Madrigal Pharmaceuticals Announces Pricing of Upsized $600 Million Public Offering

Madrigal Pharmaceuticals Announces Pricing of Upsized $600 Million Public Offering




Madrigal Pharmaceuticals Announces Pricing of Upsized $600 Million Public Offering

CONSHOHOCKEN, Pa., March 18, 2024 (GLOBE NEWSWIRE) — Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL), a biopharmaceutical company focused on delivering novel therapeutics for nonalcoholic steatohepatitis (NASH), today announced the pricing of its underwritten public offering of 750,000 shares of its common stock at a public offering price of $260.0000 per share, and, to certain investors, pre-funded warrants to purchase 1,557,692 shares of common stock at a price of $259.9999 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.0001 per share exercise price for each such pre-funded warrant. The size of the offering was increased by $100 million subsequent to the initial announcement of the offering. The gross proceeds to Madrigal from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be approximately $600 million. Madrigal has granted the underwriters of the offering a 30-day option to purchase up to an additional 346,153 shares of common stock from the company at the public offering price, less underwriting discounts and commissions.

Madrigal intends to use the net proceeds from this offering for its commercial activities in connection with the launch of Rezdiffra™ (resmetirom) in the U.S. and for general corporate purposes, including, without limitation, research and development expenditures, ongoing clinical trial expenditures, manufacture and supply of drug substance and drug products, potential ex-U.S. commercialization or partnering opportunities, potential acquisitions or licensing of new technologies, capital expenditures and working capital.

Goldman Sachs & Co. LLC, Jefferies, TD Cowen, Evercore ISI, Piper Sandler, UBS Investment Bank and Citizens JMP are acting as joint bookrunning managers of the offering. H.C. Wainwright & Co. is acting as co-manager of the offering. The offering is expected to close on or about March 21, 2024, subject to the satisfaction of customary closing conditions.

The securities are being offered by Madrigal pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the Securities and Exchange Commission (SEC) on June 1, 2021. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and the accompanying prospectus relating to the offering will be filed with the SEC and, when filed, will also be available on the SEC’s website. Alternatively, copies of the final prospectus and the accompanying prospectus may also be obtained, when available, by contacting the following: Goldman Sachs & Co. LLC, Attn: Prospectus Department, 200 West Street, New York, New York 10282, telephone: 1-866-471-2526, email: prospectus-ny@ny.email.gs.com; Jefferies LLC, Attn: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at Prospectus_Department@jefferies.com; Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by telephone at (833) 297-2926 or by email at Prospectus_ECM@cowen.com; Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, New York, New York 10055, by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com; Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at prospectus@psc.com; UBS Securities LLC, 1285 Avenue of the Americas, New York, NY 10019, Attn: ECM Syndicate, or by email: ol-prospectus-request@ubs.com; or Citizens JMP Securities, LLC, 600 Montgomery Street, Suite 1100, San Francisco, CA 94111, by telephone at (415) 835-8985, or by email at syndicate@jmpsecurities.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction.

About Madrigal Pharmaceuticals, Inc.

Madrigal Pharmaceuticals, Inc. (Nasdaq: MDGL) is a biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), a liver disease with high unmet medical need. Madrigal’s medication, Rezdiffra (resmetirom), is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH.

Forward-Looking Statements

Forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding forward-looking statements, future performance or events; include all statements that are not historical facts; and can be identified by terms such as “be,” “can,” “designed,” “expectation,” “may,” “plans,” “proposed,” “seeks,” “will,” “will be,” “would” or similar expressions and the negatives of those terms.

Statements in this release concerning Madrigal’s future expectations, plans and prospects, including, without limitation, statements about Madrigal’s public offering and use of proceeds, constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, risks associated with market conditions and the satisfaction of customary closing conditions related to the offering and the anticipated use of proceeds from the offering, as well as those risks more fully discussed under “Risk Factors” in the preliminary prospectus supplement and in the “Risk Factors” filed in Part I, Item 1A of Madrigal’s Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on February 28, 2024, and in other filings that Madrigal makes with the SEC, including those included or incorporated by reference in the preliminary prospectus supplement and accompanying prospectus supplement related to the public offering filed with the SEC. There can be no assurance that Madrigal will be able to complete the public offering. You should not place undue reliance on these forward-looking statements. In addition, any forward-looking statements represent Madrigal’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Madrigal explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

Investor Contact
Tina Ventura, Madrigal Pharmaceuticals, Inc., IR@madrigalpharma.com

Media Contact
Christopher Frates, Madrigal Pharmaceuticals, Inc., media@madrigalpharma.com

Merit Medical Systems to Announce First Quarter 2024 Results on April 30, 2024

Merit Medical Systems to Announce First Quarter 2024 Results on April 30, 2024




Merit Medical Systems to Announce First Quarter 2024 Results on April 30, 2024

SOUTH JORDAN, Utah, March 18, 2024 (GLOBE NEWSWIRE) — Merit Medical Systems, Inc. (NASDAQ: MMSI), a leading global manufacturer and marketer of healthcare technology, announced today that it will release its financial results for the quarter ended March 31, 2024, after the close of the stock market on Tuesday, April 30, 2024.

Merit will hold its investor conference call on the same day (Tuesday, April 30, 2024) at 5:00 p.m. Eastern (4:00 p.m. Central, 3:00 p.m. Mountain, and 2:00 p.m. Pacific).

To access the conference call, please pre-register using the following link. Registrants will receive confirmation with dial-in details.

A live webcast and slide deck can be accessed using this link. A link to both register for the conference call and view the webcast will be made available at merit.com.

ABOUT MERIT

Founded in 1987, Merit Medical Systems, Inc. is engaged in the development, manufacture, and distribution of proprietary disposable medical devices used in interventional, diagnostic, and therapeutic procedures, particularly in cardiology, radiology, oncology, critical care, and endoscopy. Merit serves client hospitals worldwide with a domestic and international sales force and clinical support team totaling more than 700 individuals. Merit employs approximately 7,000 people worldwide.

Contacts:  
PR/Media Inquiries:
Teresa Johnson
Merit Medical
Investor Inquiries:
Mike Piccinino, CFA, IRC
Westwicke – ICR
+1-801-208-4295 +1-443-213-0509
tjohnson@merit.com mike.piccinino@westwicke.com

 

 

Outlook Therapeutics® Announces Closing of Private Placement of up to $159 Million

Outlook Therapeutics® Announces Closing of Private Placement of up to $159 Million




Outlook Therapeutics® Announces Closing of Private Placement of up to $159 Million

ISELIN, N.J., March 18, 2024 (GLOBE NEWSWIRE) — Outlook Therapeutics, Inc. (Nasdaq: OTLK), a biopharmaceutical company working to achieve the first approval for an ophthalmic formulation of bevacizumab for the treatment of retinal diseases in the US and the EU, today announced that it has closed its previously announced private placement, for upfront gross proceeds of approximately $60 million from the issuance and sale of shares of the Company’s common stock and accompanying warrants, before deducting placement agent fees and offering expenses. Outlook Therapeutics has the potential to receive additional gross proceeds of up to $99 million upon the full cash exercise of the warrants issued in the private placement, before deducting placement agent fees and offering expenses.

The private placement was led by Great Point Partners, LLC, with participation from existing investor GMS Ventures as well as new investors Altium Capital, Armistice Capital, Caligan Partners LP, Schonfeld Strategic Advisors, Sphera Healthcare, Velan Capital, Woodline Partners LP, and an undisclosed life sciences dedicated investor.  

BofA Securities and BTIG acted as co-placement agents in connection with the financing.

About the Private Placement

Pursuant to the securities purchase agreement entered into on January 22, 2024, Outlook Therapeutics issued to purchasers in the private placement an aggregate of 8,571,423 shares of common stock and accompanying warrants to purchase an aggregate of 12,857,133 shares of common stock, at a price of $7.00 per share and accompanying warrant to purchase one and one-half shares of common stock. The warrants have an exercise price of $7.70 per share and are exercisable only for cash until their expiration on the fifth anniversary of the issuance date. The warrants include a feature that allows Outlook Therapeutics to require holders to cash exercise the warrants if certain stock price and milestone conditions are met.

All of the securities in the private placement were offered by the Company.

Outlook Therapeutics intends to use the net proceeds from the private placement to fund its ONS-5010 clinical development programs, including the ongoing NORSE EIGHT clinical trial, and for working capital and other general corporate purposes.

As previously disclosed, Outlook Therapeutics also entered into a securities purchase agreement with Syntone Ventures, another existing stockholder, to purchase $5 million in shares of common stock and warrants on the same terms as the private placement. The closing of the Syntone investment remains subject to receipt of regulatory approvals and other customary closing conditions.

The offer and sale of the foregoing securities were made by Outlook Therapeutics in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Act”), and/or Regulation D promulgated thereunder, and such securities have not been registered under the Act or applicable state securities laws. Accordingly, such securities may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws. Outlook Therapeutics has agreed to file a resale registration statement with the U.S. Securities and Exchange Commission for purposes of registering the resale of the common stock issued or issuable in connection with the private placement.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About ONS-5010 / LYTENAVA™ (bevacizumab-vikg, bevacizumab gamma)

ONS-5010/LYTENAVA™ is an investigational ophthalmic formulation of bevacizumab under development as an intravitreal injection for the treatment of wet AMD and other retinal diseases. Because no FDA or European Commission approved ophthalmic formulations of bevacizumab are available currently, clinicians wishing to treat retinal patients with bevacizumab have had to use repackaged IV bevacizumab provided by compounding pharmacies—products that have known risks of contamination and inconsistent potency and availability. If approved, ONS-5010/LYTENAVA™ would provide an approved option for physicians to treat wet AMD.

Bevacizumab-vikg/bevacizumab gamma is a recombinant humanized monoclonal antibody (mAb) that selectively binds with high affinity to all isoforms of human vascular endothelial growth factor (VEGF) and neutralizes VEGF’s biologic activity through a steric blocking of the binding of VEGF to its receptors Flt-1 (VEGFR-1) and KDR (VEGFR-2) on the surface of endothelial cells. Following intravitreal injection, the binding of bevacizumab-vikg to VEGF prevents the interaction of VEGF with its receptors on the surface of endothelial cells, reducing endothelial cell proliferation, vascular leakage, and new blood vessel formation in the retina.

About Outlook Therapeutics, Inc.

Outlook Therapeutics is a biopharmaceutical company working to achieve FDA and European Commission approval for the launch of ONS-5010/ LYTENAVA™ (bevacizumab-vikg or bevacizumab gamma) as the first approved ophthalmic formulation of bevacizumab for use in retinal indications, including wet AMD, DME and BRVO. If ONS-5010 ophthalmic bevacizumab is approved, Outlook Therapeutics expects to commercialize it as the first and only FDA and/or EC approved ophthalmic formulation of bevacizumab for use in treating retinal diseases in the United States, EU, United Kingdom, Europe, Japan, and other markets. As part of the Outlook Therapeutics’ multi-year commercial planning process, Outlook Therapeutics and Cencora entered into a strategic commercialization agreement to expand the Outlook Therapeutics’ reach for connecting to retina specialists and their patients. Cencora will provide third-party logistics (3PL) services and distribution, as well as pharmacovigilance services and other services in the United States.

Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical facts are “forward-looking statements,” including those relating to future events. In some cases, you can identify forward-looking statements by terminology such as “anticipate,” “believe,” “continue,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “target,” “will,” or “would” the negative of terms like these or other comparable terminology, and other words or terms of similar meaning. These include, among others, the private placement, including expected proceeds from the exercise of the warrants and uses of proceeds, expectations concerning the relationship with Cencora and the benefits and potential expansion thereof, and other statements that are not historical fact. Although Outlook Therapeutics believes that it has a reasonable basis for the forward-looking statements contained herein, they are based on current expectations about future events affecting Outlook Therapeutics and are subject to risks, uncertainties and factors relating to its operations and business environment, all of which are difficult to predict and many of which are beyond its control. These risk factors include those risks associated with developing pharmaceutical product candidates, risks of conducting clinical trials and risks in obtaining necessary regulatory approvals, the content and timing of decisions by the FDA, as well as those risks detailed in Outlook Therapeutics’ filings with the Securities and Exchange Commission (the SEC), including the Annual Report on Form 10-K for the fiscal year ended September 30, 2023, filed with the SEC on December 22, 2023, and future quarterly reports Outlook Therapeutics files with the SEC, which include uncertainty of market conditions and future impacts related to macroeconomic factors, including as a result of the ongoing overseas conflict, high interest rates, inflation and potential future bank failures on the global business environment. These risks may cause actual results to differ materially from those expressed or implied by forward-looking statements in this press release. All forward-looking statements included in this press release are expressly qualified in their entirety by the foregoing cautionary statements. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Outlook Therapeutics does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise, except as may be required under applicable securities law.

Investor Inquiries:       
Jenene Thomas
Chief Executive Officer
JTC Team, LLC
T: 833.475.8247 
OTLK@jtcir.com

QDOSE® Platform Partnership for Personalized Dosimetry in Radiopharmaceutical Therapy

QDOSE® Platform Partnership for Personalized Dosimetry in Radiopharmaceutical Therapy




QDOSE® Platform Partnership for Personalized Dosimetry in Radiopharmaceutical Therapy

MELBOURNE, Australia and DRESDEN, Germany, March 19, 2024 (GLOBE NEWSWIRE) — Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today announces that it has entered into an agreement to commercially partner the QDOSE® dosimetry software platform with ABX-CRO Advanced Pharmaceutical Services Forschungsgesellschaft mbH (ABX-CRO), a full-service contract research organization (CRO) based in Dresden, and its development partner, Stockholm-based Quantinm AB.

QDOSE® is a validated, versatile software platform that enables reliable estimation of patient-specific dosimetry for both therapeutic and diagnostic radiopharmaceuticals. Telix intends to integrate the QDOSE® platform into its therapeutic radiopharmaceutical programs and clinical collaborations to enable the development of personalized treatment regimens. Personalized radiopharmaceutical therapy administration based on individual patient dosimetry has the potential to improve clinical outcomes by optimizing treatment response while reducing effects on normal healthy organs and optimizing the use of isotope supply chains.

QDOSE® supports dosimetry calculations using planar imaging, tomographic imaging, single/multi time point imaging and hybrid combinations. QDOSE® incorporates additional tools for attenuation and background correction. The QDOSE® platform will be integrated with Telix’s artificial intelligence (AI) products as an accessible tool for clinicians and third-party commercial drug developers. QDOSE® has received 510(k) clearance from the United States Food and Drug Administration (FDA) and is Conformité Européenne (CE) marked for clinical use within the European Union (EU).

Dr Christian Behrenbruch, Managing Director and Group CEO of Telix said, “In nuclear medicine, dosimetry analysis is used to calculate the absorbed dose of radiation received by different parts of the body. Rapid, reliable and personalized dosimetry is becoming an increasingly important future direction of cancer care and regulatory authorities now expect to see dosimetry analysis. QDOSE® has an excellent performance track record and we are committed to positioning QDOSE® as a new industry standard in dosimetry analysis.”

The arms’ length up-front transaction consideration is non-material. Telix will pay undisclosed product royalties and fees on product sales.

About Telix Pharmaceuticals Limited

Telix is a biopharmaceutical company focused on the development and commercialization of diagnostic and therapeutic radiopharmaceuticals and associated medical devices. Telix is headquartered in Melbourne, Australia, with international operations in the United States, Europe (Belgium and Switzerland), and Japan. Telix is developing a portfolio of clinical and commercial stage products that aims to address significant unmet medical needs in oncology and rare diseases. Telix is listed on the Australian Securities Exchange (ASX: TLX).

Visit www.telixpharma.com for further information about Telix, including details of the latest share price, announcements made to the ASX, investor and analyst presentations, news releases, event details and other publications that may be of interest. You can also follow Telix on X and LinkedIn.

Telix’s lead imaging product, gallium-68 (68Ga) gozetotide injection (also known as 68Ga PSMA-11 and marketed under the brand name Illuccix®), has been approved by the FDA,1 by the Australian Therapeutic Goods Administration (TGA),2 and by Health Canada.3 Telix’s miniaturized surgical gamma probe, SENSEI®, for minimally invasive and robotic-assisted surgery, has attained a marketing authorization in the U.S., having been registered with the FDA and has attained a CE Mark for use in the European Economic Area for the intra-operative detection of sentinel lymph nodes. With the exception of Illuccix® and SENSEI® as noted above, no Telix product has received a marketing authorization in any jurisdiction.

Telix Investor Relations

Ms. Kyahn Williamson
Telix Pharmaceuticals Limited
SVP Investor Relations and Corporate Communications
Email: kyahn.williamson@telixpharma.com

Legal Notices

The information contained in this announcement is not intended to be an offer for subscription, invitation or recommendation with respect to shares of Telix Pharmaceuticals Limited (Telix) in any jurisdiction, including the United States. No representation or warranty, express or implied, is made in relation to the accuracy or completeness of the information contained or opinions expressed in the course of this announcement. The information contained in this announcement is subject to change without notification.

This announcement may contain forward-looking statements that relate to anticipated future events, financial performance, plans, strategies or business developments. Forward-looking statements can generally be identified by the use of words such as “may”, “expect”, “intend”, “plan”, “estimate”, “anticipate”, “outlook”, “forecast” and “guidance”, or other similar words. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. Forward-looking statements are based on the Company’s good-faith assumptions as to the financial, market, regulatory and other risks and considerations that exist and affect the Company’s business and operations in the future and there can be no assurance that any of the assumptions will prove to be correct. In the context of Telix’s business, forward-looking statements may include, but are not limited to, statements about: the initiation, timing, progress and results of Telix’s preclinical and clinical studies, and Telix’s research and development programs; Telix’s ability to advance product candidates into, enrol and successfully complete, clinical studies, including multi-national clinical trials; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities; the commercialisation of Telix’s product candidates, if or when they have been approved; estimates of Telix’s expenses, future revenues and capital requirements; Telix’s financial performance; developments relating to Telix’s competitors and industry; and the pricing and reimbursement of Telix’s product candidates, if and after they have been approved. Telix’s actual results, performance or achievements may be materially different from those which may be expressed or implied by such statements, and the differences may be adverse. Accordingly, you should not place undue reliance on these forward-looking statements. You should read this announcement together with our risk factors, as disclosed in our most recently filed reports with the ASX or on our website.

To the maximum extent permitted by law, Telix disclaims any obligation or undertaking to publicly update or revise any forward-looking statements contained in this announcement, whether as a result of new information, future developments or a change in expectations or assumptions.

©2024 Telix Pharmaceuticals Limited. The Telix Pharmaceuticals, Illuccix® and SENSEI® names and logos are trademarks of Telix Pharmaceuticals Limited and its affiliates – all rights reserved.


1 Telix ASX disclosure 20 December 2021.
2 Telix ASX disclosure 2 November 2021.
3 Telix ASX disclosure 14 October 2022.

Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer

Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer




Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer

– Awards Dr. Berkowitz an Inducement Grant in accordance with Nasdaq Listing Rule 5635(c)(4) –

NEW HAVEN, Conn., March 18, 2024 (GLOBE NEWSWIRE) — Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, today announced the appointment of Noah Berkowitz, M.D., Ph.D., to the role of Chief Medical Officer and a member of the Executive Committee reporting to President and Chief Executive Officer John Houston, Ph.D. Effective today, Dr. Berkowitz will lead the ongoing clinical development of Arvinas’ PROTAC® protein degrader programs in oncology and neuroscience.

“We are thrilled to have Dr. Berkowitz join Arvinas as we continue advancing multiple programs with the goal of improving the lives of patients with serious diseases,” said John Houston, Ph.D., Chairperson, President and Chief Executive Officer at Arvinas. “Noah’s vision, expertise, and proven track record in designing and launching successful clinical programs across all stages of clinical development complement our rapidly advancing portfolio of novel PROTAC protein degraders. His leadership and experience will be invaluable as we approach our first Phase 3 data readout and continue progressing multiple ongoing and planned clinical-stage programs.”

Dr. Berkowitz has more than 20 years of experience in advancing programs through early and late stages of development, with proven ability in leading clinical development, regulatory, and medical affairs. He has led multidisciplinary teams and has extensive knowledge of clinical trial design and execution. Dr. Berkowitz joins Arvinas from Bristol-Myers Squibb (BMS), where he was Senior Vice President, Development Unit Head, Hematology. While at BMS, Dr. Berkowitz’s teams achieved initial or subsequent global indications for Abecma®, Breyanzi®, Reblozyl®, Onureg®, and Inrebic®, managed development life cycles for Revlimid®, Pomalyst®, and Sprycel®, and initiated and/or designed registration trials for iberdomide, mezigdomide, and alnuctamab. Prior to BMS, Dr. Berkowitz held roles of increasing responsibility in the areas of oncology, rare diseases, and hematology at Novartis, most recently as Vice President and Clinical Development Head for Hematology. Dr. Berkowitz earned his M.D. and Ph.D. from Columbia University and trained in medical oncology at the National Cancer Institute.

“Arvinas is the leader in targeted protein degradation and is on the cusp of its first pivotal data, an important step in potentially bringing an innovative therapeutic approach to patients with serious diseases,” said Dr. Berkowitz. “Arvinas’ progress over the last several years is impressive and I look forward to working with my colleagues, including the strong leadership team at Arvinas, as we prepare for many important upcoming clinical milestones ahead.”

The Company also announced that Ron Peck, M.D., who was Chief Medical Officer since joining Arvinas in mid-2019, will be leaving to pursue other opportunities.

“In less than five years, Ron built robust clinical development and medical affairs organizations, enabling the planning and initiation of multiple clinical trials across our oncology and neuroscience portfolios,” continued Dr. Houston. “Under Ron’s leadership and in partnership with Pfizer, vepdegestrant has advanced from the start of Phase 1 to multiple ongoing and planned Phase 3 studies in advanced breast cancer. Ron’s teams also progressed our androgen receptor programs, with ARV-766 moving towards Phase 3 initiation, and initiated human dosing of ARV-102, our neuroscience program targeting LRRK2,” said Dr. Houston. “We thank Ron for his tremendous efforts and take confidence in knowing that Dr. Berkowitz is inheriting a high-performing organization.”

In connection with the commencement of Dr. Berkowitz’s employment, Arvinas granted Dr. Berkowitz an option to purchase 93,879 shares of common stock and a restricted stock unit award with respect to 63,452 shares of common stock on March 18, 2024, as an inducement material to entering into employment with Arvinas. The option and restricted stock units were granted in accordance with Nasdaq Listing Rule 5635(c)(4) and not pursuant to Arvinas’ stock incentive plan.

The option has a 10-year term and an exercise price of $42.60 per share, which is equal to the closing price per share of Arvinas’ common stock on the grant date. The option vests over four years, with 25% of the original number of shares underlying the option vesting on the one-year anniversary of the date of grant and 1/48th of the original number of shares vesting monthly thereafter, and the restricted stock unit award vests in four equal installments on each one-year anniversary of Dr. Berkowitz’s employment start date until the fourth anniversary of Dr. Berkowitz’s start date, subject to his continued service with Arvinas through the applicable vesting dates. 

About Arvinas
Arvinas is a clinical-stage biotechnology company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development, and commercialization of therapies that degrade disease-causing proteins. Arvinas uses its proprietary PROTAC® Discovery Engine platform to engineer proteolysis targeting chimeras, or PROTAC® targeted protein degraders, that are designed to harness the body’s own natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. In addition to its robust preclinical pipeline of PROTAC protein degraders against validated and “undruggable” targets, the company has four investigational clinical-stage programs: vepdegestrant (ARV-471) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer; ARV-766 and bavdegalutamide for the treatment of men with metastatic castration-resistant prostate cancer; and ARV-102 for the treatment of patients with neurodegenerative disorders. For more information, visit www.arvinas.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements with respect to Arvinas’ development plans; the anticipated data readout for Arvinas’ first Phase 3 clinical trial with vepdegestrant (ARV-471); the potential, pending regulatory approval, for vepdegestrant to address an area of significant unmet need and offer patients an innovative therapeutic approach; and the potential advantages and therapeutic benefits of vepdegestrant (ARV-471). All statements, other than statements of historical facts, contained in this press release, including statements regarding Arvinas’ strategy, future operations, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Arvinas may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements Arvinas makes as a result of various risks and uncertainties, including but not limited to: Arvinas’ and Pfizer, Inc.’s (“Pfizer”) performance of the respective obligations with respect to Arvinas’ collaboration with Pfizer; whether Arvinas and Pfizer will be able to successfully conduct and complete clinical development for vepdegestrant (ARV-471); whether Arvinas will be able to successfully conduct and complete development for its other product candidates, including ARV-766, and including whether Arvinas initiates and completes clinical trials for its product candidates and receive results from its clinical trials on its expected timelines or at all; whether Arvinas and Pfizer, as appropriate, will be able to obtain marketing approval for and commercialize vepdegestrant (ARV-471), ARV-766 and other product candidates on current timelines or at all; Arvinas’ ability to protect its intellectual property portfolio; whether Arvinas’ cash and cash equivalent resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other important factors discussed in the “Risk Factors” section of Arvinas’ Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent other reports on file with the U.S. Securities and Exchange Commission. The forward-looking statements contained in this press release reflect Arvinas’ current views with respect to future events, and Arvinas assumes no obligation to update any forward-looking statements, except as required by applicable law. These forward-looking statements should not be relied upon as representing Arvinas’ views as of any date subsequent to the date of this release.

Contacts

Investors:
Jeff Boyle
+1 (347) 247-5089
Jeff.Boyle@arvinas.com

Media:
Maurissa Messier
+1 (908) 208-9254
Maurissa.messier-c@arvinas.com

 

Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer

Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer




Arvinas Appoints Noah Berkowitz, M.D., Ph.D., as Chief Medical Officer

– Awards Dr. Berkowitz an Inducement Grant in accordance with Nasdaq Listing Rule 5635(c)(4) –

NEW HAVEN, Conn., March 18, 2024 (GLOBE NEWSWIRE) — Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, today announced the appointment of Noah Berkowitz, M.D., Ph.D., to the role of Chief Medical Officer and a member of the Executive Committee reporting to President and Chief Executive Officer John Houston, Ph.D. Effective today, Dr. Berkowitz will lead the ongoing clinical development of Arvinas’ PROTAC® protein degrader programs in oncology and neuroscience.

“We are thrilled to have Dr. Berkowitz join Arvinas as we continue advancing multiple programs with the goal of improving the lives of patients with serious diseases,” said John Houston, Ph.D., Chairperson, President and Chief Executive Officer at Arvinas. “Noah’s vision, expertise, and proven track record in designing and launching successful clinical programs across all stages of clinical development complement our rapidly advancing portfolio of novel PROTAC protein degraders. His leadership and experience will be invaluable as we approach our first Phase 3 data readout and continue progressing multiple ongoing and planned clinical-stage programs.”

Dr. Berkowitz has more than 20 years of experience in advancing programs through early and late stages of development, with proven ability in leading clinical development, regulatory, and medical affairs. He has led multidisciplinary teams and has extensive knowledge of clinical trial design and execution. Dr. Berkowitz joins Arvinas from Bristol-Myers Squibb (BMS), where he was Senior Vice President, Development Unit Head, Hematology. While at BMS, Dr. Berkowitz’s teams achieved initial or subsequent global indications for Abecma®, Breyanzi®, Reblozyl®, Onureg®, and Inrebic®, managed development life cycles for Revlimid®, Pomalyst®, and Sprycel®, and initiated and/or designed registration trials for iberdomide, mezigdomide, and alnuctamab. Prior to BMS, Dr. Berkowitz held roles of increasing responsibility in the areas of oncology, rare diseases, and hematology at Novartis, most recently as Vice President and Clinical Development Head for Hematology. Dr. Berkowitz earned his M.D. and Ph.D. from Columbia University and trained in medical oncology at the National Cancer Institute.

“Arvinas is the leader in targeted protein degradation and is on the cusp of its first pivotal data, an important step in potentially bringing an innovative therapeutic approach to patients with serious diseases,” said Dr. Berkowitz. “Arvinas’ progress over the last several years is impressive and I look forward to working with my colleagues, including the strong leadership team at Arvinas, as we prepare for many important upcoming clinical milestones ahead.”

The Company also announced that Ron Peck, M.D., who was Chief Medical Officer since joining Arvinas in mid-2019, will be leaving to pursue other opportunities.

“In less than five years, Ron built robust clinical development and medical affairs organizations, enabling the planning and initiation of multiple clinical trials across our oncology and neuroscience portfolios,” continued Dr. Houston. “Under Ron’s leadership and in partnership with Pfizer, vepdegestrant has advanced from the start of Phase 1 to multiple ongoing and planned Phase 3 studies in advanced breast cancer. Ron’s teams also progressed our androgen receptor programs, with ARV-766 moving towards Phase 3 initiation, and initiated human dosing of ARV-102, our neuroscience program targeting LRRK2,” said Dr. Houston. “We thank Ron for his tremendous efforts and take confidence in knowing that Dr. Berkowitz is inheriting a high-performing organization.”

In connection with the commencement of Dr. Berkowitz’s employment, Arvinas granted Dr. Berkowitz an option to purchase 93,879 shares of common stock and a restricted stock unit award with respect to 63,452 shares of common stock on March 18, 2024, as an inducement material to entering into employment with Arvinas. The option and restricted stock units were granted in accordance with Nasdaq Listing Rule 5635(c)(4) and not pursuant to Arvinas’ stock incentive plan.

The option has a 10-year term and an exercise price of $42.60 per share, which is equal to the closing price per share of Arvinas’ common stock on the grant date. The option vests over four years, with 25% of the original number of shares underlying the option vesting on the one-year anniversary of the date of grant and 1/48th of the original number of shares vesting monthly thereafter, and the restricted stock unit award vests in four equal installments on each one-year anniversary of Dr. Berkowitz’s employment start date until the fourth anniversary of Dr. Berkowitz’s start date, subject to his continued service with Arvinas through the applicable vesting dates. 

About Arvinas
Arvinas is a clinical-stage biotechnology company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development, and commercialization of therapies that degrade disease-causing proteins. Arvinas uses its proprietary PROTAC® Discovery Engine platform to engineer proteolysis targeting chimeras, or PROTAC® targeted protein degraders, that are designed to harness the body’s own natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. In addition to its robust preclinical pipeline of PROTAC protein degraders against validated and “undruggable” targets, the company has four investigational clinical-stage programs: vepdegestrant (ARV-471) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer; ARV-766 and bavdegalutamide for the treatment of men with metastatic castration-resistant prostate cancer; and ARV-102 for the treatment of patients with neurodegenerative disorders. For more information, visit www.arvinas.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements with respect to Arvinas’ development plans; the anticipated data readout for Arvinas’ first Phase 3 clinical trial with vepdegestrant (ARV-471); the potential, pending regulatory approval, for vepdegestrant to address an area of significant unmet need and offer patients an innovative therapeutic approach; and the potential advantages and therapeutic benefits of vepdegestrant (ARV-471). All statements, other than statements of historical facts, contained in this press release, including statements regarding Arvinas’ strategy, future operations, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

Arvinas may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements Arvinas makes as a result of various risks and uncertainties, including but not limited to: Arvinas’ and Pfizer, Inc.’s (“Pfizer”) performance of the respective obligations with respect to Arvinas’ collaboration with Pfizer; whether Arvinas and Pfizer will be able to successfully conduct and complete clinical development for vepdegestrant (ARV-471); whether Arvinas will be able to successfully conduct and complete development for its other product candidates, including ARV-766, and including whether Arvinas initiates and completes clinical trials for its product candidates and receive results from its clinical trials on its expected timelines or at all; whether Arvinas and Pfizer, as appropriate, will be able to obtain marketing approval for and commercialize vepdegestrant (ARV-471), ARV-766 and other product candidates on current timelines or at all; Arvinas’ ability to protect its intellectual property portfolio; whether Arvinas’ cash and cash equivalent resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; and other important factors discussed in the “Risk Factors” section of Arvinas’ Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent other reports on file with the U.S. Securities and Exchange Commission. The forward-looking statements contained in this press release reflect Arvinas’ current views with respect to future events, and Arvinas assumes no obligation to update any forward-looking statements, except as required by applicable law. These forward-looking statements should not be relied upon as representing Arvinas’ views as of any date subsequent to the date of this release.

Contacts

Investors:
Jeff Boyle
+1 (347) 247-5089
Jeff.Boyle@arvinas.com

Media:
Maurissa Messier
+1 (908) 208-9254
Maurissa.messier-c@arvinas.com

 

Homology Medicines Declares Distribution to Common Stockholders

Homology Medicines Declares Distribution to Common Stockholders




Homology Medicines Declares Distribution to Common Stockholders

BEDFORD, Mass., March 18, 2024 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX) today announced that it declared a distribution to its common stockholders of record as of the close of business on March 21, 2024 of the right to receive one contingent value right (CVR) for each outstanding share of Homology common stock held by such stockholder as of such record date. The payment date for such distribution is expected to be March 27, 2024 (three business days after the expected closing of the merger on March 22, 2024). Each CVR represents the non-transferable contractual right to receive certain contingent payments upon the occurrence of certain events within agreed time periods as provided in the merger agreement and agreement governing the CVRs.

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this filing may be considered forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, including statements regarding the proposed transaction involving Homology and Q32, including the conditions to, and timing of, closing of the proposed transaction, the parties’ ability to consummate the proposed transaction, among others. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” and other similar expressions among others. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: (i) the risk that the conditions to the closing of the proposed transaction are not satisfied, including the failure to timely or at all obtain stockholder approval for the proposed transaction or the failure to timely or at all obtain any required regulatory clearances; (ii) uncertainties as to the timing of the consummation of the proposed transaction and the ability of each of Homology and Q32 to consummate the proposed transaction; (iii) the ability of Homology and Q32 to integrate their businesses successfully and to achieve anticipated synergies; (iv) the possibility that other anticipated benefits of the proposed transaction will not be realized, including without limitation, anticipated revenues, expenses, earnings and other financial results, and growth and expansion of the combined company’s operations, and the anticipated tax treatment of the combination; (v) potential litigation relating to the proposed transaction that could be instituted against Homology, Q32 or their respective directors; (vi) possible disruptions from the proposed transaction that could harm Homology’s and/or Q32’s respective businesses; (vii) the ability of Homology and Q32 to retain, attract and hire key personnel; (viii) potential adverse reactions or changes to relationships with customers, employees, suppliers or other parties resulting from the announcement or completion of the proposed transaction; (ix) potential business uncertainty, including changes to existing business relationships, during the pendency of the proposed transaction that could affect Homology’s or Q32’s financial performance; (x) certain restrictions during the pendency of the proposed transaction that may impact Homology’s or Q32’s ability to pursue certain business opportunities or strategic transactions; (xi) the combined company’s need for additional funding, which may not be available; (xii) failure to identify additional product candidates and develop or commercialize marketable products; (xiii) the early stage of the combined company’s development efforts; (xiv) potential unforeseen events during clinical trials could cause delays or other adverse consequences; (xv) risks relating to the regulatory approval process; (xvi) interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; (xvii) Q32’s product candidates may cause serious adverse side effects; (xviii) inability to maintain our collaborations, or the failure of these collaborations; (xix) the combined company’s reliance on third parties, including for the manufacture of materials for our research programs, preclinical and clinical studies; (xx) failure to obtain U.S. or international marketing approval; (xxi) ongoing regulatory obligations; (xxii) effects of significant competition; (xxiii) unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; (xxiv) product liability lawsuits; (xxv) securities class action litigation; (xxvi) the impact of the COVID-19 pandemic and general economic conditions on our business and operations, including the combined company’s preclinical studies and clinical trials; (xxvii) the possibility of system failures or security breaches; risks relating to intellectual property; (xxviii) significant costs incurred as a result of operating as a public company; (xxix) whether the Company will meet the minimum bid price requirement during any compliance period or otherwise in the future, whether the Company will otherwise continue to meet the Nasdaq listing standards and whether the Company would be successful in any Nasdaq appeal process and (xxx) such other factors as are set forth in Homology’s periodic public filings with the Securities and Exchange Commission (SEC), including but not limited to those described under the heading “Risk Factors” in Homology’s Annual Report on Form 10-K for the year ended December 31,2023 and the registration statement on Form S-4 filed by Homology with the SEC. Homology can give no assurance that the conditions to the proposed transaction will be satisfied. Except as required by applicable law, Homology undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

About Homology Medicines

Homology Medicines, Inc. is a clinical-stage genetic medicines company historically focused on transforming the lives of patients suffering from rare diseases, by addressing the underlying cause of the disease. Homology Medicines has gene editing and gene therapy clinical-stage programs in phenylketonuria (PKU) and Hunter syndrome (MPS II), a preclinical pipeline that includes a gene therapy candidate for metachromatic leukodystrophy and a GTx-mAb (vectorized antibody) candidate for paroxysmal nocturnal hemoglobinuria, as well as intellectual property on its family of 15 adeno- associated viruses (AAVHSCs). Homology Medicines is not currently pursuing further development of these programs and is pursuing strategic options for the Company and its programs and platform technology. Additionally, the Company has an ownership stake in Oxford Biomedica (US) LLC (formerly Oxford Biomedica Solutions LLC), an AAV manufacturing company based on Homology Medicines’ internal process development and manufacturing formed as a joint venture between Homology Medicines and Oxford Biomedica plc. For more information, visit www.homologymedicines.com.

No Offer or Solicitation

This press release is not intended to and shall not constitute an offer to buy or sell or the solicitation of an offer to buy or sell any securities, or a solicitation of any vote or approval, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offer of securities shall be made, except by means of a prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended.

Homology Contact:
Paul Alloway
President and Chief Operating Officer
(781) 327-2633
palloway@homologymedicines.com

Aptose to Report Fourth Quarter and Year End 2023 Financial Results and Provide Corporate Update on Tuesday, March 26, 2024

Aptose to Report Fourth Quarter and Year End 2023 Financial Results and Provide Corporate Update on Tuesday, March 26, 2024




Aptose to Report Fourth Quarter and Year End 2023 Financial Results and Provide Corporate Update on Tuesday, March 26, 2024

SAN DIEGO and TORONTO, March 18, 2024 (GLOBE NEWSWIRE) — Aptose Biosciences Inc. (Nasdaq: APTO; TSX: APS), a clinical- stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, will report financial results for the fourth quarter and year ended December 31, 2023, on Tuesday, March 26, 2024, after the close of the market, and provide a corporate update.

Conference Call & Webcast:
   
Date: Tuesday, March 26, 2024
Time: 5:00 PM ET
Audio Webcast Only: link
Q&A Participant Registration Link*: link
(https://register.vevent.com/register/BIe69f08fc83634a6aa38bf7081d82c6a2)
   

*Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a unique PIN, which are required to access the conference call. They also will have the option to take advantage of a Call Me button and the system will automatically dial out to connect to the Q&A session.

The audio webcast also can be accessed through a link on the Investor Relations section of Aptose’s website here. A replay of the webcast will be available on the company’s website for 30 days.

The press release, the financial statements and the management’s discussion and analysis for the quarter ended September 30, 2023 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

About Aptose

Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company’s small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. The Company has two clinical-stage oral kinase inhibitors under development for hematologic malignancies: tuspetinib (HM43239), an oral, myeloid kinase inhibitor being studied as monotherapy and in combination therapy in the APTIVATE international Phase 1/2 expansion trial in patients with relapsed or refractory acute myeloid leukemia (AML); and luxeptinib (CG-806), an oral, dual lymphoid and myeloid kinase inhibitor in Phase 1 a/b stage development for the treatment of patients with relapsed or refractory hematologic malignancies. For more information, please visit www.aptose.com.

For further information, please contact:
   
Aptose Biosciences Inc.  LifeSci Advisors, LLC
Susan Pietropaolo Dan Ferry, Managing Director
Corporate Communications & Investor Relations 617-430-7576
201-923-2049 Daniel@LifeSciAdvisors.com
spietropaolo@aptose.com  

Avenue Therapeutics Reports Full Year 2023 Financial Results and Recent Corporate Highlights

Avenue Therapeutics Reports Full Year 2023 Financial Results and Recent Corporate Highlights




Avenue Therapeutics Reports Full Year 2023 Financial Results and Recent Corporate Highlights

– Enrollment completed in Phase 1b/2a clinical trial of AJ201 for spinal and bulbar muscular atrophy; topline data expected in second quarter of 2024 –

– Presented positive BAER-101 preclinical data at American Epilepsy Society (AES) and American Society for Experimental Neurotherapeutics (ASENT) Annual Meetings and in publication Drug Development Research

– Final agreement reached with FDA on study design and analysis approach for Phase 3 safety study of IV tramadol –

MIAMI, March 18, 2024 (GLOBE NEWSWIRE) — Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today reported financial results and recent corporate highlights for the year ended December 31, 2023.

“We made considerable progress across our pipeline of differentiated neurologic therapies in 2023,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue. “Avenue demonstrated tremendous execution capabilities with our lead clinical program AJ201, a potential first-in-class treatment for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s Disease. Within nine months of acquiring the rights to AJ201 in March 2023, we dosed the first patient in the Phase 1b/2a clinical trial, and we completed trial enrollment of 25 SBMA patients across six sites in the U.S. We remain on track to report topline data for AJ201 in the second quarter of 2024, an incredibly exciting milestone for both the Company and patients suffering from SBMA, a debilitating rare neuromuscular disorder with no approved treatments in the U.S. Additionally, we have progressed BAER-101 for the treatment of epilepsy by presenting preclinical data from a translational animal model in multiple peer-reviewed forums. We have also advanced IV tramadol, reaching final agreement with the U.S. Food and Drug Administration (FDA) on the safety study and statistical analysis approach for the final Phase 3 study. Pending additional financing, we look forward to progressing BAER-101 and IV tramadol for patients facing great unmet need. We look forward to another productive year in 2024 as we continue to make significant strides across our clinical pipeline and advance our mission of providing impactful therapies to patients suffering from neurologic diseases.”

Recent Corporate Highlights:

AJ201 (Nrf1 and Nrf2 activator, androgen receptor degradation enhancer for SBMA)

  • In January 2024, Avenue completed enrollment in the Phase 1b/2a clinical trial of AJ201 for the treatment of SBMA. The 12-week, multicenter, randomized, double-blind Phase 1b/2a clinical trial of AJ201 enrolled 25 patients randomly assigned to AJ201 (600 mg/day) or placebo. The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA. Topline data for the Phase 1b/2a study are anticipated in the second quarter of 2024. More information about this study can be found at ClinicalTrials.gov (Identifier: NCT05517603).

BAER-101 (GABAA α2/3 positive allosteric modulator)

  • Avenue presented preclinical results for BAER-101, a potentially best-in-class selective GABAA α2,3 positive allosteric modulator, in three scientific peer-reviewed settings, including the American Epilepsy Society (AES) Annual Meeting in December 2023, the publication Drug Development Research in February 2024 and the American Society for Experimental Neurotherapeutics (ASENT) Annual Meeting in March 2024. The in vivo data showcase BAER-101’s ability to significantly suppress seizures using the SynapCell’s Genetic Absence Epilepsy Rat from Strasbourg (“GAERS”) model of epilepsy. BAER-101 fully suppressed seizure activity in the GAERS model with a minimal effective dose of 0.3 mg/kg, PO, and the effect was fast in onset and stable throughout the duration of testing. The data also demonstrated BAER-101’s ability to selectively target GABAA α2 and α3 subtypes more than α1 and α5, potentially improving anti-convulsant and anxiolytic activity while minimizing the risk of tolerance and abuse associated with existing treatments in this drug class. Subject to obtaining the necessary financing, which could be provided through a strategic partnership, Avenue plans to initiate a Phase 2a clinical trial of BAER-101 to further study its anti-seizure properties in patients with common or rare epilepsies.

IV Tramadol

  • In January 2024, Avenue reached final agreement with the FDA on the safety study protocol and statistical analysis approach for the Phase 3 study for intravenous (“IV”) tramadol, which is in development for the treatment of acute post-operative pain in a medically supervised setting. The final non-inferiority study is designed to assess the theoretical risk of opioid-induced respiratory depression related to opioid stacking on IV tramadol compared to IV morphine. The study will randomize approximately 300 post bunionectomy patients to IV tramadol or IV morphine for pain relief administered during a 48-hour post-operative period. Patients will have access to IV hydromorphone, a Schedule II opioid, for rescue of breakthrough pain. Pending additional financing, Avenue aims to initiate the Phase 3 safety study as soon as feasible. The Company believes that the study can be completed and submitted to the FDA within 12 months of the study’s initiation.

General Corporate

  • In March 2024, Avenue announced that the Nasdaq Hearings Panel granted the Company’s request for an extension to evidence compliance with all applicable criteria for continued listing on The Nasdaq Capital Market, including the $1.00 bid price and $2.5 million stockholders’ equity requirements, through May 20, 2024.

2023 Financial Results:

  • Cash Position: As of December 31, 2023, cash and cash equivalents totaled $1.8 million, compared to $6.7 million at December 31, 2022, a decrease of $4.9 million. In January 2024, the Company completed a warrant inducement transaction resulting in $5.0 million in gross proceeds.
  • R&D Expenses: Research and development expenses for the full year 2023 were $6.1 million, compared to $2.7 million in 2022. Additionally, there was $4.2 million in expense for the acquisition of the AJ201 license in 2023.
  • G&A Expenses: General and administrative expenses for the full year 2023 were $4.2 million, compared to $5.3 million in 2022.
  • Net Loss: Net loss for the full year 2023 was $10.5 million, or $0.98 per share, compared to a net loss of $3.6 million, or $1.63 per share, in 2022.

About Avenue Therapeutics
Avenue Therapeutics, Inc. (Nasdaq: ATXI) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases. It is currently developing three assets including AJ201, a first-in-class asset for spinal and bulbar muscular atrophy, BAER-101, an oral small molecule selective GABAA α2, α3 receptor positive allosteric modulator for CNS diseases, and IV tramadol, which is in Phase 3 clinical development for the management of acute postoperative pain in adults in a medically supervised healthcare setting. Avenue is headquartered in Miami, FL and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.avenuetx.com.

Forward-Looking Statements
This press release contains predictive or “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of current or historical fact contained in this press release, including statements that express our intentions, plans, objectives, beliefs, expectations, strategies, predictions or any other statements relating to our future activities or other future events or conditions are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,” “should,” “would” and similar expressions are intended to identify forward-looking statements. These statements are based on current expectations, estimates and projections made by management about our business, our industry and other conditions affecting our financial condition, results of operations or business prospects. These statements are not guarantees of future performance and involve risks, uncertainties and assumptions that are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed or forecasted in, or implied by, the forward-looking statements due to numerous risks and uncertainties. Factors that could cause such outcomes and results to differ include, but are not limited to, risks and uncertainties arising from: the fact that we currently have no drug products for sale and that our success is dependent on our product candidates receiving regulatory approval and being successfully commercialized; the possibility that serious adverse or unacceptable side effects are identified during the development of our current or future product candidates, such that we would need to abandon or limit development of some of our product candidates; our ability to successfully develop, partner, or commercialize any of our current or future product candidates including AJ201, IV tramadol, and BAER-101; the substantial doubt raised about our ability to continue as a going concern, which may hinder our ability to obtain future financing; the significant losses we have incurred since inception and our expectation that we will continue to incur losses for the foreseeable future; our need for substantial additional funding, which may not be available to us on acceptable terms, or at all, which unavailability of could force us to delay, reduce or eliminate our product development programs or commercialization efforts; our reliance on third parties for several aspects of our operations; our reliance on clinical data and results obtained by third parties that could ultimately prove to be inaccurate, or unreliable, or unacceptable to regulatory authorities; the possibility that we may not receive regulatory approval for any or all of our product candidates, or that such approval may be significantly delayed due to scientific or regulatory reasons; the fact that even if one or more of our product candidates receives regulatory approval, they will remain subject to substantial regulatory scrutiny; the effects of current and future laws and regulations relating to fraud and abuse, false claims, transparency, health information privacy and security, and other healthcare laws and regulations; the effects of competition for our product candidates and the potential for new products to emerge that provide different or better therapeutic alternatives for our targeted indications; the possibility that the government or third-party payors fail to provide adequate coverage and payment rates for our product candidates or any future products; our ability to establish sales and marketing capabilities or to enter into agreements with third parties to market and sell our product candidates; our exposure to potential product liability claims; related to the protection of our intellectual property and our potential inability to maintain sufficient patent protection for our technology and products; our ability to maintain compliance with the obligations under our intellectual property licenses and funding arrangements with third parties, without which licenses and arrangements we could lose rights that are important to our business; the fact that Fortress Biotech, Inc. controls a majority of the voting power of our outstanding capital stock and has rights to receive significant share grants annually;; and those risks discussed in our filings which we make with the SEC. Any forward-looking statements speak only as of the date on which they are made, and we undertake no obligation to publicly update or revise any forward-looking statements to reflect events or circumstances that may arise after the date of this press release, except as required by applicable law. Investors should evaluate any statements made by us in light of these important factors.

Contact:
Jaclyn Jaffe
Avenue Therapeutics, Inc.
(781) 652-4500
ir@avenuetx.com

AVENUE THERAPEUTICS, INC.
Consolidated Balance Sheets
($ in thousands, except for share and per share amounts)
 
  December 31,     December 31,  
  2023     2022  
               
ASSETS              
Current assets:              
Cash and cash equivalents $ 1,783     $ 6,708  
Prepaid expenses and other current assets   67       137  
Total assets $ 1,850     $ 6,845  
               
LIABILITIES AND STOCKHOLDERS’ EQUITY              
Current liabilities:              
Accounts payable and accrued expenses $ 287     $ 949  
Accounts payable and accrued expenses – related party   323       21  
Warrant liability   586       2,609  
Total current liabilities   1,196       3,579  
               
Total liabilities   1,196       3,579  
               
Commitments and Contingencies              
               
Stockholders’ equity              
Preferred stock ($0.0001 par value), 2,000,000 shares authorized              
Class A Preferred stock, 250,000 shares issued and outstanding as of December 31, 2023 and 2022, respectively          
Common stock ($0.0001 par value)              
Common shares, 75,000,000 shares authorized and 25,597,622 shares issued and outstanding as of December 31, 2023; and 20,000,000 shares authorized and 4,773,841 shares issued and outstanding as of December 31, 2022   3        
Additional paid-in capital   92,507       84,456  
Accumulated deficit   (90,928 )     (80,551 )
Total stockholders’ equity attributed to the Company   1,582       3,905  
               
Non-controlling interests   (928 )     (639 )
Total stockholders’ equity   654       3,266  
Total liabilities and stockholders’ equity $ 1,850     $ 6,845  

AVENUE THERAPEUTICS, INC.
Consolidated Statements of Operations
($ in thousands, except for share and per share amounts)
 
   
  For the Years Ended  
  December 31,     December 31,  
  2023     2022  
Operating expenses              
Research and development $ 6,131     $ 2,698  
Research and development – licenses acquired   4,230        
General and administrative   4,179       5,345  
Loss from operations   (14,540 )     (8,043 )
               
Interest income   (126 )     (20 )
Financing costs – warrant liabilities   332       1,160  
Change in fair value of warrant liabilities   (4,258 )     (5,580 )
Net loss $ (10,488 )   $ (3,603 )
               
Net loss attributable to non-controlling interests   (111 )     (51 )
Net loss attributable to common stockholders $ (10,377 )   $ (3,552 )
               
Net loss per common share attributable to common stockholders, basic and diluted $ (0.98 )   $ (1.63 )
               
Weighted average number of common shares outstanding, basic and diluted   10,591,636       2,185,159  

Adverum Biotechnologies Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Pipeline Highlights and Corporate Updates

Adverum Biotechnologies Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Pipeline Highlights and Corporate Updates




Adverum Biotechnologies Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Pipeline Highlights and Corporate Updates

– Preliminary LUNA data presented at the Annual Meeting of the Macula Society support potential best-in-class clinical activity and an encouraging safety profile in patients with wet AMD –

– Completed $127.5 million private placement financing with new and existing institutional investors –

– Cash runway expected into late 2025 –

– 1-for-10 reverse stock split to be effective March 21, 2024 –

REDWOOD CITY, Calif., March 18, 2024 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company pioneering the use of gene therapy as a new standard of care for highly prevalent ocular diseases, today reported financial results for the fourth quarter and full year 2023. The company also provided recent pipeline highlights and corporate updates.

“We have made tremendous progress during 2023 and in 2024 to date in our development of Ixo-vec as a durable potential gene therapy treatment for the millions of patients suffering from wet AMD,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “We were pleased to present at Macula Society preliminary data from our ongoing LUNA Phase 2 trial of Ixo-vec, which demonstrated potentially best-in-class clinical activity at both the 2E11 and 6E10 doses of Ixo-vec, with the data trending similar to, or better than, the data in our first-in-human OPTIC trial. Importantly, the preliminary data demonstrated that Ixo-vec was generally well tolerated. We look forward to presenting the 26-week interim analysis of our LUNA Phase 2 program in mid-2024.”

Dr. Fischer continued, “In the lead-up to the data presentation at Macula Society, Adverum completed a $127.5 million private placement financing with new and existing investors that we expect to meaningfully extend our cash runway into late 2025, well beyond the anticipated presentation of topline 52-week data from our ongoing LUNA Phase 2 trial. In parallel, we are continuing to interact with regulators in the U.S. and E.U. to inform Phase 3 development of Ixo-vec, which we plan to initiate in the first half of 2025.”

Ixo-Vec Program Highlights:

  • Preliminary Ixo-vec LUNA Trial Data at Macula Society 2024:
    • In February 2024, we announced positive preliminary efficacy and safety data from the ongoing LUNA Phase 2 trial of Ixo-vec in patients with wet AMD at the 47th Annual Meeting of the Macula Society in Palm Springs, California.
    • LUNA patients treated with either the 2E11 or 6E10 dose of Ixo-vec experienced potentially best-in-class reduction in annualized anti-vascular endothelial growth factor (VEGF) injections and the percentage of patients receiving no supplemental injections through 26 weeks.
      • Patients at the 2E11 and 6E10 doses had mean 90% and 94% reductions, respectively, in annualized anti-VEGF injections.
      • 85% and 68% of patients at the 2E11 and 6E10 doses, respectively, received no supplemental injections through 26 weeks.
    • Maintained or improved mean BCVA and mean CST at both dose levels.
    • Ixo-vec continued to be generally well tolerated, with cases of intraocular inflammation responsive to local corticosteroids.
    • No Ixo-vec related serious adverse events, episcleritis, vasculitis, retinitis, choroiditis, vascular occlusion or hypotony were reported.
  • Long-Term Ixo-vec OPTIC Trial Data at AAO 2023:
    • In November 2023, the company announced 3-year data from the OPTIC extension study of patients with wet AMD during the Retina Subspecialty Day at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California.
    • Patients in the OPTIC extension trial continue to experience long-term benefit from Ixo-vec through 3 years of follow-up, including maintenance of vision, durability of anatomic improvements and sustained reduction in anti-VEGF treatment burden.
      • Patients at the 2E11 dose had an 84% reduction in annualized anti-VEGF injections, with 53% of the participants at the 2E11 dose receiving zero supplemental injections through three years.
      • Aflibercept protein levels have been sustained through follow-up, which is up to 4.5 years post-treatment.
      • BCVA was maintained and CST was improved through 3 years.
    • Ixo-vec was generally well tolerated, with the most common adverse event being dose-dependent inflammation that was responsive to topical corticosteroids.

Corporate Updates:

  • Approval of 1-for-10 reverse stock split: On March 8, the Board of Directors approved a 1-for-10 reverse stock split of the Company’s common stock. The Company’s common stock will begin trading on a reverse stock split-adjusted basis on March 21, 2024. Following the reverse stock split, there will be approximately 20.8 million shares of the company’s common stock outstanding.
  • $127.5 Million Private Placement Financing: In February 2024, the Company completed a private placement of 106.25 million shares of common stock (or pre-funded warrants in lieu thereof) to a select group of institutional and accredited healthcare specialist investors at a price of $1.20 per share, representing a premium of approximately 20% to Adverum’s 30-day volume-weighted average price. The financing was led by TCGX with participation from new and existing investors including 5AM Ventures, Commodore Capital, Frazier Life Sciences, Logos Capital, Samsara BioCapital, Venrock Healthcare Capital Partners and Vivo Capital, as well as two large investment management firms. The financing is expected to extend Adverum’s cash runway into late 2025.
  • Appointment of Romuald Corbau, Ph.D. as Chief Scientific Officer: In January 2024, the company announced the appointment of Romuald “Romu” Corbau, Ph.D. as Chief Scientific Officer. Dr. Corbau brings over 25 years of experience in drug development, including leading Spark Therapeutics’ translational R&D for Luxturna®, a subretinal injection gene therapy product for Leber’s congenital amaurosis, an inherited vision loss disease that routinely results in blindness

Anticipated Milestones

  • 2024: Continued FDA and EMA formal and informal regulatory interactions
  • Mid-2024: LUNA 26-week interim analysis
  • H1 2025: Initiation of Phase 3 trial

Financial Results for the Three Months Ended December 31, 2023

  • Cash, cash equivalents and short-term investments were $96.5 million as of December 31, 2023, compared to $117.1 million as of September 30, 2023, and $185.6 million as of December 31, 2022. Including proceeds from the February 2024 private placement financing, pro forma cash, cash equivalents and short-term investments was $223.8 million. Adverum expects its cash, cash equivalents and short-term investments to fund operations into late 2025.
  • Research and development expenses were $15.3 million for the three months ended December 31, 2023, compared to $22.2 million for the same period in 2022. Research and development expenses decreased due to lower clinical trial related expense, lower material production and bioanalytics, lower stock-based compensation expense, lower license fees, and lower outside research and development services. Stock-based compensation expense included in research and development expenses was $1.0 million for the fourth quarter of 2023.
  • General and administrative expenses were $10.9 million for the three months ended December 31, 2023, compared to $11.7 million for the same period in 2022. General and administrative expenses decreased due to lower facilities expense due to fewer premises leased in the current period and higher sublease income and lower depreciation expense. Stock-based compensation expense included in general and administrative expenses was $3.1 million for the fourth quarter of 2023.
  • Net Loss was $23.7 million, or $0.23 per basic and diluted share, for the three months ended December 31, 2023, compared to $32.7 million, or $0.33 per basic and diluted share for the same period in 2022.

About Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is a VEGF driven advanced form of AMD affecting approximately 10% of patients living with AMD associated with the build-up of fluid in the macula and the retina. Wet AMD is a leading cause of blindness in people over 65 years of age, with approximately 20 million individuals worldwide living with this condition. New cases of wet AMD are expected to grow significantly worldwide as populations age. AMD is expected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases. Additionally, wet AMD is a bilateral disease, and incidence of nAMD in the second eye is up to 42% in the first two to three years. The current standard of care requires frequent life-long repeated bolus injections of anti-VEGF in the eye. IVT gene therapy has the promise to preserve vision and reduce most or all injections for the life of the patient by delivering stable therapeutic levels of anti-VEGF to control macular fluid.

About Ixo-vec in Wet AMD
Adverum is developing ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), its clinical-stage gene therapy product candidate, for the treatment of wet AMD. Ixo-vec utilizes a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require surgery to administer the gene therapy under the retina (sub-retinal approach), Ixo-vec is designed to be administered as a one-time IVT injection in the physician’s office, deliver long-term efficacy, reduce the burden of frequent anti-vascular endothelial growth factor (VEGF) injections, optimize patient compliance and improve vision outcomes for patients with wet AMD. In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration granted Fast Track designation for Ixo-vec for the treatment of wet AMD. Ixo-vec has also received PRIME designation from the European Medicines Agency and the Innovation Passport from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency for the treatment of wet AMD.

About Adverum Biotechnologies
Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit www.adverum.com.

Forward-looking Statements
Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements regarding the potential benefits of Ixo-vec as a one-time IVT injection for the treatment of wet AMD, including its potential to improve vision outcomes, the potential best-in-class clinical activity of Ixo-vec, and anticipated timing of preliminary and interim data from the Phase 2 LUNA trial and initiation of a Phase 3 trial, and statements associated with the Company’s cash sufficiency and runway, and other statements containing the words “anticipates,” “believes,” “expects,” “intends,” “plans,” “target,” “will” and similar expressions. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: risks associated with market conditions. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s most recent Annual Report on Form 10-K filed with the SEC, as updated by any subsequent reports on Form 10-Q. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Corporate, Investor and Media Inquiries
Adverum Biotechnologies, Inc.
E: ir@adverum.com

 
Adverum Biotechnologies, Inc.
Selected Consolidated Balance Sheet Data
(In thousands)
         
  December 31,     December 31,
  2023     2022
         
Cash and cash equivalents, and marketable securities $ 96,526 1   $ 185,589
Total assets   173,010       308,372
Total current liabilities   24,914       32,246
Total liabilities   89,541       126,854
Total stockholders’ equity   83,469       181,518
 
(1) Cash, cash equivalents, and marketable securities exclude approximately $127.8 million in net proceeds from Adverum’s February 2024 private placement
 

 
Adverum Biotechnologies, Inc.
Consolidated Statements of Operations
(In thousands except per share data)
               
  Three months ended December 31,   Years ended December 31,
    2023       2022       2023       2022  
  (Unaudited)   (1)
               
License revenue $     $     $ 3,600     $  
               
Operating expenses:              
Research and development   15,278       22,199       77,676       99,277  
General and administrative   10,880       11,741       49,915       57,858  
Total operating expenses   26,158       33,940       127,591       157,135  
Operating loss   (26,158 )     (33,940 )     (123,991 )     (157,135 )
Other income, net   1,311       1,223       5,748       2,673  
Net loss before income taxes   (24,847 )     (32,717 )     (118,243 )     (154,462 )
Income tax benefit (provision)   1,133       (19 )     1,078       (74 )
Net loss   (23,714 )     (32,736 )     (117,165 )     (154,536 )
Net loss per share — basic and diluted $ (0.23 )   $ (0.33 )   $ (1.16 )   $ (1.56 )
Weighted-average common shares outstanding – basic and diluted   101,213       99,915       100,824       99,251  
               
(1) Derived from Adverum’s annual audited consolidated financial statements.