Mass. Biopharma Reports Positive Data and Announces New Program
Research Report

1. This article does not constitute investment advice and is not a solicitation for any investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Each reader is encouraged to consult with his or her personal financial adviser and perform their own comprehensive investment research. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company. 
2. This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

For additional disclosures, please click here.

Disclosures for Wedbush, Vor Biopharma Inc., September 6, 2024

Analyst Certification We, David Nierengarten, Martin Fan and Dennis Pak, certify that the views expressed in this report accurately reflect our personal opinions and that we have not and will not, directly or indirectly, receive compensation or other payments in connection with our specific recommendations or views contained in this report.

Company Specific Disclosures This information is subject to change at any time. 1. WS makes a market in the securities of Vor Biopharma.

Wedbush disclosure price charts are updated within the first fifteen days of each new calendar quarter per FINRA regulations. Price charts for companies initiated upon in the current quarter, and rating and target price changes occurring in the current quarter, will not be displayed until the following quarter. Additional information on recommended securities is available on request. Disclosure information regarding historical ratings and price targets is available: Research Disclosures *WS changed its rating system from (Strong Buy/ Buy/ Hold/ Sell) to (Outperform/ Neutral/ Underperform) on July 14, 2009. Applicable disclosure information is also available upon request by contacting the Research Department at (212) 833-1375, by email to [email protected]. You may also submit a written request to the following: Wedbush Securities, Attn: Research Department, 142 W 57th Street, New York, NY 10019.

OTHER DISCLOSURES The information herein is based on sources that we consider reliable, but its accuracy is not guaranteed. The information contained herein is not a representation by this corporation, nor is any recommendation made herein based on any privileged information. This information is not intended to be nor should it be relied upon as a complete record or analysis: neither is it an offer nor a solicitation of an offer to sell or buy any security mentioned herein. This firm, Wedbush Securities, its officers, employees, and members of their families, or any one or more of them, and its discretionary and advisory accounts, may have a position in any security discussed herein or in related securities and may make, from time to time, purchases or sales thereof in the open market or otherwise. The information and expressions of opinion contained herein are subject to change without further notice. The herein mentioned securities may be sold to or bought from customers on a principal basis by this firm. Additional information with respect to the information contained herein may be obtained upon request. Wedbush Securities does and seeks to do business with companies covered in its research reports. Thus, investors should be aware that the firm may have a conflict of interest that could affect the objectivity of this report. Investors should consider this report as only a single factor in making their investment decision. Please see pages 3–7 of this report for analyst certification and important disclosure information. Retail Investors The information provided is for general informational purposes only and should not be considered an individual recommendation or personalized investment advice. The companies/investments mentioned may not be suitable for everyone. Each investor needs to review their own respective situation(s) before making any investment decisions. All expressions of opinion are subject to change without notice due to shifting market(s), economic or political conditions. Investment involves risks including the risk of principal. Past performance is no guarantee of future results and the opinions presented cannot be viewed as an indicator of future performance.

Wedbush analysts Dr. David Nierengarten, Dr. Martin Fan, and Dennis Pak, in a research report published on September 6, 2024, provided an update on Vor Biopharma Inc. (VOR:NASDAQ) following the company's announcement of updated results for its Phase 1/2 VBP101 study of trem-cel + Mylotarg in AML or MDS, initial PK data for VCAR33ALLO, and the development of a new CD45 ADC. The analysts maintained their Outperform rating and a 12-month price target of US$11.00 on the stock.

The analysts highlighted the positive relapse-free survival (RFS) data from the VBP101 study, noting, "VOR presented RFS data — the median has not yet been reached in patients at a median follow-up of 169 days (6 months), which is the upper end of median RFS described in the literature for similar, high-risk patients." They added that historical data for comparable patient groups suggest median RFS of three to six months.

Regarding the new CD45 ADC program (VADC45), the analysts commented, "We view the conditioning regimen use case as having the fastest path toward commercial development, with limited competition and considerable partnership potential."

The Wedbush team also noted positive initial PK results for VCAR33ALLO, stating, "Initial PK results for VCAR33ALLO demonstrated in vivo expansion at the lowest dose of 1 x 10⁶ cells/kg, with dose escalation planned up to 1 x 10⁷ cells/kg."

Vor Biopharma's strategic focus includes advancing trem-cel towards a pivotal trial, with plans to discuss this with the FDA by year-end 2024. The company is also expanding its pipeline with the new VADC45 program.

The analysts expressed a positive outlook on Vor Biopharma's prospects, stating, "With alignment on a pivotal study possible in the next six months and significant upside potential from CD45-based depletion approaches, we view VOR as an increasingly compelling value."

Wedbush's valuation methodology for Vor Biopharma is based on a sum-of-parts approach. "Our PT is derived from a sum-of-parts valuation for the company's VOR33 and VCAR33 programs: an 8x multiple of 2030E ww sales of VOR33 (discounted back by 35%), and an 8x multiple of 2031E ww sales of VCAR33 (discounted back by 40%)," the analysts explained.

With an Outperform rating and a price target of US$11.00, representing a potential return of approximately 1,241% from the price at the time of the report of US$0.82, Wedbush sees significant upside potential for Vor Biopharma.

The analysts concluded, "Shares continue to trade near or below cash even with additional de-risking of the lead trem-cel program, and we would be buyers ahead of planned regulatory updates and likely further data readouts."