Ipsen Strengthens Its Preclinical Oncology Pipeline With Exclusive Global Collaboration With BAKX Therapeutics Inc. For BKX-001, Targeting The Apoptosis Pathway


PARIS–(COMMERCIAL THREAD) – Regulatory news:

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Ipsen (Euronext: IPN; ADR: IPSEY) and BAKX Therapeutics Inc. have signed an exclusive global collaboration agreement for the research, development, manufacture and commercialization of BKX-001 as a potential treatment for leukemia, lymphoma and solid tumors.

Apoptosis is the natural process of programmed cell death. Deregulated apoptosis can lead to uncontrolled cell division and the development of a tumor.2 The apoptosis cell signaling pathway has proven to be a target for cancer treatment with the development and approval of BCL-2 inhibitors for the treatment of certain hematologic malignancies.3 BAX is a new target in this path which is downstream of all anti-apoptotic proteins like BCL-2, BCL-XL, MCL-1 etc.4 Direct activation of BAX has several possible outcomes as an experimental cancer therapy, potentially attacking multiple tumor types and the resistance encountered while targeting only the anti-apoptotic proteins of the BCL-2 family. Activation of BAX by small molecule agonists has been shown to promote apoptosis in leukemic cell lines and human samples, while sparing healthy cells in vitro and suppressing human acute myeloid leukemia xenografts and in increasing the survival of the host without toxicity in vivo.5

Dr Howard Mayer, Executive Vice President and Head of Research and Development of Ipsen, said: “As part of our strategy, we continue to strengthen our pipeline and provide exciting external innovation opportunities. We are therefore delighted to partner with the team of experts at BAKX Therapeutics to advance BKX-001 in preclinical development, with the goal of achieving a developmental candidate that can be evaluated for the potential treatment of hematologic malignancies and solid tumors. It is important to note that this collaboration also reflects the values ​​shared between our two organizations and, as a result, we will build a strong business-to-business team to advance this innovation for people living with these forms of cancer and their care teams. ”

Sree Kant, Founder and CEO of BAKX Therapeutics Inc. said: “We are delighted to partner with Ipsen on the experimental BKX-001. This program is the result of pioneering work on the BAX protein led by our scientific co-founders Loren D Walensky and Evripidis Gavathiotis. This partnership combines Ipsen’s excellent clinical and commercial development capabilities with our state-of-the-art knowledge of the BAX protein and our unique computing platform. It is a collaboration of very distinct complementary capacities that together can lead to important treatment options in treating cancer for patients, faster. ”

Under the agreement, Ipsen will pay BAKX Therapeutics Inc. $ 14.5 million on closing, comprising an equity investment and an upfront payment, followed by up to $ 837.5 million in milestone payments . Businesses would also share equally the costs and benefits.



BKX-001 is a small molecule oral BCL-2 associated protein X (BAX) oral activator. BKX-001 binds to the BAX trigger site catalytically, stimulating a BAX conformational change to induce apoptosis in tumor cells. BKX-001 is active in hematologic malignancies, including various leukemias and lymphomas, and certain solid tumors with high unmet needs. Combinations of BKX-001 with inhibitors of the BCL-2 family have shown a strong synergistic effect suggesting an approach for the treatment of resistant tumors. Rational combination approaches based on biomarkers with other anticancer agents are also being evaluated.


Ipsen is a mid-sized global biopharmaceutical company specializing in transformative drugs in oncology, rare diseases and neuroscience; it also has a well-established activity in the field of general public health. With total sales of more than 2.5 billion euros in fiscal year 2020, Ipsen sells more than 20 drugs in more than 115 countries, with a direct commercial presence in more than 30 countries. The Company’s research and development efforts are focused on its innovative and differentiated technological platforms located at the heart of the main biotechnology and life sciences centers: Paris-Saclay, France; Oxford, United Kingdom; Cambridge, United States; Shanghai, China. Ipsen has around 5,700 employees worldwide and is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt (ADR: IPSEY) program. For more information, visit ipsen.com.

BAKX Therapeutic

BAKX Therapeutics is a New York and Boston-based biotechnology company focused on targeting pathways of mitochondrial apoptosis. BAKX was co-founded by world-renowned researchers in the field of apoptosis and computer-aided drug discovery. BAKX’s integrated platform is characterized by (i) unique knowledge of targets and pathways, combined with (ii) in-depth expertise in working with dynamic protein interactions of conformation, and (iii) computational methodologies pioneers to simulate protein interactions in the membrane. BAKX’s flagship active ingredient, BKX-001 (in partnership with Ipsen), focuses on targeting the BAX protein. Additionally, the BAKX platform is deployed to drug two other undisclosed apoptosis targets involved in various solid tumors and resistance build-up. For more information, visit bakxtx.com

Ipsen forward-looking statements

The forward-looking statements, objectives and targets contained in this document are based on Ipsen’s management strategy, its current opinions and assumptions. Such statements involve known and unknown risks and uncertainties which may cause actual results, performance or events to differ materially from those anticipated in this document. All of the above risks could affect Ipsen’s future ability to meet its financial goals, which have been set assuming reasonable macroeconomic conditions based on information available today. The use of the words “believes”, “anticipates” and “expects” and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including filings and regulatory decisions. . In addition, the objectives described in this document were established without taking into account assumptions of external growth and possible future acquisitions, likely to modify these parameters. These objectives are based on data and assumptions considered reasonable by Ipsen. These targets depend on conditions or facts that may occur in the future, and not exclusively on historical data. Actual results may deviate significantly from these objectives given the occurrence of certain risks and uncertainties, including the fact that a promising product in early development or clinical trial phase may end up never being launched on the market. or achieve its business objectives, in particular for regulatory or competition reasons. Ipsen faces or could face competition from generic products which may result in a loss of market share. In addition, the Research and Development process comprises several stages, each of which carries the significant risk that Ipsen will not be able to achieve its objectives and be forced to abandon its efforts on a product in which it has invested large sums. Therefore, Ipsen cannot be certain that the favorable results obtained in the preclinical trials will be confirmed subsequently in the clinical trials, or that the results of the clinical trials will be sufficient to demonstrate the safety and efficacy of the product concerned. There can be no guarantee that a product will receive the necessary regulatory approvals or that the product will prove to be commercially successful. If the underlying assumptions prove to be incorrect or if risks or uncertainties materialize, actual results may differ materially from those stated in forward-looking statements. Other risks and uncertainties include, but are not limited to, general industry conditions and competition; general economic factors, including fluctuations in interest rates and exchange rates; the impact of pharmaceutical industry regulation and health care legislation; global trends towards containing health care costs; technological advances, new products and patents obtained by competitors; challenges inherent in developing new products, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative products; and exposure to litigation, including patent litigation, and / or regulatory actions. Ipsen also depends on third parties to develop and market some of its products which may generate substantial royalties; these partners could behave in a way that is detrimental to Ipsen’s business and financial results. Ipsen cannot be certain that its partners will fulfill their obligations. She may not be able to derive any benefit from these agreements. Failure of one of Ipsen’s partners could generate lower than expected revenues. Such situations could have a negative impact on Ipsen’s business, financial situation or performance. Ipsen expressly disclaims any obligation or commitment to update or revise any forward-looking statements, objectives or estimates contained in this press release to reflect any change in the events, conditions, assumptions or circumstances about which such statements are made. based, unless required by law. Ipsen’s business is subject to the risk factors described in its reference documents filed with the Autorité des Marchés Financiers. The risks and uncertainties mentioned are not exhaustive and the reader is invited to refer to the Ipsen 2020 Registration Document, available on ipsen.com.

1 BAKX Therapeutic. The Apoptosis Society. Pipeline. https://bakxtx.com/pipeline/ (last accessed July 2021).

2 National Institute for Research on the Human Genome. https://www.genome.gov/genetics-glossary/apoptosis (last accessed July 2021).

3 Venclyxto SmPC. European Medicines Agency. Available here: https://www.ema.europa.eu/en/medicines/human/EPAR/venclyxto

4 ARD Delbridge and A. Strasser Cell Death Differ., 22 (2015), pp. 1071-1080. Available here: https://www.nature.com/articles/cdd201550.

5 DE Reyna, TP Garner, A. Lopez, F. Kopp, GS Choudhary, A. Sridharan, SR Narayanagari, K. Mitchell, B. Dong, BA Bartholdy et al. Cancer cell, 32 (2017), pp. 490-505. Available here: https://www.cell.com/cancer-cell/fulltext/S1535-6108(17)30364-1


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