On May 16, 2022 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 2022 (Press release, Hookipa Biotech, MAY 16, 2022, View Source [SID1234614590]).

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"We observed strong external validation of our novel arenaviral platform in the first quarter with our collaboration agreement with Gilead and our capital raise, which drew funding from new and existing top-tier investors," said Joern Aldag, Chief Executive Officer at HOOKIPA. "On the heels of our AACR (Free AACR Whitepaper) data presentations, which showcased T cell and tumor response with our technology alone and in novel combinations, we remain focused on advancing our portfolio across cancer types. We look forward to sharing Phase 1 data from our HB-200 program mid-year and early Phase 2 data on HB-200 in combination with pembrolizumab in late 2022, as well as progressing with IND preparations for our HB-300 program in prostate cancer."

Quarter Highlights

In January 2022, the first patient was dosed in the Phase 2 trial to assess HB-200 in combination with pembrolizumab as 1st-line and 2nd-line treatment for advanced head and neck squamous cell carcinoma (HNSCC). Results from the ongoing Phase 1 study have highlighted the potential additive benefits of this combination to improve anti-tumor response. Preliminary data is anticipated in the second half of 2022.

In February 2022, HOOKIPA and Gilead agreed to advance its partnered HIV program, triggering a $54 million commitment from Gilead. HOOKIPA assumed development responsibility for the HB-500 program through the completion of a Phase 1b clinical trial; Gilead has the exclusive right for further development thereafter. Financial terms included a $4 million preclinical milestone, a $15 million non-refundable initiation fee and $35 million equity commitment at a premium to market price. The $35 million equity commitment includes a first tranche of $5 million (purchased at a $3 share price on February 15) and the remaining $30 million can be drawn at a 30 percent premium in a second tranche or at market price in a third tranche. If Gilead pursues further development, HOOKIPA is entitled to potential development and sales milestone payments exceeding $237 million, as well as royalties on net product sales.

Klaus Orlinger, Ph.D. was named Chief Scientific Officer. He was promoted from his previous role as Executive Vice President, Research. Klaus has played a leading role in the development of novel arenaviral immunotherapies and advancing them to the clinic since he joined the company in 2012.

In March 2022, HOOKIPA announced the acceptance of four poster presentations on preclinical, translational and clinical biomarker data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April. The data provided further evidence of the potential of the arenaviral platform in various cancers, either alone or with other modalities. Specifically, the data showed:

the combination of co-stimulatory 4-1BB agonists with arenaviral immunotherapy in a preclinical setting increased tumor control and resulted in a higher cure rate than arenaviral immunotherapy alone;
replicating immunotherapy sequentially combined with adoptively transferred TCR transgenic T cells resulted in tumor cures in a preclinical setting;
arenaviral immunotherapy was able to overcome immune tolerance, induce potent T cell responses against two different tumor self-antigens and reduce tumor growth in these cancers in a preclinical setting;
HB-200 induced robust antigen-specific T cells that were high quality, expanding on previously reported data in patients with Human Papillomavirus 16-positive (HPV16+) head and neck cancer. Additional Phase 1 data, including the recommended Phase 2 dose for HB-202/HB-201 was recently accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June.

In April, HOOKIPA reported the addition of Tim Reilly, Ph.D. to its Board of Directors. Tim brings extensive product development experience to the Board.
Upcoming Milestones

Phase 1 HB-200 data in HPV16+ head and neck cancer: Mid-2022
Phase 2 HB-200 data in combination with pembrolizumab in HPV16+ head and neck cancer:
1st-line initial data: 2H 2022
2nd-line initial data: 2H 2022
Randomized Phase 2 HB-200 study in combination with pembrolizumab in 1st line for HPV16+ HNSCC: 1H 2023 (Fast Track designation)
Prostate cancer IND: 3Q 2022
Hepatitis B therapeutic IND: 2022 (Gilead-led)
First Quarter 2022 Financial Results
Cash Position: HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2022 was $141.8 million compared to $66.9 million as of December 31, 2021. The increase was primarily attributable to funds resulting from the amended and restated Gilead collaboration agreement, and the follow-on financing in March 2022, partly offset by cash used in operating activities.

Revenue was $1.4 million for the three months ended March 31, 2022, and $5.3 million for the three months ended March 31, 2021. The decrease was primarily due to lower cost reimbursements received under the Collaboration Agreement with Gilead and the fact that the $4.0 million milestone payment and the $15.0 million initiation fee received in the three months ended March 31, 2022 were mostly recorded as deferred revenue to be recognized in future accounting periods.

Research and Development Expenses: HOOKIPA’s research and development expenses were $16.6 million for the three months ended March 31, 2022, compared to $20.2 million for the three months ended March 31, 2021.

The decrease for the three months ended March 31, 2022 compared to the three months ended March 31, 2021 was attributable to a decrease in direct research and development expenses, partially offset by an increase in indirect research and development expenses.

The decrease in direct research and development expenses was primarily driven by lower manufacturing expenses for our HB-200 and Gilead partnered programs and lower clinical study expenses due to the completion of patient enrollment of the Phase 2 study for our HB-101 program. Indirect research and development expenses increased slightly because of an increase in professional and consulting fees, partially offset by a decrease in personnel related costs.

General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2022 were $5.0 million, compared to $4.3 million for the three months ended March 31, 2021. The increase was primarily due to an increase in professional and consulting fees, and an increase in personnel-related expenses, partially offset by a decrease in other expenses. The increase in personnel-related expenses resulted from increased stock compensation expenses, a growth in headcount along with increased salaries in our general and administrative functions. The increase in professional and consulting fees was primarily attributable to intellectual property costs incurred in connection with filing and prosecuting patent applications as well as third-party license fees.

Net Loss: HOOKIPA’s net loss was $18.0 million for the three months ended March 31, 2022 compared to a net loss of $17.2 million for the three months ended March 31, 2021. This increase was due to a decrease in revenues from collaboration and licensing, a decrease in grant income, an increase in general and administrative expenses, partially offset by a decrease in research and development expenses.

On May 16, 2022 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the first quarter ended March 31, 2022 and provided recent corporate updates (Press release, Galera Therapeutics, MAY 16, 2022, View Source [SID1234614589]).

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"We are excited to announce our plan to submit an NDA for avasopasem by year end following discussions with the FDA," said Mel Sorensen, M.D., Galera’s President and CEO. "Based on the positive data readout from our Phase 3 ROMAN trial, Galera continues to execute on its development strategy to advance its lead program, avasopasem, toward potential commercialization. To that end, we are delighted to attend the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting where our ROMAN data will be highlighted in an oral presentation. In addition, we recently announced positive topline data from our Phase 2a AESOP study of avasopasem for chemoradiotherapy-induced esophagitis in patients with lung cancer. We are encouraged by these results, which further demonstrate avasopasem’s ability to reduce radiation toxicity in high-risk patient populations."

Recent Corporate Updates

Radiotherapy-Induced Toxicity Programs:

Severe Oral Mucositis (SOM)

The Company announced plans to submit a New Drug Application (NDA) for avasopasem, its lead product candidate, for the treatment of radiotherapy-induced SOM to the U.S. Food and Drug Administration (FDA) by the end of 2022.
An abstract on the Phase 3 ROMAN data of avasopasem for SOM was accepted for an oral presentation on June 3rd at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Esophagitis

The Company reported positive topline data from the Phase 2a AESOP trial of avasopasem evaluating its ability to reduce the incidence of severe acute radiation-induced esophagitis in patients with lung cancer receiving concurrent chemoradiotherapy. Overall, avasopasem was well tolerated and the incidence of Grade 3 esophagitis was substantially reduced in comparison to literature. No patients experienced Grade 4 or 5 esophagitis at any point during the trial.
Anti-Cancer Programs:

Locally Advanced Pancreatic Cancer (LAPC)

Enrollment is ongoing in the Phase 2b, 160-patient randomized, multicenter, placebo-controlled GRECO-2 trial of rucosopasem, Galera’s second dismutase mimetic product candidate, in combination with stereotactic body radiation therapy (SBRT) in patients with LAPC. The primary endpoint of the trial is overall survival.
A Trials in Progress abstract on GRECO-2 was accepted for presentation at the upcoming 2022 ASCO (Free ASCO Whitepaper) Annual Meeting.
Non-Small Cell Lung Cancer (NSCLC)

Enrollment is ongoing in the Phase 1/2 GRECO-1 trial of rucosopasem in combination with SBRT in patients with NSCLC. The Company expects to report initial data from this trial in the first half of 2022.
First Quarter 2022 Financial Highlights

Research and development expenses were $8.1 million in the first quarter of 2022, compared to $12.4 million for the same period in 2021. The decrease was primarily attributable to a decrease in avasopasem development costs, partially offset by an increase in rucosopasem development costs.
General and administrative expenses were $5.0 million in the first quarter of 2022, consistent with the first quarter of 2021.
Galera reported a net loss of $(15.4) million, or $(0.58) per share, for the first quarter of 2022, compared to a net loss of $(18.7) million, or $(0.75) per share, for the same period in 2021.
As of March 31, 2022, Galera had cash, cash equivalents and short-term investments of $60.9 million. Galera expects that its existing cash, cash equivalents and short-term investments will enable Galera to fund its operating expenses and capital expenditure requirements into the second half of 2023.

On May 16, 2022 Flamingo Therapeutics, a biotechnology company pioneering RNA-targeting therapies in oncology, reported that Mike Garrett, Chief Executive Officer, will present a company overview at the BioEquity Europe and Knowledge for Growth meetings, taking place May 16-18 in Milan, Italy and May 18-19 in Ghent, Belgium (Press release, Flamingo Therapeutics, MAY 16, 2022, View Source;utm_medium=rss&utm_campaign=flamingo-therapeutics-to-present-at-the-bioequity-europe-and-knowledge-for-growth-conferences-in-may-2022 [SID1234614588]). The Flamingo management team will also participate in one-on-one meetings with investors during both conferences.

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The presentations for both conferences will be available on-demand for registered attendees.

On May 16, 2022 Emergent BioSolutions Inc. (NYSE: EBS) reported that it has entered into a definitive agreement with Chimerix, Inc. (NASDAQ: CMRX), to acquire Chimerix’s exclusive worldwide rights to TEMBEXA (brincidofovir), the first antiviral approved by the U.S. Food and Drug Administration (FDA) for all age groups for the treatment of smallpox (Press release, Emergent BioSolutions, MAY 16, 2022, View Source [SID1234614587]). TEMBEXA was approved in June 2021 and is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates.

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"The addition of TEMBEXA to Emergent’s portfolio of medical countermeasures builds upon our core capabilities and leverages our long and successful history partnering with the U.S. government to address dangerous public health threats," said Robert G. Kramer, president and CEO of Emergent. "It exemplifies our thoughtful M&A strategy as part of our 2024 growth plan and positions us better to deliver value for our shareholders."

"This transaction expands and further diversifies our medical countermeasures business with the addition of a small molecule therapeutic that aligns with the government’s smallpox preparedness strategy," said Paul Williams, SVP government/MCM business at Emergent. "It is expected to be accretive upon first product delivery under the anticipated BARDA contract within three to six months from closing."

Transaction Details
Under the terms of the agreement, Emergent will pay Chimerix a $225 million one-time upfront payment in cash upon closing and up to a total of $100 million in milestone payments contingent on the potential exercise by the U.S. government of procurement options following the base period. The closing payment and the milestone payments may be adjusted based on the actual procurement value. The terms also include sales-based royalties contingent on future potential worldwide procurement during the exclusivity period of TEMBEXA on a market-to-market basis. Chimerix remains eligible to receive a portion of the regulatory milestone payments associated with the license to SymBio Pharmaceuticals Ltd. for indications other than orthopox infections.

Emergent anticipates that the transaction will be funded using currently available funds.

Closing Conditions
This transaction is subject to customary closing conditions, including expiration or early termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (HSR Act).

The transaction is further conditioned on the execution of an anticipated procurement contract between Chimerix and the Biomedical Advanced Research and Development Authority (BARDA) as well as receipt of any required consent from BARDA to a pre-novation agreement to be entered into with Emergent, upon which time Emergent would be poised to deliver the first shipment of TEMBEXA to the U.S. Strategic National Stockpile (SNS) upon completion of customary pre-shipment obligations. In December 2021, BARDA issued a sole source request for proposal (RFP) to procure up to 1.7 million treatment courses of TEMBEXA. Chimerix expects a BARDA procurement contract award as early as second quarter of 2022.

Subject to the satisfaction or waiver of the closing conditions, the companies expect the transaction to close as early as the end of the second quarter of 2022.

ABOUT TEMBEXA
TEMBEXA is an oral antiviral formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. TEMBEXA is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates. TEMBEXA is not indicated for the treatment of diseases other than human smallpox disease.

In June 2021, the FDA approved TEMBEXA tablets and oral suspension for the treatment of smallpox. TEMBEXA is approved for adult and pediatric patients and is the first and only smallpox therapy approved for neonates. The oral suspension formulation is particularly important for patients who have difficulty swallowing due to age or medical status. Please read full prescribing information here.

About Smallpox
Smallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30%. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention (CDC), variola virus is ranked in the highest risk category for bioterrorism agents (Category A) due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption. Based on a recent report – The Department of Health and Human Services Fiscal Year 2023 Public Health and Social Services Emergency Fund Justification of Estimates for Appropriations Committee – smallpox remains a threat of high concern to both the domestic and international community. BARDA’s goal is to ensure adequate vaccine supply for all Americans, including special populations, and to make available at least two different therapeutic agents as recommended by the National Academy of Medicine of the National Academies of Sciences, Engineering, and Medicine.

On May 16, 2022 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update on key events since the start of 2022 (Press release, Compugen, MAY 16, 2022, View Source [SID1234614586]).

"I am excited about the outlook of Compugen’s immune checkpoint inhibitors based on their unique characteristics, encouraging preliminary clinical data and our differentiated clinical development strategy," said Anat Cohen-Dayag, Ph.D., President, and Chief Executive Officer of Compugen. We are the first to evaluate the triple blockade of the DNAM-1 axis, targeting PVRIG, TIGIT and PD-1 in the clinic. Based on the totality of the data we have to date on the PVRIG pathway, we believe triple blockade of PVRIG/TIGIT/PD-1 may be required for optimizing clinical responses in both inflamed and less inflamed tumors where other checkpoint inhibitors have so far been unsuccessful. Our Phase 1 clinical data demonstrated durable disease control rates, consistent immune activation, and good tolerability. With COM902, we are the first company to present clinical data with an IgG4 anti-TIGIT antibody, with low Fc-effector function. Having over a decade of expertise in this space, we believe this is the optimal design for an anti-TIGIT antibody. COM902 achieved a disease control rate of 50%. Unlike some other anti-TIGIT antibodies, to date studies have shown that COM902 avoids depletion of the CD8+ T cells, crucial for efficacy and we believe the IgG4 backbone may come with additional safety benefits. We look forward to proving our DNAM-1 axis hypothesis through our robust differentiated clinical strategy with studies designed to maximize the potential of COM701."

Dr. Cohen-Dayag also commented, "Continuing on our excellent track record in execution, our first quarter of 2022 has been focused on execution of our differentiated clinical strategy to further enhance our leadership in DNAM-1 axis evaluation. Enrollment continues in our well designed, comprehensive Phase 1 clinical studies with our pioneering immunotherapy drug candidates COM701, targeting PVRIG, and COM902, targeting TIGIT. In addition, we continue to be data-driven, invest in cutting edge research and innovate as reflected by presentations at the AACR (Free AACR Whitepaper) and Keystone Symposium as well as expansion of COM701 intellectual property portfolio with a new U.S. patent covering triple combination use with anti-PD-1 and anti-TIGIT antibodies. Our $107 million cash balance affirms our financial discipline, with bold execution on our DNAM-1 axis hypothesis."

Dr. Cohen-Dayag continued, "Our clinical program is comprised of three ongoing cohort expansion combination studies, with overlapping indications, in patients with relapsed disease and indications so far insensitive to immunotherapy. The program was designed to focus on patients with limited treatment options and indications with greatest unmet need to efficiently demonstrate proof-of-concept for our novel immune therapies. Our intention is to report data from fully enrolled cohorts of each of these studies, taking into consideration that certain cohorts/indications enroll faster than others. We are on track to provide results from these studies starting with the microsatellite stable colorectal cancer, COM701/nivolumab combination expansion cohort, in the fourth quarter of 2022. We are also planning to report results from the other cohorts throughout 2023. The data from these studies will guide our regulatory strategy on a cohort-by-cohort basis."

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Dr. Cohen-Dayag concluded, "We are committed and we look forward to updating the medical and investment communities with our progress."

Financial Results

As of March 31, 2022, cash, cash equivalents, short-term bank deposits and restricted cash totaled approximately $107 million, compared with approximately $118 million as of December 31, 2021. The Company expects its existing cash and cash related balances to be sufficient to fund its operating plan into 2024, at the current rate of expenses. Compugen does not have any debt.

R&D expenses for the first quarter ended March 31, 2022, were approximately $7.2 million compared with approximately $7.3 million for the comparable period in 2021.

General and administrative expenses for the first quarter ended March 31, 2022, were approximately $2.6 million compared with approximately $2.7 million for the comparable period in 2021.

Net loss for the first quarter ended March 31, 2022, was approximately $9.7 million, or $0.11 per basic and diluted share, compared with a net loss of approximately $9.9 million, or $0.12 per basic and diluted share, in the comparable period of 2021.