2021.
Fourth Quarter and Full Year 2020 Financial Results

Research and development expenses were $28.5 million in the fourth quarter of 2020 as compared to $16.6 million in the same period in 2019, an increase of $11.9 million. Research and development expenses were $105.5 million for the full year 2020 as compared to $61.9 million in the same period in 2019, an increase of $43.6 million.

General and administrative expenses were $15.8 million in the fourth quarter of 2020 as compared to $10.3 million in the same period in 2019, an increase of $5.5 million. General and administrative expenses were $51.5 million for the full year 2020 as compared to $29.6 million in the same period in 2019, an increase of $21.9 million.

Allakos reported a net loss of $44.3 million in the fourth quarter of 2020 as compared to $24.6 million in the same period in 2019, an increase of $19.7 million. Net loss per basic and diluted share was $0.86 for the fourth quarter of 2020 compared to $0.51 in the same period in 2019. Net loss was $153.5 million for the full year 2020 as compared to $85.4 million in the same period in 2019, an increase of $68.1 million. Net loss per basic and diluted share was $3.10 for the full year 2020 compared to $1.89 in the same period in 2019.

Allakos ended the fiscal year 2020 with $659.0 million in cash, cash equivalents and marketable securities.

On March 1, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported it has entered into a clinical development collaboration with AgonOx, Inc. to develop novel T cell-based cancer immunotherapies using Phio’s lead INTASYL based product candidate PH-762 and AgonOx’s "double positive" (DP) tumor-infiltrating lymphocyte (TIL) technology (Press release, Phio Pharmaceuticals, MAR 1, 2021, View Source [SID1234575827]). The companies have shown that the combination of their respective technologies can result in enhanced TIL therapeutics, and based on these data, the collaboration will focus on conducting a clinical study for PH-762 treated DP TILs. The study is expected to start enrolling patients later this year.

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!

AgonOx in collaboration with the Earle A. Chiles Research Institute, a division of the Providence Cancer Institute, developed a method for the identification, isolation and expansion of tumor-specific CD8 T cells from cancer patients. AgonOx has also shown that "double positive" (DP) CD8 T cells isolated from human solid tumors have increased tumor killing activity when compared to CD8 TIL that were not enriched prior to expansion. Preclinical data presented at SITC (Free SITC Whitepaper) 2020 by AgonOx in collaboration with Phio show that treating the DP CD8 TIL with Phio’s PH-762, increases the tumor killing activity of the CD8 DP TIL even further (two-fold increase). As a result, the use of PH-762 treated DP CD8 TIL is expected to enhance therapeutic responses in cancer patients.

Under the terms of the collaboration agreement, AgonOx will receive financial support for the clinical trial from Phio and Phio is entitled to certain future development milestones and sales related royalty payments from AgonOx’s DP TIL technology.

"Autologous T cell therapies hold a lot of promise, however, there is still a lot of research needed to be done to unlock its full therapeutic potential, including ways to improve upon the first generation of TIL products, and ways to use TIL therapy in more types of cancer," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals. "By joining forces with AgonOx, we believe our collaboration can fulfil these unmet needs, without the need for complex and costly technologies, such as genetic engineering."

"Our collaboration with Phio is based on data showing that PH-762 increases the activity of our CD8 DP TIL technology, therefore we believe this combination should increase the therapeutic efficacy of this first-in-man study," stated by Dr. Andrew Weinberg, President/CSO of AgonOx, Inc. and Full Member at the Earle A. Chiles Research Institute.

Dr. James Cardia, VP of Business Operations of Phio Pharmaceuticals, commented: "Both the clinical community and the investment community are embracing the broad potential of cell-based immunotherapy, and TIL therapy in specific, based on recent clinical and corporate development activities in this field. Our data show the important role that INTASYL based products can play in improving adoptive cell therapy, and we look forward to working with AgonOx to bring better cell therapies to patients."

About TIL Therapy and "DP" TILs

In TIL therapy, T cells are extracted from a patient’s own tumor. These cells are then expanded ex-vivo, and infused back into the patient. The infused TIL naturally recognize the tumor and then attack the cancer cells. Currently, clinical applications focus on indications such as metastatic melanoma and cervical cancer. Increasing the frequency of tumor-reactive cells within TIL products should allow for improved response rates in these indications and expand the use of this form of treatment to patients with locally advanced, recurrent or metastatic cancers including head and neck, non-small cell lung cancer and other solid tumor types. AgonOx developed methods to enrich for tumor-killing T cells, which could greatly improve the immune response in cancer patients – potentially leading to successful treatment of tumors. Their research in collaboration with the Earle A. Chiles Research Institute, entitled "Co-expression of CD39 and CD103 identifies tumor-reactive CD8 positive T cells in human solid tumors" was published in Nature Communications and forms the basis of this clinical study (Duhen, T., Duhen, R., Montler, R. et al, 2018: Co-expression of CD39 and CD103 identifies tumor-reactive CD8 T cells in human solid tumors. Nat Commun 9, 2724).

About PH-762

PH-762 is a self-delivering RNAi compound that targets the checkpoint protein PD-1. Checkpoint proteins, such as PD-1, normally act as a type of "off switch" that prevents T cells from attacking certain cells, such as cancer cells, in the body. PH-762 silences PD-1 checkpoint expression, thereby removing the "off switch" and resulting in enhanced T cell activation and tumor cytotoxicity. Experimental data shows that PH-762 can silence the expression of PD-1 in target human T cells in a potent and durable manner, and can increase function of patient derived TILs or engineered cells, such as CAR T-cells for use in adoptive cell therapy. PH-762 use does not involve genetic engineering, and its cell delivery does not require special formulations or other complex delivery tools.

On March 1, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that Jeff Baxter, President and Chief Executive Officer, and David E. Anderson, Ph.D., Chief Scientific Officer, will participate in an analyst-led fireside chat at the Raymond James 42nd Annual Institutional Investors Conference on Tuesday, March 2, 2021, at 4:40 PM ET (Press release, VBI Vaccines, MAR 1, 2021, View Source [SID1234575815]).

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!

Presentation Details

Event: Raymond James 42nd Annual Institutional Investors Conference
Presenters: Jeff Baxter, President and CEO, and David E. Anderson, Ph.D., Chief Scientific Officer
Date: Tuesday, March 2, 2021
Time: 4:40 – 5:20 PM ET
Webcast: View Source

A recording of the webcast will be available on the "Events and Presentations" page of the Company’s website: View Source

On March 1, 2021 Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the full year 2020 (Press release, Morphic Therapeutic, MAR 1, 2021, View Source [SID1234575814]).

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!

2020 and Recent Corporate Highlights

Filed first IND of an oral integrin drug candidate, MORF-057, generated by Morphic’s MInT Platform and initiated first clinical study of MORF-057 in healthy volunteers in Phase 1 clinical trial of oral IBD candidate, MORF-057, after acceptance of IND by Food and Drug Administration
Announced positive preliminary results from Phase 1 single ascending dose portion of MORF-057 Phase 1 clinical trial including:
MORF-057 well tolerated in all five dose cohorts ranging from 25 mg to 400 mg
MORF-057 achieved greater than 95% mean receptor occupancy of α4β7 at the three highest dose levels
Phase 1 multiple ascending dose and food effect trials ongoing with full data anticipated to be presented mid-year 2021
Received $20 million payment upon AbbVie exercise of a license option under the companies’ research and development collaboration agreement to develop Morphic’s αvβ6 integrin inhibitors, including the compounds MORF-720 and MORF-627
Expanded research and development collaboration with Janssen through a third integrin program
Presented promising preclinical data supporting MORF-057 as an oral integrin targeting α4β7 at UEG Week 2020, Digestive Disease Week 2020, European Crohn’s and Colitis Organization (ECCO)
Advanced novel integrin-targeted candidates generated by the MInT Platform against integrins αvβ1 and αvβ8 for the treatment of fibrosis and cancer
Ended the year with $228.3 million in cash and equivalents and marketable securities, providing runway into 2023
"The past year challenged how we work and live but the Morphic team came together to drive tremendous advances in the creation of oral integrin therapies. Most notably in 2020, our lead oral candidate in IBD, MORF-057, completed preclinical testing with strong proof-of-concept and entered the clinic. In an important milestone for Morphic, we have already delivered positive preliminary results from the MORF-057 Phase 1 trial. The data show a favorable tolerability profile as well as strong pharmacodynamic data that suggest α4β7 inhibition may be on par with the approved intravenous blockbuster, vedolizumab. Further, we expanded our strategic collaborations with AbbVie and Janssen to explore a broader scope of integrin drug targets and potentially boost our partnered pipeline," commented Praveen Tipirneni, M.D., president and chief executive officer of Morphic Therapeutic. "In the year ahead, with a strong financial base, we are able to focus on advancing the clinical development of MORF-057 and our promising preclinical programs targeting αvβ1 and αvβ8, as well as continuing to expand the MInT Platform that generates this pipeline of novel integrin therapeutic candidates."

Financial Results for the Full Year 2020

Net loss for the year ended December 31, 2020, was $45.0 million or $1.47 per share compared to a net loss of $43.3 million or $2.69 per share
Revenue was $44.9 million for the year ended December 31, 2020 compared to $17.0 million for the year ended December 31, 2019. The increase was mainly due to AbbVie’s option exercise on our αvβ6 integrin inhibitor program in the third quarter of 2020 for $20 million
Research and development expenses were $73.6 million for the year ended December 31, 2020 as compared to $53.7 million for the year ended December 31, 2019. The increase was primarily attributable to higher development and manufacturing costs associated with our lead product candidates, MORF-057 and MORF-720, as well as increased personnel-related costs to support continued progress with the company’s pipeline
General and administrative expenses were $18.5 million for the year ended December 31, 2020, compared to $10.2 million for the year ended December 31, 2019. The increase was primarily attributable to increased headcount and higher professional and consulting fees associated with ongoing business activities and Morphic’s operating as a public company
As of December 31, 2020, Morphic had cash, cash equivalents and marketable securities of $228.3 million, compared to $237.0 million as of December 31, 2019. Morphic believes its cash, cash equivalents and marketable securities as of December 31, 2020, will be sufficient to fund operating expenses and capital expenditure requirements into 2023.

On March 1, 2021 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported that it will report its fourth quarter and full year 2020 financial and operating results on Thursday, March 11, 2021, following the close of the U.S. financial markets (Press release, Lineage Cell Therapeutics, MAR 1, 2021, View Source [SID1234575813]). Lineage management will also host a conference call and webcast on Thursday, March 11, 2021, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its fourth quarter and full year 2020 financial and operating results and to provide a business update.

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!

Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through March 19, 2021, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 4176568.