On January 10, 2025 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported updated guidance for the planned first- and second-line combination clinical trials for vepdegestrant in patients with locally advanced or metastatic estrogen receptor positive (ER+)/human epidermal growth factor receptor 2 negative (HER2-) breast cancer, highlighted key upcoming milestones and provided a corporate update (Press release, Arvinas, JAN 10, 2025, View Source [SID1234649587]).

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"We are on the cusp of a major transformation in 2025, with the potential to provide significant benefit to patients and meaningful value to our stockholders," said John Houston, Ph.D., Chairperson, Chief Executive Officer and President at Arvinas. "We are on track to report topline results from our first Phase 3 trial in the first quarter and to initiate two additional Phase 3 trials by the end of the year. In the first half of 2025, we plan to present the first-in-human data from ARV-102, our PROTAC LRRK2 degrader, which we believe will highlight the potential value that our PROTAC degraders may offer for patients with neurodegenerative diseases. And finally, we plan to share initial data from our Phase 1 trial with ARV-393, our PROTAC BCL6 degrader, which will provide an early look the tolerability and efficacy in patients with B-cell lymphomas."

Select milestones anticipated in 2025

Vepdegestrant: Oral PROTAC ER degrader

As part of Arvinas’ global collaboration with Pfizer, in 2025 the companies plan to:

Announce topline data for the VERITAC-2 Phase 3 monotherapy clinical trial in patients with second-line-plus ER+/HER2- metastatic breast cancer (mBC) (1Q25).
Initiate two new Phase 3 combination trials in patients with ER+/HER2- mBC (pending emerging data and regulatory feedback):
First-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib.
Second-line Phase 3 combination trial with a CDK4/6 inhibitor.
With the prioritization of the vepdegestrant plus atirmociclib combination for the first-line setting, the VERITAC-3 trial evaluating vepdegestrant plus palbociclib in the first-line will not proceed beyond the study lead-in.

ARV-102: Oral PROTAC LRRK2 degrader

Present single-ascending dose data from the ongoing Phase 1 clinical trial in healthy volunteers in an oral session at the Alzheimer’s Disease/Parkinson’s Disease (AD/PD) conference in Vienna, Austria (April 1-4, 2025).
Complete enrollment and present initial data from the ongoing Phase 1 clinical trial in patients with Parkinson’s disease.
ARV-393: Oral PROTAC BCL6 degrader

Present initial data from the ongoing Phase 1 clinical trial in patients with B-cell lymphomas (NCT06393738).
Novel PROTAC KRAS G12D degrader

File an Investigational New Drug (IND) application.
Corporate update
Alex Santini, Arvinas’ Senior Vice President, Global and U.S. Market Access, has been appointed interim Chief Commercial Officer, effective January 17, 2025. Mr. Santini joined Arvinas in 2023 with more than 30 years of experience managing and leading commercial organizations. Previously, he was Executive Vice President, Chief Commercial Officer, at Lexicon Pharmaceuticals. Prior to Lexicon, Mr. Santini was Executive Vice President, U.S. Market Access at Bayer, where he served on the U.S. Executive Committee. Mr. Santini’s prior experience also includes serving as Senior Vice President, Market Access at Nektar Therapeutics, and he began his career at Berlex Laboratories, where he served in roles of increasing responsibility in the commercial organization.

John Northcott, Chief Commercial Officer, is leaving the Company for personal reasons, effective January 17, 2025.

"Our commercial organization couldn’t be in a better position, and I look forward to working closely with Alex," continued Dr. Houston. "He has been a highly valued member of the Arvinas team for multiple years, and his well-established ability to build and lead an outstanding commercial team will be invaluable as we prepare for our potential first launch alongside our partners at Pfizer. We thank John for his contributions to the business, particularly his efforts to begin building a strong commercial organization for launch."

About Vepdegestrant
Vepdegestrant is an investigational, orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with ER positive (ER+)/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer. Vepdegestrant is being developed as a potential monotherapy and as part of combination therapy across multiple treatment settings for ER+/HER2- metastatic breast cancer.

In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will share worldwide development costs, commercialization expenses, and profits.

The U.S. Food and Drug Administration (FDA) has granted vepdegestrant Fast Track designation as a monotherapy in the treatment of adults with ER+/HER2- locally advanced or metastatic breast cancer previously treated with endocrine-based therapy.

On January 10, 2025 Quanterix Corporation (NASDAQ: QTRX), a company fueling scientific discovery through ultra-sensitive biomarker detection, and Akoya Biosciences (NASDAQ: AKYA), The Spatial Biology Company, reported a definitive merger agreement under which Quanterix will acquire Akoya in an all-stock transaction (Press release, Akoya Biosciences, JAN 10, 2025, View Source [SID1234649586]). The transaction will create the first integrated solution for ultra-sensitive detection of blood- and tissue-based protein biomarkers.

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"Liquid biopsy will eventually surpass the market size of all other diagnostics testing combined. Enabling early disease detection, before symptoms appear, using non-invasive methods is our mission and will be the majority of Quanterix’s long-term value," said Masoud Toloue, PhD, Chief Executive Officer of Quanterix. "This transaction accelerates our progress by creating the first platform that lets researchers and clinicians track disease progression from tissue to blood. By starting with tissue and detecting early signs of complementary proteins in blood using leading ultra-sensitive SIMOA technology, we are uniquely positioned to speed up market development of new liquid biopsy tests."

Brian McKelligon, Chief Executive Officer of Akoya, said, "Joining forces with Quanterix marks a pivotal step in our journey to revolutionize the way we understand and treat disease. We are thrilled to be part of an established leader in the life science tools and diagnostics market that not only strengthens our presence in critical markets but also accelerates our ability to scale, innovate and ultimately bring to market products that impact human health. With the capital structure and significant synergies facilitated through this transaction, the combined company is well-positioned for future growth and profitability. We look forward to being part of the Quanterix team to better serve the needs of researchers and clinicians and bring substantial value to our customers and shareholders."

Strategic and Financial Benefits of the Combination

Creates first integrated solution for liquid and tissue proteomic biomarkers: The integration of Akoya’s spatial biology capabilities in tissue with Quanterix’s advanced tools for the ultra-sensitive detection of biomarkers in blood will establish the first fully integrated technology ecosystem to identify and measure biomarkers across tissue and blood. Quanterix will be better positioned to serve research customers and ultimately clinicians with a broader set of technologies to improve diagnostic relevance and accuracy and enhance patient outcomes through biomarker-driven treatment decisions.
Expands technology offering across high growth markets in neurology, oncology and immunology: With Quanterix’s industry-leading position in neurology and Akoya’s focus within oncology and immunology, Quanterix will expand its technology offerings across these high-growth markets. The addition of Akoya’s cutting-edge spatial biology capabilities will enable Quanterix to capitalize on growth opportunities in a $5 billion serviceable addressable market.
Expanded lab services and clinical diagnostic market opportunity: Leveraging Akoya’s established clinical partnerships and CLIA-certified lab services, Quanterix is now strategically positioned to drive significant value creation through an expanded portfolio of lab service offerings. This collaboration establishes a clear path for Quanterix to participate in the rapidly emerging spatial biology clinical market, particularly in oncology.
Increases commercial reach and maximizes cross-selling opportunities: Quanterix and Akoya have complementary offerings and deep customer relationships across discovery, translational, and clinical research. When offered as an integrated solution, Quanterix expects significant cross-selling opportunities to a combined 2,300 instrument install-base driving strong double-digit organic revenue growth in 2026.
Accelerates path to profitability through realization of substantial cost savings: The transaction is expected to generate approximately $40 million in annual cost synergies by the end of 2026, with $20 million expected to be realized within the first year following close. These cost savings will be driven primarily by the elimination of duplicative corporate structures, streamlined commercial infrastructure, increased operational efficiencies, process improvements and footprint optimization. The synergies will be additive to the cost savings initiatives already implemented by the two organizations. Quanterix’s previous cost initiatives combined with the expected cost synergies from the transaction are expected to accelerate its path to profitability, including generating positive free cash flow in 2026.
Significant combined cash balance: For the trailing 12 months ending September 30, 2024, the combined company generated revenue of approximately $220 million. With more than $300 million in combined cash today, Quanterix expects to have approximately $175 million in cash with no expected debt at the time of closing, after accounting for debt repayment, transaction costs, and a $20 million payment for its recently announced EMISSION acquisition. Quanterix will have financial flexibility to advance the Company’s global diagnostic testing infrastructure, including for Alzheimer’s disease and other growth opportunities such as Akoya’s advancement into the companion diagnostics segment.
Transaction Terms

Under the terms of the agreement, which was approved by the Boards of Directors of both companies, Akoya shareholders will receive 0.318 shares of Quanterix common stock for each share of Akoya common stock owned. This represents a 19% premium to Akoya’s unaffected stock price on November 14, 2024, the last full trading day prior to Akoya’s announcement of its review of strategic alternatives.

Following the close of the transaction, Quanterix shareholders will own approximately 70% of the combined company and Akoya shareholders will own approximately 30%, on a fully diluted basis.

Timing, Approvals and Governance

The transaction is expected to close in the second quarter of 2025, subject to approval by both companies’ shareholders, expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and satisfaction of other customary closing conditions.

Quanterix has entered into voting agreements supporting the transaction with certain shareholders owning more than 50% of Akoya’s common stock.

Following close, Masoud Toloue will serve as Chief Executive Officer and Vandana Sriram will serve as Chief Financial Officer. The combined company will continue to operate under the Quanterix name.

Upon closing, the Quanterix Board will consist of nine members. Two current Quanterix directors will resign, and Quanterix will appoint two directors designated by Akoya from their current Board.

Conference Call and Webcast

Quanterix will host a conference call and webcast today at 8:30 a.m. E.T. to discuss the transaction. For audio, use the following dial-in number and passcode: USA & Canada – Toll-Free (800) 715-9871 Conference ID: 9092934. Interested investors can also access the live webcast from the News & Events page within the Investors section of the Quanterix website at View Source

Advisors

Goldman Sachs & Co. LLC is serving as financial advisor to Quanterix and Covington & Burling LLP is serving as its legal counsel. Perella Weinberg Partners LP is serving as financial advisor to Akoya and DLA Piper LLP is serving as its legal counsel.

On January 10, 2025 Rein Therapeutics ("Rein") (NASDAQ: RNTX), formerly known as Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported that it has changed its name to Rein Therapeutics, Inc (Press release, Aileron Therapeutics, JAN 10, 2025, View Source [SID1234649585]). The new name, logo, website, and branding elements reflect the Company’s mission to develop first-in-class treatments to rein in diseases. The Company’s common stock will begin trading under the Nasdaq ticker symbol "RNTX" effective January 13, 2025. The CUSIP number for the Company’s common stock is not affected by the name change. The Company also highlighted its recent clinical and corporate achievements and provided its key strategic objectives for 2025.

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"Our rebrand to Rein Therapeutics reflects our unwavering commitment to address the critical needs of underserved patients with fibrotic diseases," said Brian Windsor, Ph.D., President and Chief Executive Officer. "This new chapter for the Company underscores our focus on reining in fibrosis and advancing our pipeline of novel candidates. We look forward to the initiation of a Phase 2 clinical trial for our lead candidate, LTI-03, in the first half of this year, aiming to bring hope to those affected by idiopathic pulmonary fibrosis."

Recent Clinical Achievements

LTI-03, a Caveolin-1 related peptide:
In November 2024, the Company announced positive topline data from Cohort 2 of the Phase 1b clinical trial evaluating the safety and tolerability of inhaled high dose LTI-03 (5 mg BID) and a set of exploratory biomarkers in patients with idiopathic pulmonary fibrosis (IPF). Four biomarkers showed statistical significance in the combined Cohort 1 and Cohort 2 dataset, and five demonstrated dose dependence with respect to low dose (2.5 mg BID) Cohort 1, indicative of active pharmacodynamics. High dose LTI-03 continued to exhibit a favorable safety profile.
2024 Corporate Highlights:
In March 2024, Brian Windsor, Ph.D., was appointed Chief Executive Officer of the Company, marking the Company’s sole focus on advancing a pipeline of first-in-class therapies for orphan pulmonary and fibrosis indications.

In May 2024, the Company completed an underwritten registered direct offering of its common stock and accompanying warrants raising net proceeds of approximately $17.7 million.
2025 Strategic Objectives and Anticipated Milestones

LTI-03: A Phase 2 trial of LTI-03 for the treatment of IPF is anticipated to be initiated in the first half of this year.

LTI-01: The Company’s Phase 2b-ready asset is a first-in-class therapy for the treatment of loculated pleural effusions (LPEs). It holds Orphan Drug Designation for the treatment of pleural empyema in the U.S and E.U. and Fast Track Designation in the U.S.

On January 09, 2025 Light Horse Therapeutics Inc., a developer of first-in-class small molecule therapeutics, reported a $62 million Series A financing (Press release, Light Horse Therapeutics, JAN 9, 2025, View Source [SID1234649637]). The financing was led by founding investor Versant Ventures and included Mubadala Capital as well as three strategic investors: Bristol-Myers Squibb Co, Taiho Ventures, and AbbVie. Light Horse is the latest company started by Versant’s Inception Discovery Engine, and its scientific co-founders are Brian Liau, PhD, Ben Cravatt, PhD, and Nathanael Gray, PhD.

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Light Horse has developed a proprietary platform that uncovers novel functional sites in disease-critical targets. The company integrated distinct approaches to build this precision genetic editing-based platform that interrogates proteins in their native environment, providing a faster discovery of clinical candidates.

The company’s technology turns conventional drug discovery on its head by first identifying novel functional domains and then screening for chemistry. This "function-first" approach is the converse of traditional "screening-first" methodologies.

Led by Light Horse’s CSO, Laure Escoubet, PhD, the Light Horse team has translated this methodology into first-in-class preclinical programs against high-value oncology targets and has enabled a discovery collaboration with Novartis that is described in a separate press release issued today. Light Horse internal programs are now progressing through preclinical testing, paving the way towards the development of disruptive therapies.

"We are very impressed with Light Horse’s chemical genomics capabilities to rapidly investigate protein complexes at scale and to uncover unprecedented functional sites in high-value targets," said Rami Hannoush, PhD, venture partner at Versant and a Light Horse board member. "We believe the talented team at Light Horse and its technology will enable a pipeline of first-in-class drug candidates."

"We have a unique capability to interrogate proteins and pathways, identifying functionally critical sites that can drive the development of groundbreaking first-in-class therapies," said Light Horse CEO Markus Renschler, MD. "Our initial focus addresses high-value, historically challenging oncology targets with the opportunity to apply the technology to other therapeutic areas in the future."

Leadership team and scientific founders

Light Horse is led by experienced executives who possess expertise in oncology drug discovery and development. They are complemented by scientific founders who are world leaders in chemoproteomics and chemical biology.

Thomas Daniel, MD, Chairman
Dr. Daniel has more than 25 years of experience leading and building research and development organizations and leading investments in new companies at the forefront of innovative application of science to healthcare. He served as President of Global Research and Early Development at Celgene and previously held senior leadership roles at AmbRx, Amgen and Immunex.
Markus Renschler, MD, CEO
Dr. Renschler is a proven biotechnology executive with more than 25 years’ experience in building successful companies. Prior to Light Horse, he was President and CEO of Cyteir Therapeutics, Inc. leading it from a private discovery stage startup to a publicly traded clinical-stage company. Prior to Cyteir, he held leading roles in clinical R&D, business development and medical affairs at Celgene, Pharmion and Pharmacyclics, where he helped to develop and/or launched some of the most successful cancer drugs approved to date.
Laure Escoubet, PhD, CSO
Dr. Escoubet has almost 20 years of experience in building successful drug discovery teams and driving drug discovery programs into the clinic. Prior experiences include Vice President, Biology, at Zentalis, Head of Epigenetic Drug Discovery at Bristol-Myers Squibb/Celgene and other leading roles in early drug discovery in oncology.
Brian Liau, PhD, scientific co-founder
Dr. Liau is an Associate Professor of Chemistry and Chemical Biology at Harvard University. His research group develops chemical genomic approaches to interrogate the mechanisms of gene regulatory complexes and their promise as therapeutic targets.
Ben Cravatt, PhD, scientific co-founder
Dr. Cravatt is the Gilula Chair of Chemical Biology and Professor in the Department of Chemistry at Scripps Research. His research group develops and applies chemical proteomic technologies for protein and drug discovery on a global scale and has particular interest in studying biochemical pathways in the nervous system and cancer. Dr. Cravatt is a co-founder of multiple successful biotech companies.
Nathanael Gray, PhD, scientific co-founder
Dr. Gray is the Krishnan-Shah Family Professor of Chemical and Systems Biology at Stanford, Co-Director of Cancer Drug Discovery Co-Leader of the Cancer Therapeutics Research Program, Member of Chem-H, and Program Leader for Small Molecule Drug Discovery for the Innovative Medicines Accelerator. His research uses the tools of synthetic chemistry, protein biochemistry, and cancer biology to discover and validate new strategies for the inhibition of cancer targets.
"Light Horse has deep scientific talent, a very experienced management team, and a strong syndicate of strategic and institutional investors to enhance its delivery of exceptional drug candidates addressing unmet medical need" said Dr. Daniel.

On January 09, 2025 Light Horse Therapeutics Inc., a developer of first-in-class small molecule therapeutics, announced a multi-target collaboration with Novartis to identify and develop potentially first-in-class therapeutics using the biotech’s platform (Press release, Light Horse Therapeutics, JAN 9, 2025, View Source [SID1234649563]). Under the terms of the agreement, Light Horse will receive a $25 million upfront payment. The company is also eligible for $1 billion in further research, development and sales milestones, in addition to royalties on licensed therapeutics. Further details are undisclosed.

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"Light Horse’s high-caliber team has built a cutting-edge discovery platform that rapidly enables the exploration of core biological areas of interest, the identification of novel targets and the functionalization of high-value targets," said John Tallarico, Head of Discovery Sciences at Novartis. "This collaboration has the potential to create first-in-class therapeutics capable of driving meaningful benefit for patients."

"The Novartis collaboration presents an extraordinary opportunity to leverage our unbiased genetic screening platform and proprietary chemical libraries to address novel, high-value targets previously considered hard to drug," said Light Horse CEO Markus Renschler, MD. "We are excited to work with the Novartis team to potentially accelerate the development of innovative cancer treatments over the coming years."