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CLEVELAND - Abeona Therapeutics Inc. (NASDAQ:ABEO) has announced significant progress towards the resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), an investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). Following a Type A meeting on August 8, 2024, with the U.S.
The company has completed data generation for most of the CMC deliverables required by the FDA and is validating two outstanding items related to sterility and identity assays. Abeona expects to resubmit the BLA in the latter half of 2024 and, if accepted, anticipates a Prescription Drug User Fee Act (PDUFA) action date six months from submission.
Abeona's CEO, Vish Seshadri, stated that the CRL did not raise concerns about the clinical efficacy or safety data of pz-cel and did not require new clinical trials. The company has also presented new long-term safety data and data on wound healing after pz-cel treatment at recent medical meetings.
In preparation for a potential U.S. launch of pz-cel, Abeona is engaging with treatment sites and medical and payer communities while building supply chain capabilities. Additionally, the company closed a $75 million securities offering in May 2024 and reports a net income of $7.4 million for the Q2 of 2024, a significant turnaround from the net loss reported in the same period of 2023.
As of June 30, 2024, Abeona's financial position includes $123.0 million in cash, cash equivalents, short-term investments, and restricted cash, with an estimated runway to fund operations into 2026, not accounting for potential revenue from pz-cel or proceeds from a Priority Review Voucher, if awarded.
The company's pipeline also features AAV-based gene therapies for ophthalmic diseases and a non-exclusive agreement with Beacon Therapeutics to evaluate Abeona’s patented AAV204 capsid for ophthalmology indications.
Abeona Therapeutics has been making significant strides in its operations. The biopharmaceutical company has recently announced a partnership with Beacon Therapeutics to explore the potential of a novel gene therapy technology for treating eye diseases.
H.C. Wainwright reaffirmed its Buy rating for Abeona Therapeutics, highlighting the potential of Abeona's adeno-associated viral vector (AAV) platform, known as AIM. The firm suggests that this partnership with Beacon could expand and hasten the development of new pipeline programs utilizing the AIM vector platform.
Financial services company, Stifel, has initiated coverage of Abeona Therapeutics, assigning a 'buy' rating with a price target of $21.00. Stifel's optimism is primarily based on the potential of Abeona's therapy, Prademagene zamikeracel, a treatment for a rare genetic skin disorder.
However, Abeona has faced challenges with the FDA requesting additional data for its Biologics License Application (BLA) for this therapy. Despite this, the company anticipates resubmitting the BLA in the third quarter of 2024.
In a separate development, Abeona has set terms for a $75 million stock offering, expected to close in May 2024. The proceeds will be used to fund preparations for the BLA resubmission, commercialization efforts for its product candidate, and other general corporate purposes.
InvestingPro Insights
Abeona Therapeutics Inc. (NASDAQ:ABEO) has shown a noteworthy turnaround in its financial performance, as evidenced by the company's net income of $7.4 million for the second quarter of 2024. This positive shift in profitability is particularly significant given the company's historical financial challenges. InvestingPro data reveals a substantial 227.72% revenue growth in the last twelve months as of Q1 2024, demonstrating Abeona's ability to increase its revenue base substantially. However, this growth comes with the backdrop of a negative gross profit margin of -810.34% in the same period, highlighting the costs associated with the expansion.
Investors should note that Abeona's market capitalization stands at $181.06M, reflecting the market's valuation of the company's potential. Despite the impressive revenue growth, the company's stock price has experienced considerable volatility, with a 19.78% decline over the last month, aligning with one of the InvestingPro Tips that points out the stock's price movements are quite volatile. This volatility could be attributed to the market's reaction to the company's financial data and its ongoing efforts to bring its gene therapy product to market.
On a strategic note, Abeona holds more cash than debt on its balance sheet, which is a positive sign of financial health and stability. This is supported by the fact that as of June 30, 2024, the company reported having $123.0 million in cash and equivalents, which could provide a runway to fund operations well into 2026. This aligns with another InvestingPro Tip indicating that the company's liquid assets exceed its short-term obligations. Investors can find more detailed analysis and additional InvestingPro Tips on Abeona, with a total of 10 tips available, by visiting https://www.investing.com/pro/ABEO.
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