On May 15, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the first quarter of 2024 and recent corporate progress (Press release, Abeona Therapeutics, MAY 15, 2024, View Source [SID1234643313]).

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"We are grateful to our existing as well as new investors who have demonstrated their support through the recent financing, which has extended our cash runway into 2026, well beyond anticipated regulatory milestones," said Vish Seshadri, Chief Executive Officer of Abeona. "We now remain focused on working with the FDA to address the CMC deficiencies noted in the CRL and making the BLA resubmission to bring pz-cel to RDEB patients as soon as possible."

First Quarter and Recent Progress

Corporate highlights

● On May 7, 2024, Abeona closed a $75 million underwritten securities offering with participation from both new and existing investors.
● In January 2024, Abeona entered into a $50 million credit facility and received the first tranche of $20 million.

Pz-cel for RDEB

● In April 2024, Abeona received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Company’s Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB), based on the need for additional Chemistry Manufacturing and Controls (CMC) information. In the CRL, the FDA noted that certain additional information needed to satisfy CMC requirements must be resolved before the application can be approved. The information needed to satisfy the CMC requests in the CRL pertains to validation requirements for certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The Company anticipates completing the BLA resubmission in the second half of 2024.
● New pz-cel data will be presented at upcoming medical meetings. At the Society for Investigative Dermatology (SID) Annual Meeting, being held on May 15-18, 2024, new long-term safety data with up to 11 years of follow-up has been accepted as a late-breaking presentation.

U.S. commercial launch preparations for pz-cel

● Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with epidermolysis bullosa treatment sites, conducting medical and payer engagement, as well as building supply chain and enterprise capabilities to support the Company’s transition to a commercial stage company.

First Quarter Financial Results and Cash Runway Guidance

Cash, cash equivalents, restricted cash and short-term investments totaled $62.7 million as of March 31, 2024, compared to $52.6 million as of December 31, 2023. Net cash used in operating activities was $14.5 million for the three months ended March 31, 2024.

Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility, combined with the net proceeds from the underwritten securities offering, are sufficient resources to fund operations into 2026, before accounting for any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher or PRV, if awarded by the FDA.

Research and development expenses for the three months ended March 31, 2024 were $7.2 million, compared to $8.0 million for the same period of 2023. General and administrative expenses were $7.1 million for the three months ended March 31, 2024, compared to $4.0 million for the same period of 2023. Net loss was $31.6 million for the first quarter of 2024, or $1.16 loss per common share, including a change in the fair value of warrant liabilities due to remeasurement of the Company’s issued stock purchase warrants. Net loss in the first quarter of 2023 was $9.1 million, or $0.54 loss per common share.

Conference Call Details

The Company will host a conference call and webcast on Wednesday, May 15, 2024, at 8:30 a.m. ET, to discuss the first quarter results. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 496484 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at View Source The archived webcast replay will be available for 30 days following the call.

On May 14, 2024 Eagle Pharmaceuticals, Inc. reported the company entered into a Third Amendment to Third Amended and Restated Credit Agreement (the "Third Amendment Agreement") with JPMorgan Chase Bank, N.A., as administrative agent (the "Agent"), and the lenders party thereto (the "Lenders"), with an effective date of May 13, 2024, which amends the terms of (i) the Company’s Third Amended and Restated Credit Agreement, dated as of November 1, 2022 (as amended by the First Amendment Agreement (defined below), the "Original Credit Agreement") and (ii) the Limited Waiver and First Amendment to Third Amended and Restated Credit Agreement, dated as of January 12, 2024 (as amended by the Second Amendment Agreement (defined below), the "First Amendment Agreement") (Press release, Eagle Pharmaceuticals, MAY 15, 2024, View Source [SID1234643301]). The Original Credit Agreement as amended by that certain Second Amendment to Third Amended and Restated Credit Agreement, dated as of February 29, 2024 (the "Second Amendment Agreement"), is referred to herein as the "Credit Agreement," and the Credit Agreement as amended by the Third Amendment Agreement is referred to herein as the "Amended Credit Agreement."

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As previously disclosed (i) in the Company’s Current Report on Form 8-K filed with the U.S. Securities and Exchange Commission (the "SEC") on January 16, 2024, incorporated by reference herein, the Company, the Agent and the Lenders entered into the First Amendment Agreement, which provided a waiver of defaults and events of default that occurred and were continuing under the Original Credit Agreement at such time and (ii) in the Company’s Current Report on Form 8-K filed with the SEC on March 1, 2024, incorporated by reference herein, the Company, the Agent and the Lenders entered into the Second Amendment Agreement, which amended certain terms of the Original Credit Agreement and the First Amendment Agreement.

Pursuant to the Credit Agreement and the First Amendment Agreement, (i) the Company is required to deliver to the Agent and the Lenders, by not later than May 13, 2024, (a) annual audited financial statements for the fiscal year ended December 31, 2023, reported on by the Company’s independent public accountant (without a "going concern" or like qualification or exception and without any qualification or exception as to the scope of such audit) to the effect that such consolidated financial statements present fairly in all material respects the financial condition and results of operations of the Company and its consolidated subsidiaries in accordance with GAAP (the "Annual Financial Statement Requirement"), (b) restated quarterly financial statements for the fiscal quarter ended June 30, 2023 and (c) quarterly financial statements for the fiscal quarter ended September 30, 2023, (ii) the Company is required to deliver to the Agent and the Lenders, by not later than June 3, 2024, quarterly financial statements for the fiscal quarter ended March 31, 2024, (iii) the Company is required to deliver to the Agent and the Lenders, concurrently with delivery of the financials statements referred to in the foregoing clauses (i)(b), (i)(c) and (ii), a certificate by one of its officers that such financial statements present fairly in all material respects the financial condition and results of operations of the Company and its consolidated subsidiaries in accordance with GAAP for the respective quarter (clauses ((i)(b), (i)(c) and (ii), the "Quarterly Financial Statement Requirement" and together with the Annual Financial Statement Requirement, the "Financial Statement Requirement") and (iv) until the Quarterly Financial Statement Requirement has been satisfied, (a) availability under the revolving facility under the Credit Agreement is reduced from $100 million to $50 million (the "Availability Restriction"), (b) the Company is not permitted to utilize any negative covenant flexibility that is based on a pro forma compliance with any of the Fixed Charge Coverage Ratio, Senior Secured Net Leverage Ratio and/or the Total Net Leverage Ratio test (each as defined in the Credit Agreement), which restricts the Company’s flexibility to, among other things, incur certain additional indebtedness, complete certain corporate transactions, including certain acquisitions and dispositions, or make certain additional restricted payments (the "Covenant Flexibility Restriction") and (c) compliance with the minimum liquidity covenant is waived (the "Liquidity Covenant Waiver").

Pursuant to the terms of the Third Amendment Agreement, (i) the delivery deadline with respect to each Financial Statement Requirement has been extended to July 31, 2024, (ii) the Availability Restriction has been superseded by a permanent reduction of availability under the revolving facility under the Amended Credit Agreement from $100 million to $50 million, (iii) the Covenant Flexibility Restriction has been revised to continue until the Company has satisfied each Financial Statement Requirement, (iv) the Liquidity Covenant Waiver has been terminated and (v) the minimum liquidity covenant in the Amended Credit Agreement has been reduced from $50 million to $10 million.

Pursuant to the terms of the Amended Credit Agreement, failure to timely satisfy the Financial Statement Requirement will result in an event of default. During the continuance of an event of default, the Agent may, with the consent of the required lenders, and shall, at the request of the required lenders, by notice to the Company, terminate undrawn commitments, declare the loans then outstanding to be due and payable in full and/or exercise other remedies available to it, among other things. In addition, the Company’s obligations under the Amended Credit Agreement are secured by a pledge of substantially all of the Company’s assets. If the Company is unable to pay its obligations, the Agent on behalf of the lenders could proceed to protect and enforce their rights under the Amended Credit Agreement, including by foreclosure on the assets securing the Company’s obligations under the Amended Credit Agreement. The foregoing would materially and adversely affect the Company’s business and financial condition. There can be no assurance that the Company will be able to satisfy the Financial Statement Requirement on the required timing or at all, or comply with the terms of the Third Amendment Agreement and the Amended Credit Agreement.

The foregoing descriptions of the Third Amendment Agreement, the Second Amendment Agreement, the First Amendment Agreement and the Original Credit Agreement are not intended to be complete and are qualified in their entirety by reference to the full text of the Third Amendment Agreement, the Second Amendment Agreement, the First Amendment Agreement and the Original Credit Agreement, which are filed as Exhibit 10.1 to this Current Report on Form 8-K, Exhibit 10.1 to the Company’s Current Report on Form 8-K filed on March 1, 2024, Exhibit 10.1 to the Company’s Current Report on Form 8-K filed on January 16, 2024, and Exhibit 10.1 to the Company’s Current Report on Form 8-K filed on November 3, 2022, respectively.

On May 14, 2024 Bayer reported its first quarter 2024 results (Presentation, Bayer, MAY 14, 2024, View Source [SID1234644682]).

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On May 16, 2024 HEPHAISTOS-Pharma, a biotechnology company developing next generation immunotherapies against cancer targeting innate immunity to increase the cure rate of patients, reported its €4.5 million seed round to finance industrialization and advance its lead candidate ONCO-Boost towards the clinic (Press release, HEPHAISTOS-Pharma, MAY 14, 2024, View Source [SID1234643393]). Elaia leads a consortium previously initiated by xista science ventures, the Fondation Fournier-Majoie and Noshaq.

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Florian DENIS, Investment Director at Elaia, stated: "Elaia is very proud to join and strengthen the syndication. HEPHAISTOS’ core technology based on immunostimulants has the potential to turn cold tumors into hot tumors in hard-to-treat cancers. Frédéric CAROFF and his team have generated impressive preclinical data that will be scaled up towards additional value-creating milestones."

"ELAIA has a proven track record in supporting biotech companies through to the clinic and success, and we are delighted to have them on board " said Frederic CAROFF, CEO and co-founder of HEPHAISTOS-Pharma. "With xista science ventures, Fondation Fournier-Majoie and Noshaq, our consortium of seed investors brings not only the necessary funds, but also complementary expertise and a network that will structure the company. They will play a crucial role in the success of HEPHAISTOS. "

HEPHAISTOS’ innovative approach to innate modulation has received significant validation and support from prestigious programs, with a total of €5.8 million awarded to accelerate the development of ONCO-Boost for the treatment of hard-to-treat solid tumors, where there is a high unmet medical need, BPIFRANCE iNov national competition, EIC Accelerator European competition and RHU clinical grant from France 2030, that will include a clinical phase 1/2 in collaboration with prestigious clinical centers.

This seed round will mainly finance the industrialization of drug production and regulatory toxicity studies to file for the CTA in Europe. HEPHAISTOS already has a lot of preclinical data in many cancer indications, but they plan to generate new additional data in monotherapy against unmet medical needs and in combination with promising drugs from other pharmaceutical companies. Finally, it will also help to recruit new talents and complete the structuring of the board.

On May 14, 2024 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, reported financial results for the quarter ended March 31, 2024 and provided a corporate update (Press release, Syros Pharmaceuticals, MAY 14, 2024, View Source [SID1234643280]).

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"In 2024, we are acutely focused on execution across clinical and pre-commercial activities as we advance tamibarotene toward critical milestones, including additional data from the Phase 2 SELECT-AML-1 trial in the third quarter and pivotal CR data from the Phase 3 SELECT-MDS-1 trial in the fourth quarter," said Conley Chee, Chief Executive Officer of Syros. "We are particularly pleased to share today that an independent data monitoring committee recently completed a pre-specified interim futility analysis on 50% of the patients enrolled in the SELECT-MDS-1 trial to support our primary endpoint analysis, and recommended that our study continue without modification. This recommendation, together with the FDA’s decision to grant Fast Track Designation to tamibarotene in AML, reinforces our confidence in the potential for our RARα agonist to offer improved clinical outcomes to HR-MDS and AML patients with RARA gene overexpression, supported by our belief that tamibarotene has a differentiated safety profile well suited for use in these patients."

Mr. Chee continued, "In addition, following the completion of enrollment in the first quarter of 2024 of the 190 patients necessary for our primary endpoint analysis in the Phase 3 SELECT-MDS-1 trial, we have begun preparing for our first New Drug Application filing and subsequent launch in the United States. We look forward to engaging further with the medical community to drive awareness of tamibarotene and the companion diagnostic to identify RARA overexpression in patients with higher-risk MDS, as we work to deliver tamibarotene as the new frontline standard-of-care for patients with RARA overexpression."

Syros reported plans to host a webcast event on June 25, 2024 to discuss disease biology and the current treatment landscape in HR-MDS, as well as the design of the ongoing pivotal Phase 3 SELECT-MDS-1 trial and opportunity for tamibarotene. The event will feature presentations from medical experts, in addition to Syros management. The event will be webcast live on the Investors & Media section of Syros’ website, www.syros.com. More details for the event are forthcoming.

UPCOMING MILESTONES

Report pivotal complete response (CR) data from the SELECT-MDS-1 Phase 3 trial in newly diagnosed HR-MDS patients with RARA gene overexpression by the middle of the fourth quarter of 2024.
Report clinical activity and tolerability data from a prespecified analysis of over 40 patients from the SELECT-AML-1 Phase 2 trial in unfit AML patients with RARA overexpression in the third quarter of 2024.
RECENT PIPELINE HIGHLIGHTS

In March, the Phase 3 SELECT-MDS-1 clinical trial of tamibarotene passed a pre-specified interim futility analysis based on the CR rate, which was conducted by an Independent Data Monitoring Committee (IDMC). There were no concerning safety signals noted in the analysis and the IDMC recommended SELECT-MDS-1 continue without modification. Syros remains blinded to the data.
In April, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to tamibarotene in combination with venetoclax and azacitidine for the treatment of newly diagnosed AML with RARA overexpression, as detected by an FDA approved test in adults who are over age 75 and who have comorbidities that preclude the use of intensive induction chemotherapy. The FDA previously granted this designation to tamibarotene in combination with azacitidine for the treatment of adults with HR-MDS and RARA overexpression in January 2023.
First Quarter 2024 Financial Results

The Company did not recognize any revenue in the first quarter of 2024, as compared to $3.0 million for the first quarter of 2023. The decrease reflects the termination of Syros’ collaboration agreement with Pfizer.
Research and development (R&D) expenses were $24.7 million for the first quarter of 2024, as compared to $28.8 million for the first quarter of 2023. The decrease was primarily due to the reduction in external R&D consulting, contract manufacturing, and a reduction in headcount and related expenses.
General and administrative (G&A) expenses were $6.3 million for the first quarter of 2024, as compared to $7.4 million for the first quarter of 2023. The decrease was primarily due to a reduction of headcount and related expenses, consulting fees, and facilities expenses.
For the first quarter of 2024, Syros reported a net loss of $3.7 million, or $0.10 per share, compared to a net loss of $23.8 million, or $0.85 per share, for the same period in 2023.
Cash and Financial Guidance

On May 9, 2024, the Company agreed to amend its Loan Agreement with Oxford Finance LLC. The amendment will increase the amount of term loans available to Syros from $40 million to $100 million with tranches totaling $40.0 million in the aggregate becoming available upon the achievement of certain clinical development, regulatory and equity-raising milestones, and $20.0 million becoming available at Oxford’s discretion. In addition, Oxford will extend the interest only period from September 1, 2024 to November 1, 2025 with further extensions to as late as November 1, 2026 upon achievement of certain milestones.

Cash, cash equivalents and marketable securities as of March 31, 2024, were $108.3 million, as compared with $139.5 million as of December 31, 2023.

Based on its current plans and the recently executed amendment with Oxford, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its anticipated operating expenses and capital expenditure requirements into the third quarter of 2025, beyond pivotal Phase 3 data from the SELECT-MDS-1 trial and additional data from the randomized portion of the SELECT-AML-1 trial.

Conference Call and Webcast

Syros will host a conference call today at 8:30 a.m. ET to discuss the first quarter 2024 financial results and provide a corporate update.

To access the live conference call, please dial (800) 549-8228 (domestic) or (289) 819-1520 (international) and refer to conference ID 16518. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.