Share Price Appreciation of Biotech Due Over Next Six Months
Research Report

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Disclosures for Wedbush, Beam Therapeutics, April 6, 2025

Valuation Sum of parts: 8x multiple to 2030-2034E US/EU sales for each program (T-ALL, AML, SCD, TDBT, AATD, GSD1a), with 30-35% discount rates applied. Company Description Beam Therapeutics is a Cambridge, MA-based preclinical-stage biotechnology company developing gene editing candidates, with their novel base editing platform, for rare genetic diseases and cancer. Risks to the Attainment of Our Price Target and Rating: Risks to our valuation and price target include poorer-than-expected preclinical or clinical data for BEAM’s product candidates, particularly if greater-than-anticipated off-target activity or any other unanticipated safety signals are shown, as this would have significant negative read-through to each of the company’s product candidates. Longer-term risks include greater-than-expected competition, which could adversely and materially impact each product candidate’s market potential and valuation. There is also political, regulatory or commercial risk if BEAM is unable to command favorable reimbursement and adoption of its marketed products. 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. Mentioned Companies Investment Rating System: OUTPERFORM: Expect the total return of the stock to outperform relative to the median total return of the analyst's (or the analyst's team) coverage universe over the next 6-12 months. NEUTRAL: Expect the total return of the stock to perform in-line with the median total return of the analyst's (or the analyst's team) coverage universe over the next 6-12 months. UNDERPERFORM: Expect the total return of the stock to underperform relative to the median total return of the analyst's (or the analyst's team) coverage universe of the next 6-12 months. The Investment Ratings are based on the expected performance of a stock (based on anticipated total return to price target) relative to the other stocks in the analyst's coverage universe (or the analyst's team coverage).* Rating distribution (as of April 6, 2025) Investment Banking Relationships (as of April 6, 2025) OUTPERFORM: 65.41% OUTPERFORM: 7.44% NEUTRAL: 32.16% NEUTRAL: 3.36% UNDERPERFORM: 2.43% UNDERPERFORM: 0.00% The Distribution of Ratings is required by FINRA rules; however, WS' stock ratings of Outperform, Neutral, and Underperform most closely conform to Buy, Hold, and Sell, respectively. Please note, however, the definitions are not the same as WS' stock ratings are on a relative basis. The analysts responsible for preparing research reports do not receive compensation based on specific investment banking activity. The analysts receive compensation that is based upon various factors including WS' total revenues, a portion of which are generated by WS' investment banking activities. Company Specific Disclosures This information is subject to change at any time. 1. WS makes a market in the securities of Beam Therapeutics. 

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 leslie.lippai@wedbush.com. 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.

Beam Therapeutics Inc. (BEAM:NASDAQ) delivered follow-up data for BEAM-302, an investigational in vivo gene therapy, or base editing medicine, intended to correct the underlying cause of alpha-1 antitrypsin deficiency (AATD), reported Wedbush Analyst Dr. David Nierengarten in an April 6 research note.

"With a clear pathway toward commercial entry in sickle cell disease and continuing room for differentiation in AATD, we anticipate considerable share price movement in the next six months," Nierengarten wrote.

273% Return Potential

Following the recent news, Wedbush reiterated its US$57 per share target price on the biotech, noted Nierengarten. In comparison, Beam was trading at the time of the report at about US$15.27 per share. Thus, the implied return for investors is 273%.

"[We] see recent price action as a buying opportunity," Nierengarten wrote.

Beam is rated Outperform.

New Info Provided

According to the life sciences firm, updated biomarker results from the 60 milligram (60 mg) cohort showed decreasing levels of mutant Z-AAT and a high proportion of corrected M-AAT (mean 91%) at 28 days. The mean percent change in Z-AAT at Day 28 was 11% among patients on the 15 mg dose of BEAM-302, 38% for those on 30 mg, and 79% for individuals receiving 60 mg. Beam reported other data last month.

The biotech also indicated that 75 mg will be the final dose evaluated, the others having been 15, 30, and 60 mg.

"We continue to regard 11 microns of serum AAT, already achieved at the 60 mg dose of BEAM-302 but not by competing gene therapies, as the threshold necessary for regulatory approval," Nierengarten wrote. "We view increases from these levels as providing upside, noting the existing benchmark does not consider proportion of corrected M-AAT."

Events To Watch For

Beam has multiple catalysts slated for this year, the analyst pointed out and listed them. Launch of a Phase 1/2 trial of BEAM-301 in GSD1a, an autosomal recessive disorder, is expected by this quarter's end. Updated results are due out midyear from the Phase 1/2 BEACON trial evaluating BEAM-101, an investigational, base-edited cell therapy, in sickle cell disease.

Anticipated events in H2/25 include the release of updated results from the Phase 1/2 trial of BEAM-302 in AATD and the start of patient enrollment for this study's liver disease cohort.

Late this year, the trial of BEAM-103 in healthy volunteers will be launched. BEAM-103 is an anti-CD117 monoclonal antibody designed to suppress hematopoietic stem and progenitor cells expressing CD117, part of the biotech's ESCAPE technology. Also coming up later in 2025 is the release of further data on nonhuman primates for ESCAPE.