Decibel Therapeutics Announces Extension of Research Term under Strategic Collaboration with Regeneron to Discover and Develop Gene Therapies for Hearing Loss

On November 22, 2021 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported that Regeneron has extended the research term of its collaboration with the Company to discover and develop gene therapies for hearing loss (Press release, Decibel Therapeutics, NOV 22, 2021, View Source [SID1234595908]). The research term will be extended to November 15, 2023, and Regeneron will pay Decibel an extension fee of $10 million in Q4 of 2022.

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Under the collaboration launched in 2017, Decibel is developing three gene therapy programs targeting congenital, monogenic hearing loss with Regeneron. Decibel plans to initiate in 2022 a Phase 1/2 clinical trial of DB-OTO, the Company’s lead gene therapy product candidate, designed to provide hearing to individuals born with profound hearing loss due to mutation of the otoferlin gene. Decibel is also advancing AAV.103 and AAV.104, gene therapy programs targeting other monogenic forms of hearing loss, with Regeneron. AAV.103 aims to restore hearing in individuals with mutations in the GJB2 gene, and AAV.104 aims to restore hearing in individuals with mutations in the STRC gene.

"Decibel and Regeneron scientists have worked closely to advance our gene therapy pipeline for the treatment of congenital, monogenic hearing loss, bringing our lead program, DB-OTO, within sight of the clinic. We are pleased that Regeneron has elected to extend the research term, which extends our access to Regeneron’s world-leading genomic and genetic technologies, and therapeutic discovery and development expertise," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "This collaboration, which plays to each party’s strengths, is an important part of our ability to accelerate the discovery and development of innovative genetic therapies for patients in need."

"The inner ear is a highly promising frontier for gene therapy, and we believe that the programs being developed in collaboration with Decibel could lead to novel medicines that help people with congenital, monogenic hearing loss," said George D. Yancopoulos, M.D., Ph.D., Co-Founder, President and Chief Scientific Officer, Regeneron. "We’re pleased to extend this successful collaboration, which is an important component of Regeneron’s growing genetics medicine portfolio. Regeneron and Decibel will continue to work together to combine novel gene therapy technologies with deep biologic expertise, so as to bring life-changing medicines to those suffering with hearing loss."

Through the collaboration, Regeneron provides Decibel with broad access to its proprietary suite of technologies to support Decibel’s goal of discovering new medicines for congenital, monogenic hearing loss. Regeneron also directly participates in and provides financial support for Decibel’s research and development efforts under the collaboration through milestone payments and reimbursement intended to fund approximately half of the costs of the collaboration programs. Decibel retains worldwide development and commercialization rights to the product candidates being developed in the collaboration and will pay Regeneron tiered royalties based on net sales.

Entry into a Material Definitive Agreement

On November 22, 2021 BioCryst Pharmaceuticals, Inc. (the "Company") reported that entered into the following material definitive agreements: (i) a Purchase and Sale Agreement entered into with RPI 2019 Intermediate Finance Trust ("RPI") (the "2021 RPI Royalty Purchase Agreement"), pursuant to which the Company sold to RPI the right to receive certain royalty payments from the Company for a purchase price of $150 million in cash, and (ii) a Purchase and Sale Agreement entered into with OCM IP Healthcare Holdings Limited, an affiliate of OMERS Capital Markets ("OMERS") (the "OMERS Royalty Purchase Agreement", together with the 2021 RPI Royalty Purchase Agreement, the "Royalty Purchase Agreements"), pursuant to which the Company sold to OMERS the right to receive certain royalty payments from the Company for a purchase price of an additional $150 million in cash (Filing, 8-K, BioCryst Pharmaceuticals, NOV 22, 2021, View Source [SID1234595907]).

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Under the 2021 RPI Royalty Purchase Agreement, RPI is entitled to receive tiered, sales-based royalties on net product sales of ORLADEYO in the United States and certain key European markets (collectively, the "Key Territories"), and other markets where the Company sells ORLADEYO directly or through distributors (collectively, the "Direct Sales") in an amount equal to: (i) 0.75% of aggregate annual net sales of ORLADEYO for annual net sales up to $350 million and (ii) 1.75% of annual net sales of ORLADEYO for annual net sales between $350 million and $550 million (with no royalty payments payable on annual net sales over $550 million). RPI is also entitled to receive a tiered revenue share on amounts generally received by the Company on account of ORLADEYO sublicense revenue or net sales by licensees outside of the Key Territories (the "Other Markets") in an amount equal to: (i) 3.0% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets, (ii) 3.0% of proceeds received by the Company on annual net sales of up to $150 million in the Other Markets, and (iii) 2.0% of proceeds received by the Company on annual net sales between $150 million and $230 million in the Other Markets (with no royalty payments payable on annual net sales above $230 million in the Other Markets).

Under the 2021 RPI Royalty Purchase Agreement, RPI is also entitled to receive tiered, sales-based royalties on net product sales of BCX9930 and another earlier stage Factor D inhibitor in an amount equal to: (i) 3.0% of worldwide aggregate annual net sales up to $1.5 billion and (ii) 2.0% of worldwide aggregate annual net sales between $1.5 billion and $3 billion (with no royalty payments payable on annual net sales above $3 billion). RPI is also entitled to receive tiered profit share amounts of up to 3.0% from certain other permitted sales in certain other markets.

The royalties payable under the 2021 RPI Royalty Purchase Agreement are in addition to the royalties payable to RPI under that certain Purchase and Sale Agreement by and between the Company and RPI dated as of December 7, 2020.

Under the OMERS Royalty Purchase Agreement, commencing with the calendar quarter beginning October 1, 2023, OMERS will be entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 7.5% of aggregate annual net sales of ORLADEYO for annual net sales up to $350 million and (ii) 6.0% of annual net sales of ORLADEYO for annual net sales between $350 million and $550 million (with no royalty payments payable on annual net sales over $550 million) (the "Regime A Royalty Rate"). If annual Direct Sales for calendar year 2023 reach a specified amount set forth in the OMERS Royalty Purchase Agreement, then for each calendar quarter beginning on or after January 1, 2024, OMERS will be entitled to receive the Regime A Royalty Rate. If annual Direct Sales for calendar year 2023 are less than the specified amount, OMERS will be entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 10.0% of aggregate annual net sales of ORLADEYO for annual net sales up to $350 million and (ii) 3.0% of annual net sales of ORLADEYO for annual net sales between $350 million and $550 million (with no royalty payments payable on annual net sales over $550 million) (the "Regime B Royalty Rate").

In addition, OMERS is also entitled to receive a tiered revenue share on amounts generally received by the Company on account of ORLADEYO sublicense revenue or net sales by licensees in the Other Markets in an amount equal to: (i) 20.0% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets, (ii) 20.0% of proceeds received by the Company on annual net sales of up to $150 million in the Other Markets, and (iii) 10.0% of proceeds received by the Company on annual net sales between $150 million and $230 million in the Other Markets (with no royalty payments payable on annual net sales above $230 million in the Other Markets). OMERS is also entitled to receive profit share amounts of up to 10% from certain other permitted sales in certain other markets.

The Company will be required to make payments (i) to RPI in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2021, and (ii) to OMERS in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2023. OMERS will no longer be entitled to receive any payments on the date in which aggregate payments actually received by OMERS equals either 142.5% or 155.0% of the $150 million purchase amount, depending on sales levels in calendar year 2023.

The transactions contemplated by each of the Royalty Purchase Agreements are referred to herein as the "Royalty Sales."

Under the Royalty Purchase Agreements, the Company has agreed to specified affirmative and negative covenants, including without limitation covenants regarding periodic reporting of information by the Company to RPI and OMERS, as applicable, third-party audits of royalties paid under the Royalty Purchase Agreements, and restrictions on the ability of the Company or any of its subsidiaries to incur indebtedness (which restrictions are eliminated after the achievement of certain milestones specified in the Royalty Purchase Agreements) other than certain royalty sales and as is permitted to be incurred under the terms of the Company’s Credit Agreement with Athyrium (each defined below), as amended. The Royalty Purchase Agreements also contain representations and warranties, other covenants, indemnification obligations, and other provisions customary for transactions of this nature.

The foregoing description of the Royalty Purchase Agreements does not purport to be complete and is qualified in its entirety by reference to the complete text of each of the Royalty Purchase Agreements, which will be filed as exhibits to the Company’s Annual Report on Form 10-K for the year ending December 31, 2021.

Common Stock Purchase Agreement

On November 19, 2021, concurrent with entering into the Royalty Purchase Agreements, the Company and RPI entered into a Common Stock Purchase Agreement (the "Stock Purchase Agreement"), pursuant to which the Company agreed to issue 3,846,154 shares of the Company’s common stock (the "Shares") for an aggregate purchase price of approximately $50 million, at a price of $13.00 per share, calculated based on the 20-day volume weighted average price. The Stock Purchase Agreement also contains representations and warranties, other covenants, indemnification obligations, and other provisions customary for transactions of this nature.

The foregoing description of the Stock Purchase Agreement does not purport to be complete and is qualified in its entirety by reference to the Stock Purchase Agreement, which will be filed as an exhibit to the Company’s Annual Report on Form 10-K for the fiscal year ending December 31, 2021.

Amendment Number One to Credit Agreement

On November 19, 2021, the Company entered into an amendment to its Credit Agreement, dated as of December 7, 2020, by and among the Company, as borrower; BioCryst Ireland Limited, a wholly-owned subsidiary of the Company, as guarantor; the other guarantors from time to time party thereto (all such guarantors, together with the borrower, the "Loan Parties"); the lenders from time to time party thereto; and Athyrium Opportunities III Co-Invest 1 LP ("Athyrium"), as lender and as administrative agent for the lenders (the "Existing Credit Agreement"). The Existing Credit Agreement provided for (i) an initial term loan in the principal amount of $125 million (the "Term A Loan") funded on December 7, 2020 and (ii) two additional term loans in the respective principal amounts of $25 million (the "Term B Loan") and $50 million (the "Term C Loan" and, collectively with the Term A Loan and the Term B Loan, the "Term Loans" and each, a "Term Loan").

The Amendment Number One to Credit Agreement, by and among the Loan Parties, the lenders party thereto, and Athyrium, amends the Existing Credit Agreement (the Existing Credit Agreement as so amended, the "Credit Agreement") (i) to permit the Company to enter into the 2021 RPI Royalty Purchase Agreement, the OMERS Royalty Purchase Agreement and the other definitive documentation related thereto and to perform its obligations thereunder; (ii) to require the Company to pay to Athyrium, for the account of the lenders, a make-whole premium plus certain fees set forth in the Credit Agreement in the event that the Company does not draw the Term B Loan or the Term C Loan, as applicable, by the end of the applicable period available to draw the Term B Loan or the Term C Loan, subject to certain exceptions set forth in the Credit Agreement; and (iii) to require the Company to pay to Athyrium, for the account of the lenders, a make-whole premium plus certain fees set forth in the Credit Agreement in the event that the Company either (x) terminates the commitments in respect of the Term B Loan or the Term C Loan, as applicable, on or prior to the end of the applicable period available to draw the Term B Loan or the Term C Loan, or (y) prepays or repays, or is required to prepay or repay, voluntarily or pursuant to a mandatory prepayment obligation under the Credit Agreement (e.g., with the proceeds of certain asset sales, certain ORLADEYO out-licensing or royalty monetization transactions (excluding the Royalty Sales), extraordinary receipts, debt issuances, or upon a change of control of the Company and specified other events, subject to certain exceptions), all of the then-outstanding Term Loans, in each case, subject to certain exceptions set forth in the Credit Agreement.

The foregoing description of the material terms of the Credit Agreement does not purport to be complete and is subject to, and is qualified in its entirety by reference to, the full text of the Credit Agreement, which will be filed as an exhibit to the Company’s Annual Report on Form 10-K for the year ending December 31, 2021.

The information set forth in Item 1.01 of this Current Report on Form 8-K under the caption "Common Stock Purchase Agreement" is incorporated by reference into this Item 3.02. The Shares were offered and sold without registration under the Securities Act of 1933, as amended (the "Securities Act"), in reliance on the exemptions provided by Section 4(a)(2) of the Securities Act as transactions not involving a public offering and Rule 506 promulgated under the Securities Act as sales to an accredited investor.

Cogent Biosciences to Present at Piper Sandler 33rd Annual Virtual Healthcare Conference

On November 22, 2021 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported Andrew Robbins, Chief Executive Officer and President will participate in a fireside chat and one-on-one investor meetings during the Piper Sandler 33rd Annual Healthcare Conference, taking place virtually November 29 – December 2, 2021 (Press release, Cogent Biosciences, NOV 22, 2021, View Source [SID1234595905]).

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The pre-recorded fireside chat will be available to conference attendees starting at 10:00 a.m. ET today and will remain available through the end of the conference on Thursday, December 2. Access to the recording will be available under the "Events" tab on the investor relations section of the Cogent Biosciences website at: View Source

Eagle Pharmaceuticals to Present at Piper Sandler 33rd Annual Healthcare Conference 2021

On November 22, 2021 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that Scott Tarriff, Chief Executive Officer, and Brian Cahill, Chief Financial Officer, will present virtually at the Piper Sandler 33rd Annual Healthcare Conference 2021 as follows (Press release, Eagle Pharmaceuticals, NOV 22, 2021, View Source [SID1234595904]):

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View Source

The webcast of the previously recorded presentation will be accessible on demand and archived for 90 days thereafter, via the Company’s website at www.eagleus.com, under the Investors section.

Eagle will be participating in 1×1 meetings on December 1. Meetings can be requested exclusively via Piper Sandler.

Apollomics Inc. Doses First Patient in Phase 3 Clinical Trial with APL-106 (Uproleselan Injection) in Chinese Patients with Relapsed/Refractory Acute Myeloid Leukemia

On November 22, 2021 Apollomics Inc., an innovative biopharmaceutical company committed to the discovery and development of mono- and combination- oncology therapies, reported that the first patient has been successfully dosed in a Phase 3 clinical trial of APL-106 (uproleselan injection) for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) in China (Press release, Apollomics, NOV 22, 2021, View Source [SID1234595902]). Apollomics licensed the Greater China rights for uproleselan from GlycoMimetics.

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"As our first Phase 3 clinical trial, dosing of the first patient with APL-106 is a major inflection point for Apollomics as we now become a company in late-stage development," said Guo-Liang Yu, PhD, Co-Founder, Chairman and Chief Executive Officer. "AML is a highly aggressive hematological cancer, and the prognosis of patients with relapsed or refractory disease is extremely poor. APL-106 acts via an innovative mechanism that drives cancer cells out of the bone marrow, making them a prime target to be killed by chemotherapy. This combined treatment approach could have a meaningful impact on the lives of patients living with relapsed or refractory AML."

The Phase 3 trial with APL-106 is part of the overall development program for Apollomics in China that also includes an ongoing Phase 1 pharmacokinetics (PK) and tolerability study. The Phase 3 clinical trial is a randomized, double-blind, placebo controlled, bridging study that will evaluate the efficacy of uproleselan in combination with chemotherapy, compared to chemotherapy alone, for treating relapsed/refractory AML, in Chinese patients. The trial will enroll approximately 140 adult patients with primary refractory AML or relapsed AML (first or second untreated relapse) and eligible to receive induction chemotherapy.

The primary endpoint for the trial is overall survival. Secondary outcome measures include the rate and duration of remission, and whether uproleselan could reduce the rate of oral mucositis, a chemotherapy-related side effect. Apollomics expects to conduct this study at approximately 20 blood cancer clinical research centers across China. Additional information on the Phase 3 trial can be found on clinicaltrials.gov (NCT05054543)

About APL-106 (uproleselan injection)

APL-106 (uproleselan injection) is an innovative drug discovered and developed by GlycoMimetics. Uproleselan (yoo’ pro le’ sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells, thereby disrupting the mechanism of leukemic cell resistance within the bone marrow microenvironment. Apollomics licensed uproleselan from GlycoMimetics, and Apollomics has the rights to clinical development, production and commercial sales in the Greater China market (Mainland China, Hong Kong, Macau and Taiwan). The U.S. Food and Drug Administration granted Breakthrough Therapy Designation to uproleselan for the treatment of adults with relapsed or refractory acute myeloid leukemia. APL-106 has also been granted Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is an aggressive disease that causes the bone marrow to produce immature cells that are unable to carry out their normal function and develop into leukemia cells. In the U.S., there are approximately 20,000 new cases of AML each year, and the 5-year survival rate is 28.7%1. The annual incidence of AML in China in 2019 is approximately 26,9002, and relapsed/refractory AML has an extremely poor prognosis.

1National Cancer Institute Surveillance, Epidemiology, and End Results (SEER) Program
2CIC Report