On May 6, 2021 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported financial results for the first quarter of 2021 (Press release, Agenus, MAY 6, 2021, View Source [SID1234579398]).

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"We have delivered on multiple key initiatives since our last update and expect to achieve additional impactful milestones during the remainder of the second quarter and the rest of 2021," said Garo Armen, PhD, Chief Executive Officer of Agenus. "Among important developments are the continuing clinical responses we are seeing with AGEN1181. We intend to advance AGEN1181 in combination with balstilimab in cancers for which current immunotherapies have shown no activity given AGEN1181’s positive clinical responses in these tumors. Treating these cancers successfully will be of substantial value to patients while potentially representing large commercial opportunities for Agenus."

Balstilimab (anti-PD-1): BLA submitted to U.S. FDA for recurrent/metastatic cervical cancer

A Biologics License Application (BLA) was submitted to the U.S. Food and Drug Administration (FDA) for the accelerated approval of balstilimab for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy.

The submission was based on an update to data presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020 and published in an Oncogene editorial. This updated dataset includes maturation of late patient responses, with the overall data showing response rates of 20% in PD-L1 positive tumors, 15% in all tumors (PD-L1 positive and negative), and a median duration of response of 15.4 months.

Data demonstrating that balstilimab is a potentially differentiated anti-PD-1 antibody will be presented at the 2021 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 4 – 8, 2021.

Discussions with the FDA regarding accelerated BLA filing for balstilimab plus zalifrelimab are ongoing; additional guidance and updated response rate data will be provided upon the FDA acceptance of the balstilimab monotherapy BLA.
AGEN1181 (anti-CTLA-4): Data demonstrate continued strong activity, including in tumors unresponsive to immunotherapy, as presented at AACR (Free AACR Whitepaper) 2021

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, a new partial response with AGEN1181 monotherapy was reported in the first and only melanoma patient treated to date, as well as a new conversion to complete response with AGEN1181 plus balstilimab in an ovarian cancer patient.

Continued clinical activity in patients with biomarkers which indicate a poor prognosis with approved immunotherapies, including patients with microsatellite stable (MSS) tumors and melanoma, endometrial, and ovarian cancer with the low-affinity FcyRIIIA allele. No immune-mediated hypophysitis, pneumonitis, or hepatitis were reported.

As of AACR (Free AACR Whitepaper) 2021, a total of seven confirmed objective responses were achieved in a Phase 1/2 trial of AGEN1181 in solid tumors out of 52 evaluable patients: 2 confirmed responses among 21 treated with monotherapy, and 5 confirmed responses among 31 treated with AGEN1181 in combination with balstilimab.

Phase 2 trial in colorectal cancer was initiated; registrational trials are targeted to commence in 2021 with a focus on indications enabling a rapid path to BLA submission. Further data updates expected later this year.
AGEN1777 (anti-TIGIT bispecific): Phase 1 anticipated 2021

IND submission is planned for the current quarter.

Phase 1 study is expected to commence in the third quarter.
Intelligent cell platform: Phase 1 study ongoing with iNKT cell therapy in patients with cancer and ARDS secondary to COVID-19

Phase 1 trial in hematologic cancers was initiated; expansion into solid tumors is expected this year.

Preliminary Phase 1 data for acute respiratory distress syndrome (ARDS) secondary to COVID-19 suggest iNKTs (invariant natural killer T cells) can be dosed without adverse events attributable to the therapy and may demonstrate early signals of activity. Dose escalation is expected to be completed this year with data readouts to be presented at upcoming conferences.
Additional programs and initiatives continue to advance

A data update on a Phase 1 trial of AGEN2373 (a CD137 agonist antibody) will be presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting.

Agenus entered into a clinical collaboration with Nelum Pharmaceuticals for zalifrelimab in combination with NLM-001, Nelum’s small molecule hedgehog inhibitor, and chemotherapy for first-line advanced pancreatic cancer.
First Quarter Financial Results

We ended our first quarter 2021 with a cash balance of $119 million as compared to $100 million at December 31, 2020.

Cash used in operations for the three months ended March 31, 2021 was $43 million compared to $35 million for the quarter ended March 31, 2020. Net loss for the quarter ended March 31, 2021 was $54 million or $0.27 per share which includes non-cash expenses of $12 million compared to a net loss for the same period in 2020 of $45 million, or $0.31 per share which includes non-cash expenses of $3 million.

We recognized revenue of $12 million and $15 million for the quarters ended March 31, 2021 and 2020, respectively, which includes revenue related to non-cash royalties earned and revenue recognized under our collaboration agreements.

On May 6, 2021 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Lipocine, MAY 6, 2021, View Source [SID1234579415]).

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First Quarter Recent Corporate Highlights

Announced positive topline results from the Phase 2 LiFT ("Liver Fat intervention with oral Testosterone") clinical study, investigating LPCN 1144 in biopsy-confirmed NASH male subjects
Both LPCN 1144 treatment arms met the primary endpoint, change in hepatic fat fraction via magnetic resonance imaging proton density fat fraction ("MRI-PDFF"), with statistical significance
36-week biopsy data from the LiFT study are expected in August 2021
Continued enrolling patients into an open label extension to the LiFT clinical study in which all patients will have access to LPCN 1144
Announced the publication of results supporting the therapeutic potential of LPCN 1144 in the treatment of non-alcoholic steatohepatitis ("NASH") and hepatic fibrosis
The results were featured in a paper entitled "Treatment Potential of LPCN 1144 on Liver Health and Metabolic Regulation in a Non–Genomic, High Fat Diet Induced NASH Rabbit Model" (Comeglio et al), published in the Journal of Endocrinological Investigation
Continued to evaluate commercial options for TLANDO, our oral testosterone product for testosterone replacement therapy ("TRT") in adult males with hypogonadism
Posters featuring clinical data on TLANDO were presented at the ENDO 2021 Conference
Raised gross proceeds of approximately $28.7 million in a public offering of approximately 16.4 million shares of common stock
First Quarter Ended March 31, 2021 Financial Results
Lipocine reported a net loss of $3.4million, or ($0.04) per diluted share, for the first quarter ended March 31, 2021, compared with a net loss of $5.8 million, or ($0.14) per diluted share, for the first quarter ended March 31, 2020.

Research and development expenses were $1.6 million for the first quarter ended March 31, 2021, compared with $2.5 million for the first quarter ended March 31, 2020. The decrease in research and development expenses during the three months ended March 31, 2021 was primarily due to a decrease in contract research organization expense and outside consulting costs related to the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects and a decrease in raw material costs of TLANDO XR offset by increases in personnel expense and other R&D expenses.

General and administrative expenses were $1.5 million for the first quarter ended March 31, 2021, compared with $2.1 million for the first quarter ended March 31, 2020. The decrease in general and administrative expenses during the three months ended March 31, 2021 was primarily due to a decrease in legal costs due to less activity in 2021 as compared to 2020 in the following activities: lawsuit filed against Clarus Therapeutics Inc. for patent infringement in April 2019 and the on-going class action lawsuit defense. Additionally, personnel costs decreased in 2021. These decreases were offset by an increase in corporate insurance expenses.

As of March 31, 2021, the Company had $50.0 million of unrestricted cash, cash equivalents, and marketable investments, compared to $19.7 million of unrestricted cash, cash equivalents and marketable investment securities as of December 31, 2020.

On May 6, 2021 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Harpoon Therapeutics, MAY 6, 2021, View Source [SID1234579438]).

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"We continue to make solid progress across all our clinical programs for our TriTAC portfolio and continue to expand our ProTriTAC efforts following our presentation at AACR (Free AACR Whitepaper) this year," said Jerry McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "We expect to announce or present data on all four of our clinical TriTAC programs throughout the course of 2021."

First Quarter 2021 Business Highlights and Other Recent Developments

In April 2021, Harpoon presented encouraging data on the biologic effects of the TriTAC and ProTriTAC platforms at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data presented underscore the potential to produce novel tri-specific T cell activating construct (TriTAC) molecules targeting additional tumor types and highlighted the properties of the ProTriTAC platform, conditionally active T cell engager prodrugs, which may lead to greater tumor specificity, enhanced efficacy, and improved tolerability for patients. In addition, preclinical combination approaches of TriTACs with checkpoint inhibitors were explored. Key findings include:
FLT3-targeting TriTACs are T cell engagers with a potential role in the treatment of acute myeloid leukemia. Data showed that FLT3 TriTACs bind FLT3 in preclinical models and can direct T cells to kill FLT3 expressing cells in vitro and in vivo.
ProTriTAC is a modular and robust T cell engager prodrug platform with therapeutic index expansion observed across multiple tumor targets. The data presented showed the consistency and robustness of the platform in vivo and in vitro as demonstrated by cell-based assays, pharmacokinetic studies, and therapeutic index assessments.
T cell activation generally leads to the induction of PD1 on T cells, which may lead to a reduction of the biologic activity of TriTAC activated T cells. The data demonstrated the potential utility of PD1/PD-L1 blockade to enhance the potency of TriTAC mediated tumor cell killing.
In March 2021, Harpoon appointed experienced biotech leader Alan Colowick, M.D., to its board of directors. Throughout his career, Dr. Colowick has had a broad impact across the healthcare landscape with a focus on clinical stage companies in oncology, rare diseases, and other specialty therapeutic areas.

In January 2021, Harpoon received Orphan Drug designation by the U.S. Food and Drug Administration for HPN217 for the treatment of multiple myeloma. HPN217 targets B-cell maturation antigen (BCMA).
First Quarter 2021 Financial Results

Harpoon ended the first quarter of 2021 with $239.4 million in cash, cash equivalents, and marketable securities compared to $150.0 million as of December 31, 2020. The cash balance at the end of the first quarter includes Harpoon’s follow-on financing that closed on January 11, 2021 resulting in net proceeds of approximately $107.6 million.

Revenue for the first quarter ended March 31, 2021 was $9.0 million compared to $3.3 million for the quarter ended March 31, 2020. The increase in revenue was primarily due to a $4.3 million increase in revenue recognized due to the delivery of the second initial target under Harpoon’s Amended and Restated Discovery Collaboration Agreement with AbbVie, where all remaining deferred revenue associated with that target was recognized as we had no further continuing performance obligations, as well as a $1.5 million increase in revenue recognized related to Harpoon’s Development and Option Agreement with AbbVie, which was entered into in November 2019, for research and development services performed.

Research and development expense for the first quarter ended March 31, 2021, was $16.2 million compared to $12.5 million for the quarter ended March 31, 2021. The increase primarily arose from higher personnel-related expense due to an increase in headcount and clinical development expense, which included conducting preclinical studies and ongoing clinical development for HPN424, HPN536, HPN217 and HPN328.

General and administrative expense for the first quarter ended March 31, 2021 was $4.6 million compared to $3.9 million for the quarter ended March 31, 2020. The increase was primarily attributable to an increase in personnel-related expenses due to an increase in headcount and other professional services to support Harpoon’s operations as a public company.

Litigation settlement for the first quarter ended March 31, 2021 was $50.0 million. On May 5, 2021, Harpoon entered into a settlement agreement with Millennium Therapeutics, Inc. pursuant to which Harpoon agreed to pay the damages awarded by the Delaware Court of Chancery issued in its memorandum opinion issued on April 23, 2021, equal to $38.2 million in damages plus $11.8 million in pre-judgment interest.

Net loss for the first quarter ended March 31, 2021 was $61.7 million compared to $12.6 million for the quarter ended March 31, 2020.
Anticipated 2021 Milestones

HPN424 – present interim data from the dose escalation phase of the ongoing Phase 1/2a trial in the first half of 2021, and initiate the dose expansion cohort mid-year 2021
HPN536 – in the second half of 2021, initiate the dose expansion cohort of the ongoing Phase 1/2a trial and, by year end 2021, present interim Phase 1 data from the dose escalation phase of the trial
HPN217 – in the second half of 2021, initiate the dose expansion cohort of the ongoing Phase 1/2 trial, and present interim data from the dose escalation phase of the trial
HPN328 – in the second half of 2021, present interim data from the dose escalation phase of the ongoing Phase 1/2 trial
COVID-19 Business Update

In response to the ongoing COVID-19 pandemic, Harpoon has established testing and other protocols for personnel access to its headquarter offices and laboratory although the majority of the company’s employees continue to telecommute. Harpoon is currently continuing its clinical trials, and has not yet experienced any material delays or impacts as a result of the COVID-19 pandemic. In addition, Harpoon’s third-party contract manufacturers continue to operate at or near normal levels. Harpoon continues to assess the potential impact of the COVID-19 pandemic on its business and operations, including its programs, expected timelines, expenses, manufacturing activities and preclinical and clinical trials. The full extent to which the COVID-19 pandemic may have a negative impact on Harpoon’s business, assets, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted.

On May 6, 2021 Targovax ASA (OSE: TRVX), a clinical stage biotechnology company developing immune activators to target hard-to-treat solid tumors, reported its first quarter 2021 results (Press release, Targovax, MAY 6, 2021, View Source [SID1234579261]).

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An online presentation by Targovax’s management to investors, analysts and the press will take place at 10:00 CET today (details below).

FIRST QUARTER HIGHLIGHTS
REASEARCH & DEVELOPMENT
Reported continued survival benefit in Targovax’s ONCOS-102 trial in mesothelioma at the 21-month follow-up
Median Overall Survival (mOS) has still not been met for randomized first-line patients receiving ONCOS-102 plus chemotherapy
mOS will be at least 20.5 months for randomized first-line patients receiving ONCOS-102 plus chemotherapy, compared to mOS of 13.5 months in the chemotherapy-only control group
Received Fast-Track designation from the US FDA for ONCOS-102 in malignant pleural mesothelioma. This opens the potential for expedited development path and review
Entered a research collaboration with Papyrus Therapeutics to develop novel ONCOS viruses with receptor tyrosine kinase (RTK) inhibitor functionality
CORPORATE
Announced Dr Sonia Quaratino as a new member of the Board of Directors
Obtained US Patent for ONCOS-102 in combination with checkpoint inhibitors
Maintained TG + chemo patent as granted after opposition in European Patent Office
FINANCIALS

The interim financial information has not been subject to audit

Øystein Soug, CEO commented: "Targovax is at the beginning of a new and exciting development phase. Based on impressive ONCOS-102 clinical data, our main priority going forward is to start a next trial in PD1 refractory melanoma. At the same time, it is also important that we do it right and discuss our strategy with the FDA, since the aim of this trial is to support an accelerated approval. Moreover, based on the strength and breadth of the clinical and immune data, we believe our technology warrants a broader application. Hence, we envision several expansion possibilities beyond melanoma in other indications, with other novel combinations, and for our next generation pipeline products."

Presentation
We invite to a live webcast today at 10.00 CET. You can join the webcast here. It will be possible to ask questions during the presentation.

On May 6, 2021 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial results for the first quarter 2021 (Press release, Acorda Therapeutics, MAY 6, 2021, View Source [SID1234579333]).

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"We were pleased to see the increase in INBRIJA net sales in the first quarter of 2021 over the same quarter of 2020. In addition, our organic growth, measured by the increase in dispensed cartons, was 25% compared to the first quarter of 2020 and also an acceleration versus last quarter. This is an encouraging sign that we may be starting to see the impact of a receding pandemic on our business," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "We continue to see renewed interest in ex-US commercial partnerships for INBRIJA, owing to the reduced cost of goods that resulted from the sale of our manufacturing operations to Catalent earlier this year, as well as clarity from the GBA in Germany indicating that an early benefit assessment would not be required. We are in active discussions with several parties for commercialization both in Europe and around the world."

"We were also pleased to see stable year-over-year quarterly sales for AMPYRA for the first time since it went generic in September 2018 and believe this is due to the strategies we have executed to maintain the brand. The strength of the AMPYRA brand is an important contributor to Acorda’s financial stability and to our goal of becoming cash-flow neutral by the end of 2022," Cohen continued. "We also plan to address our $69 million convertible debt payment that is coming due in June of 2021."

First Quarter 2021 Financial Results

For the quarter ended March 31, 2021, the Company reported INBRIJA net revenue of $5 million, compared to $4.4 million for the same quarter in 2020.

For the quarter ended March 31, 2021, the Company reported AMPYRA net revenue of $20.3 million compared to $20.1 million for the same quarter in 2020. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended March 31, 2021 were $4.7 million, including $0.2 million of share-based compensation compared to $7.7 million, including $0.4 million of share-based compensation for the same quarter in 2020.

Sales, general and administrative (SG&A) expenses for the quarter ended March 31, 2021 were $34.0 million, including $0.5 million of share-based compensation compared to $41.1 million, including $1.5 million of share-based compensation for the same quarter in 2020.

Change in fair value of derivative liability for the quarter ended March 31, 2021 was $0.2 million compared to $(26.5) million for the same quarter in 2020.

Benefit from income taxes for the quarter ended March 31, 2021 was $3.2 million compared to a benefit from income taxes of $7.0 million for the same quarter in 2020.

The Company reported a GAAP net loss of $33.5 million for the quarter ended March 31, 2021, or $3.53 per diluted share. GAAP net loss in the same quarter of 2020 was $6.5 million, or $0.81 per diluted share.

Non-GAAP net loss for the quarter ended March 31, 2021 was $23.3 million, or $2.46 per diluted share. Non-GAAP net loss in the same quarter of 2020 was $24.4 million, or $3.06 per diluted share. This quarterly non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, asset impairment charges, changes in the fair value of derivative liability related to our 2024 convertible senior secured notes, and expenses that pertain to non-routine corporate restructurings. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At March 31, 2021, the Company had cash, cash equivalents, short-term investments, and restricted cash of $148.4 million, compared to $102.9 million at year end 2020. Restricted cash includes $31.1 million in escrow related to the 6% semi-annual interest portion, payable in cash or stock, of the 2024 convertible senior secured notes. If the Company elects to pay interest due in stock, the restricted cash will be released from escrow.

Financial Guidance

For the full-year 2021, Acorda continues to expect AMPYRA net revenue to be $75 – $85 million, and operating expenses to be $130 – $140 million. The operating expense guidance is a non-GAAP projection that excludes restructuring costs and share-based compensation as more fully described below under "Non-GAAP Financial Measures."
Webcast and Conference Call

The Company will host a conference call and webcast in conjunction with its first quarter 2021 update and financial results today at 4:30 p.m. EDT.

To participate in the Webcast/Conference Call, please note there is a new pre-registration process.

To register for the Webcast, use the link below:
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To register for the Conference Call, use the link below:
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**When registering please type your phone number with no special characters**

Once you have registered, you will receive a confirmation email with Webcast/Conference Call details. For the Webcast you will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 7:30 p.m. EDT on May 6, 2021 until 11:59 p.m. EDT on June 3, 2021. To access the replay, please dial (800) 585-8367 (domestic) or (416) 621-4642 (international); reference code 2996776. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.