Initiation of Research Coverage of Two Gene Therapy Companies

Tuesday, July 10, 2018

William Blair & Company initiated research coverage of two gene therapy companies—Krystal Biotech, Inc. (KRYS $15.07) and Rocket Pharmaceuticals, Inc. (RCKT $19.17).

Analyst Raju Prasad said that shares of Krystal Biotech hold a positive risk/reward profile ahead of clinical data expected by the end of the year. Krystal is developing novel gene therapies for skin diseases; lead candidate KB103 is in a Phase I/II trial for the treatment of dystrophic epidermolysis bullosa (DEB), a rare and severe genetic disease affecting the skin and mucosal tissues caused by one or more mutations in the COL7A1 gene, with no currently approved therapies. KB103 leverages Krystal’s Skin TARgeted Delivery (STAR-D) platform, which consists of a proprietary engineered viral vector based on herpes simplex virus 1 (HSV-1) that is both non-integrating and non-replicating.

Prasad said that the positive risk/reward profile is “a result of KB103’s mechanism of action and preclinical data, recent proof-of-concept with gene-corrected autologous skin grafts, and Amgen’s Imlygic, an HSV-1-based product already approved for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery.”

Prasad noted that Rocket Pharmaceuticals’ lead candidate, RP-L102, is an ex vivo lentiviral gene therapy being developed for the treatment of Fanconi anemia (FA), with three additional ex vivo lentivirus programs in earlier development for the treatment of leukocyte adhesion deficiency-I (LAD-I), pyruvate kinase deficiency (PKD), and infantile malignant osteopetrosis (IMO). He also expects the company to disclose its in vivo adeno-associated virus (AAV) gene therapy indication by year end, as the company anticipates having up to four programs in the clinic (likely FA, LAD-I, PKD, AAV) and data from two (likely FA, LAD-I) in 2019.

In his report, Prasad wrote, “The company is targeting rare diseases with a focus on bone marrow health and monogenic disorders with little to no competition. We believe this sound strategic approach somewhat de-risks the company’s preclinical programs. Rocket expects to have at least four clinical programs in 2019 (FA, LAD-I, PKD, and AAV indication), with data available from at least two of them during the year (we anticipate FA and LAD-I), which we view as ambitious goals for a relatively young company, and execution on this strategy could unlock significant value creation for shareholders.”

William Blair is a premier global boutique with expertise in investment banking, investment management, and private wealth management. We provide advisory services, strategies, and solutions to meet our clients’ evolving needs. As an independent and employee-owned firm, together with our strategic partners, we operate in more than 20 offices worldwide.* 

View our research coverage list

*Includes strategic partnerships with Allier Capital, BDA Partners, and Poalim Capital Markets.


William Blair or an affiliate does and seeks to do business with companies covered in its research reports. As a result, investors should be aware that the firm may have a conflict of interest that could affect the objectivity of this report. This report is not intended to provide personal investment advice. The opinions and recommendations herein do not take into account individual client circumstances, objectives, or needs and are not intended as recommendations of particular securities, financial instruments, or strategies to particular clients. The recipient of this report must make its own independent decisions regarding any securities or financial instruments mentioned herein.

William Blair or an affiliate is a market maker in the securities of Krystal Biotech, Inc. and Rocket Pharmaceuticals, Inc.

William Blair or an affiliate expects to receive or intends to seek compensation for investment banking services from Krystal Biotech, Inc. and Rocket Pharmaceuticals, Inc. or an affiliate within the next three months.

Officers and employees of William Blair or its affiliates (other than research analysts) may have a financial interest in securities of Krystal Biotech, Inc. and Rocket Pharmaceuticals, Inc.

Please contact us at +1 800 621 0687 or view disclosures on our coverage list.

Additional information is available upon request.

Current Ratings Distribution (as of 7/9/18)

Coverage Universe
Outperform (Buy): 66%
Market Perform (Hold): 32%
Underperform (Sell): 1%

Inv. Banking Relationships*
Outperform (Buy): 20%
Market Perform (Hold): 9%
Underperform (Sell): 0%

* Percentage of companies in each rating category that are investment banking clients, defined as companies for which William Blair has received compensation for investment banking services within the past 12 months.

The compensation of the research analyst is based on a variety of factors, including the quality and accuracy of research, client feedback, contributions to other firm departments, competitive factors, and firm profitability.

OTHER IMPORTANT DISCLOSURES

Stock ratings and valuation methodologies: William Blair & Company, L.L.C. uses a three-point system to rate stocks. Individual ratings reflect the expected performance of the stock relative to the broader market (generally the S&P 500, unless otherwise indicated) over the next 12 months. The assessment of expected performance is a function of near-, intermediate-, and long-term company fundamentals, industry outlook, confidence in earnings estimates, valuation (and our valuation methodology), and other factors. Outperform (O) – stock expected to outperform the broader market over the next 12 months; Market Perform (M) – stock expected to perform approximately in line with the broader market over the next 12 months; Underperform (U) – stock expected to underperform the broader market over the next 12 months; not rated (NR) – the stock is not currently rated. The valuation methodologies include (but are not limited to) price-to-earnings multiple (P/E), relative P/E (compared with the relevant market), P/E-to-growth-rate (PEG) ratio, market capitalization/revenue multiple, enterprise value/EBITDA ratio, discounted cash flow, and others. Stock ratings and valuation methodologies should not be used or relied upon as investment advice. Past performance is not necessarily a guide to future performance.

The ratings and valuation methodologies reflect the opinion of the individual analyst and are subject to change at any time.

Our salespeople, traders, and other professionals may provide oral or written market commentary, short-term trade ideas, or trading strategies—to our clients, prospective clients, and our trading desks—that are contrary to opinions expressed in this research report. Certain outstanding research reports may contain discussions or investment opinions relating to securities, financial instruments and/or issuers that are no longer current. Always refer to the most recent report on a company or issuer. Our asset management and trading desks may make investment decisions that are inconsistent with recommendations or views expressed in this report. We will from time to time have long or short positions in, act as principal in, and buy or sell the securities referred to in this report. Our research is disseminated primarily electronically, and in some instances in printed form. Research is simultaneously available to all clients. This research report is for our clients only. No part of this material may be copied or duplicated in any form by any means or redistributed without the prior written consent of William Blair & Company, L.L.C.

This is not in any sense an offer or solicitation for the purchase or sale of a security or financial instrument. The factual statements herein have been take from sources we believe to be reliable, but such statements are made without any representation as to accuracy or completeness or otherwise, except with respect to any disclosures relative to William Blair or its research analysts. Opinions expressed are our own unless otherwise stated and are subject to change without notice. Prices shown are approximate.

This material is distributed in the United Kingdom and the European Economic Area (EEA) by William Blair International, Ltd., authorized and regulated by the Financial Conduct Authority (FCA). William Blair International, Limited is a limited liability company registered in England and Wales with company number 03619027. This material is only directed and issued to persons regarded as Professional investors or equivalent in their home jurisdiction, or persons falling within articles 19 (5), 38, 47, and 49 of the Financial Services and Markets Act of 2000 (Financial Promotion) Order 2005 (all such persons being referred to as “relevant persons”). This document must not be acted on or relied on by persons who are not “relevant persons.”

William Blair” and “R*Docs” are registered trademarks of William Blair & Company, L.L.C. Copyright 2018, William Blair & Company, L.L.C. All rights reserved.

News Alerts

Stay connected to your favorite publications and news features.

Subscribe Now