On May 12, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported financial results and business highlights for the first quarter of 2021 (Press release, Hookipa Pharma, MAY 12, 2021, View Source [SID1234579783]).

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"We had a strong start to the year as we drive our pipeline forward to deliver a new class of arenavirus-based immunotherapeutics. As we shared at AACR (Free AACR Whitepaper), one initial dose of our lead oncology candidates, HB-201 or HB-202, each induced a robust increase in antigen-specific T cells, including an increase of up to 8% of antigen-specific circulating CD8+ T cells, in people with advanced Human Papillomavirus 16-positive (HPV16+) cancers," said Joern Aldag, Chief Executive Officer at HOOKIPA. "We believe these data are impressive, and they are consistent with the pre-clinical data published in Cell Reports Medicine in March. Both data sets highlight the potential of our engineered arenavirus platform to redefine success in cancer immunotherapy. As our clinical programs progress, we’re excited about the oral abstract presentation at ASCO (Free ASCO Whitepaper) (#2502) and other future data presentations at upcoming conferences."

Program Highlights

In April 2021, HOOKIPA announced positive preliminary Phase 1 immunogenicity data for its lead oncology candidates, HB-201 and HB-202, for the treatment of advanced HPV16+ cancers, reinforcing the promising anti-tumor activity reported from the Phase 1/2 clinical trial in December 2020. The preliminary immunogenicity data demonstrated a robust increase in HPV16+-specific T cells, including an increase of up to 8% of antigen-specific circulating CD8+ T cells, after one dose of HB-201 or HB-202. Early HB-201 monotherapy data also highlighted immune system activation of increasing interferon-gamma and other immune stimulatory cytokines with a single dose. The data were presented at a late-breaker poster session at the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

In March, Cell Reports Medicine published pre-clinical data on HOOKIPA’s arenaviral therapeutics. The peer-reviewed article showed that intravenous HB-201 administration induced single digit percentage of antigen-specific CD8+ T cells, while alternating administration of HB-201 and HB-202 induced a potent CD8+ T cell response, exceeded 50% of the circulating T cell pool. The two-vector cancer therapeutic approach also resulted in tumor cures and long-term immunity in a pre-clinical setting.

HOOKIPA’s prophylactic Cytomegalovirus, or CMV, vaccine candidate, HB-101, continued to enroll patients awaiting kidney transplantation in a Phase 2 clinical trial. We expect to conclude trial enrollment in mid-2021 and to report additional safety, immunogenicity, and efficacy data from evaluable patients in the second half of 2021.
Upcoming Milestones

Oral abstract presentation at ASCO (Free ASCO Whitepaper) (#2502 at 3:00pm EDT on June 7): First report of the safety/tolerability and preliminary antitumor activity of HB-201 and HB-202, an arenavirus-based cancer immunotherapy, in patients with HPV16+ cancers
Initial HB-201/HB-202 Phase 1/2 efficacy data in HPV16+ cancers in mid-2021
Additional HB-101 CMV Phase 2 efficacy data in H2 2021
Advancing our HB-300 to IND for the treatment of metastatic prostate cancer
HBV and HIV collaboration with Gilead Sciences advancing towards clinical studies
First Quarter 2021 Financial Results

Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2021 was $128.1 million compared to $143.2 million as of December 31, 2020. The decrease was primarily attributable to cash used in operating activities.

Revenue was $5.3 million for the three months ended March 31, 2021, and $3.7 million for the three months ended March 31, 2020. The increase was primarily due to higher cost reimbursements received under the Collaboration Agreement with Gilead and the recognition of cost reimbursements initially recognized as deferred revenue.

Research and Development Expenses: HOOKIPA’s research and development expenses were $20.2 million for the three months ended March 31, 2021, compared to $11.5 million for the three months ended March 31, 2020.

The primary drivers of the increase in direct research expenses were an increase in clinical trial expenses of $1.5 million and an increase in manufacturing and quality control expenses of $4.6 million.

The increase was mainly due to the progress in our HB-201 and HB-202 clinical trial, the increased patient recruitment in our HB-201 and HB-202 clinical trial, monitoring and testing activities and manufacturing and quality control work in preparation of a further extension of the trial. Manufacturing and quality control expenses were also driven by the progress towards clinical development in our Gilead partnered programs. This increase in HB-201/HB-202 and Gilead related direct expenses was partially offset by a decrease in direct costs related to our HB-101 program due to slower patient recruitment as a result of the COVID pandemic.

The increase in internal research and development expenses was mainly due to an increase of personnel-related research and development expenses, resulting primarily from a higher headcount, while stock-based compensation expenses included in personnel-related research and development expenses decreased. In addition, an increase in facility related costs and an increase in other internal costs contributed to the overall increase in internal research and development expenses.

General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2021 were $4.3 million, compared to $4.6 million for the three months ended March 31, 2020. The decrease was primarily due to a decrease in personnel-related expenses, partially offset by an increase in professional and consulting fees. The decrease in personnel-related expenses resulted from decreased stock compensation expenses, partially offset by increased salaries and a growth in headcount in our general and administrative functions.

Net Loss: HOOKIPA’s net loss was $17.2 million for the three months ended March 31, 2021 compared to a net loss of $10.9 million for the three months ended March 31, 2020. This increase was due to an increase in research and development expenses, partially offset by an increase in revenues from collaboration and licensing, a decrease in general and administrative expenses, and an increase in grant income.

On May 12, 2021 Qu Biologics Inc., a private clinical stage biopharmaceutical company developing Site Specific Immunomodulators (SSIs), a novel platform of immunotherapies designed to restore innate immune function, reported that expanded its patent portfolio to include the perioperative use of Qu’s SSIs to overcome immune dysfunction in patients undergoing cancer surgery (Press release, Qu Biologics, MAY 12, 2021, View Source [SID1234579799]).

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It has long been observed that "immune paralysis" is a phenomenon that occurs following the physical stress of surgery performed for the removal of cancer. During this post-surgical period, cells of the immune system are suppressed, and this immune suppression provides a dangerous window of opportunity for any cancer cells that have been left behind to spread or metastasize unchecked. As a result, post-operative immune dysfunction can lead to cancer recurrence in cancer patients who have undergone surgery to remove their tumours. There are currently no effective treatments that prevent or relieve this post-surgical immune suppression, leaving patients vulnerable to cancer recurrence and metastasis.

The unique ability of Qu’s SSIs to simultaneously program multiple important immune cells to effectively fight malignancy was hypothesized to offer a potential solution for this critical problem. To test this hypothesis, Qu scientists worked with Dr. Rebecca Auer, a world renown surgical oncologist at the Ottawa Hospital Research Institute, whose research is focused on determining how to overcome post-surgical immune dysfunction to improve outcomes of cancer patients. Preclinical studies performed in Dr. Auer’s lab showed that perioperative administration of Qu’s lung-directed SSI preserved anti-cancer immunity and markedly reduced lung metastases in a surgical model.

Dr. Auer is enthusiastic about the prospect of leading a clinical trial to test the perioperative application of SSIs to improve cancer outcomes, "Post-surgical immune suppression continues to present a critical challenge in oncology. To date very few trials have been conducted in the perioperative period, specifically designed to alleviate this immune dysfunction, which can adversely impact outcomes for patients having undergone cancer excision surgery. Given the large number of patients who undergo surgery for cancer every year, this is a significant unmet clinical need. I am hopeful that the SSI approach can transform perioperative management of cancer patients." Immunologist Dr. Shirin Kalyan, Qu’s Vice President of Scientific Innovation, said, "We have previously shown that SSI treatment, given in the absence of surgery, overcomes cancer-induced immune suppression through innate immune training and NK cell activation, and increases susceptibility of cancer cells to be targeted by immune effector cells. The new evidence from Dr. Auer’s lab that shows perioperative SSI treatment can also serve to overcome post-surgical immune suppression is really akin to hitting two birds with one stone for cancer immunotherapy."

Dr. Hal Gunn, Qu’s CEO, said, "We are very excited about this important discovery. Since the large majority of cancer patients undergo surgery, perioperative SSI treatment has the potential to transform outcomes for cancer surgery patients. Qu looks forward to working with Dr. Auer and her colleagues in conducting a Phase 2 clinical trial to assess the unique potential of SSIs in the context cancer surgery and we welcome discussions with potential investors and licensing partners."

On May 12, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported an abstract presentation related to AUTO1 in relapsed / refractory (r/r) indolent B cell lymphomas (IBCL) at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress to be held June 9-17, 2021 (Press release, Autolus, MAY 12, 2021, View Source [SID1234579817]).

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Title: Early safety and efficacy findings of AUTO1 (CAT19), a fast-off rate CD19 CAR, in Relapsed/Refractory Indolent B Cell Lymphomas

Presenter: Clare Roddie, MD, PhD, FRCPath, Consultant Haematologist and Honorary Senior Lecturer, Cancer Institute, University College London (UCL)

Date and Time: All e-poster presentations will be made available on the on-demand Virtual Congress platform as of Friday, June 11 at 9.00 AM CEST.

As of the data cut-off date of February 18, 2021, 10 r/r IBCL patients had received AUTO1 and nine patients were evaluable. AUTO1 demonstrated a tolerable safety profile in adult patients with r/r low grade B-cell lymphoma despite high disease burden. Early data shows 100% complete remission rates and excellent CAR engraftment and expansion. Grade 1 CRS was reported in 4 patients and Grade 2 CRS in 1 patient. No Immune effector cell-associated neurotoxicity syndrome (ICANS) was observed on study. At a median of 3.1 months (range 1-5.6m), 8/9 patients are in ongoing remission. One patient died in complete remission at month 5.6 of COVID-19.

On May 12, 2021 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, reported that the company’s Hong Kong subsidiary, Pangu BioPharma Limited (Pangu), together with the Hong Kong University of Science and Technology (HKUST), have achieved the milestones set forth for the first year of a $750,000 grant received from the Hong Kong Government’s Innovation and Technology Commission (ITC) (Press release, aTyr Pharma, MAY 12, 2021, View Source [SID1234579834]). The two-year project, which is in part funded by the ITC’s Partnership Research Program (PRP), is intended to develop a high-throughput platform for the development of bispecific antibodies with an initial focus on diseases in which Neuropilin-2 (NRP2) overexpression is strongly implicated, including cancer.

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Key milestones achieved for the first year of the project included building out a highly skilled research team to establish an innovative antibody discovery platform at HKUST. An integral part of this project was the development and implementation of a novel single-cell antibody discovery approach which has so far yielded numerous candidate high-affinity NRP2/co-receptor antibodies that are currently being screened in functional assays. The second year of the project aims to identify the most productive pairings, optimize mid-scale production/purification and prioritize lead candidate bispecific antibodies based on activity in therapeutically relevant cell-based assays.

"We are very pleased with the progress of Pangu and HKUST in the first year of this important project," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "We continue to learn more about NRP2 as a target for diseases, including immunology and cancer. Based on its role in regulating inflammatory responses and interaction with various co-receptors, we believe there are several potential options to therapeutically target NRP2 and that bispecific antibodies present a unique approach to create highly-specific agonists of this system which may be therapeutically relevant in certain disease states. We look forward to the outcome from the second year of the project."

Mingjie Zhang, Ph.D., Chair Professor of the Division of Life Science and Kerry Holdings Professor of Science at HKUST and project coordinator of the Pangu collaboration, commented, "We are excited to have implemented this new antibody discovery platform as part of the collaboration between Pangu and HKUST. We look forward to utilizing the additional capabilities related to bispecific antibody development that this platform supports to enable us to achieve our goals in the second year of the project."

About NRP2

Neuropilin-2 (NRP2) is a cell surface receptor that plays a key role in lymphatic development and in regulating inflammatory responses. In many forms of cancer, high NRP2 expression is associated with worse outcomes. NRP2 can interact with multiple ligands and co-receptors through distinct domains to influence their functional roles, making it a potential drug target with multiple distinct therapeutic applications. NRP2 interacts with type 3 semaphorins and plexins to impact inflammation and with forms of vascular endothelial growth factor (VEGF) and their receptors, to impact lymphangiogenesis. In addition, NRP2 modulates interactions between CCL21 and CCR7 potentially impacting homing of dendritic cells to lymphoid organs. aTyr is currently investigating NRP2 receptor biology, both internally and in collaboration with key academic thought leaders, as a novel target for new product candidates for a variety of diseases, including cancer and inflammation.

On May 12, 2021 Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported financial results for the first quarter ended March 31, 2021 (Press release, TapImmune, MAY 12, 2021, View Source [SID1234579880]).

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"We had a productive first quarter supported by our recently completed financing, strengthening our overall cash position and enabling continued growth and expansion of our Multi-TAA pipeline," said Peter L. Hoang, President & CEO of Marker Therapeutics. "In addition, we continue to make strong progress on both the clinical and manufacturing fronts. In March, we dosed the first patient in the safety lead-in portion of our Phase 2 trial in post-transplant acute myeloid leukemia, or AML, and continue to activate clinical sites. In parallel, we continue to optimize the MT-401 cell therapy manufacturing process, which we believe could result in an increase in the number of T cells available for patient administration—among other benefits—as we prepare to operationalize our new in-house cGMP facility in the first half of the year."

PROGRAM UPDATES

In March 2021, Marker dosed the first patient in the safety lead-in portion of its Phase 2 trial in AML, which is expected to enroll a total of six patients: three of which will be treated with MT-401 manufactured with a legacy reagent, and the remaining three to be treated with study drug manufactured with a new reagent from an alternate supplier.
The clinical operations team has made considerable progress in opening sites to enroll patients for the safety lead-in portion of the AML trial. The Company has also received commitments from additional clinical sites to participate in the Phase 2 AML trial following the safety lead-in phase and anticipates activating a total of approximately 20 sites.
Marker continues to streamline and simplify the MT-401 manufacturing process, which could potentially result in a product with a superior T cell phenotype and improved antigen specificity as compared to the original process. The new process improvements have been updated in the CMC section of the IND and will be used for all patients in the Marker AML Phase 2 clinical trial.
Marker presented early results of robotic automation of T cell generation for the treatment of AML at the American Society of Gene & Cell Therapy 24th Annual Meeting on May 11, 2021. The results will also be presented at the upcoming International Society for Cell & Gene Therapy 2021 Annual Meeting, taking place virtually May 26-28, 2021.
BUSINESS UPDATES

In March, Marker closed an underwritten public offering of 32,282,857 shares of its common stock at a public offering price of $1.75 per share. The gross proceeds to Marker from the offering, before deducting the underwriting discounts and commissions and other offering expenses, were approximately $56.5 million.
The Company is preparing to open its new cGMP manufacturing facility in Houston, TX, which will be used to manufacture study drug for Marker’s Phase 2 AML trial (MT-401) and for future hematological and solid tumor trials, in addition to the potential commercialization of any approved products. Marker initiated the technology transfer from Baylor College of Medicine to the cGMP facility in Q1 2021 and expects the facility to be fully operational in the first half of 2021.
ANTICIPATED PROGRAM MILESTONES

AML Trial Milestones

Complete safety lead-in (6 patients) in Q2 2021
Initiate main portion of Phase 2 trial in Q3 2021
Complete enrollment of 20 patients in main portion of Phase 2 trial in Q4 2021
Topline readout of Group 2 (active disease) in Q1 2022
Manufacturing Milestones

Receive regulatory approval for Marker cGMP in Q2 2021
Manufacture MT-401 at Marker cGMP for Phase 2 AML trial in Q3 2021
FIRST QUARTER 2021 FINANCIAL RESULTS

Cash Position and Guidance: At March 31, 2021, Marker had cash and cash equivalents of $64.5 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses and capital expenditure requirements into Q1 2023.
R&D Expenses: Research and development expenses were $5.6 million for the quarter ended March 31, 2021 compared to $3.8 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in headcount-related expenses and infrastructure expenses due to growth of research and development operations.
G&A Expenses: General and administrative expenses were $3.1 million for the quarter ended March 31, 2021 compared to $2.8 million for the quarter ended March 31, 2020.
Net Loss: Marker reported a net loss of $8.8 million for the quarter ended March 31, 2021, compared to a net loss of $6.5 million for the quarter ended March 31, 2020.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its first quarter 2021 financial results and provide a corporate update today at 5:00 p.m. EDT. The webcast will be accessible in the Investors section of the Company’s website at markertherapeutics.com. Individuals can participate in the conference call by dialing 877-407-8913 (domestic) or 201-689-8201 (international) and referring to the "Marker Therapeutics First Quarter 2021 Earnings Call."