BOOST Pharma

Treating the untreatable

BOOST Pharma awarded RPD

On October 14th 2022, FDA awarded the Rare Paediatric Disease (RPD) designation to BOOST Pharma for its novel cell therapy for the treatment of children suffering from Osteogenesis Imperfecta (OI). OI is a serious rare genetic disease that can lead to severe physical and functional disability, and for which there is currently no approved therapy. The RPD designation allows BOOST Pharma to receive a priority review voucher (at the time of marketing authorization) that can expedite product approval. RPD designation follows the recently awarded Orphan Drug Designation in both Europe and USA.

October 2022

BOOST Pharma at OIFE's Annual Educational Webinar

Dr Cecilia Götherström, Associate Professor in Stem Cell Science at Karolinska Institute and CSO of B​OOST Pharma, is presenting at at the OIFE’s Annual Educational Webinar, giving an update on the ongoing BOOSTB4 clinical trial.

June 2022

Orphan Drug Designation in the US

BOOST Pharma receives U.S. FDA Orphan Drug Designation for its innovative therapy to treat Osteogenesis Imperfecta.

May 2022

Leadership and clinical update

 

Evert Kueppers appointed CEO and co-founder Dr Lilian Walther Jallow transitioned to Chief Development Officer. Significant progress seen for ongoing BOOSTB4 clinical study.

February 2022

 

Orphan Drug Designation for OI

BOOST Pharma receives an Orphan Drug Designation (ODD) for Osteogenesis Imperfecta (OI) in Europe,  strengthening the  company’s position in the OI field.

December 2021

BOOST Pharma receives Innobooster Grant

BOOST Pharma have received an additional €200K funding from the the Danish Innovation Foundation in the form of an Innobooster Grant. The funding is dedicated to develop the IP around BOOST Cells, stem cells with unique bone forming capabilities.

March 2021

Additional funding for BOOST Pharma

BOOST Pharma have received an additional €800K funding from the Danish Growth Foundation (Vækstfonden). This funding will allow  BOOST Pharma to strengthen CMC development and manufacturing of the bone forming BOOST Cells.

February 2021

PreSeed funding received from BII

BOOST Pharma has been  granted PreSeed funding from BioInnovation Institute in Copenhagen. The funding of €470K will facilitate the establishment of BOOST Pharma as an active player in the development of Stem Cell Therapies. The funding will be used to increase the team behind BOOST Pharma and further develop BOOST Cell treatment.

November 2020

BOOST Pharma – Treating the untreatable

BOOST Pharma is based on many years of collaborative research from Karolinska Institute in Stockholm with the focus on a novel cell therapy treatment for Osteogenesis Imperfecta. The research teams of associate professor Cecilia Götherström and professor Magnus Westgren have shown that that treatment with BOOST Cells greatly enhanced the quality of life for patients suffering from this otherwise extremely debilitating disease. 

Osteogenesis Imperfecta

Brittle Bone Disease

The greatest wish of every parent, every family, is to have their children grow up with a bright future, the ability to pursue their dreams, and life in perfect, or at the very least modest, health.

Unfortunately, for parents with children suffering from Osteogenesis Imperfecta, this is not the case. This devastating inherited disease, which is usually abbreviated as OI, leaves patients bound to a life filled with pain, fear, and disability. This disease is due to mutations, often in the collagen gene, that cause collagen to either not be present in sufficient amounts or not be of sufficient quality.

Patients have:

  • Very fragile bones.
  • Experience constant fractures and breakage of their bones.
  • Deformities of long bones, spinal curvature, and deformed ribs.

There are very few therapies available and those that exist, such as physiotherapy, rodding surgery, and bisphosphates (BPs), are merely palliative and fail to reduce the frequency of fractures. Generally, OI sufferers have an almost normal life span with severe disabilities due to bone defects and hundreds of painful bone fractures, even during fetal life, causing irreversible damage.

OI affects more than 56.000 people in the United States of America (USA) and the European Union (EU) with an annual incidence rate of 1.000 newborns globally.

There is no cure, no treatment.

BOOST Pharma was founded based science of the Karolinska Institutet, which is a world-leader in cell therapy research. BOOST Pharma is developing a cell therapy treatment for Osteogenesis Imperfecta (OI) using mesenchymal stem cells (MSC).

Kid in blue shirt-unsplash

Boost Cell Therapy

Technology

BOOST Pharma is developing a first-in-class therapy to treat Osteogenesis Imperfecta. This treatment is based on novel Mesenchymal Stem Cells (MSC), with high bone-forming capabilities – dubbed BOOST Cells. 

In mice models, BOOST Cells has shown that cell therapy leads to higher calcium deposition, higher alkaline phosphatase activity, and a high ectopic bone formation.

Once injected, these BOOST Cells will migrate to the bone of patients with OI where they will engraft, and start bone formation. BOOST Pharma has human proof-of-concept studies from four children with Type III and IV that were treated with BOOST Cell therapy.

This treatment showed great promise in the effectiveness of treating children with OI; the children followed their own growth curve, they had increased lengthwise growth compared to contemporary OI patients, and a significant reduction of fractures was observed. The cells showed great safety, with no adverse reactions and no immune responses towards the donor MSC.

BOOST Cell Therapy start already at the prenatal stage, when OI is first diagnosed, or as early as possible after the child is born. By treating this early, BOOST Pharma are addressing the disease at the earliest possible stage and increasing the benefits for the patient in later years. The boost cells are given as one prenatal infusion dose. Postnatally, treatment schemes will vary from patient to patient, but a typical treatment scheme will involve postnatal infusions of BOOST Cells every 4 month during childhood and early adolescence. Patients will be monitored to see if they follow their normal growth curve and monitored for fracture rates.

BOOST Pharma major milestones

The next steps for BOOST Pharma are to further develop our treatment cell therapy in the ongoing clinical trials sponsored by Karolinska Institute in Sweden, prepare for a pivotal trial, and upscale our manufacturing capabilities and develop our CMC package. We have ongoing fundraising activities to support these developments.

 

Key focus areas

  • Finalizing European BOOSTB4 trial
  • Finalizing Indian BOOST2B trial
  • Obtaining orphan drug designation for OI in US
  • Upscaling manufacturing capabilities
  • Start of pivotal clinical trial

Clinical Trials

The two ongoing clinical studies – one in Europe (BOOSTB4) and one in India (BOOST2B), are funded under a Horizon 2020 academic grant, Vinnova, and the Swedish Research Council. Both of these clinical trials started in the first half of 2020, with expected positive outcomes in 2022.

The BOOSTB4 phase I/II clinical trial is a multicentre, open-label, and multiple doses for safety and efficacy of postnatal or pre-and/or postnatal dosing. The treatment regime will be four doses of allogeneic MSC every 4th month during the duration of the trials. The inclusion age is ≤18 months. For the non-treatment arm, we will use historical and untreated perspective controls. The BOOST2B phase I/II clinical trial is a single-center, open-label, and multiple doses for safety and efficacy postnatal dosing. In this trial, older children will be treated (1-4 years of age). We will also examine another type of administration; direct injection into the bone, in addition to intravenous injection.

These studies will ensure the investigation of the safety and efficacy of multiple (dosing) to show a clear clinical effect of BOOST Cells on bone development. Additional safety will be assessed to support findings from the human proof-of-concept case studies as well as the effect. Evaluation of the safety and effect of prenatal treatment will also be determined.

Click Logo for European study: BOOSTB4

Click Logo for India study: BOOST2B

Executive Team

Evert Küppers

Evert Küppers

CEO - MBA & M.Sc. Biomedical Sciences

 

Serial biotech CEO ao. Pieris Pharmaceuticals (NASDAQ: PIRS)

Lasse Nørregaard

Lasse Nørregaard

CBO - MBA & M.Sc. Biotechnology

 

 Experienced Bioentrepreneur & Life Science Consultant

Lilian Walther Jallow

Lilian Walther Jallow

Co-founder & CDO - PhD

 

Project manager of Drug Development and Advanced Therapy Medicinal Products, CMC/RA experience

Bo Danner Henriksen

Bo Danner Henriksen

CFO - BA

 

CFO/Financial Manager within the Biotech and Life science industry

Cecilia Götherström

Cecilia Götherström

Co-founder and CSO - PhD

 

Research Leader and Associate Professor in Stem Cell Science at Karolinska Institutet. Manages the BOOSTB4 consortium and trial.

Board of Directors 

Ingelise Saunders

Ingelise Saunders

Chairman of BoD and Strategic Advisor - M.Sc. Pharm.

 

More than 35 years experience from biotech and pharmaceutical industry at C-level positions as well as chairman/member of boards both in SME and large pharma companies

Magnus Westgren

Magnus Westgren

Co-founder and member of BoD and of SAB - MD & PhD

 

Senior Professor at Karolinska Institutet. Board Member in several biotech companies. Established fetal therapies in Sweden and performed the first prenatal MSC transplantation in the world.

Hervør Lykke Olsen

Hervør Lykke Olsen

Observer, PhD

 

Representative of BioInnovation Institute on the Board and gives input on strategic, scientific and commercial strategy. 

Scientific Advisors

Magnus Westgren

Magnus Westgren

Co-founder and member of BoD and of SAB - MD & PhD

 

Senior Professor at Karolinska Institutet. Board Member in several biotech companies. Established fetal therapies in Sweden and performed the first prenatal MSC transplantation in the world

Jörg Oliver Semler

Jörg Oliver Semler

Member of SAB - MD & PhD

 

One of the world’s foremost OI experts. Head of the Department of Skeletal Dysplasias and associate professor of pediatrics at University Hospital in Cologne. Chair of the medical advisory board of the national and the European patient organization for OI

Willem E. Fibbe

Willem E. Fibbe

Member of SAB - MD & PhD

 

Chairman of the Department of Immunohematology and Blood Transfusion at Leiden University. An expert within Stem Cell Therapeies and has vast experience with clinical program using Advanced Therapy Medicinal Products (ATMP) for a variety of clinical indications 

General

Publications

Docs try new stem cell therapy to help children with ‘brittle bones’

Chennai: When Devi’s baby was just 45 days old, he fractured ..

Svenska Dagbladet, SvD, article about the first child treated in the European study. The article is in Swedish and requires payment.

Varje gång Tellef börjar gråta får hans mamma en klump i magen. Har han brutit något? Föräldrarna uppmanades att ta farväl när sonen föddes med en ovanlig ben­sjukdom och knappt kunde andas.

Scientific

Publications

Clinical trial article on treatment in children with OI:

Götherström C, Westgren M, et.al Pre- and postnatal transplantation of fetal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience. Stem Cells Transl Med. 2014 Feb;3(2):255-64. doi: 10.5966/sctm.2013-0090. Epub 2013 Dec 16. PMID: 24342908; PMCID: PMC3925052.

Case report on stem cell engraftment in OI patient:

Le Blanc, Katarina et al. Fetal mesenchymal stem-cell engraftment in bone after in utero transplantation in a patient with severe osteogenesis imperfecta. Transplantation vol. 79,11 (2005): 1607-14. doi:10.1097/01.tp.0000159029.48678.93

Review article on prenatal stem cell therapies:

Ekblad-Nordberg Å, Walther-Jallow L, Westgren M, Götherström C. Prenatal stem cell therapy for inherited diseases: Past, present, and future treatment strategies. Stem Cells Transl Med. 2020;9(2):148-157. doi:10.1002/sctm.19-0107

Review article on stem cell therapy for OI:

Götherström C, Walther-Jallow L. Stem Cell Therapy as a Treatment for Osteogenesis Imperfecta. Curr Osteoporos Rep. 2020 Aug;18(4):337-343. doi: 10.1007/s11914-020-00594-3. PMID: 32710427; PMCID: PMC7419362.

About BOOST Pharma

BOOST Pharma is based on years of collaborative research from Karolinska Institute in Stockholm with a focus on novel cell therapy treatments for Osteogenesis Imperfecta.

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