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UCB Media Room: Final Phase II Results for Rozanolixizumab in ITP Published in Blood Advances


[09/09/2020 | 07:03]

Final Phase I=I Results for UCB=E2=80=99s Rozanolixizumab in Primary Immune Thrombocytope=nia (ITP) Published in Blood Advances

  • Phase II data demonstrated clinically meaningful pl=atelet count increases with meaningful decreases in IgG concentration =
  • Rozanolixizumab=E2=80=99s subcutaneous route of adm=inistration shows potential to deliver targeted individualized patient care=for people living with Primary Immune Thrombocytopenia

<=span style=3D"font-size:12pt">Brussels, Belgium: 9th Sep=tember 2020, 07:00 CEST =E2=80=93 UCB, a global=biopharmaceutical company, today announced positive results from a Phase I=I study (TP0001; NCT02718716) of its investigational treatment, rozanolixiz=umab, the first subcutaneously infused monoclonal FcRn antibody being inves=tigated for patients with primary immune thrombocytopenia (ITP). The result=s were published in the September online issue of Blood Advances.<=sup>1 

ITP is a rare, often chronic autoimmune disease that is chara=cterized by unpredictable and debilitating symptoms, including spontaneous =bruising, bleeding and extreme fatigue, that can greatly impact patients=E2==80=99 activities of daily life.2 

=E2=80=9CPatients are at the core of everything we do at =UCB, and through the scientific discovery of novel therapies like rozanolix=izumab, we are dedicated to developing new solutions that can help improve =health outcomes for people living with primary ITP and other rare, IgG auto=antibody-mediated diseases,=E2=80=9D said Dr. Iris Loew-Friedrich, Chi=ef Medical Officer, Executive Vice-President, UCB. =E2=80=9CThese resul=ts are a critical step forward for rozanolixizumab, which has one of the la=rgest clinical trial programmes worldwide in ITP.=E2=80=9D 

In this study1, clinically relevant improvements i=n platelet count (i.e., reaching =E2=89=A550x109/L) and meaningful decrease=s in immunoglobin G (IgG) levels were observed across all dose groups, with=advantages seen in the single-dose cohorts (15 and 20 mg/kg) compared with=the multiple-dose cohorts (5x4, 3x7 and 2x10 mg/kg weekly, cumulative dose=of approximately 20mg/kg). Specifically, platelet counts of =E2=89=A550x10=9/L were achieved by more patients following  a single infu=sion of  15 or 20 mg/kg: (66.7% and 54.5% patients, respectively) vs m=ultiple infusions to achieve an approximate cumulative dose of 20mg/kg &nbs=p;(5x4, 3x7, or 2x10 mg/kg: 35.7%, 35.7% and 45.5% patients, respectively),=and occurred more rapidly in single-dose cohorts. Minimum mean IgG occurre=d by Day 8 in higher (15 and 20 mg/kg) single dose cohorts, and by Day =E2==89=A515 in multiple dose cohorts. The subcutaneous administration of rozan=olixizumab demonstrated a generally tolerated safety profile across all rep=orted dose groups,1 consistent with other rozanolixizumab studie=s.3,4 The most commonly reported adverse event was mild to moder=ate headache, with highest occurrence in the 20mg/kg cohort; other reported=adverse events included diarrhea and vomiting, the latter only observed in=single dose cohorts. All were managed with standard medication (if require=d) of short duration, and all resolved without clinical sequelae. No patien=t discontinued the study due to side effects.1

=E2=80=9CPeople who have primary ITP may experience low p=latelet count that puts them at risk for severe bleeding, and there are lim=ited options to reduce this risk,=E2=80=9D said Professor Tadeusz Roba=k, Professor of Hematology at the Medical University of Lodz, Poland. =E2==80=9CNew treatment options for ITP that have the potential to provide =improvement in platelet count are urgently needed, and I am encouraged by t=he results in this phase 2 study, which is now being tested also in a chron=ic use program in phase 3.=E2=80=9D

Current treatment options for people with ITP are limited and=can be time-consuming and invasive. There continues to be a need for new t=reatment options that can improve patients=E2=80=99 health outcomes and qua=lity of life. Rozanolixizumab is an investigational, advanced SC anti-neona=tal Fc receptor (FcRn) therapy that has the potential to provide targeted i=ndividualized patient care. 

=E2=80=9CThese data build on the growing body of evidence=that targeting the FcRn pathway has the potential to treat people with rar=e IgG autoantibody-mediated diseases such as primary ITP=E2=80=9D, sai=d James Bussel*, MD, professor emeritus of pediatrics at Weill Cornell Medi=cine. =E2=80=9CPublication of these findings in Blood Advances encourag=es us to continue to deepen our understanding of primary ITP and the ways r=ozanolixizumab may help treat people living with this disease=E2=80=9D=. 

About the rozanolixizumab clinical study
TP0001 (NCT02718716) is a Phase II, multi-center, open-label, multiple-dose=study of rozanolixizumab in adult patients with persistent/chronic primary=ITP. Sixty-six patients were assigned to one of five groups with different=dosing regimens (5 x 4 mg/kg, 3 x 7 mg/kg, 2 x 10 mg/kg, 1 x 15 mg/kg or 1=x 20 mg/kg; multiple doses were administered at weekly intervals), receivi=ng rozanolixizumab by SC infusion. All patients were monitored for an 8-wee=k observation period after completion of treatment. The primary objective o=f the study assessed safety and tolerability of subcutaneous rozanolixizuma=b infusion in patients with persistent/chronic primary ITP, and the seconda=ry objective considered the clinical efficacy (platelet count) and pharmaco=dynamic (total IgG) effects. The study was designed to explore a range of t=herapeutic doses in order to develop an appropriate dosing regimen for pati=ents with ITP. 

Rozanolixizumab was generally tolerated across all dose group=s (4=E2=80=9320 mg/kg) with mild-to-moderate headaches seen at higher doses=; no patient discontinued the study due to side effects.
In the study, clinically relevant improvements in platelet count (to =E2=89==A550x109/L) were observed in patients with primary ITP receivin=g rozanolixizumab across all dose groups as were decreases in serum IgG con=centration. More patients receiving a single, higher-dose infusion achieved=platelet counts of =E2=89=A550x109/L at least once at any time (66.7% and =54.5% in the 1 x 15 mg/kg and 1 x 20 mg/kg dose groups, respectively) compa=red with patients in the multiple-dose cohorts (35.7%, 35.7% and 45.5% in t=he 5 x 4 mg/kg, 3 x 7 mg/kg, and 2 x 10 mg/kg groups, respectively). Minimu=m mean IgG levels and maximum mean platelet counts both occurred by Day 8 i=n the higher (15 and 20 mg/kg) single dose cohorts, and maximum mean platel=et count occurred from day 11 onwards in the multiple dose cohorts. 

About primary immune thrombocytopenia =;
Primary ITP is an acquired autoimmune disorder characterized, in most cases=, by the presence of pathogenic IgG autoantibodies, with an estimated preva=lence of approximately 10 people per 100,000 (USA).5 Pathogenic =IgG autoantibodies target platelets and megakaryocytes (platelet precursors=), leading to the removal and destruction of both circulating and newly for=med platelets6,7,8 ,ultimately resulting in a propensity for ble=eding in patients with ITP. The standard of care for patients with newly di=agnosed ITP consists of corticosteroids or intravenous immunoglobulin (IVIg=).9 Patients intolerant to corticosteroids or with contraindicat=ions are treated with IVIg or anti-D (where appropriate). Subsequent treatm=ents include thrombopoietin receptor agonists,  rituximab, immunosuppr=essive agents or splenectomy.10     

About rozanolixizumab <=/strong>
Rozanolixizumab is a subcutaneously administered, humanized monoclonal anti=body that specifically binds, with high affinity, to human FcRn. It has bee=n designed to block the interaction of FcRn and IgG, inhibiting IgG recycli=ng and inducing the removal of pathogenic IgG autoantibodies.1,11 

Rozanolixizumab is under clinical development with the aim of=improving the lives of people with pathogenic IgG-autoantibody-driven auto=immune diseases, including ITP, myasthenia gravis (MG) and chronic inflamma=tory demyelinating polyneuropathy (CIDP), by driving removal of pathogenic =IgG autoantibodies. 

Rozanolixizumab, an investigational monoclonal antibody, was =granted orphan drug designation for the treatment of ITP by the US Food and=Drug Administration on 30 April 2018 and by the European Commission on 11 =January 2019.12,13 The safety and efficacy of rozanolixizumab ha=s not been established; it is not currently approved by any regulatory auth=ority worldwide.

About UCB in Rare Diseases
At UCB, we don=E2=80=99t just see patients or population sizes, we see peop=le in need. Through decades of serving the neurology and immunology communi=ties, we have improved lives with impactful medicines and by enhancing the =social and emotional well-being of patients. As a continuation of our herit=age, we are now expanding our efforts to tackle rare neurological and immun=ologic diseases where current options offer little hope, including investig=ational treatments for primary immune thrombocytopenia (ITP), myasthenia gr=avis (MG), chronic inflammatory demyelinating polyneuropathy (CIDP) and pro=gressive supranuclear palsy (PSP).

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company =focused on the discovery and development of innovative medicines and soluti=ons to transform the lives of people living with severe diseases of the imm=une system or of the central nervous system. With 7,600 people in approxima=tely 40 countries, the company generated revenue of =E2=82=AC4.9 billion in=2019. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitt=er: @UCB_news 

For further information, UCB: 

Brand Communications
Jim Baxter, 
Neurology Communications, UCB
T+32.2.473.78.85.01 jim.baxter@ucb.com 

Corporate Communications
Laurent Schots 
Media Relations, UCB  
T+32.2.559.92.64  Laurent.schots@ucb.com 

Investor Relations
Antje Witte          
Investor Relations, UCB
T +32.2.559.94.14 antje.witte@ucb.com

Isabelle Ghellynck,
 Investor Relations, UCB
T+32.2.559.9588, isabelle.ghellynck@ucb.com 

Forward-looking statements 
This press release contains forward-looking statements based on current pla=ns, estimates and beliefs of management. All statements, other than stateme=nts of historical fact, are statements that could be deemed forward-looking=statements, including estimates of revenues, operating margins, capital ex=penditures, cash, other financial information, expected legal, political, r=egulatory or clinical results and other such estimates and results. By thei=r nature, such forward-looking statements are not guarantees of future perf=ormance and are subject to risks, uncertainties and assumptions which could=cause actual results to differ materially from those that may be implied b=y such forward-looking statements contained in this press release. Importan=t factors that could result in such differences include: changes in general=economic, business and competitive conditions, the inability to obtain nec=essary regulatory approvals or to obtain them on acceptable terms, costs as=sociated with research and development, changes in the prospects for produc=ts in the pipeline or under development by UCB, effects of future judicial =decisions or governmental investigations, product liability claims, challen=ges to patent protection for products or product candidates, changes in law=s or regulations, exchange rate fluctuations, changes or uncertainties in t=ax laws or the administration of such laws and hiring and retention of its =employees. 
Additionally, information contained in this document shall not constitute a=n offer to sell or the solicitation of an offer to buy any securities, nor =shall there be any offer, solicitation or sale of securities in any jurisdi=ction in which such offer, solicitation or sale would be unlawful prior to =the registration or qualification under the securities laws of such jurisdi=ction. UCB is providing this information as of the date of this document an=d expressly disclaims any duty to update any information contained in this =press release, either to confirm the actual results or to report a change i=n its expectations.
There is no guarantee that new product candidates in the pipeline will prog=ress to product approval or that new indications for existing products will=be developed and approved. Products or potential products which are the su=bject of partnerships, joint ventures or licensing collaborations may be su=bject to differences between the partners. Also, UCB or others could discov=er safety, side effects or manufacturing problems with its products after t=hey are marketed.
Moreover, sales may be impacted by international and domestic trends toward=managed care and health care cost containment and the reimbursement polici=es imposed by third-party payers as well as legislation affecting biopharma=ceutical pricing and reimbursement.

*Dr. James Bussel is a paid consultant for various comp=anies in the ITP space including Amgen, Argenx, Dova, Johnson and Johnson, =Momenta, Principia, Rigel and UCB.


  1. Robak T., Ka=C5=BAmierczak M., Jarque I., Musteata V., Trel=i=C5=84ski J. et al. Phase 2 multiple-dose study of an FcRn inhibitor, roza=nolixizumab, in patients with primary immune thrombocytopenia. Blood advanc=es 2020; 4(17):4136=E2=80=9346
  2. Kohli, R, Chaturvedi, S (2019) Epidemiology and Clinical Ma=nifestations of Immune Thrombocytopenia. Hamostaseologie 2019; 39(3):238=E2==80=93249
  3. Kiessling, P, Lledo-Garcia, R. S, Watanabe et al. (2017) Th=e FcRn inhibitor rozanolixizumab reduces human serum IgG concentration: A r=andomized phase 1 study. Sci Transl Med. 9(414)
  4. Bril, V, Benatar, M.  Brock, M et al. (2019) Proof-of-=Concept and Safety of the Anti-FcRn Antibody Rozanolixizumab in Patients wi=th Moderate-to-Severe Generalized Myasthenia Gravis (GMG): A Phase 2a Study=. Neurology (Abstracts: AAN 71th Annual Meeting, Philadelphia)  Neurol=ogy 2019; 92(15 Suppl.): abs S43.001
  5. National Organization for Rare Disorders (NORD). Immune thr=ombocytopenia. Retrieved from: https://rarediseases.org/rare-diseases/immun=e-thrombocytopenia/ Accessed September 2020 
  6. Cortelazzo, S., Finazzi G., Buelli, M., Molteni, A.,Viero P=. and Barbui, T. (1991) High risk of severe bleeding in aged patients with =chronic idiopathic thrombocytopenic purpura. Blood. 77(1):p31-
  7. Chang, M., Nakagawa P.A., Williams S.A. et al. (2003) Immun=e thrombocytopenic purpura (ITP) plasma and purified ITP monoclonal autoant=ibodies inhibit megakaryocytopoiesis in vitro. Blood. 102(3):p887-95=
  8. Chan, H., Moore J.C., Finch C.N., Warkentin T.E., and Kelto=n J.G. (2003) The IgG subclasses of platelet-associated autoantibodies dire=cted against platelet glycoproteins IIb/IIIa in patients with idiopathic th=rombocytopenic purpura. Br J Haematol. 122(5):p818-24
  9. Neunert, C., Terrell, D.R., Arnold, D.M., et al. American S=ociety of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv=. 2019;3(23):3829-3866.
  10. Provan, D., Arnold D.M., Bussel, J.B., et al. (2019) Update=d international consensus report on the investigation and management of pri=mary immune thrombocytopenia. Blood Adv. (2019) 3 (22): 3780=E2=80=933817.<=/span>
  11. Smith B, Kiessling A, Lledo-Garcia R, et al. Generation and=characterization of a high affinity anti-human FcRn antibody, rozanolixizu=mab, and the effects of different molecular formats on the reduction of pla=sma IgG concentration. MAbs2018;10:1111-30
  12. U.S. Food and Drug Administration (FDA). (2018). Orphan dru=g designations and approvals. Retrieved from:  https://www.accessdata.=fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=3D636618 Access=ed September 2020
  13. European Medicines Agency (EMA). (2019). Public summary of =opinion on orphan designation. Retrieved from: https://www.ema.europa.eu/en=/medicines/human/orphan-designations/eu3182131 Accessed September 2020

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