MolMed TK therapy submitted for Conditional Marketing Authorisation in EU

MolMed S.p.A. (Milan:MLM) announces that the Company has filed to the European Medicines Agency an application for Conditional Marketing Authorisation for T ! its no"el in"estigational cell#gene therapy. T is an ad$uncti"e treatment in hematopoietic stem cell transplantation (%SCT) for patients affected &y high risk leukaemia.

It is estimated that in EU approximately 5,000 leukaemia patients/year lack a fully compatible donor: TK offers to these patients the option of transplantation from a mismatched donor ithout post!transplant immunosuppression" If appro#ed, TK ould be the first cell!$ene therapy product on the market for this life threatenin$ condition" The %onditional &arketin$ 'uthorisation application is supported by cumulati#e efficacy and safety results obtained from a completed (hase I!II, multicentre trial )TK00*+" These data ere included in presentations $i#en at the ,-th annual con$ress of the 'merican .ociety of %linical /ncolo$y )'.%/+ and at the 55th 'nnual &eetin$ of the 'merican .ociety of 0ematolo$y )'.0+ in 1023" 4esults sho that TK can reduce transplant!related mortality and increase o#erall sur#i#al in hi$h risk leukaemia patients transplanted from a mismatched 0.%T donor" The application also includes initial data from the currently on$oin$ pi#otal randomised (hase III study )TK005+, hich ha#e been submitted to the next '.%/ con$ress takin$ place in &ay in %hica$o" The TK therapy as $ranted /rphan 6ru$ 6esi$nation by the European %ommission" %laudio 7ordi$non, %hairman of the 7oard and %E/ of &ol&ed, commented: 8'ith the su&mission of the Conditional Marketing Authorisation! (e ha"e achie"ed an important milestone (hich represents a turning point in the clinical de"elopment of our T technology. 'e &elie"e that &y pro"iding fast immune reconstitution and eliminating the need for post#transplant immune# suppression! T may represent a ne( and po(erful tool a&le to pro"ide an efficacious treatment option for those high risk leukaemia patients (ho lack a fully matched donor. 'ith more than )*+ patients treated (orld(ide and an ongoing randomised ,hase --- trial! our efforts in the implementation of the T technology (itnesses our dedication to the treatment of leukaemia patients lacking a "alid treatment option! and MolMed competiti"eness in the challenging field of immune#gene therapy of cancer."

Conditional Marketing Authorisation The %onditional &arketin$ 'uthorisation represents an expedite path for early market authorisation ahead of completion of the pi#otal re$istration studies" .uch anticipated authorisation is mainly based on efficacy and safety e#idences accumulated in early studies" ' %onditional &arketin$ 'uthorisation may be $ranted only if all the follo in$ re9uirements are met: 2" the risk!benefit balance of the medicinal product is positi#e: 1" it is likely that the applicant ill be in a position to pro#ide the comprehensi#e clinical data: 3" unmet medical needs ill be fulfilled: ," the benefit to public health of the immediate a#ailability on the market of the medicinal product concerned out ei$hs the risk inherent in the fact that additional data are still re9uired" ' %onditional &arketin$ 'uthorisation is #alid for one year, on a rene able basis" The holder is re9uired to complete on$oin$ studies or to conduct ne studies ith a #ie to confirmin$ that the benefit!risk balance is positi#e"

About TK TK is a cell therapy product, based on the use of $enetically en$ineered donor T cells carryin$ a ;suicide $ene8" These cells are administered to patients durin$ the haematopoietic stem cell transplantation for the treatment of hi$h risk leukaemia" TK therapy allo s to eliminate the post! transplant immunosuppression treatment thus acceleratin$ the immune reconstitution and controllin$ the immunolo$ical conse9uences arisin$ from the $enetic differences ith the donor, kno n as <raft #ersus 0ost 6isease )<#06+"

In #irtue of this approach, 0.%T from partially compatible donors is a safer and more effecti#e option, thus potentially increasin$ the number of candidates for transplantation"

TK008 study TK005 is a pi#otal randomised (hase III trial )TK005+ in adult patients affected by hi$h!risk leukaemia under$oin$ transplant of haematopoietic stem cells collected from partially compatible )haploidentical+ family donors" The trial desi$n has disease!free sur#i#al as the primary end!point ! hich includes both transplant! related mortality and disease relapse ! e#aluated on a patient population of 2*0 patients" The trial ill compare the outcome of haplo!transplants ith or ithout TK add!backs, ith a 3:2 randomisation ratio in fa#our of the TK arm" .econdary end!points include o#erall sur#i#al, reduction of transplant!related mortality, safety and patients= 9uality of life" >ith the aim to pro#ide additional clinical benefit to patients and to si$nificantly increase the potential participation of centres in the trial, the %ompany implemented in 1021 t o important chan$es in the protocol desi$n of (hase III trial TK005" The first consists in broadenin$ the enrolment criteria to include patients in leukaemic relapse, in addition to those in disease remission: the second chan$e pro#ides for the introduction of a further treatment option in the control arm, based on the use of an unmanipulated transplant follo ed by cyclophosphamide administration durin$ the post!transplantation period"

This press release is written in compliance with public disclosure obligations established by C !" # $%talian securities & e'change commission( resolution no) **+,* of *- May *+++. as subse/uently amended)

About MolMed
MolMed S.p.A. is a &iotechnology company focused on research! de"elopment and clinical "alidation of no"el anticancer therapies. MolMed/s pipeline includes t(o antitumour therapeutics in clinical de"elopment: T ! a cell# &ased therapy ena&ling &one marro( transplants from partially compati&le donors! in a&sence of post#transplant immune#suppression! in ,hase --- in high#risk acute leukaemia0 123#hT14! a no"el "ascular targeting agent! in ,hase --- in malignant pleural mesothelioma and in ,hase -- in si5 more indications: colorectal! lung (small#cell and non# small#cell)! li"er and o"arian cancer! and soft tissue sarcomas. MolMed also offers top#le"el e5pertise in cell and gene therapy to third parties to de"elop! conduct and "alidate pro$ects from preclinical to ,hase --- trials! including scale#up and c2M, production of clinical#grade "iral "ectors! and manufacturing of patient#specific genetically engineered cells. MolMed is head6uartered at the San 3affaele 7iomedical Science ,ark in Milan! -taly. The Company/s shares are listed on the main market (MTA) of the Milan Stock E5change. (Ticker 3euters: MLM8.M-)

?/@TE: MolMed)com