On March 7, 2022 Targovax ASA (OSE: TRVX), a clinical-stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, and Agenus (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported that they have entered into a clinical collaboration and supply agreement to combine Targovax’s TG mutant KRAS cancer vaccines with Agenus´s clinically validated and FDA approved QS-21 STIMULON adjuvant (Press release, Targovax, MAR 7, 2022, View Source [SID1234609599]).

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The TG vaccines are peptide mixtures designed to elicit polyvalent immune responses against the most frequent mutations in the RAS gene family, which drive up to 30% of all cancers. TG01 has shown promise in phase 1/2 clinical trials, and Targovax has recently been awarded two prestigious research grants providing up to NOK 18m in funding to advance the TG program. Cancer vaccines require combination with an adjuvant in order to stimulate antigen processing and T-cell priming, and Targovax has selected QS-21 STIMULON as the adjuvant of choice for continued clinical development.

QS-21 STIMULON is a purified, natural saponin proprietary to Agenus. It has been widely studied in over 120 clinical trials and consistently demonstrated powerful antibody and cell-mediated immune responses as well as a favorable safety profile. QS-21 STIMULON is a critical component of multiple vaccines, including GSK’s FDA approved Shingrix shingles vaccine with protection exceeding 9 years, and the world’s first malaria vaccine Mosquirix.

Under the collaboration with Targovax, Agenus will supply QS-21 STIMULON and scientific support for up to three initial TG01 clinical trials in different cancer indications. If the clinical results meet certain pre-specified performance targets, the collaboration will be extended into an exclusive pivotal development and commercialization agreement where Agenus will be entitled to tiered double-digit royalties on future TG vaccine sales.

Dr. Garo H. Armen, Chief Executive Officer of Agenus Inc., said: "We look forward to collaborating with Targovax to extend the benefit of QS-21 STIMULON to new patient populations such as those harboring KRAS-mutated cancers. It highlights the broad applicability of our QS-21 STIMULON technology and could unlock a sizeable commercial opportunity in KRAS-mutated cancers."

Dr. Erik Digman Wiklund, Chief Executive Officer of Targovax ASA, added: "QS-21 STIMULON is arguably the most clinically and commercially successful FDA approved adjuvant for protein- and peptide-based vaccines, and has proven highly effective in eliciting immune responses. We expect that this unique adjuvant will further enhance the efficacy of our TG01 and TG02 products, and see it as a critical component of our development strategy as we prepare to bring the TG vaccines back into the clinic in multiple mutant KRAS cancers."

On March 7, 2022 Mission Therapeutics ("Mission"), a drug discovery and development company focused on protein homeostasis by selectively inhibiting deubiquitylating enzymes (DUBs), reported that its VP of Translational Science, Dr Andy Pearce, will attend and present at the Babraham Research Campus Signalling Event on 9 March 2022 (Press release, Mission Therapeutics, MAR 7, 2022, View Source [SID1234609598]).

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In his presentation, titled ‘Targeting Deubiquitinase Enzymes for Treatment of a Broad Spectrum of Diseases’, Andy will discuss the Company, discovery platform, cross disease interest, and data from its lead program, USP30. The presentation at 13.35 will include time for questions and Andy will be available for one-to-one meetings.

Mission Therapeutics has built a world-leading proprietary DUB platform for the discovery and development of first-in-class drugs that selectively target specific disease-associated DUBs. As the VP of Translational Science, Dr Pearce leads the Translational Science department and drug discovery projects. He previously led projects and research groups for over 15 years at Novartis and GSK.

The Signalling Event will focus on the range of R&D ongoing across the Babraham campus, as well as highlighting scientific services and capabilities available on site.

On March 7, 2022 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported financial results for the three and twelve months ended December 31, 2021 (Press release, Eagle Pharmaceuticals, MAR 7, 2022, View Source [SID1234609597]).

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Business and Recent Highlights:

Announced the commercial availability of its novel product, PEMFEXY (pemetrexed for injection), a branded alternative to ALIMTA. PEMFEXY is a ready-to-use liquid with a unique J-code and is approved in the United States to treat nonsquamous non-small cell lung cancer and mesothelioma. Eagle received approval from the U.S. Food and Drug Administration ("FDA") in February 2020 of its new drug application ("NDA") for PEMFEXY, following the settlement agreement of patent litigation with Eli Lilly and Company in December 2019. The agreement provided for a release of all claims by the parties and allows for an initial entry of PEMFEXY into the market (equivalent to approximately a three-week supply of ALIMTA utilization) on February 1, 2022, and a subsequent uncapped entry on April 1, 2022. The ALIMTA U.S. market totaled $1.2 billion for the 12 months ended December 31, 2021, as reported by Eli Lilly and Company
Commenced shipment of vasopressin on January 18, 2022 with 180 days of marketing exclusivity. Vasopressin, an A-rated generic alternative to Vasostrict, is indicated for use to increase blood pressure in adults with vasodilatory shock (e.g., post-cardiotomy or sepsis) who remain hypotensive despite fluids and catecholamines. U.S. sales of Vasostrict totaled $901.7 million for the 12 months ended December 31, 2021, as reported by Endo International plc.
AOP Orphan Pharmaceuticals GmbH, Member of the AOP Health Group, ("AOP Health"), with whom Eagle entered into a licensing agreement in August 2021, has engaged with the FDA to obtain alignment on the content and format of the pre-clinical and clinical data required to support an NDA seeking approval of Landiolol, a novel therapeutic, for the short-term reduction of ventricular rate in patients with supraventricular tachycardia, including atrial fibrillation and atrial flutter. Based on the FDA’s responses to AOP Health’s communications, Eagle remains on track to support AOP Health’s NDA filing for Landiolol in the second quarter of 2022.
1 The Company’s expectations with respect to the first quarter of 2022 are based on its estimates and assumptions as of March 7, 2022 and are subject to substantial uncertainty. The Company’s first quarter of 2022 is ongoing and not complete, and the Company’s expectations with respect to revenues, earnings per share and adjusted non-GAAP earnings per share for the first quarter of 2022 are estimates. Actual revenue, earnings per share and adjusted non-GAAP earnings per share for the Company’s first quarter of 2022 are subject to completion of the quarter as well as financial closing procedures for the period, and the actual and reported financial results for the Company’s first quarter of 2022 may materially differ. As such, the Company’s expectations with respect to the first quarter of 2022 are inherently unpredictable and actual results and outcomes could differ materially for a variety of reasons, including the factors discussed below under "Forward-Looking Statements".

* Adjusted non-GAAP net income, adjusted non-GAAP earnings per share, adjusted non-GAAP R&D expense and adjusted non-GAAP SG&A expense are non-GAAP financial measures. For descriptions and reconciliations of these non-GAAP financial measures to their most comparable GAAP financial measures, please see below and the tables at the end of this press release.

Financial Highlights

Fourth Quarter 2021

Total revenue for Q4 2021 was $42.3 million, compared to $49.9 million in Q4 2020, primarily reflecting lower product sales of BELRAPZO and RYANODEX and lower royalty revenue of BENDEKA, partially offset by higher product sales of TREAKISYM.
Q4 2021 net loss was $6.2 million, or $(0.48) per basic and diluted share, compared to net income of $8.1 million, or $0.62 per basic and $0.60 per diluted share, in Q4 2020.
Q4 2021 adjusted non-GAAP net income* was $11.2 million, or $0.87 per basic and $0.85 per diluted share, compared to adjusted non-GAAP net income* of $12.8 million, or $0.98 per basic and $0.96 per diluted share, in Q4 2020.
Cash and cash equivalents were $97.7 million, net accounts receivable was $41.1 million, and debt was $26.0 million as of December 31, 2021.
Full Year 2021

Total revenue for the 12 months ended December 31, 2021 was $171.5 million, compared to $187.8 million in 2020. 2020 included a $5.0 million milestone payment from SymBio Pharmaceuticals Limited for regulatory approval of TREAKISYM ready-to-dilute ("RTD") (250 ml) liquid bendamustine formulation.
Net loss for the 12 months ended December 31, 2021 was $8.6 million, or $(0.66) per basic and diluted share, compared to net income of $12.0 million, or $0.89 per basic and $0.87 per diluted share, in 2020.
Adjusted non-GAAP net income* for the 12 months ended December 31, 2021 was $34.4 million, or $2.64 per basic and $2.59 per diluted share, compared to $48.7 million, or $3.62 per basic and $3.54 per diluted share, in 2020.
From August 2016 through December 31, 2021, Eagle has repurchased $228.1 million of its common stock.
"With two important launches in early 2022, Eagle is off to a great start. The initial impressive revenue generated from vasopressin and PEMFEXY, each with significant periods of exclusivity, together with our royalties from bendamustine sales in Japan, position us to more than double our earnings this year. Based on early 2022 trends, we believe that our Q1 2022 earnings per share should approximate $4.00. Our pipeline is advancing as expected, and our balance sheet remains healthy. The period ahead will be exciting for us as we plan to deploy our cash to strengthen our product offerings and grow the company," stated Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals.

Q4 2021 BELRAPZO product sales were $5.5 million, compared to $10.2 million in Q4 2020.

Q4 2021 RYANODEX product sales were $6.1 million, compared to $7.9 million in Q4 2020.

Royalty revenue was $26.2 million in the fourth quarter of 2021, compared to $27.0 million in the fourth quarter of 2020. BENDEKA royalties were $24.2 million in the fourth quarter of 2021, compared to $27.0 million in the fourth quarter of 2020.

Gross margin was 71% during the fourth quarter of 2021, as compared to 75% in the fourth quarter of 2020. The decrease in gross margin for the fourth quarter of 2021 was driven by revenue mix.

R&D expense was $3.8 million for the fourth quarter of 2021, compared to $9.4 million for the fourth quarter of 2020. The decrease was primarily due to the non-recurrence of development cost on vasopressin and lower spend on RYANODEX related projects. Excluding stock-based compensation and other non-cash and non-recurring items, adjusted non-GAAP R&D expense* during the fourth quarter of 2021 was $2.6 million.

SG&A expenses in the fourth quarter of 2021 totaled $20.3 million compared to $18.2 million in the fourth quarter of 2020. This increase was primarily related to employee related costs and consulting costs partially offset by a decrease in stock-based compensation expense. Excluding stock-based compensation and other non-cash and non-recurring items, fourth quarter 2021 adjusted non-GAAP SG&A expense* was $14.6 million.

Net loss for the fourth quarter of 2021 was $6.2 million, or $(0.48) per basic and diluted share, compared to net income of $8.1 million, or $0.62 per basic and $0.60 per diluted share, in the fourth quarter of 2020, as a result of the factors discussed above.

Adjusted non-GAAP net income* for the fourth quarter of 2021 was $11.2 million, or $0.87 per basic and $0.85 per diluted share, compared to adjusted non-GAAP net income* of $12.8 million or $0.98 per basic and $0.96 per diluted share in the fourth quarter of 2020.

Full Year 2021 Financial Results

Product sales decreased by $7.3 million in the year ended December 31, 2021, primarily driven by decreases in product sales of Bendeka by $4.3 million, coupled with decreases in Belrapzo product sales of $3.8 million, due to price decreases and Ryanodex product sales by $3.0 million, due to volume decreases. The decreased sales were partially offset by increases in product sales of $3.9 million for TREAKISYM.

Gross margin was 75% in 2021, as compared to 76% in 2020. The decrease in gross margin in 2021 was driven by revenue mix.

R&D expense increased to $51.3 million in 2021, compared to $30.8 million in 2020, primarily reflecting a $10.0 million upfront payment related to our license agreement with Combioxin SA for CAL02, a $5.0 million upfront payment related to our licensing agreement with AOP Orphan for Landiolol, and increases of $2.3 million in development costs for vasopressin, $2.1 million in employee related costs, and $1.6 million related to PEMFEXY launch preparedness and regulatory costs. Excluding stock-based compensation and other non-cash and non-recurring items, adjusted non-GAAP R&D expense* in 2021 was $32.5 million.

SG&A expenses decreased by $3.3 million to $75.3 million in 2021, compared to $78.6 million in 2020. The decrease primarily reflects lower stock-compensation expense and the non-recurrence of expense related to Tyme Technologies, Inc. ("Tyme"), partially offset by increases in external legal fees and employee related costs. Excluding stock-based compensation and other non-cash and non-recurring items, adjusted non-GAAP SG&A expense* in 2021 was $54.9 million.

Net loss for the year ended December 31, 2021 was $8.6 million, or $(0.66) per basic and diluted share, as compared to net income of $12.0 million or $0.89 per basic and $0.87 per diluted share for the year ended December 31, 2020, as a result of the factors discussed above.

Adjusted non-GAAP net income* for the year ended December 31, 2021 was $34.4 million, or $2.64 per basic and $2.59 per diluted share, compared to adjusted non-GAAP net income* of $48.7 million, or $3.62 per basic and $3.54 per diluted share, for 2020.

First Quarter 2022 Expected Revenue and EPS1

Revenue for the first quarter 2022 is expected to be in the range of $120 million – $130 million.
Adjusted non-GAAP earnings per share* for the first quarter 2022 is expected to be in the range of $3.80 – $4.10.
2022 Full Year Expense Guidance

Adjusted non-GAAP R&D expense* is expected to be in the range of $46 million to $50 million, as compared to $32.5 million in 2021.
Adjusted non-GAAP SG&A expense* is expected to be in the range of $54 million to $58 million, as compared to $54.9 million in 2021.
Liquidity

As of December 31, 2021, Eagle had $97.7 million in cash and cash equivalents plus $41.1 million in net accounts receivable, and $26.0 million in outstanding debt. Therefore, as of December 31, 2021, Eagle had net cash plus receivables of $112.8 million.

In the fourth quarter of 2021, Eagle purchased $8.6 million of its common stock as part of its current $160.0 million Share Repurchase Program. From August 2016 through December 31, 2021, Eagle has repurchased $228.1 million of its common stock.

Conference Call

As previously announced, Eagle management will host its fourth quarter 2021 conference call as follows:

A replay of the conference call will be available for one week after the call’s completion by dialing 800-839-0866 (US) or 402-220-0662 (International) and entering conference call ID EGRXQ421. The webcast will be archived for 30 days at the aforementioned URL.

On March 7, 2022 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported the presentation of findings from a collaborative study with Tempus investigating the prevalence of Class II and Class III alterations among patients with BRAF-mutated solid tumors (Press release, Kinnate Biopharma, MAR 7, 2022, View Source [SID1234609596]). These findings were presented as an e-Poster at the virtual European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress (TAT), taking place March 7-9, 2022.

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"Advances in genomic profiling have significantly increased our ability to define emerging patient populations, and enable the development of effective targeted therapies for patients who do not currently benefit from precision medicine approaches," said Richard Williams, MBBS, Ph.D., Chief Medical Officer at Kinnate. "Through our collaboration with Tempus, and other leading precision medicine companies, we have found that there is a substantial number of cancer patients with BRAF Class II or Class III alterations, none of whom have access to approved targeted cancer therapies. We appreciate the opportunity to share insights on this urgent unmet need with the clinical research community gathered for the ESMO (Free ESMO Whitepaper) TAT 2022 conference."

This study utilized a de-identified clinico-genomic database of more than 55,000 cancer patients whose tumors were profiled using the Tempus xT next generation sequencing assay, a 648-gene DNA panel coupled with transcriptome RNA sequencing. A cohort of more than 1,100 patients was identified with BRAF Class II or Class III oncogenic alterations representing approximately 2% of patients. Among the patients with BRAF Class II or Class III alterations, those diagnosed with melanoma or non-small cell lung cancer (NSCLC) were most commonly treated with immunotherapy or chemotherapy +/- immunotherapy, respectively. At least 70% of patients with BRAF alterations had stage IV (metastatic) disease, and the distribution of cancer stages was similar across BRAF classes. Compared to BRAF Class I alterations, BRAF Class II and Class III alterations were more likely to co-occur with other gene alterations in the MAPK pathway such as NRAS, KRAS and NF1. Within the NSCLC and melanoma cohort, tumors with BRAF Class II or Class III alterations had a higher tumor mutation burden (TMB) than BRAF wild-type tumors. Additionally, patients with NSCLC and Class II or Class III alterations experienced shorter time to treatment discontinuation in first line and second line of therapy compared to patients with NSCLC and BRAF Class I alterations which is suggestive of inferior treatment outcomes.

"These findings are of particular interest given that BRAF Class II and Class III alterations are prevalent oncogenic drivers with no approved targeted therapy. Tumors with BRAF Class II or Class III alterations have been shown to be associated with unique tumor characteristics, including higher TMB and more frequent co-occurrence with other MAPK pathway alterations. Our real-world data study indicates that patients with NSCLC and BRAF Class II or Class III alterations experience shorter time to treatment discontinuation relative to patients with NSCLC and BRAF Class I alterations," said Paul Severson, Ph.D., Senior Director of Translational Medicine and Bioinformatics at Kinnate.

The e-Poster (Presentation #40P), titled "Real-World Clinical Genomic Analysis of Patients with BRAF Mutated Cancers Identifies BRAF Class II and III as a Population of Unmet Medical Need," will be presented by Dr. Severson and is available to registered attendees for on-demand viewing at: www.esmo.org/meetings/esmo-tat-2022.

On March 7, 2022 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported the presentation of safety and efficacy results from Cohort 4 of the ZENITH20 clinical trial (Press release, Spectrum Pharmaceuticals, MAR 7, 2022, View Source [SID1234609595]). This data is from 70 first-line patients with non-small lung cancer (NSCLC) with HER2 exon 20 insertion mutations who received 16 mg daily, given as 16 mg once daily (48 patients) or 8 mg twice daily (22 patients) of oral poziotinib. These results showed a confirmed objective response rate (ORR) of 41% (95% CI:30%-54%), as evaluated centrally by an independent image review committee using RECIST 1.1 criteria. The evaluable patient population showed an ORR of 50%. The study met its primary endpoint as the observed lower bound of 30% exceeded the pre-specified lower bound of 20%. The safety profile was consistent with the TKI class. The data is part of an oral presentation at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress 2022 being held virtually March 7-8, 2022.

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"Cohort 4 from our ZENITH 20 study demonstrated positive results for treatment naïve lung cancer patients harboring HER2 Exon 20 insertion mutations. There is currently no approved treatment for NSCLC patients with these mutations," said Francois Lebel, M.D., Chief Medical Officer of Spectrum. "We are encouraged by these findings and look forward to further discussions with the FDA on the regulatory path forward."

The presentation is available on the Spectrum Pharmaceuticals website at: View Source

Safety and Efficacy Data for Cohort 4 of the ZENITH20 Clinical Trial

Cohort 4 enrolled treatment-naïve NSCLC patients with HER2 exon 20 insertion mutations. This cohort investigated the efficacy of poziotinib and included patients dosed either with a QD or BID dosing strategy. Poziotinib 16 mg was administered orally once daily for the first 48 patients followed by an additional 22 patients dosed at 8 mg twice daily. Both dosing regimens allowed dose reductions/interruptions for toxicity. The primary endpoint was ORR evaluated centrally by an independent image review committee using RECIST 1.1 criteria. Secondary endpoints included disease control rate (DCR), duration of response (DoR), progression-free survival (PFS) and safety.

The primary endpoint of ORR was 41% (95% CI:30-54%) in the 70 treated patients including one complete response. The DCR was 73% (95% CI:61-83%). DoR was 5.7 months (range 1.2-19.1+). Median progression free survival (PFS) was 5.6 months (range 0-20.2+). 90% of patients had dose interruptions and 79% had reductions from the 16 mg QD starting dose, while 64% had reductions from the 8mg BID starting dose. The most common treatment related Grade ≥ 3 adverse events were rash (30%), stomatitis (19%), diarrhea (14%), and paronychia (7%). In addition, the incidence of Grade ≥ 3 pneumonitis was low at 3%. The safety profile was consistent with the TKI class and tolerability, dose reductions and interruptions were less frequent with BID dosing.

About the ZENITH20 Clinical Trial

The ZENITH20 study consists of seven cohorts of NSCLC patients. Cohorts 1 (EGFR) and 2 (HER2) in previously treated NSCLC patients with exon 20 mutations, Cohort 3 (EGFR) in first-line patients and Cohort 4 (HER2) in first-line NSCLC patients with exon 20 mutations have completed patient enrollment. Cohorts 1-4 are each independently powered for a pre-specified statistical hypothesis and the primary endpoint is ORR. Cohort 5 includes previously treated or treatment-naïve NSCLC patients with EGFR or HER2 exon 20 insertion mutations. Cohort 6 includes NSCLC patients with classical EGFR mutations who progressed while on treatment with first-line osimertinib and developed an additional EGFR mutation. Cohort 7 includes NSCLC patients with a variety of less common mutations in EGFR or HER2 exons 18-21 or the extracellular or transmembrane domains.