Axelar AB

The Challenge

Non-small cell lung cancer (NSCLC) is the most common form of lung cancer and it is among the most deadly of all cancers. Surgery, and to some extent radiotherapy, is used as a therapy for the very few patients with localized tumors. However, most NSCLC patients present with advanced metastatic disease already at the time of diagnosis, and the median survival time with first-line therapy is only around 10 months in spite of recent  advances in treatment methods and introduction of new pharmaceuticals(1). The need for new effective targeted treatments is huge.

Axelar’s Solution

Axelar has discovered AXL1717, which is a  dual-action compound. AXL1717 targets the signaling pathway of the Insulin-like Growth Factor 1-receptor (IGF- 1R). IGF-1R is overexpressed on cancer cells and its signaling pathway is believed to be of great importance for cancer cell growth, tumor cell survival and resistance to therapy(2). The IGF-1R signaling pathway is therefore an excellent target for cancer drug development and a range of major tumors including NSCLC may be addressed through inhibition of this receptor. At the same time, AXL1717 also suppress tumor cell division by arresting cells in mitosis through a non-IGF-1R dependent mechanism.

Competitive Advantages

Because IGF-1R is structurally closely related to the insulin receptor, most of the small molecule IGF-1R inhibitors in clinical development also inhibit the insulin receptor with ensuing adverse effects. Axelar’s lead candidate AXL1717 is the first small molecule inhibitor of the IGF-1R signaling pathway in clinical development that clearly does not simultaneously inhibit the insulin receptor. AXL1717 has shown pronounced anti-tumor activity in a range of animal studies, demonstrating tumor regression or inhibition of tumor growth.

Based on promising Phase I/II data, a randomized, open-label Phase II study with AXL1717 versus standard regimen chemotherapy (docetaxel) was performed in NSCLC patients that had progressed from first line treatment. Results from this study showed efficacy, however no statistically significant difference in PFS, the primary endpoint, between the arms. Data indicated that patients with adenocarcinoma responded better to AXL1717 than patients with squamous cell carcinoma. The main side effect for both trial arms was neutropenia, reported in 22% of the patients treated with AXL1717 and 53% in those treated with docetaxel.

The Market

Each year 1.1 million patients are diagnosed with lung cancer in the major pharmaceutical markets and the disease accounts for 950,000 deaths annually in these territories. Around 85 % of those patients have NSCLC(4). Axelar’s lead product is focusing on the two most common types of NSCLC, squamous cell carcinoma and adenocarcinoma, which each year affect approximately 30 % each of the NSCLC patients(5). In the United States alone, about 130,000 new patients with these types of lung cancers are diagnosed annually (4,5).

The global NSCLC market is estimated at $5.71 billion in 2012 and it is projected that the market will grow to $7.52 billion in 2022 (3). Major drivers of the growth of the NSCLC market over the forecast period include (i) a growing aging population, (ii) increasing NSCLC incidence in several markets, especially in Japan and the emerging markets of China and India, as well as (iii) an increased adoption of premium-priced new therapies.

With respect to AXL1717, it is noteworthy that common cancers such as brain tumors, prostate, breast and colorectal cancer may also be targeted by the compound.


•   AXL1717 has shown potent anti-tumor effect against a wide range of tumors in various animal models

•  In a Phase I/II study, AXL1717 demonstrated a good safety profile and signs that suggest clinical benefit in some patients with NSCLC were observed

•  Results from a randomized Phase II clinical trial indicated that AXL1717 has activity in patients in NSCLC

•   An investigator-led Phase I/II study in malignant brain tumors has been initiated at Rush University Medical Hospital in Chicago

Planned Milestones

•  Further elucidation of the AXL1717 mechanism of action to be presented

•  Continuation of the clinical program with AXL1717 together with a strategic partner Patent Status

The lead compound AXL1717 is protected by several medical use patents, and by a process patent for making this compound. Patent applications are also pending to novel polymorphs of AXL1717.


Axelar intends to find one or more partners to continue the clinical development of AXL1717.
1. Source: Goffin et al., J Thorac Oncol.
5(2):260-274, 2010
2. Source: Pollak, Nat Rev Cancer.
8(12):915-928, 2008
3. Source: NSCLC: Global drug forecast and market analysis to 2022. Globaldata Report 2014
4. Source: Datamonitor, Epidemiology:
Non-Small Cell Lung Cancer, 2011
5. Source: Travis et al., Cancer. 75(12):2979, 1995


Axelar AB
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