Entry into a Material Agreement

On January 8, 2023, Ionis Pharmaceuticals, Inc. (the "Company") and its wholly-owned subsidiary, Akcea Therapeutics, Inc. (the "Subsidiary") reported to have entered into a royalty purchase agreement (the "Purchase Agreement") with Royalty Pharma Investments 2019 ICAV, an Irish collective asset-management vehicle ("Royalty Pharma"), pursuant to which the Company and the Subsidiary sold to Royalty Pharma (Filing, 8-K, Ionis Pharmaceuticals, JAN 9, 2023, View Source [SID1234626074]):

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(i) the Company’s right, title and interest in and to: (A) in respect of net sales by Biogen Idec International Holding Ltd ("Biogen Idec") and Biogen MA ("Biogen MA", and together with Biogen Idec, "Biogen") between January 1, 2023 and December 31, 2027, 25%, and (B) in respect of net sales by Biogen Idec commencing on January 1, 2028, 45% of the royalties payable to the Company on annual worldwide net sales up to $1,500,000,000 pursuant to that certain Development, Option and License Agreement by and between the Company and Biogen Idec dated January 3, 2012, as amended (the "2012 Biogen License") and that certain Research Collaboration, Option and License Agreement by and between the Company and Biogen MA dated December 19, 2017 (the "2017 Biogen License") (the "Purchased SMA Royalties"), subject to an overall cap of either $475,000,000 or $550,000,000, depending on the timing of FDA approval of Pelacarsen, or IONIS-APO(a)-LRx, as described in the Purchase Agreement; and

(ii) the Subsidiary’s right, title and interest in and to 25% of the royalties payable to the Subsidiary in respect of net sales by Novartis Pharma AG ("Novartis") pursuant to that certain Strategic Collaboration, Option and License Agreement by and between the Subsidiary and Novartis dated January 5, 2017, as amended (the "Novartis License") (the "Purchased Pelacarsen Royalties" and together with the Purchased SMA Royalties, the "Purchased Royalties").

In consideration for the sale of the Purchased Royalties, Royalty Pharma paid to the Company an initial purchase price of $500,000,000 and has agreed to pay the Company certain additional payments totaling up to $625,000,000, subject to the achievement of specified milestones set out in the Purchase Agreement.

Under the Purchase Agreement, and in connection with its sale of the Purchased Royalties, each of the Company and the Subsidiary has agreed to certain covenants with respect to the exercise of its rights under the 2012 Biogen License, 2017 Biogen License and Novartis License, including with respect to the Company and the Subsidiary’s right to amend, assign and terminate such agreements. The Company and Subsidiary will also provide certain additional payments to Royalty Pharma in the event the Company or the Subsidiary commercializes certain products directly competitive with Pelacarsen. The Purchase Agreement contains other customary terms and conditions, including representations and warranties, covenants and indemnification obligations in favor of each party.

The foregoing summary of the Purchase Agreement is not complete and is qualified in its entirety by reference to the complete text of the Purchase Agreement, which the Company intends to file as an exhibit to its Quarterly Report on Form 10-Q for the quarter ending March 31, 2023.

Corporate Overview

On January 9, 2023 IO Biotech presented its corporate presentation (Presentation, IO Biotech, JAN 9, 2023, View Source [SID1234626073]).

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Hepion Pharmaceuticals Receives $3.2 Million in Aggregate Non-Dilutive Funding

On January 9, 2023 Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical stage biopharmaceutical company focused on Artificial Intelligence ("AI")-driven therapeutic drug development for the treatment of fibrotic diseases, including non-alcoholic steatohepatitis ("NASH"), hepatocellular carcinoma ("HCC"), and other chronic diseases, reported the receipt of $2.9 million in net proceeds from the sale of tax benefits pursuant to the Company’s participation in the New Jersey Economic Development Authority ("NJEDA") NOL program under the New Jersey Economic Recovery Act of 2020, and receipt of a C$416,415 (US$309,000) Alberta Innovation Employment Grant (Press release, Hepion Pharmaceuticals, JAN 9, 2023, View Source [SID1234626063]).

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NJEDA’s NOL program enables qualified, New Jersey-based technology or biotechnology companies to sell net operating losses to unrelated profitable corporations. This allows qualifying technology and biotechnology companies with NOLs to turn their tax losses and credits into cash proceeds to fund growth and operations, including research and development ("R&D") or other allowable expenditures.

Alberta’s Innovation Employment Grant program encourages economic growth by supporting small and medium-sized businesses that invest in R&D with a grant worth up to 20% of qualifying expenditures. The program promotes investment and diversification by rewarding all R&D spending in Alberta, Canada, regardless of the industry.

"We appreciate the support of both the State of New Jersey and the Province of Alberta to support innovation within their respective business communities," said Robert Foster, PharmD, PhD, Hepion’s CEO. "This non-dilutive funding adds to the approximate $59.1 million in cash we had as of the end of Q3-2022, further strengthening the Company’s balance sheet as we continue to advance rencofilstat, our lead oral drug candidate for the treatment of NASH and HCC."

Guardant Health Announces Preliminary Fourth Quarter and Full Year 2022 Results

On January 9, 2023 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company focused on helping conquer cancer globally through use of its proprietary tests, vast data sets and advanced analytics, reported preliminary, unaudited results for the year ended December 31, 2022 (Press release, Guardant Health, JAN 9, 2023, View Source [SID1234626062]).

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Full year 2022 preliminary unaudited financial results

For the twelve-month period ended December 31, 2022, as compared to the same period of 2021:

Revenue of between $447 million and $450 million, an increase of 20%
Reported 124,800 tests to clinical customers and 26,000 tests to biopharma customers, an increase of 42% and 40%, respectively
Fourth quarter 2022 preliminary unaudited financial results

For the three-month period ended December 31, 2022, as compared to the same period of 2021:

Revenue of between $124 million and $127 million, an increase of between 15% and 17%
Reported 36,000 tests to clinical customers and 8,200 tests to biopharma customers, an increase of 41% and 24%, respectively
Cash, cash equivalents and marketable debt securities were $1.0 billion as of December 31, 2022.

"We are very pleased with the strong finish to 2022 that enabled us to post annual volume growth for both clinical and biopharma above 40%, and believe we are well positioned for continued strong double-digit revenue growth in 2023." said Helmy Eltoukhy, co-founder and co-CEO.

"During the fourth quarter we delivered on a long-term ambition with the positive readout of our ECLIPSE trial. We are thrilled with the strong and positive feedback expressed by guideline members, key opinion leaders, and patient advocacy leaders about the performance of the Shield test in the ECLIPSE trial," said AmirAli Talasaz, co-founder and co-CEO. "Fueled by this success, we will expand this test to many other cancer types, including lung cancer, the leading cause of death from cancer."

Guardant Health has not completed preparation of its financial statements for the fourth quarter or full year of 2022. The revenue ranges and test volumes presented in this release for the fourth quarter and the year ended December 31, 2022 are preliminary and unaudited and are thus inherently uncertain and subject to change as we complete our financial results. The company is in the process of completing its customary year-end close and review procedures as of and for the year ended December 31, 2022, and there can be no assurance that final results for this period will not differ from these estimates. During the course of the preparation of the Guardant Health’s consolidated financial statements and related notes as of and for the year ended December 31, 2022, the company’s independent registered public accountants may identify items that could cause final reported results to be materially different from the preliminary financial estimates presented herein.

Upcoming events

Guardant Health plans to report its fourth quarter and full year audited financial results for the period ended December 31, 2022 during its February 2023 earnings call.

G1 Therapeutics Announces Upcoming 2023 Readouts from its Phase 2 and Pivotal Phase 3 Clinical Trials of Trilaciclib

On January 9, 2023 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported the 2023 data readouts that are expected to drive its near-term and long-term indications and potential future treatment paradigms for some of the most aggressive and refractory cancers, including metastatic colorectal (mCRC), bladder or urothelial cancer (mUC), and triple negative breast cancer (TNBC) (Press release, G1 Therapeutics, JAN 9, 2023, View Source [SID1234626060]).

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"Cytotoxic therapy remains the backbone of treatment for many metastatic tumors, either alone or in combination with targeted therapies," said Raj Malik, M.D., G1’s Chief Medical Officer. "But the high toxicity of these cytotoxic agents poses a risk of serious adverse events that can compromise patient well-being and patient outcomes. Trilaciclib represents a novel approach to protect against the side effects of cytotoxic therapy, but it also holds the potential to extend survival in certain cancer types, especially in combination with other novel anti-cancer interventions such as ADCs and checkpoint inhibitors—a benefit we are currently exploring in a variety of clinical trials with important readouts throughout 2023."

Trilaciclib, an IV-administered transient CDK4/6 inhibitor, is a first-in-class therapy designed to preserve bone marrow and immune system function during cytotoxic therapy to improve patient outcomes. Depending on the tumor type and the chemotherapy backbone, this mechanistic profile can drive patient benefits of myeloprotection and/or anti-tumor efficacy. Its mechanism of action of improving overall immune response by improving long term immune surveillance lends itself to longer term endpoints, such as progression free and overall survival.

Clinical Trial Results Expected in the First Quarter of 2023

PRESERVE 1: 1L mCRC registrational Phase 3 trial

February 2023: Primary endpoint (myeloprotection). The Company expects to release initial results in February 2023 from the ongoing PRESERVE1 trial in patients with mCRC who received trilaciclib administered prior to treatment with FOLFOXIRI and bevacizumab. These data will include the primary myeloprotection endpoints of severe neutropenia during induction and duration of severe neutropenia in Cycles 1-4; the impact of trilaciclib on Grade 3 or 4 diarrhea; and initial patient reported outcome data. If positive, these data will serve as the basis for working closely with the FDA and other regulatory health authorities to extend the label to trilaciclib as a myeloprotective agent in patients with mCRC.

Compared to the extensive-stage small cell lung cancer market, the colorectal market is significantly larger with a longer duration of therapy (ES-SCLC: 3 months vs mCRC: 6-12 months) and CRC patients often have a better prognosis that facilitates the more aggressive therapeutic approach of FOLFOXIRI triplet therapy.

"Triplet therapy with FOLFOXIRI and bevacizumab is highly efficacious compared to doublet therapy, but it is also the most myelotoxic regimen with higher rates of neutropenia, febrile neutropenia, and diarrhea – which limits its use to a small population of 1L CRC patients," said Dr. Malik. "Trilaciclib may be an important addition to this regimen to enable a broader population of patients to benefit from this therapy and potentially further improve anti-tumor efficacy by allowing increased duration and exposure to chemotherapy."

Clinical Trial Results Expected in the Second Quarter of 2023

Phase 2 ADC trial in patients with refractory TNBC

Anti-tumor efficacy. Additional data from G1’s ongoing trial of trilaciclib administered prior to the ADC sacituzumab govitecan-hziy in patients with unresectable locally advanced or metastatic TNBC are expected in 2Q23 and will include anti-tumor efficacy endpoints as measured by the primary endpoint of progression-free survival (PFS).

Initial results released in 4Q22 demonstrated the potential of trilaciclib to reduce adverse events associated with sacituzumab govitecan-hziy. Initial data on the first 18 patients show a clinically meaningful on-target effect of trilaciclib to reduce (>50%) the rates of multiple adverse events compared to the previously published sacituzumab govitecan-hziy single agent safety profile from the ASCENT trial, including myelosuppression (neutropenia, anemia, thrombocytopenia), and diarrhea and potentially alopecia due to the presence of CDK4/6-expressing cells in the intestinal crypt and hair follicles.

Phase 2 mechanism of action (MOA) trial in patients with neoadjuvant TNBC

Pathologic complete response (pCR) and immune enhancements in tumor microenvironment: Presentation of a more comprehensive data set from this 24 patient Phase 2 trial are expected in 2Q23 from analyses of the secondary endpoint of pathologic complete response rate at the time of definitive surgery and the safety of trilaciclib combined with neoadjuvant chemotherapy. Additional analyses will further elucidate the immune-based mechanism of action of a single dose of trilaciclib.

Initial results from the primary endpoint of immune-based MOA presented at the 2022 San Antonio Breast Cancer Symposium showed favorable alterations in the tumor microenvironment from a single dose of trilaciclib monotherapy as measured by an increase in the ratio of CD8+ T cells compared to regulatory T cells (Tregs).

Clinical Trial Results Expected in the Midyear 2023

PRESERVE 3: 1L bladder cancer (mUC) Phase 2 trial

Anti-tumor efficacy. Additional safety and efficacy data, including the primary endpoint of the anti-tumor efficacy of trilaciclib when combined with platinum-based chemotherapy and the checkpoint inhibitor avelumab maintenance therapy as measured by PFS are anticipated in mid-2023.

Initial results provided in January 2023 indicate that the confirmed objective response rate as of the cutoff date was comparable between arms. Longer-term follow-up is required to characterize additional anti-tumor endpoints including duration of confirmed objective response and PFS. Safety is reviewed by the data monitoring committee (DMC) on an ongoing basis, and it has recommended that the study continue as planned. Though early, the safety and tolerability profile of trilaciclib administered prior to chemotherapy is generally consistent with that expected in patients treated with gemcitabine plus cisplatin/carboplatin and avelumab maintenance for previously untreated advanced or metastatic urothelial carcinoma.

Clinical Trial Results Expected in the Second Half of 2023

PRESERVE 2: 1L metastatic TNBC registrational Phase 3 trial

2H23: Overall survival. An interim overall survival (OS) analysis at 70% of events is currently anticipated in 2H23 to evaluate the effect of trilaciclib on overall survival in patients with TNBC when administered prior to treatment with gemcitabine and carboplatin (GC). If the interim OS analysis achieves the threshold of statistical significance required for the interim assessment showing that trilaciclib has superior efficacy in overall survival, the trial will terminate, and the data will be reported. In addition, we will discuss the data with regulatory health authorities regarding filing for potential approval of this indication.

This trial builds on the foundational Phase 2 data showing a statistically significant and medically important improvement in survival in the two arms that received trilaciclib prior to GC compared to an arm that received GC alone (HR: 0.31 and 0.40, respectively).

PRESERVE 1: 1L mCRC registrational Phase 3 trial

4Q23: Anti-tumor efficacy. G1 expects to release initial PFS data from PRESERVE 1 on the combination of trilaciclib administered prior to FOLFOXIRI and bevacizumab in 4Q23.

These data from the five ongoing Phase 2 and pivotal Phase 3 trials of trilaciclib will inform the Company’s strategic direction, clarify the potential synergistic potential of trilaciclib, and, if positive, serve as the basis for seeking additional indications beyond extensive-stage small cell lung cancer, starting with mCRC which could be approved in early 2024.