A concerted collaboration to advance treatment options for people living with rare diseases

Advancing our understanding of rare diseases

As we focus on developing treatment options in conditions where Hansa’s technology may help bring meaningful benefit to people living with rare diseases, we see strong value in collaborating with researchers and institutions at the forefront of scientific advancement through investigator-initiated studies. By advancing the knowledge on rare diseases, we can reach a better understanding of the potential added value of our therapies in different therapeutic areas.

In anti-glomerular basement membrane (anti-GBM) disease, an acute and very severe inflammatory disease, an open-label Phase 2 investigator-initiated study (GOOD-IDES-01) led by Professor Mårten Segelmark at Linköping University highlighted new key insights on the correlation between the inactivation of IgG autoantibodies in patients experiencing an acute autoimmune attack and the improved outcomes and kidney function.¹³ Hansa Biopharma is now following up on the encouraging evidence with an ongoing pivotal Phase 3 trial (GOOD-IDES-02).

"Our collaboration with Mårten Segelmark and all the participating investigators on GOOD-IDES-01 was instrumental to understand how we could bring added benefit to treatment strategies" said Elisabeth Sonesson, Global Franchise Lead, Autoimmune, Hansa Biopharma. "Now, our phase 3 trial GOOD-IDES-02 is the first and currently only pivotal randomized trial in anti-GBM disease, remarking the need to bring innovation in the treatment of this debilitating disease."

Similarly, Hansa’s first-generation enzyme is now being investigated in an investigator-initiated phase 2 study in anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, a group of conditions with a high unmet need, where one in four patients progresses to end stage kidney disease and respiratory failure in worst cases.^14,15

Bringing new opportunities to more patients

By teaming up with other industry players focusing on bringing new treatment options to patients, we can contribute to the evolving field of rare disease therapies, as the opportunities for treatment access also evolve.

For people living with rare, monogenic diseases, gene therapies may offer a cure to a life-long condition by introducing genetic material that compensates for a defective gene. However, for therapies based on the use of modified Adeno Associated Viruses (AAV) vectors, a challenge is posed by the presence of pre-formed antibodies targeting the AAV vectors and currently prevent up to 1 in 3 people from benefiting from these treatments.^16-20

We are frequently exposed to wild-type AAVs as they are the cause of many common infections. Many people, therefore, develop antibodies following a viral infection with AAVs, and these antibodies become part of their immune response. If the person then receives a gene therapy based on the same type of AAV vector, the anti-AAV antibodies trigger an immune response against the viral vectors that prevents the gene therapy to enter the body cells and deliver the genetic material.

Sarepta Therapeutics is partnering with Hansa Biopharma on preclinical and clinical programs to advance the use Hansa’s enzyme as a pre-treatment to their gene therapies in Duchenne Muscular Dystrophy and in Limb-Girdle Muscular Dystrophy. The combination of the gene therapy and Hansa’s enzymes can expand access to Sarepta’s treatments to patients carrying anti-AAV antibodies.

Similarly, in the spring of 2023, Hansa formed a partnership with Généthon, a leading gene therapy research non-profit organization. Hansa and Généthon are conducting a study to enable gene therapy treatment in patients with Crigler-Najjar syndrome and pre-formed antibodies towards the AAV-based gene therapy. Crigler-Najjar syndrome is a genetic disease leading to bilirubin accumulation in the body and subsequent organ damage.

"There are growing opportunities for industry players to work together and advance the science and understanding of rare disease treatments. Our primary focus is patients, and our priority is ensuring we can enable all patients, including those with anti-AAV antibodies, to receive innovative treatments" said Lena Winstedt, Global Franchise Lead, Gene Therapy, Hansa Biopharma.

Ensuring access to innovation for all those in need

At Hansa we aspire to be a leader in treating rare immunologic diseases by delivering lifesaving and life-altering medicines to people with high medical unmet need. We take a multi-pronged approach to ensuring we work together with a constellation of stakeholders – patients, clinicians, industry players and more –   to create new opportunities for people living with rare diseases, faster.

We know that delivering innovative treatments is not enough. We must collaborate with a spectrum of stakeholders including clinical, research, payer and patient community members to quickly discover, develop and make innovative new medicines available.