Participating Companies


Abide Tx
Serine hy­dro­lase-tar­get­ed ther­a­peu­tics har­ness­ing the en­do­cann­abi­noid sys­tem to ad­dress neu­ro­log­i­cal and pain di­s­or­ders w. limit­ed or no treat­ment op­tions. Ph 2: Tourette Syn­drome on­go­ing with da­ta ex­pect­ed in 1Q20; Ph 1b: neu­ro­path­ic pain com­plete, with plans to ini­ti­ate Ph 2 in 1H19. [more in­for­ma­tion]
Acceleron Pharma Inc [XLRN] US$2,279 MM MCap
Lead ther­a­peu­tic can­di­date in he­ma­tol­o­gy, lus­pa­ter­cept, un­der a glob­al part­n­er­ship with Cel­gene, re­port­ed pos­i­tive Phase 3 ME­DAL­IST and BE­LIEVE trial re­sults, and cont­in­ues to ad­vance mul­ti­ple Ph2 and Ph3 trials for the treat­ment of chron­ic ane­mia in myelodys­plas­tic syn­dromes, be­ta-tha­lassemia, and myelo­fi­bro­sis. [more in­for­ma­tion]
AcelRx Pharmaceuticals, Inc. [ACRX] US$229 MM MCap
Re­cent­ly launched DSU­VIA; rev-gen­er­at­ing in Q1. Plans to re­sub­mit Za­lvi­so NDA in US by YE [more in­for­ma­tion]
Adamas Pharmaceuticals, Inc. [ADMS] US$145 MM MCap
Launched GO­COV­RI™ for LID in pa­tients w/ Parkin­son’s dis­ease. Late-stage pipe­line in­cludes ADS-5102 in P3 for MS walk­ing im­pair­ment and ADS-4101, high-dose, mod­i­fied re­lease la­cosamide, which com­plet­ed P1 for par­tial onset seizures in pa­tients with epilep­sy. [more in­for­ma­tion]
Affibody
Pro­pri­e­tary Af­fi­body® molecules and Al­bu­mod plat­forms. 4 clin­i­cal / late-stage, pro­pri­e­tary pro­grams in­clud­ing pso­ri­a­sis, B-cell au­toim­mune dis­eas­es, & liv­er dis­eas­es. Pipe­line al­so in­cludes Dx imag­ing pro­gram tar­get­ing me­tastat­ic breast can­cer. [more in­for­ma­tion]
Affimed N.V. [AFMD] US$187 MM MCap
Mo­d­u­lar ROCK™plat­form en­abling mul­ti-spe­cif­ic NK / T-cell bi-and tri-spe­cif­ic Abs. Aug 2018 deal w Ge­nen­tech ($96M up­front / $5B po­ten­tial). – AFM13 in P1b (com­bo) in HL and P2a(mono) in CD30+lym­pho­ma [more in­for­ma­tion]
Allele Biotechnology
​Lead­ing in­de­pen­dent Nanoanti­boy (nAb) tech plat­form. Mul­ti­ple whol­ly-owned hu­man­ized nAb ther­a­peu­tics in late-stage pre-clin­i­cal studies. Shares the sci­en­tif­ic foun­da­tion Ab­l­ynx used to de­vel­op its pipe­line. IBD (TN­Fa) and On­col­o­gy (HER2) are among ini­tial tar­gets. [more in­for­ma­tion]
Amarin Corporation [AMRN] US$5,713 MM MCap
Vas­ce­pa: 1st and on­ly pure EPA ome­ga-3 fat­ty acid ap­proved to low­er trig­lyc­eride lev­els in adults. Pre­sent­ed (+) da­ta from RE­DUCE-IT CV Out­comes trial at AHA [more in­for­ma­tion]
Antev
Ph3 ready hor­mone ther­a­py com­pany with an in­jectable, long-act­ing GnRH an­ta­g­on­ist for pros­tate can­cer and be­nign pro­stat­ic hy­per­pla­sia. An in­jectable en­ables ex­tend­ed drug ac­tiv­i­ty, which is im­por­tant for in­di­ca­tions with ac­tive oral an­ta­g­on­ists in late-stage de­vel­op­ment and al­so ap­proved agent(s). The safe­ty pro­file looks good with over 415 sub­jects dosed over 805 times. [more in­for­ma­tion]
Apexigen
CD40 ag­on­ist mAb; 170+ pts dosed (mono & com­bi sett­ings). 7 on­go­ing Ph 2s, undis­closed oc­u­lar dis­ease pro­gram in Ph 3. AACR: Ph 1b da­ta (w/Park­er Inst); col­labs w/BMY, MDACC, Yale, et al [more in­for­ma­tion]
Aptorum Group [APM] US$161 MM MCap
Ap­to­rum is a phar­ma­ceu­ti­cal com­pany ded­i­cat­ed to de­vel­op­ing and com­mer­cial­iz­ing a broad range of ther­a­peu­tic and di­ag­nos­tic tech­nolo­gies to tack­le un­met med­i­cal needs. Ap­to­rum is pur­su­ing ther­a­peu­tic and di­ag­nos­tic pro­jects in neu­rol­o­gy, in­fec­tious dis­eas­es, gas­troen­terol­o­gy, on­col­o­gy and other dis­ease ar­eas; [more in­for­ma­tion]
Arcus Biosciences [RCUS] US$404 MM MCap
Ar­cus has sev­er­al pro­grams tar­get­ing im­por­tant im­muno-on­col­o­gy path­ways, in­clud­ing a du­al adeno­sine re­cep­tor an­ta­g­on­ist AB928, which is in a Phase 1/1b pro­gram to eval­u­ate AB928 in com­bi­na­tion with other agents in mul­ti­ple tu­mor types, and an an­ti-PD-1 anti­body AB122, which is be­ing eval­u­at­ed in a Phase 1 trial and is be­ing test­ed in com­bi­na­tion with Ar­cus’s other prod­uct can­di­dates. [more in­for­ma­tion]
AsclepiX Therapeutics
Pri­vate, oph­thal­mol­o­gy fo­cused ear­ly stage com­pany; tech­nol­o­gy out of Johns Hop­kins. Plan­n­ing 4Q19 IND and 1Q20 Ph1/2 ini­ti­a­tion (DME and Wet AMD). AX­T107: nov­el pep­tide in­hibit­ing VEGF and ac­ti­vat­ing Tie2; de­mon­s­trat­ed ef­fi­ca­cy and dura­bil­i­ty in rab­bit and mouse mod­els. [more in­for­ma­tion]
Asklepion Pharmaceuticals
Pri­vate, fo­cused on the de­vel­op­ment of IV Cit­rul­line for rare pe­di­a­tric dis­eas­es. First in­di­ca­tion, the pre­ven­tion of sig­ni­f­i­cant se­que­lae of pe­di­a­tric acute car­diopul­mo­nary by­pass-in­duced lung in­jury, Ph 3 trial is on­go­ing. [more in­for­ma­tion]
Assembly Biosciences, Inc. [ASMB] US$396 MM MCap
Nov­el, oral, live bio­ther­a­peu­tics ad­dress­ing dis­eas­es as­soc w mi­cro­biome. HBV pro­gram ad­vanc­ing mul­ti­ple drug can­di­dates; ful­ly in­te­grat­ed mi­cro­biome plat­form in­cludes ro­bust strain ID and se­lec­tion pro­cess (for strain iso­la­tion & growth). [more in­for­ma­tion]
Aurinia Pharmaceuticals, Inc. [AUPH] US$595 MM MCap
De­vel­op­ing vo­clos­porin ad­dress­ing lu­pus nephri­tis (LN), fo­cal seg­men­tal glomeru­los­cle­ro­sis (FS­GS) & dry eye syn­drome (DES). - Dry eye top-line da­ta: ear­ly Q1 19 - LN top line da­ta: late Q4 19 - FS­GS da­ta: open la­bel da­ta: 2H 19 [more in­for­ma­tion]
Avacta [AVCT:LN] £32 MM MCap
It is based on a na­t­u­ral­ly oc­cur­ring hu­man pro­tein, en­gi­neered to be­have like an anti­body, but small­er, sim­pler, more ro­bust, easy to for­mat, cheap­er and quick­er to pro­duce, high­ly spe­cif­ic to the tar­get and non-im­muno­genic. [more in­for­ma­tion]
AxoGen [AXGN] US$846 MM MCap
Da­m­age or tran­sec­tion to pe­ripher­al nerves. Q1 revs $23.3M, 35% growth YoY; exp 2019 rev bt $109-$114M [more in­for­ma­tion]
Ayala Pharma
Tar­get­ed ther­a­pies for on­co in­di­ca­tions w ac­ti­vat­ed Notch path­way (vali­dat­ed in pro­lif­er­a­tion, stem­ness, an­gio­ge­n­e­sis & me­tas­ta­sis; over­ex­pressed in cer­tain ACC, TN­BC & he­ma. can­cers). Lead as­set in Ph 2 for ACC; in-li­censed/BMS gen­er­at­ed Ph 1 da­ta. [more in­for­ma­tion]
Bellicum Pharmaceuticals Inc. [BLCM] US$115 MM MCap
Molec­u­lar switch tech­nol­o­gy and co-stim do­mains en­ables phar­ma­co­log­ic con­trol over cells for en­hanced ef­fi­ca­cy and safe­ty. Lead (reg­is­tra­tio­n­al EU) pro­gram: Ph2 for ri­vo-cel (al­pha/be­ta ad­d­back T-cell ther­a­py for post al­lo­gene­ic HSTC in non-ma­lig­nant and ma­lig­nant dis­eas­es). Pos­i­tive late in­ter­im anal­y­sis on ri­vo-cel exp at ASH 2018; [more in­for­ma­tion]
BerGenBio [BGBIO:OS] NKr1,242 MM MCap
De­vel­op­ing first-in-class se­lec­tive Axl ki­nase in­hibi­tors for liquid and solid can­cers, lead can­di­date is bem­cen­tinib (BG­B324) n four com­pany-spon­sored trials. Stage 2 of Ph 2 com­bi­na­tion trial with bem­cen­tinib/KEYTRU­DA in NS­CLC ex­pect­ed in 1H19. Bem­cen­tinib/TARCE­VA Ph 2 com­bi­na­tion trial in first line EGFRm pa­tients re­port­ed that me­dian PFS sur­passed TARCE­VA monother­a­py. [more in­for­ma­tion]
Biohaven Pharmaceuticals, Inc [BHVN] US$2,677 MM MCap
Lead de­vel­op­ment pro­grams in­clude mul­ti­ple com­pounds across its CGRP re­cep­tor an­ta­g­on­ist and glu­ta­mate mo­d­u­la­tion plat­forms. In­ter­nal de­vel­op­ment and re­search with in­tel­lec­tu­al prop­er­ty li­censed from com­pa­nies and in­sti­tu­tions in­clud­ing BMS, As­traZene­ca, Yale Uni­ver­si­ty, Ca­ta­l­ent, ALS Bio­phar­ma and Mas­sachusetts Gen­er­al Hos­pi­tal. [more in­for­ma­tion]
BioPharmX [BPMX] US$9 MM MCap
De­vel­op­ing BPX-04 for rosacea, us­ing pro­pri­e­ta­ty hy­drophil­ic top­i­cal for­mu­la­tion tech­nol­o­gy. En­rolling Ph2b w da­ta exp in 2019. Com­plet­ed Ph 2b with BPX-01 in acne. [more in­for­ma­tion]
BioShin
New­ly in­cor­po­rat­ed (Nov 2018), whol­ly owned sub­si­diary of Bio­haven Phar­ma­ceu­ti­cals. Shang­hai-based, led by ex-BMY ex­ec and Bio­haven’s Chief of Corp Strat­e­gy & BD, Don­nie Mc­Grath, Bioshin aims to de­vel­op and com­mer­cial­ize Bio­haven’s late-stage mi­graine and neu­rol­o­gy port­fo­lio in Chi­na and other APAC mar­kets. [more in­for­ma­tion]
BioTime, Inc [BTX] US$159 MM MCap
Lead (Ph 2) cell re­place­ment can­di­date: OpRe­gen®, reti­nal pig­ment epithe­li­um tran­s­plant ther­a­py: Dry AMD. Lead cell de­liv­ery clin­i­cal pro­gram: Rene­via®: med de­vice de­vel­oped as al­ter­na­tive to adi­pose tis­sue trans­fer pro­ce­dures. Equi­ty in As­te­rias Bio­ther­a­peu­tics (NYSE: AST), On­co­Cyte Cor­po­ra­tion (NYSE: OCX), and AgeX Ther­a­peu­tics (pri­vate) [more in­for­ma­tion]
BlinkBio, Inc.
They have in­no­vat­ed a plat­form based on Tun­able Drug Con­ju­gate (TDCs) ther­a­pies used to di­rect­ly tar­get the de­liv­ery of toxins to tu­mors. The lead ther­a­peu­tic pro­gram tar­gets Fo­late Re­cep­tor al­pha and has been vali­dat­ed in the clin­ic by ADCs, pro­vid­ing a de-risked bench­mark for plat­form vali­da­tion and can­di­date de­vel­op­ment. [more in­for­ma­tion]
BrainStorm Cell Therapeutics Inc. [BCLI] US$111 MM MCap
NurOwn® au­tol­o­gous cell ther­a­py plat­form for neu­rode­gen­er­a­tive dis­eas­es. Lead can­di­date for ALS, Ph2 eval­u­at­ed 60 ALS pts w fa­vor­able safe­ty/tol­er­a­bil­i­ty. Ph3 cur­rent­ly en­rolling 200 pts across 6 US cen­ters; top-line da­ta ex­pect­ed in mid-2020. Se­cond in­di­ca­tion is pro­gres­sive MS - IND ac­cept­ed Dec 2018. [more in­for­ma­tion]
Camurus AB [CAMX:ST] SKr3,440 MM MCap
De­vel­op­ing/com­mer­cial­iz­ing in­no­va­tive/long-act­ing medicines for se­vere & chron­ic con­di­tions (e.g. opi­oid de­pen­dence, pain, can­cer & en­docrine di­s­or­ders). Fluid­Crys­tal® drug de­liv­ery tech­nolo­gies for im­proved Q of life, treat­ment out­comes and re­source uti­l­iza­tion. [more in­for­ma­tion]
Caris Life Sciences
Pre­ci­sion medicine com­pany uti­l­iz­ing pro­pri­e­tary plat­forms to pro­vide on­col­o­gists with ac­tion­able treat­ment da­ta for can­cer and other com­plex dis­eas­es. [more in­for­ma­tion]
CASI Pharmaceuticals Inc. [CASI] US$302 MM MCap
Re­ceived MN­PA (f.k.a. CF­DA) ap­pro­val for EVOMELA® (mel­pha­lan for in­jec­tion: mul­ti­ple myelo­ma) on Dec 3, 2018; CF­DA re­view in progress for ZE­VALIN® and MAR­QI­BO® (in-li­censed from Spec­trum Phar­ma). Ac­qd 25 US FDA-ap­proved AN­DAS from San­doz in Jan 2018. [more in­for­ma­tion]
Catalyst Pharmaceuticals, Inc. [CPRX] US$365 MM MCap
Fir­dapse ap­proved 11/18, Q1 sales of $12m: 380 ac­tive LEMS pa­tients on ther­a­py (a/o May 9) [more in­for­ma­tion]
CellMax Life
The com­pany is cur­rent­ly fo­cused on col­orec­tal can­cer (CRC) screen­ing us­ing the clin­i­cal­ly vali­dat­ed CMx plat­form, a blood biop­sy test plat­form that de­tects pre-can­cer­ous le­sions (ade­no­mas) in as lit­tle as two tea­spoons of blood (less than 10mL). [more in­for­ma­tion]
Cellular Biomedicine Group, Inc. [CBMG] US$332 MM MCap
This vali­dates CB­MG's in-house cell man­u­fac­tur­ing ca­pa­bil­i­ties de­vel­oped in part­n­er­ship with Gen­er­al Elec­tric and Ther­mo Fish­er. CB­MG's pro­pri­e­tary im­muno-on­col­o­gy pipe­line in­cludes BC­MA, CD22, TCR-T for HCC and TIL for the treat­ment of NS­CLC and other solid tu­mors. [more in­for­ma­tion]
Context Therapeutics
Con­text’s lead pro­gram is Apris­tor®, an in­vesti­ga­tio­n­al oral full pro­ges­terone re­cep­tor (PR) an­ta­g­on­ist that is in Phase 2 de­vel­op­ment for PR+ breast, ovarian, and en­dome­trial can­cers. In ad­di­tion, Con­text is ad­vanc­ing CTX-30916, a pre­clin­i­cal oral pro­ges­terone re­cep­tor an­ta­g­on­ist, and a dis­cov­ery-stage pro­gram tar­get­ing Sig­ma1. [more in­for­ma­tion]
CTI BioPharma [CTIC] US$50 MM MCap
Lead can­di­date pa­cri­tinib, oral ki­nase in­hibi­tor w spe­ci­fic­i­ty for JAK2, FLT3, IRAK1, and CS­F1R in Ph 2 for treat­ment of myelo­fi­bro­sis. Up­com­ing ca­t­alysts: pa­cri­tinib PAC203 Ph 2 da­ta - mid'19, ini­ti­a­tion of ph3 - Q3. [more in­for­ma­tion]
CureVac AG
Found­ing Co of mR­NA space (last round $1bn+). In­tra­tu­mo­ral ther­a­py Ph1 (mul­ti­ple onc in­di­ca­tions) ini­ti­at­ed Q4 17, ear­ly da­ta rep at SITC. First pt en­rolled Ph 1 pro­phy­lac­tic mR­NA ra­bies vax Oct 2018. Pro­phy­lac­tic in­fluen­za vax ini­ti­a­tion Ph 1 2020, OTC de­fi­cien­cy Ph 1 ini­ti­a­tion H219. In­ter­im da­ta /all trials: 2019. [more in­for­ma­tion]
Cytosen
Next-gen NK Cell ther­a­py; en­ter­ing Ph 2 trials in 1H19 (AML HSCT tran­s­plant) with RMAT po­ten­tial in 2H20. Ph 1/2 de­mon­s­trat­ed sig­nif re­duc­tion in re­lapse rates (MD An­der­son). Pa­tent­ed nano­par­ti­cle pro­cess­ing tech en­ables high-dose, high-po­ten­cy NK cells from com­mer­cial CD­MO. Self-fund­ed through en­roll­ment of ini­tial Ph 1/2 dos­ing co­horts. [more in­for­ma­tion]
DBV Technologies [DBVT] US$675 MM MCap
The com­pany has al­so de­vel­oped Vi­askin Milk, which re­port­ed pos­i­tive pre­lim­i­nary Phase II re­sults in 2018 and Vi­askin Egg, which is in pre-clin­i­cal de­vel­op­ment. The Com­pany is ex­plor­ing Vi­askin in other ar­eas of un­met need in­clud­ing vaccines, in­flam­ma­to­ry con­di­tions and au­toim­mune dis­eas­es. [more in­for­ma­tion]
Enzo Biochem, Inc. [ENZ] US$155 MM MCap
$100m revs in F18. Pi­oneer in molec­u­lar di­ag­nos­tics (Dx), lead­ing con­ver­gence of clin­i­cal lab­o­ra­to­ries, life sci­ences and IP through de­vt of unique Dx plat­form tech­nolo­gies pro­vid­ing nu­mer­ous ad­van­tages over pre­vi­ous stan­dards. [more in­for­ma­tion]
Epigenomics AG [EPGNY] US$75 MM MCap
Blood-based can­cer de­tec­tion com­pany with DNA methy­la­tion bio­mark­er tech­nol­o­gy. De­vel­ops/com­mer­cial­izes Dx prod­ucts across mul­ti­ple can­cer in­di­ca­tions w/high med­i­cal need. Lead, Epi pro­Colon: blood-based screen­ing test de­tect­ing col­orec­tal can­cer. FDA ap­proved, mar­ket­ed in US, EU, Chi­na et al. [more in­for­ma­tion]
Erytech Pharma SA [ERYP] US$127 MM MCap
Clin pro­grams ad­dress­ing solid tu­mors, PC pro­grams: en­zyme ther­a­pies be­yond on­col­o­gy, PC pro­grams in I-O. Lead Ph 3, eryas­pase (L-as­parag­i­nase en­cap­su­lat­ed in RBCs): pan­cre­at­ic can­cer (de­mon­s­trat­ed ef­fi­ca­cy/safe­ty in ran­domized Ph 2b in this tu­mor type); ini­ti­at­ing Ph 2 in TN­BC in 2018. [more in­for­ma­tion]
ESSA Pharma Inc. [EPIX] US$12 MM MCap
Sm molecule drugs: cas­tra­tion-re­sis­tant pros­tate can­cer. EPI-506, exp to en­ter clin­ic YE19 (se­cond-gen com­pound). De­vel­op­ing com­pounds bind­ing to N-ter­mi­nus of AR and block sig­nal­ing. MOA po­ten­tial­ly over­comes re­sis­tance mech­anisms cur­rent­ly seen in me­tastat­ic pros­tate can­cer. [more in­for­ma­tion]
Evofem Biosciences, Inc. [EVFM] US$172 MM MCap
Se­cured $30M strate­g­ic in­vest­ment from PDL Bio­S­ciences (PDLI) in April 2019 for 6.7M shares at $4.50 (26% pre­mi­um to pri­or day close) and 25% war­rant cov­er­age. [more in­for­ma­tion]
Forbius
Se­lec­tive in­hibi­tors of TGF Be­ta & EGFR path­ways. AACR: (+) Ph 1 da­ta. 3 on­go­ing Ph 2 trials (SCCHN, NS­CLC, TN­BC); ad­dl Ph 1 trials in IO, Sys­temic Scle­ro­sis & Myelo­fi­bro­sis [more in­for­ma­tion]
Genfit SA [GNFTF] US$875 MM MCap
$155m Mar 2019 US IPO; FDA BTD for Ela­fi­branor (4/19); (+) 36-mo DSMB rec for cont­in­u­a­tion of Ph 3 study in NASH; da­ta by YE [more in­for­ma­tion]
GenSight Biologics [SIGHT:PA] €43 MM MCap
Gene ther­a­py: reti­nal & CNS de­gen­er­a­tive patholo­gies and mi­to­chon­drial dis­ease. Fil­ing for MAA exp Q4 2019 in EU and 2020 in US. Op­to­ge­net­ic pro­gram and tar­get­ed dis­ease im­me­di­ate­ly trans­fer­able to dry AMD. Plat­form com­bines mono-ge­net­ic ther­a­py-based ap­proach w core tech­nol­o­gy plat­form and pro­pri­e­tary Mi­to­chon­drial Tar­get­ing Se­quence. [more in­for­ma­tion]
GW Pharmaceuticals PLC [GWPH] US$5,751 MM MCap
GW has estab­lished a world lead­ing po­si­tion in the de­vel­op­ment of plant-de­rived cann­abi­noid ther­a­peu­tics through its proven drug dis­cov­ery and de­vel­op­ment pro­cess­es, in­tel­lec­tu­al prop­er­ty port­fo­lio and reg­u­la­to­ry and man­u­fac­tur­ing ex­per­tise. GW has de­vel­oped an oral for­mu­la­tion of pu­ri­fied cann­abi­di­ol (CBD), ap­proved as Epi­di­olex® in the U.S. by the U.S. [more in­for­ma­tion]
HaemaLogiX
I-O; nov­el tgts in Mul­ti­ple Myelo­ma (col­lab w Take­da). Kap­paMab (MM) Ph 2b, da­ta 1H20. Kap­paMab Car-T en­ter­ing clin­ic 2019. Kap­paMab & Lamb­daMab tar­get cell sur­face pro­teins KMA and LMA on ab­nor­mal plas­ma cells. Kap­paMab: com­plete & par­tial re­spons­es in Ph 1 & 2a. Kap­pa-mab Car-T en­ter­ing clin­ic 2019. [more in­for­ma­tion]
Hua Medicine [2552:HK]
Hua Medicine is a clin­i­cal stage drug de­vel­op­ment com­pany cur­rent­ly fo­cused on de­vel­op­ing a glob­al first-in-class oral drug, Dorza­gli­atin or HM­S5552, for the treat­ment of Type 2 Di­a­betes (T2D). Dorza­gli­atin is a fourth gen­er­a­tion glu­cok­i­nase ac­ti­va­tor, orig­i­nat­ed at Roche AG, and in-li­censed by Hua Medicine as a pre-clin­i­cal stage com­pound. [more in­for­ma­tion]
Hutchison China MediTech Ltd ("Chi-Med") [HCM] US$3,596 MM MCap
Fruquin­tinib (VEG­FR in­hibi­tor, LLY-part­nered) for col­orec­tal can­cer was ap­proved in Chi­na in Septem­ber 2018 and Phase 3 to­p­line re­sults in NS­CLC is ex­pect­ed in late 2018. AZN has the de­ci­sion on strat­e­gy for Phase 3 reg­is­tra­tion and po­ten­tial Break­through Ther­a­py in NS­CLC in com­bi­na­tion with Ta­gris­so. [more in­for­ma­tion]
I-Mab Biopharma
The com­pany built its strong pipe­line with strong in­ter­nal re­search sup­ple­ment­ed with ac­tive li­cens­ing from lead­ing glob­al bio­phar­ma part­n­ers such as best in class sg­p130 molecules from Fer­ring, unique CD38 mAb from Mor­phosys, and mul­ti­ple late stage molecules from Genexine. [more in­for­ma­tion]
Idera Pharmaceuticals, Inc. [IDRA] US$78 MM MCap
Lead can­di­date Til­so­toli­mod (TLR 9 ag­on­ist) used in comb w Ipi in PD1 re­frac­to­ry me­lano­ma pts. En­rolling ad­dl pts in Ph 2 ex­pan­sion and Phase 3 en­roll­ment ex­pect­ed to com­plete Q4 2019. Com­bo of ipi and Til­so­toli­mod ap­pears to ac­ti­vate im­mune re­sponse in pts who have ex­haust­ed all op­tions, e.g. PD1/CPIs. Fast Track des for Til­so­toli­mod (Nov’17). [more in­for­ma­tion]
ImmuneSensor, LLC
Chen was award­ed the 2018 Lurie Prize in Biomed­i­cal Sci­ences and the 2019 Break­through Prize in Life Sci­ences for his dis­cov­ery of cGAS and its prod­uct cGAMP, which ac­ti­vates ST­ING to sti­m­u­late in­nate im­mune re­spons­es against can­cers and in­fec­tions. [more in­for­ma­tion]
Iovance Biotherapeutics [IOVA] US$2,054 MM MCap
AS­CO: new in­ter­im da­ta from TIL ther­a­py LN-145 in ad­vanced cer­vi­cal can­cer (ORR 44%, 1 CR, 9 PRs, 2 uPRs) & TIL ther­a­py in ad­vanced me­lano­ma (ORR 38%, 2 CRs, 18 PRs, 1 uPR). [more in­for­ma­tion]
Kadmon Holdings, Inc. [KDMN] US$282 MM MCap
Plat­form of ROCK in­hibi­tors. ROCK2 in­hibi­tor in reg­is­tra­tion trial cGVHD; en­roll­ment com­ple­tion exp 2H19; ini­tial anal­y­sis exp YE. Ph 2 ini­ti­a­tion in scle­ro­der­ma exp in 2019. [more in­for­ma­tion]
Marker Therapeutics [MRKR] US$264 MM MCap
A clin­i­cal-stage im­muno-on­col­o­gy com­pany with the po­ten­tial to sig­ni­f­i­cant­ly dis­rupt the cur­rent cell ther­a­py land­s­cape. Mark­er's cur­rent Mul­ti­TAA cell ther­a­py is look­ing to ini­ti­ate a Phase II study in AML in 2019. Ad­di­tio­n­al­ly, TPIV200, a T-cell vaccine is cur­rent­ly in Phase 2 for ovarian can­cer and tri­ple-neg­a­tive breast can­cer. [more in­for­ma­tion]
Medigene AG [MDG1:DB] €202 MM MCap
blue­bird bio col­lab; BLUE ini­ti­at­ing trial w/MDG MAGEA-4 can­di­date in 2020. TCRs tar­get­ing HA-1 & MAGE-A1 in de­vt. . DC vax in on­go­ing Ph 1/2; fi­nal read­out in 2019. [more in­for­ma­tion]
Medivir AB [MVIR.B-SE]
Late stage broad pipe­line: remeti­no­s­tat, Ph 3 ready top­i­cal HDAC in­hibi­tor for ear­ly-stage cu­ta­neous T-cell lym­pho­ma; MIV-711, cathepsin K in­hibi­tor for OA, Ph 2 da­ta showed dis­ease mod­i­fy­ing ef­fect on knee struc­ture; birini­pant, bi­va­lent SMAC mimet­ic read out pos­i­tive Ph 1/2 com­bo (keytru­da) da­ta Oct 18: con­firmed PR, good safe­ty. [more in­for­ma­tion]
MEI Pharma, Inc. [MEIP] US$217 MM MCap
4 clin-stage can­di­dates, in­cl 1 in on­go­ing glob­al reg­is­tra­tion trial (pra­ci­no­s­tat), another (ME-401) en­ter­ing Ph 2; in­tends to sub­mit to FDA to sup­port ac­cel­er­at­ed ap­pro­val mar­ket­ing ap­pli­ca­tion. Can­di­dates tar­get dif­fer­ent mech­anisms crit­i­cal to over­com­ing can­cer pro­gres­sion and drug re­sis­tance. [more in­for­ma­tion]
Minerva Neurosciences, Inc. [NERV] US$211 MM MCap
Ph 3 tar­get­ing neg symp­toms as­soc w/ schi­zophre­nia; to­p­line re­sults exp 4Q19. 3 Ph 2b trials on­go­ing w/Janssen MDD/in­som­nia. (+) to­p­line Ph 2 b da­ta w/20mg dose; in­som­nia read­out exp 2Q19 [more in­for­ma­tion]
Modus Therapeutics AB
A se­cond for­mu­la­tion, a sub­cu­ta­neous for­mu­la­tion, will be a first-in class op­tion for SCD pa­tients and will al­low dos­ing at home. By the end of 2018 we ex­pect that the Phase II IV study will be ful­ly en­rolled and a Phase I PK study for the SC op­tion will al­so be com­plete. [more in­for­ma­tion]
MolMed [MLM:MI] €179 MM MCap
Owns one of the first au­tho­rized cel­lu­lar ther­a­pies (Zal­moxis®, first pt-spe­cif­ic cell ther­a­py for treat­ment of adult pa­tients af­fect­ed by leukaemia or other high-risk hae­ma­to­log­i­cal ma­lig­nan­cies) au­tho­rized by EMA for all EU Coun­tries, as well as on­ly fa­cil­i­ty au­tho­rized in EU for com­mer­cial ex-vi­vo cell & gene prod­ucts (Strimvelis and Zal­moxis) man­u­fac­tur­ing, serv­ing rel­e­vant int'l part­n­ers, e.g. [more in­for­ma­tion]
Motif Bio plc [MTFB] US$30 MM MCap
PD­U­FA date: Feb 13, 2019 for Antibi­ot­ic Iclaprim. Two Phase 3 trials in pts w acute bac­te­rial skin and skin struc­ture in­fec­tions (ABSS­SI) achieved pri­mary and se­condary end­points. Com­pared to SoC (van­comycin), no ob­served nephro­tox and a fixed dose. Po­ten­tial other in­di­ca­tions in­clude HABP/VABP and S. au­reus lung in­fec­tions in CF (or­phan). [more in­for­ma­tion]
Nanobiotix SA [NANO:PA] €231 MM MCap
Nano­bi­otix is a lead­ing, clin­i­cal-stage nanomedicine com­pany pi­oneer­ing new ap­proach­es to sig­ni­f­i­cant­ly change pa­tient out­comes by bring­ing nano­physics to the heart of the cell. Nano­bi­otix’s Im­muno-On­col­o­gy pro­gram has the po­ten­tial to bring a new di­men­sion to can­cer im­munother­a­pies. [more in­for­ma­tion]
NeuroproteXeon
The blurb: NPXe is a phase III phar­ma­ceu­ti­cal and drug de­liv­ery de­vice com­pany which is de­vel­op­ing XENEXTM (xenon gas for in­ha­la­tion) for post car­di­ac ar­rest syn­drome. [more in­for­ma­tion]
NeuroRx
De­vel­op­ing first Glx tar­get­ed an­tide­pres­sant to ad­dress sui­ci­dal­i­ty in bipo­lar de­pres­sion. Lead can­di­date, NRX-101 in Ph 2b/3 de­vt (Break­thru Desig­na­tion, SPA and Fast Track Desig­na­tion by FDA) for Se­vere Bipo­lar De­pres­sion in pts w Acute Sui­ci­dal Idea­tion and Be­havior. Plans to pre­sent Ph 2 POC da­ta lat­er this year. [more in­for­ma­tion]
NexImmune, Inc.
Anti­gen-spe­cif­ic T cell ther­a­py. Syn­thet­ic nano­par­ti­cles de­c­o­rat­ed w a com­bo of mul­ti­ple tu­mor rel­e­vant anti­gens and co-sti­m­u­la­to­ry sig­nals en­gag­ing di­rect­ly w tar­get­ed T cells. Mul­ti­ple IND fil­ings exp H1'19. [more in­for­ma­tion]
Oncimmune [ONC:LN] £59 MM MCap
Two mar­ket­ed prod­ucts, Ear­ly­CDT®-Lung launched in 2012 and Ear­ly­CDT®-Liv­er launched in 2018, and a broad ad­vanced pipe­line of ear­ly can­cer de­tec­tion tests, in­cl. pros­tate, breast and ovarian. 156,000 Ear­ly­CDT®-Lung tests sold since launch) and Chi­na. broad and com­pre­hen­sive pa­tent port­fo­lio. [more in­for­ma­tion]
Oncopeptides AB [ONCO:ST] SKr7,492 MM MCap
Melflufen (Yga­lo): alky­lat­ing pep­tide se­lec­tive­ly tar­gets MM; su­pe­ri­or ef­fi­ca­cy vs. SOC. In­ter­im da­ta at EHA (2018) from on­go­ing Ph2 study in late stage r/r MM showed ORR of ~32%. [more in­for­ma­tion]
Oryzon Genomics [ORY:MA]
Tar­get­ing ly­sine spe­cif­ic demethy­lase 1 (LS­D1, aka KD­M1A). Lead CNS com­pound in 3 Phase 2a trials in MS, AD and bas­ket trial. Onc com­pound in 2 com­bo Phase 2a trials in AML and SCLC. Da­ta exp mid-2019 [more in­for­ma­tion]
Oventus Medical
In 2013, worked with the CSIRO to de­vel­op pa­tent­ed 3D print­ed air­way tech­nol­o­gy clin­i­cal­ly proven to de­liv­er sig­ni­f­i­cant clin­i­cal ad­van­tages over ex­ist­ing treat­ments with po­ten­tial to help mil­lions of snor­ers and sleep ap­nea suf­fer­ers in a fast-grow­ing multi­bil­lion dol­lar mar­ket with FDA clear­ance and ex­ist­ing re­im­burse­ment codes launch­ing in North Amer­i­ca Q1 2019. [more in­for­ma­tion]
PAION AG [PAIOF] US$153 MM MCap
Ph 3 study in pro­ce­du­ral se­da­tion in US. Safe­ty da­ta in ASA III/IV pa­tients in Ph 3 colonos­copy study and Ph 3 bron­chos­copy study. US fil­ing exp by part­n­er Cos­mo Phar­ma­ceu­ti­cals in 4Q18/1Q19. Ini­ti­at­ed Ph 3 in gen­er­al anes­th­e­sia in EU in Ju­ly 2018, exp com­ple­tion 2019. Fa­vor­able safe­ty pro­file of mi­da­zo­lam and at­trac­tive half-life of propo­fol: best of both in­dus­try stan­dards. [more in­for­ma­tion]
Perrigo Company [PRGO] US$6,496 MM MCap
Per­ri­go Com­pany is ded­i­cat­ed to mak­ing lives bet­ter by bring­ing high Qual­i­ty, Af­ford­able Self-Care Prod­ucts™ that con­sumers trust ev­ery­where they are sold. The Com­pany is a lead­ing provider of over-the-coun­ter health and well­ness so­lu­tions that en­hance in­di­vi­d­u­al well-be­ing by em­pow­er­ing con­sumers to proac­tive­ly pre­vent or treat con­di­tions that can be self-ma­n­aged. [more in­for­ma­tion]
PharmaMar SA [PHM:ES]
2018 to­tal rev­enues of &eu­ro;162M. Yon­delis (soft tis­sue sar­co­ma and ovarian can­cer) com­mer­cial­ized w di­rect sales-force in EU and part­n­er­ship in US w Janssen. Zep­syre in Ph 3 trials for SCLC - da­ta due 2H19. MOA: Op­ti­mized ana­log of Yon­delis. [more in­for­ma­tion]
Pharming Group NV [PHARM:AS] €492 MM MCap
On­ly prod­uct with fu­ture po­ten­tial to be ap­proved for both pro­phy­laxis and treat­ment of at­tacks of HAE. An­nounced pos­i­tive re­sults from a ph2 study of RU­CON­EST in Con­tract-in­duced Nephro­pa­thy for pa­tients re­ceiv­ing elec­tive coro­nary an­giog­ra­phy with or with­out a per­cu­ta­neous coro­nary in­ter­ven­tion (PCI). [more in­for­ma­tion]
Pharvaris
Phar­varis is a pre­clin­i­cal stage com­pany fo­cused on bring­ing an oral bradykinin B2 re­cep­tor an­ta­g­on­ist to pa­tients for use as an al­ter­na­tive to in­ject­ed ther­a­pies for hered­i­tary an­gioede­ma (HAE) and other B2 re­cep­tor-me­di­at­ed in­di­ca­tions. [more in­for­ma­tion]
PhaseBio Pharmaceuticals, Inc. [PHAS] US$303 MM MCap
De­vel­op­ing ther­a­pies for treat­ment of or­phan dis­eas­es; Al­so de­vel­op­ing PB1046, a week­ly va­soac­tive in­testi­nal pep­tide re­cep­tor ag­on­ist for treat­ment of PAH. PB1046 lev­er­ages pro­pri­e­tary elastin-like po­lypep­tide (ELP) biopo­ly­mer tech­nol­o­gy plat­form to de­vel­op ther­a­pies with po­ten­tial for less-fre­quent dos­ing and bet­ter pa­tient com­pliance. [more in­for­ma­tion]
Phosphagenics [PPGNY] US$1 MM MCap
Plat­form en­hances us­a­bil­i­ty char­ac­teris­tics for in­jecta­bles, patch and gel prod­ucts. Part­n­er­ships w No­var­tis and Themis: Do­clofenac Gel for Pain and in­flam­ma­tion due to mus­cle, ten­don and joint in­juries; mar­ket­ed in 16 coun­tries. Teru­mo part­n­er­ship: Propo­fol in­jec­tion; My­lan part­n­er­ship: Dap­to­mycin Lyophil­ized Pow­der for IV In­jec­tion. [more in­for­ma­tion]
Photocure ASA
op­ti­cal imag­ing agent for im­proved de­tec­tion, re­duced dis­ease re­cur­rence and pro­gres­sion rates of blad­der can­cer. Re­cent­ly an­nounced FDA ap­pro­val of sN­DA to ex­tend in­di­ca­tion to in­clude Flex­i­ble Cy­to­s­copes for on­go­ing surveil­lance of blad­der can­cer. The com­pany is fo­cused on grow­ing Cysview rev­enues in the US and re­port­ed rev­enue growth in the US of 45% year to date 3Q18. [more in­for­ma­tion]
Pieris Pharmaceuticals, Inc. [PIRS] US$184 MM MCap
An­ti­calins (en­gi­neered lipo­calin pro­teins) for re­sp & on­co dis­eas­es. Im­mi­nent da­ta for AZN-part­nered PRS-060 in­haled IL4 for mod-to-se­vere asth­ma. Cash of $128MM in the bank as of March 2019. [more in­for­ma­tion]
Pliant Therapeutics, Inc.
Lead pro­gram is a small molecule du­al se­lec­tive in­hibi­tor of avb6/avb1 in­te­grins for IPF, pri­mary scle­ros­ing cholan­gi­tis (PSC), and re­nal fi­bro­sis - re­ceived or­phan drug desig­na­tion and will en­ter Ph1 in De­cem­ber 2018. Se­cond pro­gram is a se­lec­tive avb1 in­hibi­tor for the treat­ment of end stage liv­er fi­bro­sis and has en­tered in­to de­vel­op­ment can­di­date se­lec­tion. [more in­for­ma­tion]
Poxel SA [POXEL:PA] €181 MM MCap
Imeglimin (mi­to­chon­dria-based MOA) for T2D be­gan Ph 3 in Ja­pan with Asian part­n­er Su­m­i­t­o­mo Dainip­pon. Com­ple­tion of Phase 3 on track for 2019 and JN­DA sub­mis­sion in 2020. In 1Q18, an­nounced agree­ment w Roi­vant for Imeglimin (Ph 3 ready) in US, EU, and ad­dl coun­tries WW. [more in­for­ma­tion]
Protagonist Therapeutics [PTGX] US$256 MM MCap
PTG-300, in­jectable hep­cidin mimet­ic in Ph 2 Be­ta-Tha­lassemia study with ini­tial re­sults ex­pect­ed 2H19. PTG-200, part­nered w Janssen, oral GI-re­strict­ed IL-23R an­ta­g­on­ist for Crohn’s dis­ease com­plet­ed Ph 1 in Novem­ber 2018. PN-943, oral GI-re­strict­ed al­pha4­be­ta7 in­te­grin-spe­cif­ic an­ta­g­on­ist in UC in Ph 1, with to­p­line re­sults ex­pect­ed 1H19. [more in­for­ma­tion]
Protalix BioTherapeutics [PLX] US$59 MM MCap
Lead as­set in mul­ti Ph 3 trials for Fab­ry dis­ease, part­nered glob­al­ly w Chie­si - In­ter­im Ph 3 (PRX-102) da­ta: im­prove­ment in kid­ney func­tion in pts switched from Re­pla­gal®. Ad­dl Ph 2 (in Cys­tic Fi­bro­sis) [more in­for­ma­tion]
PsiOxus Therapeutics
The ther­a­py (NG-348), us­es virus to tran­s­port two ther­a­peu­tic genes di­rect­ly in­to tu­mors to re­cruit im­mune cells in­to at­tack­ing tar­get­ed can­cer cells. Up­com­ing Mile­s­tones: - 2 pro­grams in clin­ic with read­outs in 2019 (ovarian can­cer in comb w Pa­cl­i­tax­el, car­ci­no­mas in comb w nivolumab) - 2 INDs in 2018 (NG-348 and NG-350A) - 2 INDs in 2019 (NG-641 and NG-347) [more in­for­ma­tion]
Pulse Biosciences, Inc. [PLSE] US$257 MM MCap
Med de­vice: Nano-Pulse Elec­tro-Sig­nal­ing [more in­for­ma­tion]
Quentis Therapeutics
Lead pro­gram is a first-in-class IRE1&al­pha; in­hibi­tor de­signed to boost an­ti-tu­mor im­mu­ni­ty in can­cer. Quen­tis is pur­su­ing ther­a­pies to ad­dress mul­ti­ple ER stress path­way tar­gets in the tu­mor mi­cro-en­vi­ron­ment, as well as in other dis­eas­es where ER stress plays an im­por­tant role. Quen­tis launched in Fe­bruary 2018 with a $48 mil­lion Se­ries A fi­nanc­ing. [more in­for­ma­tion]
ReNeuron Group plc [RENE:LN] £91 MM MCap
Clin-stage, UK-based cell ther­a­py; al­lo­gene­ic, off-the-shelf can­di­dates: chron­ic stroke dis­a­bil­i­ty (Ph3 de­sign ap­proved by FDA); re­tini­tis pig­men­tosa (Ph1/2 on­go­ing), cone rod dys­tro­phy (Ph2 ini­ti­a­tion); PC ex­o­some nanomedicine plat­form pro­gram, first in­di­ca­tion GMBM. GMP com­mer­cial scale cell pro­duc­tion ca­pa­bil­i­ty. [more in­for­ma­tion]
Rigel Pharmaceuticals [RIGL] US$338 MM MCap
The com­pany's first FDA ap­proved prod­uct is TA­VALISSE™ (fos­ta­ma­tinib di­s­odi­um hex­ahy­drate), an oral spleen ty­ro­sine ki­nase (SYK) in­hibi­tor, for the treat­ment of adult pa­tients with chron­ic im­mune throm­bo­cy­tope­nia who have had an in­suf­fi­cient re­sponse to a pre­vi­ous treat­ment. [more in­for­ma­tion]
Savara Inc. [SVRA] US$421 MM MCap
Or­phan lung dis­ease; Mol­gradex, Ph 3, in­haled GM-CSF: au­toim­mune pul­mo­nary alve­o­lar pro­tei­no­sis (aPAP), Ph 2a: non­tu­ber­cu­lous my­cobac­te­ria (NTM) lung in­fec­tion [more in­for­ma­tion]
Selvita S.A
PIM/FLT3 ki­nase in­hibi­tor in Ph 1/2 in AML (part­nered w Me­nari­ni) and sm molecule se­lec­tive CD­K8 in­hibi­tor for leukemias, lym­pho­mas, and solid tu­mors: Ph 1/2 ini­ti­a­tion exp H1 2019 [more in­for­ma­tion]
Sernova [SEOVF] US$31 MM MCap
Ser­no­va Corp. is a clin­i­cal-stage re­gen­er­a­tive medicine com­pany com­mer­cial­iz­ing prod­ucts which ben­e­fit pa­tients with chron­ic metabolic, neu­ro­log­i­cal and he­ma­to­log­i­cal dis­eas­es with first ther­a­peu­tic ap­pli­ca­tions in di­a­betes and he­mophilia. The com­pany is cur­rent­ly con­duct­ing a PI/II trial in di­a­bet­ic pa­tients with Hy­po­g­lycemia un­aware­ness. [more in­for­ma­tion]
Solebury Capital
Sole­bury Cap­i­tal LLC is a glob­al ad­vi­so­ry firm and reg­is­tered bro­k­er deal­er ex­clu­sive­ly fo­cused on the equi­ty cap­i­tal mar­kets. We pro­vide our cor­po­rate and fi­nan­cial spon­sor clients with in­de­pen­dent ad­vice, ex­pe­ri­enced judg­ment and tran­s­ac­tion ma­n­age­ment ser­vices – em­pow­er­ing them to make more ful­ly in­formed de­ci­sions at all points in the un­der­writ­ing pro­cess. [more in­for­ma­tion]
Solebury Trout
With al­most two de­cades of ex­pe­ri­ence in the biotech­nol­o­gy, phar­ma­ceu­ti­cal, med­i­cal tech­nol­o­gy and life sci­ence tech­nol­o­gy sec­tors, the Trout Group of­fers its clients the knowl­edge base need­ed to clar­i­fy in­vest­ment themes and lev­er­age key re­la­tion­ships for in­creased ex­po­sure to the prop­er au­di­ence. [more in­for­ma­tion]
Stemline Therapeutics, Inc. [STML] US$661 MM MCap
EL­ZON­RIS, tar­get­ed ther­a­py di­rect­ed to IL-3 re­cep­tor (CD123, ap­proved for BPDC; SL-801 an oral small molecule re­v­ersi­ble in­hibi­tor of XPO1 in Ph 1 trial; and SL-701 an im­munother­a­peu­tic that com­plet­ed Ph 2 trial in pts w/2nd line GBM [more in­for­ma­tion]
STRATEC Biomedical AG [SBS:FF] €757 MM MCap
Au­to­ma­tion sys­tems and soft­ware for di­ag­nos­tics in­dus­try. In­s­talled base of >13,000 sys­tems across 20 fam­i­lies in­clud­ing PAN­THER (Ho­log­ic), Li­a­son XL (Di­a­Sorin), VI­DAS (BioMerieux) and AD­VIA (Sie­mens). Rev gen­er­at­ing and pro­f­itable; growth drivers in­cl prod­uct launch­es/ramp-ups, new tests; de­mo­graph­ics and out­sourc­ing in IVD in­dus­try. [more in­for­ma­tion]
Strongbridge Biopharma [SBBP] US$161 MM MCap
Fo­cused on rare dis­eas­es; Keveyis for PPP 2019 rev­enue gui­dance of $18-20M; Re­cor­lev 2nd Phase 3 top-line re­sults by end of 1Q 2020 with NDA sub­mis­sion planned for 3Q 2020 [more in­for­ma­tion]
Sutro Biopharma [STRO] US$242 MM MCap
Fo­cused on de­vel­op­ing next gen­er­a­tion anti­body drug con­ju­gates (ADCs) and mul­ti-spe­cif­ic anti­body-based ther­a­peu­tics for can­cer ther­a­py. STRO-001 for mul­ti­ple myelo­ma and lym­pho­mas is in Ph 1 and ex­pects ini­tial safe­ty da­ta in mid-2019. IND sub­mis­sion for STRO-002 for ovarian and en­dome­trial can­cer in Q4 2018 with the ini­ti­a­tion of a Ph 1 trial planned for ear­ly 2019. [more in­for­ma­tion]
SutroVax
Gen­er­at­ed pre-clin­i­cal proof-of-con­cept with its PCV in head-to-head studies against cur­rent vaccines and has ad­vanced the pro­gram in­to IND-en­abling de­vel­op­ment to de­mon­s­trate proof-of-con­cept in adults and chil­dren. [more in­for­ma­tion]
Taiwan Liposome Company [TLC] US$173 MM MCap
TLC is a clin­i­cal-stage spe­cial­ty phar­ma­ceu­ti­cal com­pany ded­i­cat­ed to the de­vel­op­ment and com­mer­cial­iza­tion of nov­el nanomedicines that com­bine pro­pri­e­tary lipid-as­sem­bled drug de­liv­ery plat­forms with ap­proved ac­tive phar­ma­ceu­ti­cal in­gre­di­ents (APIs). All pro­grams ex­pect­ed to be in late or piv­o­tal stage by the end of 2020. [more in­for­ma­tion]
Targovax [TRVX:OS] NKr421 MM MCap
En­gi­neered on­co­lyt­ic virus armed w im­mune-sti­m­u­lat­ing trans­genes for pts w solid tu­mors. Ph 1 and Ph 1/2 trials on­go­ing with mul­ti­ple da­ta read­outs in 2019-2020. CMO de­vel­oped Yer­voy at BMS. [more in­for­ma­tion]
Tessa Therapeutics
On­go­ing Ph III – (na­sopha­ryn­geal can­cer), Ph I – (cer­vi­cal, mov­ing in­to Ph II); mul­ti­ple other as­sets en­ter­ing clin­ic. Key col­labs: Bay­lor, Park­er Inst, St. Jude Chil­dren’s [more in­for­ma­tion]
The Jackson Laboratory
Found­ed in 1929, The Jack­son Lab­o­ra­to­ry has over 9 de­cades of ex­per­tise in hu­man and mice ge­net­ics. With ev­er-in­creas­ing pre­ci­sion, The Jack­son Lab­o­ra­to­ry iden­ti­fies the ge­net­ic and molec­u­lar bas­es of dis­ease and mar­shals its strengths in ge­nomics and mod­el­ing to dis­cov­er in­di­vi­d­u­al­ized treat­ments and cures. [more in­for­ma­tion]
Valbiotis [AVAL-FR]
Lead pro­gram VALE­DIA®, tar­get­ing the un­ad­dressed pre­di­a­betes mar­ket, is cur­rent­ly in a Ph 2a trial for the re­duc­tion of risk fac­tors for de­vel­op­ing Type 2 di­a­betes with re­sults ex­pect­ed mid 2019. [more in­for­ma­tion]
Vericel Corporation [VCEL] US$690 MM MCap
Ad­vanced cell ther­a­py; mar­ket­ing MA­CI (car­ti­lage re­pair) and Epi­cel (skin re­place­mentt) in TAMs of $2b+. Re­cent­ly ac­quired US com­mer­cial rights for Nexo­Brid. [more in­for­ma­tion]
Verseon [VSN-LON]
In-house drug dis­cov­ery plat­form, built w 10+ years of R&D. Po­ten­tial­ly first sys­te­m­at­ic, com­pu­ta­tio­n­al­ly-driv­en so­lu­tion to achieve molec­u­lar mod­elling ac­cu­ra­cy ne­ces­sary for rapid /cost-ef­fec­tive drug dis­cov­ery. Drug pro­grams in­clude an­ti­co­ag­u­la­tion, di­a­bet­ic mac­u­lar ede­ma, hered­i­tary an­gioede­ma, and solid tu­mors. [more in­for­ma­tion]
Veru Inc. [VERU] US$110 MM MCap
Zu­clomiphene in Ph 2 treat­ing hot flash­es caused by hor­mone treat­ment for pros­tate can­cer. Ad­vanc­ing 4 new drug for­mu­la­tions in urol­o­gy: Ta­dala­fil/fi­nas­teride com­bo tablets for symp­toms of BPH and erec­tile dys­func­tion (NDA fil­ing exp 2H19). Tam­su­losin DRS gran­ules and XR cap­sules for be­nign pro­stat­ic hy­per­pla­sia (BPH) treat­ment w/o food ef­fect (NDA fil­ing exp 2019). [more in­for­ma­tion]
Viela Bio
Lead pro­gram, Ine­bi­l­izumab, is a CD19 mAb which met the pri­mary end­point in the Ph 2b piv­o­tal study for the treat­ment of Neu­romyeli­tis Op­ti­ca. Pri­mary anal­y­sis de­mon­s­trat­ed a 77% re­duc­tion in the risk of de­vel­op­ing NMOSD at­tack in pa­tients treat­ed with ine­bi­l­izumab monother­a­py com­pared to place­bo. [more in­for­ma­tion]
vTv Therapeutics, Inc. [VTVT] US$95 MM MCap
Eval­u­at­ing TT­P399 as an add-on ther­a­py to in­sulin ther­a­py in a 12 week phase 1/2 trial in pa­tients with type 1 di­a­betes. An­a­lyz­ing re­cent Azeli­ra­gon phase 3 da­ta in or­der to move de­vel­op­ment of the ther­a­py for­ward. Re­sults from a sub­group anal­y­sis of the STEAD­FAST Alzheimer’s trial to be pre­sent­ed at CTAD dis­cuss­ing the ef­fect of Azeli­ra­gon in pa­tients with De­men­tia and Di­a­betes. [more in­for­ma­tion]
Wave Life Sciences Ltd. [WVE] US$854 MM MCap
Top-line da­ta from first 3 clin­i­cal pro­grams, 1 in DMD and 2 in Hunt­ing­ton’s dis­ease, exp 2019. Da­ta read­outs from ad­dl pro­grams in DMD, ALS, and fron­totem­po­ral de­men­tia exp in 2020. Lev­er­ag­ing plat­form to ad­vance pro­grams across oph­thal­mol­o­gy and he­p­a­tol­o­gy. [more in­for­ma­tion]
Yisheng Biopharma
Prod­ucts in de­vt in­clude YS-ON-001 (Ph 1), an IO prod­uct tar­get­ing solid tu­mors and new gen of bi­o­log­ics for pre­ven­tive and ther­a­peu­tic ben­e­fits, in­cl YS-HBV-001 (Ph 1) and PI­KA ra­bies vaccine (Ph 3), tar­get­ing hep B and ra­bies in­fec­tions. PC pro­grams in­cl bi­o­log­ics tar­get­ing in­fluen­za, her­pes zoster, ebo­la, et al. 2 bi­o­log­ics prod­ucts on mkt. [more in­for­ma­tion]
ZIOPHARM Oncology Inc [ZIOP] US$687 MM MCap
Cur­rent­ly en­rolling pa­tients in a phase 1 trials of Ad-RTS-hIL-12 as monother­a­py and in com­bi­na­tion with OP­DI­VO for the treat­ment of rGBM. [more in­for­ma­tion]