Project portfolio

Idogen´s technology enables development of tolerogenic cell therapy in a number of areas. Idogen has chosen to develop, as a first application, a treatment method for patients with severe haemophilia Type A who currently lack any treatment option, after developing antibodies to their vital factor VIII therapy.

When the body’s immune system attacks factor VIII, a critical medication

IDO 8 is Idogen’s most advanced development program, with the objective of developing a tolerogenic cell therapy for patients with severe hemophilia A who have developed neutralizing antibodies against their ongoing treatment. Hemophilia A is caused by a hereditary lack of coagulation factor VIII, and the standard treatment for patients with a severe form of hemophilia is to treat them with drugs that consist of the missing coagulation factor. Approximately 30% of the patients treated with factor VIII, however, develop neutralizing antibodies, or inhibitors, which inhibit the effect of the coagulation factor and render the treatment ineffective. This complication can often be managed by intensifying factor VIII treatment to induce tolerance, which means frequent injections of a high dose of factor VIII. This is the recommended treatment today under guidelines from the World Hemophilia Federation (WHF) and can last anywhere from six months up to three years. Unfortunately, these antibodies remain in approximately one third of patients, which leaves them without an alternative for a therapy that could prevent bleeding. Idogen’s initial development program, IDO 8, is aimed at meeting the need among these patients. The goal of IDO 8 is to treat these patients with tolerogenic cell therapy, thereby inducing long-term tolerance for FVIII and thus recovering the desired effect of the life-saving treatment with factor VIII.

The company is working to be ready to recruit the first patient in the Phase 1/2a clinical trial in the second quarter of 2022

Hemophilia A is a rare disease and Idogen has been granted European orphan drug designation for the treatment – a key step for the company since orphan drug designation provides several regulatory advantages such as less extensive requirements for clinical trials, scientific guidance from the regulator during development and ten-year market exclusivity in Europe after launch. Idogen also intends to apply for orphan drug designation for hemophilia A in the US and Japan as well. In the US, orphan drug designation would entail seven years of market exclusivity after launch.

Potential market for IDO8

When the body’s immune system attacks a transplanted organ

Idogen intends to use the same method that has now been developed for the treatment of hemophilia in other therapeutic areas with only minor adjustments to the production process. Therefore, the company is now also developing IDO T, a product candidate for transplantation. The treatment could work for several types of organs, and the company intends to focus initially on kidney transplantation. The basic principle is to “teach” the immune system to recognize and tolerate the transplanted organ so that it is not rejected in conjunction with the transplantation. This could eventually increase the lifespan of the transplanted organ and reduce or eliminate entirely the need for treatment, often lifelong, with immunosuppressive drugs that do not selectively suppress the immune system. A successful product would be expected to decrease the side effects that result from the immunosuppressive treatment itself such as cancer and severe infections. The company is of the opinion that there is a great need for a long-acting, efficient and safe treatment that induces tolerance for the transplanted organ in order to avoid the risk of organ rejection. Initially, Idogen intends to target patients about to undergo kidney transplantation with organs from living donors. With a living donor, there is often the possibility of planning the transplant well in advance and therefore also being able to plan for the production of the prophylactic treatment using Idogen’s cell therapy. The cells from the organ donor are added to the patient’s own cells together with Idogen’s proprietory tolerance inducer in conjunction with the transplantation being performed. This means that the tolerogenic cell therapy that facilitates tolerance for the tranplanted kidney can be built up in the patient in parallel with the transplantation.

Preclinical development for IDO T is ongoing with the expectation of  being able to submit an application to initiate a Phase I/IIa clinical trial during 2023.

Kidney transplants are the most common type of organ transplant and almost 80,000 kidney transplants are performed globally per year, including about 20,000 in Europe [2]. The largest and most serious complication is when the transplant recipient’s immune system attacks, destroys and rejects the donated organ. To prevent this, the transplanted patients – with few exceptions – are given lifelong treatment with a combination of drugs to suppress their immune system. While the proportion of patients who retain a functional transplanted kidney during the first year of transplantation has increased in recent decades, there has not been any improvement in the long-term survival of transplant recipients [3].

Immunosuppressive therapy also carries a risk of serious infections and cancer. Transplantation is therefore an indication with a major unmet medical need. Idogen’s tolerogenic cell therapy has the opportunity to reduce the need for immunosuppressive drugs and improve transplant survival.

Idogen’ intention is to treat patients before transplantation.

Potential market for IDOT

When the body’s immune system attacks the body’s own proteins

Idogen has also planned a third therapeutic area focused on severe and rare autoimmune diseases, IDO AID. Idogen has identified a group of autoimmune diseases where there is a major unmet medical need and a treatment could be granted orphan drug designation. Patients with autoimmune diseases are often treated for long periods of time with powerful broad-spectrum immunosuppressive drugs. However, the effect on the underlying disease is rarely optimal and at the same time, the treatment can lead to undesirable side effects in a manner similar to that in conjunction with transplantation. The medical need for improved therapies is therefore high. By drastically shortening the treatment, Idogen’s tolerogenic cell therapy could reduce the need for immunosuppressive drugs, with improvements to patient health as a result. The company is looking for partners in these fields of therapy to move further.

[1] https://www.fiercepharma.com/regulatory/roche-nabs-pair-blockbuster-fda-approvals-for-hemlibra-gazyva
[2] Global Observatory on Donation & Transplantation in collaboration with WHO.
[3] Afzali B, Taylor AL, Goldsmith DJA. What we CAN do about chronic allograft nephropathy: Role of immunosuppressive modulations. Kidney International, 2005, 68, 2429-2443.
Wang JH, Skeans MA, Israni AK. Current status of kidney transplant outcomes: dying to survive. Adv Chronic Kidney Dis, 2016, 23, 5, 281-286.

For further information or interest regarding collaborations,
please contact Acting CEO Christina Herder, christina.herder[at]idogen.com