PRESS RELEASE

Biotest achieves another important clinical milestone for IgG Next Generation

• Phase III study in Primary Immunodeficiency Disease (PID) successfully completed
• IgG Next Generation meets all clinical endpoints
• Results will serve as basis for worldwide approval

Dreieich, 1 September 2020. Biotest AG today announced the completion of study no. 991 - a phase III study investigating IgG Next Generation as replacement therapy in patients diagnosed with primary immunodeficiency disease (PID), a group of disorders in which a part of the body's immune system is missing or does not function properly.

In the study, 67 patients were treated, including 18 children. The last patient completed the study beginning of April 2020. Biotest is currently finalizing the clinical study report.
Study no. 991 examined the efficacy and safety of IgG Next Generation. The study has met its primary and secondary endpoints in patients with PID and the treatment was well tolerated by all age groups. The treatment emergent adverse events observed in the study are in line with those known for other IVIGs with regard to both type, severity, and frequency. The overall benefit-risk profile of IgG Next Generation was favorable in all age groups assessed.

"We are very pleased to have successfully completed this milestone in the development plan for our new intravenous immunoglobulin and that the results with IgG Next Generation have met the EMA/FDA guideline requirements for both safety and efficacy," said Dr Silke Aigner, responsible Head of Clinical Strategy & Development for IgG Next Generation. "This is the second pivotal clinical study generating positive phase III results. In 2019, we completed study no. 992 - a phase III study investigating IgG Next Generation as immunomodulatory therapy in patients diagnosed with chronic primary immune thrombocytopenia (ITP)".

Results of both studies will be submitted together to the health authorities. Following approval, IgG Next Generation can be marketed in additional established indications of intravenous immunoglobulins, in particular replacement therapy in secondary immunodeficiency disease (e.g. chronic lymphocytic leukaemia, multiple myeloma, after allogeneic haematopoietic stem cell transplantation) and as immunomodulatory therapy in Guillain Barré Syndrome (GBS), Kawasaki disease, Multifocal motor neuropathy (MMN) and Chronic inflammatory demyelinating polyneuropathy (CIDP).

In 2019, the market volume of immunoglobulins solely for the indication PID in US and EU amounted to approximately $3.0 billion.

IgG Next Generation is manufactured using an innovative production process and will be the master product of the new Biotest Next Level manufacturing facility currently under construction. This new manufacturing facility, which uses state of the art technologies, represents Biotest's latest commitment to the global immunoglobulin markets.

About study no. 991
Study no. 991 is a phase III, open-label, prospective, multicentre trial and will investigate the clinical efficacy, safety, and pharmacokinetic properties of IgG Next Generation as replacement therapy in paediatric and adult patients diagnosed with primary immunodeficiency disease (PID). Patients previously receiving IVIg (immunoglobulins for intravenous use) treatment are switched to infusions with IgG Next Generation and treated for 12 months. The initial dose and dosage intervals will be kept consistent with the patient's pre-study IVIg treatment. The primary objective of this study is to demonstrate that IgG Next Generation provides effective protection against infection by supplementing and maintaining a normal level of immunoglobulin in the patients' body. According to EMA/FDA guidelines on the clinical investigation of immunoglobulin, the rate of acute serious bacterial infections (SBI) per patient year should be less than one to provide substantial evidence of efficacy. An SBI is defined as bacterial pneumonia, bacteraemia and septicaemia, osteomyelitis/septic arthritis, bacterial meningitis or visceral abscess. More information about the study design can be found at (ClinicalTrials.gov Identifier: NCT02810444).

About Primary Immunodeficiency Disease (PID)
Primary Immunodeficiency Diseases are a group of currently 200 identified hereditary and genetic disorders, in which some of the body's immune defences are absent or dysfunctional. It is estimated that worldwide at least 10 million children and adults may be affected by PID. One of the most common signs of PID is an increased susceptibility to a wide range of germs like bacteria, viruses and fungi that can cause infections. Infections in patients diagnosed with PID are more frequent, longer lasting or harder to treat than are the infections of someone with a normal immune system. Patients with PID typically require life-long immunoglobulin replacement therapy to prevent recurrent infections.

About Biotest
Biotest is a provider of plasma proteins and biological drugs. With a value added chain that extends from pre-clinical and clinical development to worldwide sales, Biotest has specialised primarily in the areas of clinical immunology, haematology and intensive care medicine. Biotest develops and markets immunoglobulins, coagulation factors and albumin based on human blood plasma. These are used for diseases of the immune and haematopoietic systems. Biotest has more than 1,800 employees worldwide. The ordinary and preference shares of Biotest AG are listed in the Prime Standard on the German stock exchange.

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