ThromboGenics Business Update – FY 2016

16 Mar 2017

Innovative Diabetic Eye Disease Clinical and Pre-Clinical Portfolio
Provides “Multiple Shots on Goal”

First Patients Enrolled in Phase II Clinical Study
Evaluating THR-317 (anti-PlGF) for Diabetic Macular Edema (DME)

Cash Position - €80.1 Million at end of December 2016

Highlights

ThromboGenics continues to advance its attractive portfolio of innovative medicines for the treatment of diabetic eye disease: 4 novel treatments for diabetic retinopathy (DR) - non-proliferative diabetic retinopathy (NPDR) and proliferative diabetic retinopathy (PDR), in the presence or absence of diabetic macular edema (DME).

Clinical Update

Phase II study: THR-409 (Ocriplasmin) for Non Proliferative Diabetic Retinopathy (CIRCLE)

  • The Phase II study (CIRCLE) is assessing the ability of multiple doses of THR-409 to induce a total PVD in patients with NPDR.
  • CIRCLE will also explore whether generating a total PVD in patients with non-proliferative diabetic retinopathy could prevent the disease from progressing to proliferative diabetic retinopathy, a serious sight threatening condition.
  • In December, the CIRCLE study protocol was amended to allow inclusion of patients with less severe non-proliferative diabetic retinopathy. This change was made to broaden the patient pool for this study to recruit from.

Phase II study: THR-317 (anti-PlGF) for Diabetic Macular Edema (DME)

  • A phase II study evaluating the safety and efficacy of 3 intravitreal injections of 2 dose levels of THR-317 (4 mg or 8 mg) recruited its first patients in January 2017. The trial is assessing THR-317’s ability to improve best-corrected visual acuity (BCVA) and to reduce central retinal thickness in subjects with DME.
  • The study aims at enrolling a total of 50 patients (including 10 anti-VEGF treatment resistant patients) over a period of about 12 months. The first results from the study are expected in Q1 2018.
  • ThromboGenics believes THR-317 could be used as a stand-alone therapy or as an add-on treatment to anti-VEGF medicines, for the treatment of DME or DR.

Phase I/II clinical trials in the works: THR-687 and THR- 149

Pre-clinical development of THR-687 and THR-149 is making good progress

  • THR-687 is being developed to treat a broad range of patients with diabetic retinopathy, with or without DME. The Company plans to start a Phase I/II clinical trial around end of 2017
  • THR-149 is being developed to treat edema associated with diabetic retinopathy and a Phase I/II clinical trial is expected to start in 2018.

Oncurious NV

  • Oncurious and its development partner BioInvent International signed a partnership agreement with the Neuroblastoma and Medulloblastoma Translational Research Center (NMTRC) for the clinical development of TB-403 for the treatment of medulloblastoma in the US in March 2016.
  • A Phase I/IIa study was initiated with TB-403 in May 2016. The study aims to recruit 27 patients with Relapsed or Refractory Medulloblastoma.
  • In January 2017, the European Commission confirmed orphan drug designation for TB-403 for medulloblastoma following a positive opinion issued by the European Medicine Agency (EMA).

Financial

  • ThromboGenics generated overall revenues of €7.1 million in 2016. This includes a €2.5 million in royalty income.
  • ThromboGenics US business achieved its goal of an operational break even at the end of 2016.
  • In order to align the JETREA® intangibles and goodwill value with the continued lower than expected sales levels, the Company has decided to take an impairment charge of €26.6 million. This charge has no impact on the Company’s cash position.
  • Cash and investments were €80.1 million as of the end of December 2016, compared with €101.4 million at the end of December 2015.

 

Leuven, Belgium – 16 March 2017 - ThromboGenics NV (Euronext Brussels: THR), an integrated biopharmaceutical company, focused on developing novel medicines for diabetic eye disease, today issues a business update and its financial update for the year ending December 31, 2016.

ThromboGenics is developing novel medicines for diabetic eye disease, diabetic retinopathy (DR) with or without diabetic macular edema (DME).

ThromboGenics’ innovative pipeline targeting diabetic eye disease, which is one of the strongest in the industry, includes:

THR-409 – an ongoing Phase IIa (CIRCLE) clinical study is evaluating the efficacy and safety of multiple doses of ocriplasmin in inducing total posterior vitreous detachment (PVD) in patients with non-proliferative diabetic retinopathy (NPDR).  

THR-317 – a PLGF neutralizing monoclonal antibody is being developed for DME and/or for use in combination therapy with current anti-VEGF drugs for the treatments of DME or DR. The Company is currently enrolling patients in a Phase II clinical study.

THR-687 – a small molecule integrin antagonist being developed to treat a broad range of patients with diabetic retinopathy, with or without DME. The Company is preparing for the start of a Phase I/II clinical trial around end of 2017

THR-149 – a plasma kallikrein inhibitor is being developed to treat edema associated with diabetic retinopathy. Company is preparing for a Phase I/II clinical trial to start in 2018

In addition, ThromboGenics, through its oncology subsidiary Oncurious NV, is conducting a Phase I/IIa clinical trial in children assessing TB-403 for the treatment of medullablastoma, a pediatric brain tumor.

 

Dr. Patrik De Haes, ThromboGenics’ CEO, said: “We are happy with the progress we are making with our pipeline of potential new disease modifying medicines for the treatment of diabetic eye disease. Diabetic Retinopathy and Diabetic Macular Edema (DME) are significant indications where there are clear unmet medical needs. THR-317 (anti-PlGF) in a Phase II study to assess its ability to improve best-corrected visual acuity (BCVA) and to reduce central retinal thickness in subjects with DME. We are also conducting the CIRCLE study, a Phase II clinical trial to assess THR-409 as a potential treatment to reduce the risk of patients with NPDR developing sight-threatening PDR. We are also making excellent progress with our pre-clinical pipeline, including 2 molecules which could be in the clinic by next year.

With our current cash resources of around €80 million, we can support our Company’s development activities for the foreseeable future, allowing us to demonstrate the value of our exciting pipeline of innovative drug candidates.”