REGULATED INFORMATION
New clinical studies for JETREA® confirm positive OASIS results: 28 days post injection resolution rates ranging from around 40% to 58%
New JETREA® ready-diluted formulation received final EU approval
Philippe Vlerick nominated Non-Executive Director ThromboGenics NV
Highlights
JETREA® in the US
- ThromboGenics reported positive top-line results from the OASIS study. This randomized controlled study met its primary endpoint with 41.7% of patients treated with JETREA® achieving VMA resolution at day 28 post injection compared with only 6.2% of patients who received a sham injection (p<0.001). This was a positive result which is in line with the JETREA® real-world experiences of the retina community and more favourable than the results of the pivotal Phase III program where VMA resolution was seen in only 26.5% of patients. The study also showed that the JETREA® safety profile in this 24 month follow-up study was consistent with the drug's overall safety profile as known from the approved label
- ThromboGenics, and its partner Alcon, presented a number of new data posters on JETREA® at the ARVO (The Association for Research in Vision and Ophthalmology) meeting in Denver May 3-7. The majority of these posters showed additional positive real world data on JETREA®, which provided further important insights into the value that this novel medicine provides, reinforcing the findings of the OASIS study
- ThromboGenics' sales team is focused on key accounts. These accounts comprise retina specialists who have already achieved experience and satisfactory clinical or real-world results with JETREA®. This approach is designed to allow ThromboGenics to capitalize on the real-world data that has and will continue to emerge during the course of 2015 to drive the adoption of JETREA®
JETREA® outside the US
- A new ready-diluted formulation of JETREA® (ocriplasmin), which is easier to use as it eliminates a dilution step in the product's preparation by physicians, received a positive opinion from the CHMP in Europe in March. This new formulation gained its final EU approval at the end of April.
- ThromboGenics' partner Alcon continues to gain new marketing and reimbursement approvals and to execute commercial launches of JETREA® across Europe and Rest of the World (RoW).
- JETREA® is now approved in 52 countries globally. Patients are being treated and reimbursed in over 20 countries
Research & Development
- ThromboGenics is on track to recruit the first patient in its Phase IIa clinical study assessing JETREA® for the treatment of diabetic retinopathy (DR) before the end of 2015. DR is the second indication that ThromboGenics is developing JETREA® for
- ThromboGenics has also announced the start of the evaluation and development of JETREA® for the treatment of Retinal Vein Occlusion (RVO). RVO is the third indication the Company will develop JETREA® for. The Company has received an IWT grant to conduct pre-clinical research related to this indication. RVO is known to be the second most common retinal vascular disease, and thought to negatively impact the quality of life of 16 million patients worldwide
Corporate
- ThromboGenics' spun out its oncology research activities into a new company Oncurious NV of which ThromboGenics is the majority shareholder. This company has been created in conjunction with the VIB (Flanders Institute for Biotechnology). This new venture is initially focusing on developing TB-403 for the treatment of medulloblastoma, the most common form of brain cancer in children
Appointments
- Dominique Vanfleteren was appointed as ThromboGenics Chief Financial Officer (CFO) in January 2015
- Emmanuèle Attout was appointed Independent Non-Executive Director of the Board of ThromboGenics NV on May 5, 2015
- Philippe Baron Vlerick was nominated as a Non-Executive Director of the Board of ThromboGenics NV. It is expected that he will be appointed by an extraordinary shareholders' assembly in June/July
Financial
- Cash and investments of €121.4 million as of the end of March 2015, compared with €127.1 million at the end of December 2014
- On May 19, Philippe Baron Vlerick informed ThromboGenics that on May 14th his ownership interest in ThromboGenics' share capital has exceeded the 3% threshold.
Leuven, Belgium - 21 May 2015 - ThromboGenics NV (Euronext Brussels: THR), an integrated biopharmaceutical company focused on developing and commercializing innovative ophthalmic medicines, today issues a business and financial update for the three months ending 31 March, 2015.
ThromboGenics developed JETREA®, the first and only pharmacological treatment indicated for an important sight-threatening condition, symptomatic vitreomacular adhesion (VMA)/vitreomacular traction (VMT) as known in the US and Europe respectively. Symptomatic VMA/VMT is a progressive, sight-threatening condition that may lead to visual distortion, decreased visual acuity and central blindness.
ThromboGenics' strategy is focused on:
- Driving the sales of JETREA® in the US
- Supporting Alcon to develop the sales of JETREA® outside the US
- Creating value by generating further real-world clinical data on the use of JETREA® in its approved indication, confirming its efficacy and safety profile.
- Evaluating and developing JETREA® in new indications: Diabetic Retinopathy & Retinal Vein Occlusion.
- Progressing its pipeline in earlier stage projects focused on developing treatments for diabetes-related vitreo-retinal diseases
Dr Patrik De Haes, ThromboGenics' CEO, said: "The positive top-line results from the OASIS study and real-world data that have been reported in recent months gives us great confidence that we can demonstrate to the broader retina community the clear benefits and value of using JETREA®. With our US sales and marketing organization now fully focused on key accounts we believe we are well placed to drive the adoption of JETREA® for the treatment of the many patients with symptomatic VMA who could benefit from pharmacological intervention.
"We are also looking to expand the medium term potential of JETREA® by evaluating and developing it for two further important retinal diseases, DR and RVO. These are two conditions where there is a clear unmet need for improved treatment options. We are convinced that by developing JETREA® for a broader range of indications we will help the global retina community to gain a deeper understanding of this novel medicine and its potential to treat a number of important vitreo-retinal diseases".
JETREA® in the US
ThromboGenics' U.S. commercial team is focusing its efforts on key retinal accounts, which are typically clinics that have successfully treated patients with JETREA® over a longer period of time (12 - 24 months). Longer term experience and new data - both efficacy and safety - are critically important in shaping a retinal physician's perspectives on this novel pharmacological treatment option for symptomatic VMA.
This longer term experience has helped those specialists build their own clinical data sets and develop a good understanding of JETREA®'s efficacy, safety and side effects. This knowledge and experience ultimately leads to improved patient selection and improved treatment outcomes and in turn to more patients being treated with this novel medicine.
ThromboGenics' commercial strategy is to build on these experiences by reaching out to the broader ophthalmic community near those retina centers which are using JETREA® on a regular basis. At the same time, ThromboGenics and its partner Alcon, are working to ensure that real world data that is being reported is appropriately communicated to the global retina community.
The generation of real world data is designed to support/reinforce the findings of a post-hoc data analysis of the Phase III program with JETREA® which has shown that certain patient characteristics, such as a focal VMA (< 1,500 µm) or the absence of an epiretinal membrane (ERM), are independently associated with successful VMA resolution.
In clinical practice, many retinal specialists have started to use these criteria to select the patients with symptomatic VMA whom they treat with JETREA®, leading to higher rates of VMA resolution.
Generating real-world JETREA® data
ThromboGenics has already and continues to generate real-world data with JETREA® as part of its commitment to ensuring that the most suitable patients with symptomatic VMA are treated with this novel medicine. In addition, many retinal practices are generating and publishing their own real-world data, leading to an increasing body of knowledge on the clinical outcomes that JETREA® can deliver.
OASIS study - Positive Top-Line Results
In April, ThromboGenics announced positive top-line results from its OASIS study "Ocriplasmin for Treatment for Symptomatic Vitreomacular Adhesion including Macular Hole" with JETREA® (ocriplasmin).
The OASIS study (n= 220) is a randomized, sham controlled, double masked study that followed patients for 24 months post injection. The study was designed to provide long term and well-controlled efficacy and safety data for JETREA® in patients being treated for symptomatic vitreomacular adhesion (sVMA).
The OASIS study is the first controlled study with JETREA® since the results of the pivotal Phase III program were announced in 2011. The study includes 24 month follow up data, the longest period patients have been studied post-treatment with this novel medicine.
The key findings of the OASIS study were as follows:
- 41.7% of patients treated with JETREA® achieved VMA resolution at Day 28 post injection compared with only 6.2% of patients who received a sham injection (p<0.001); and
- The JETREA® safety profile in this 24 month follow up study was consistent with the drug's overall safety profile as known from the approved label. No new types of safety events were identified.
The OASIS data compare favourably with the results from the pivotal Phase III program with JETREA® where VMA resolution was seen in 26.5% of patients at Day 28 post injection. In the Phase III program 10.1% of patients treated with a placebo injection achieved VMA resolution (p<0.001).
The OASIS data show the importance of improved patient selection in order to generate higher rates of VMA resolution with JETREA®. Recent real world data confirm that access to more advanced diagnostic technology, such as SD-OCT, enables retina physicians to improve patient selection. As a result they have been able to select patients with focal VMA and an absence of Epiretinal Membrane (ERM), two criteria which have been shown to lead to better treatment outcomes with JETREA®.
It is known that an ERM adversely impacts the efficacy of JETREA®. Approximately 20% of the recruited patients in the OASIS study had an epiretinal membrane (ERM) (despite it being one of the exclusion criteria), suggesting that the 41.7% overall resolution rate at day 28 post-injection could have been even higher. This underscores the message that proper patient selection will lead to better treatment outcomes.
Further analysis of all of the OASIS data, which will be interpreted with the help of retina physicians, is ongoing. The results from these analyses are planned to be shared with the retina community at the American Academy of Ophthalmology conference of November 14 - 17, 2015.
ORBIT study
In March 2014, ThromboGenics launched the "Ocriplasmin Research to Better Inform Treatment" (ORBIT) study.
This prospective, observational study is designed to assess clinical outcomes and the safety of JETREA® administered in a real-world setting for the treatment of symptomatic VMA by assessing both anatomical and functional outcomes.
The study is looking at a number of parameters including resolution of VMA, full thickness macular hole (FTMH) closure, changes in visual acuity (VA) and occurrence and time to vitrectomy. It will also monitor adverse drug reactions (ADRs) and changes from baseline in ocular signs and symptoms over time, such as metamorphopsia. These data will further characterize the efficacy and safety profile of the product.
Patients will be followed for up to 12 months following a single treatment with JETREA®. The ORBIT study is due for completion in mid-2016.
Six month data from the ORBIT study, were presented in a poster at the Association for Research in Vision and Ophthalmology (ARVO) meeting May 3-7 in Denver, Colorado.
The study showed that 58.1% of patients experienced VMT resolution within one month post treatment. The study also showed that the safety of JETREA® was consistent with the product's label and the data from the Phase III clinical trials.
More JETREA® real-world data was the subject of several scientific posters presentations that were delivered at the recent (ARVO) 2015 meeting, which took place in Denver from May 3-7.
The results outlined in these posters were consistent with those from previous real world analyses and with the top line data from the OASIS study.
The posters showed VMA resolution 28 days post injection with JETREA® ranged from around 40% to as high as 58.1% demonstrating that physicians have started using criteria such as focal VMA and an absence of ERM when selecting patients for treatment with this novel medicine.
All of the JETREA® posters that were presented at ARVO can be accessed using the following link: http://www.arvo.org/webs/am2015/abstract/166.pdf
Education of the Wider Ophthalmic Community on Symptomatic VMA
ThromboGenics has begun implementation of a new program to educate general ophthalmologists and optometrists about symptomatic VMA. Patients who first notice the symptoms of VMA often have the initial discussion about their condition with their general ophthalmologist or optometrist, and this program is intended to help these front-line eye care practitioners decide whether symptomatic VMA patients should be considered for referral to a specialist retina clinic with JETREA® experience.
A number of seminars in this program have already taken place with a total of more than 1,000 ophthalmologists and optometrists participating.
JETREA® in Europe and RoW
ThromboGenics' partner Alcon is continuing to commercialize JETREA® across Europe using a similar commercial approach to that being used by ThromboGenics in the U.S. - i.e. a focus on key retinal accounts.
In March 2015, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for a new ready-diluted formulation of JETREA®.
Meanwhile, the new JETREA® ready-diluted formulation gained final EU approval in late April.
The forthcoming introduction of the new formulation of JETREA® will eliminate the preparatory dilution steps prior to injection. At the point of administration into the eye, the strength, potency, composition and pharmaceutical form of the ready-diluted formulation remain identical to the currently available formulation after dilution.
Regulatory Update
In February, JETREA® was granted approval in Argentina, Israel and the Philippines.
In April, JETREA® was granted approval in New Zealand. This was the product's 52nd approval globally.
Research & Development Update
Diabetic Retinopathy - 2nd Indication for JETREA®
ThromboGenics remains on track to start its planned Phase IIa trial with JETREA® in diabetic retinopathy. This study is designed to assess the utility of the product in this significantly underserved patient population.
ThromboGenics intends to enroll the first patient into this study in the second half of 2015.
Retinal Vein Occlusion - 3rd Indication for JETREA®
In April, ThromboGenics announced that it would be evaluating and developing JETREA® for the treatment of retinal vein occlusion. RVO is the third indication ThromboGenics has underway for JETREA® (ocriplasmin).
With this new vitreo-retinal project, ThromboGenics aims to demonstrate the potential of using locally delivered ocriplasmin for lysing the blood clots (in the retinal veins) that are responsible for this sight threatening condition.
In support of this research, ThromboGenics has secured a €0.6 million grant from the Flemish Agency for Innovation by Science and Technology (IWT). ThromboGenics will use this grant to evaluate ocriplasmin's ability to lyse the clots that cause RVO by local intravenous administration of this thrombolytic agent in pre-clinical models of the disease. ThromboGenics will collaborate with the Ophthalmology Department of the University Hospital UZLeuven in Belgium on this study.
The IWT grant will also support a partnership with the Mechanical Engineering Department of the KU Leuven. The Department is developing a robotics-assisted system which has the ability to deliver the local administration of ocriplasmin directly in the retinal veins.
At present, there are no treatment options for clearing clots from the retinal veins of RVO-patients.
Corporate
Oncology R&D transfer into new company
In April, ThromboGenics announced the formation of Oncurious NV, a new oncology company that will develop TB-403 for the treatment of pediatric brain tumors. Oncurious is a joint venture between ThromboGenics and VIB, the leading life sciences institute in Flanders (Belgium). ThromboGenics is the majority shareholder.
TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF). PlGF is expressed in several types of cancer, including medulloblastoma. High expression of the PlGF receptor neuropilin 1 has been shown to correlate with poor overall survival. Medulloblastoma is a rare, life-threatening brain tumor that mainly affects children.
Treatment with TB-403 in relevant animal models for medulloblastoma has demonstrated beneficial effects on tumour growth and survival.
The favourable safety profile of TB-403 has already been demonstrated in clinical trials in patients with other diseases.
Oncurious will initiate a Phase I/IIa program with TB-403 in medulloblastoma patients with the first patient expected to be enrolled by the end of 2015. BioInvent International will act as a co-development partner.
Appointments
Philippe Baron Vlerick - Nominated Non-Executive Director
In May, Philippe Baron Vlerick has been nominated as a new Non-executive member of the Board of ThromboGenics nv.
It is expected that Mr Vlerick will be appointed by an extraordinary shareholders' assembly in June/July.
Philippe Vlerick is owner, Chairman and CEO of several businesses in Belgium and abroad. He currently serves as the Chairman and Chief Executive Officer of Vlerick Group (Belgium). He also serves as the Chairman and CEO of UCO Textiles NV. In addition, he is the Vice-chairman of KBC Group, Corelio, smartphoto Group and Durabilis. Baron Vlerick is also a member of the Board of Directors of Exmar, Hamon & Cie, Besix Group and I.V.C. (Belgium).
Mr Vlerick holds a Degree in Philosophy and Law from the University of Leuven, and an MBA General Management (PUB) (Ghent, Vlerick School of Management - 1979). He also holds a Masters Degree in Business Administration from Indiana University, Bloomington (USA - 1980).
He was elected 2006 Manager of the Year by Trends, a leading business magazine in Belgium. He was granted the title of Baron in 2008, and became Commander of the Order of Leopold in 2013.
Emmanuèle Attout - Appointed Non-Executive Director
In January, ThromboGenics nominated Emmanuèle Attout to be its new Independent non-executive director. Mrs. Attout was appointed during the ThromboGenics Annual Shareholders Meeting of May 5, 2015. She will also join the Audit Committee of the Company.
Mrs. Attout has been an audit partner at PricewaterhouseCoopers since 1994. She has been in charge of the audits for a wide range of clients, including in recent years being in charge of the audits of publicly listed pharmaceutical companies and life sciences businesses.
Dominique Vanfleteren - Appointed Chief Financial Officer
Dominique Vanfleteren was appointed as ThromboGenics' new Chief Financial Officer (CFO) in January 2015.
Dominique Vanfleteren has over 25 years of experience in senior finance, operational, control and reporting roles with quoted international biopharmaceutical companies. Before joining ThromboGenics, Mr. Vanfleteren spent 12 years at UCB, where he held a number of international managerial finance positions, the latest being the CFO of UCB's Asia Pacific Operations, operating from Brussels and Shanghai. Prior to joining UCB, Dominique worked for GSK for 16 years.
Financial review
At the end of March 2015, ThromboGenics had €121.4 million in cash and investments, compared with €127.1 million at the end of December 2014.
Cash usage in the first quarter was far below the forecast of €11 million per quarter partly due to positive impact of a reduction in receivables and favorable exchange rate effects which together amounted to €2.9million.
ThromboGenics believes that it has the financial resources needed to sustain the commercialization of JETREA® in the US, to research and develop new indications and formulations of JETREA® for the US market, and to expand its R&D pipeline as it delivers on its commitment to become a leading ophthalmology company.
END
For further information please contact:
| ThromboGenics Wouter Piepers, Global Head of Corporate Communications/ IR +32 16 75 13 10 / +32 478 33 56 32 wouter.piepers@thrombogenics.com | Citigate Dewe Rogerson David Dible/Malcolm Robertson Tel: +44 20 7638 9571 David.Dible@citigatedr.co.uk |
About JETREA® (ocriplasmin)
JETREA® (ocriplasmin) is a truncated form of human plasmin. JETREA® acts as a selective proteolytic enzyme that cleaves fibronectin, laminin and collagen, three major components of the vitreoretinal interface that play an important role in vitreomacular adhesion.
In the US, JETREA® is indicated for the treatment of symptomatic vitreomacular adhesion. In Europe, JETREA® is indicated for the treatment of vitreomacular traction (VMT), including when associated with macular hole of diameter less than or equal to 400 microns.
JETREA® was evaluated in two multi-center, randomized, double-masked Phase III trials conducted in the U.S. and Europe involving 652 patients with vitreomacular adhesion. Both studies met the primary endpoint of resolution of VMA at day 28. This Phase III program found that 26.5% of patients treated with ocriplasmin saw resolution of VMA, compared with 10.1% of patients receiving placebo (p<0.01). The Phase III program also showed that JETREA was generally well tolerated with most adverse events being transient and mild in severity.
In March 2015, ThromboGenics reported top line results from OASIS, a Phase IIIb study. This randomized, sham controlled, double masked study followed-up patients for 24 months post injection. In this study, retina physicians were able to use SD-OCT to select patients with focal VMA and without Epiretinal Membrane (ERM), two criteria which have been shown to lead to better treatment outcomes with JETREA®. Despite this, OASIS data showed over 20% of the patients recruited into study had ERM.
The trial showed that 41.7% of patients treated with JETREA® achieved VMA resolution at Day 28 post injection compared with only 6.2% of patients who received a sham injection (p<0.001); and that the drug's safety profile in the 24 month follow period was consistent with the drug's overall safety profile as known from the approved label.
About ThromboGenics
ThromboGenics is an integrated biopharmaceutical company focused on developing and commercializing innovative ophthalmic medicines. The Company's lead product, JETREA® (ocriplasmin) was approved by the US FDA for the treatment of symptomatic VMA and was launched by ThromboGenics in January 2013.
ThromboGenics signed a strategic partnership with Alcon, a division of Novartis, for the commercialization of JETREA® outside the United States in 2012. To-date JETREA® has been approved in over 50 countries globally.
JETREA® is currently being evaluated for two further indications, diabetic retinopathy (DR) and retinal vein occlusion (RVO).
ThromboGenics is the major shareholder in Oncurious NV, a cancer focused company that was formed in April 2015 in collaboration with VIB, a leading life science institute in Flanders (Belgium). Oncurious was created to develop TB-403, initially for medulloblastoma, the most frequent form of pediatric brain cancer. ThromboGenics has retained the exclusive rights to use TB-403 for ophthalmic indications.
ThromboGenics is headquartered in Leuven, Belgium, and has offices in Iselin, NJ (US) and Dublin, Ireland. The Company is listed on the NYSE Euronext Brussels exchange under the symbol THR. More information is available at www.thrombogenics.com.
Important information about forward-looking statements
Certain statements in this press release may be considered "forward-looking". Such forward-looking statements are based on current expectations, and, accordingly, entail and are influenced by various risks and uncertainties. The Company therefore cannot provide any assurance that such forward-looking statements will materialize and does not assume an obligation to update or revise any forward-looking statement, whether as a result of new information, future events or any other reason. Additional information concerning risks and uncertainties affecting the business and other factors that could cause actual results to differ materially from any forward-looking statement is contained in the Company's Annual Report.
This press release does not constitute an offer or invitation for the sale or purchase of securities or assets of ThromboGenics in any jurisdiction. No securities of ThromboGenics may be offered or sold within the United States without registration under the U.S. Securities Act of 1933, as amended, or in compliance with an exemption therefrom, and in accordance with any applicable U.S. state securities laws.