Acceleron Pharma Inc [XLRN] US$5,941 MM MCap

The com­pa­nies have mul­ti­ple Ph2 and Ph3 trials on­go­ing for chron­ic ane­mia in ad­di­tio­n­al pa­tient pop­u­la­tions of MDS and be­ta-tha­lassemia, as well as myelo­fi­bro­sis. The com­pany ex­pects to re­port to­p­line re­sults from Ph2 clin­i­cal trials in PAH (so­tater­cept) and CMT (ACE-083) in Q1 2020. [more in­for­ma­tion]

AcelRx Pharmaceuticals, Inc. [ACRX] US$85 MM MCap

Launched DSU­VIA (sufen­tanil sublin­gual tablet 30 mcg) in the US in 1Q19 for acute pain se­vere enough to re­quire opi­oid anal­gesic (adult pa­tients in cer­ti­fied med­i­cal­ly su­per­vised health­care sett­ings). Al­so de­vel­op­ing Za­lvi­so (Ph 3 com­plet­ed) in the US as an in­no­va­tive pa­tient-con­trolled anal­ge­sia sys­tem for mod­er­ate-to-se­vere acute pain in med­i­cal­ly su­per­vised sett­ings. [more in­for­ma­tion]

Achilles Therapeutics

cNeT (clo­n­al neoanti­gen) pro­cess builds on TILs, com­bined with abil­i­ty to iden­ti­fy pt-spe­cif­ic clo­n­al neoanti­gens and gen­er­ate per­so­n­al­ized T-cell ther­a­pies. Sep19 GBP100 mil­lion Se­ries B w/ RA, Syn­co­na, For­bion, In­vus, Per­cep­tive + Red­mile. [more in­for­ma­tion]

Adrenomed AG

Car­dio­vas­cu­lar and Sep­sis, Adre­cizumab Ph 2, bio­mark­er-guid­ed, mon­o­clo­n­al anti­body for sep­tic shock; to­p­li­nee da­ta re­leased late Feb. Adre­cizumab showed low­er all-cause mor­tal­i­ty for treat­ed pts com­pared to place­bo (28% mor­tal­i­ty rate in 28-day fol­low-up). De­tailed da­ta com­ing in fol­low­ing few months. [more in­for­ma­tion]

Affibody

Pro­pri­e­tary Af­fi­body® molecules and Al­bu­mod plat­forms. Lead can­di­date is an IL-17 with pos­i­tive Ph2 12wk da­ta in pso­ri­a­sis. Al­so has a Ph1 FcRn for b-cell au­toim­mune dis­eas­es part­nered with ALXN. Rev­enue gen­er­at­ing from ear­li­er li­censed as­sets. Backed by In­ves­tor AB. [more in­for­ma­tion]

Affimed N.V. [AFMD] US$336 MM MCap

NK cell en­gager AFM13 (CD30/CD16A) to en­ter a Phase 2 reg­is­tra­tion study in CD30-pos­i­tive pe­ripher­al T cell lym­pho­ma and an in­vesti­ga­tor-spon­sored study by MDACC of AFM13 com­bined with al­lo­gene­ic NK cells in re­lapsed/re­frac­to­ry CD30-pos­i­tive lym­pho­ma. [more in­for­ma­tion]

Aileron Therapeutics, Inc. [ALRN] US$44 MM MCap

Clin­i­cal stage on­col­o­gy com­pany con­duct­ing 3 clin­i­cal trials for its lead can­di­date, AL­RN-6924, a first-in-class/best-in-class drug that tar­gets the p53 path­way. AL­RN-6924 is from the Sta­pled pep­tide plat­form and is the on­ly clin­i­cal drug can­di­date that binds equipo­tent­ly to both of the p53 sup­pres­sor pro­teins, MD­MX and MD­M2. [more in­for­ma­tion]

AM-Pharma B.V.

Treat­ing sep­sis as­so­ci­at­ed acute kid­ney in­jury (AKI) with pro­pri­e­tary re­com­bi­nant al­ka­line phos­pha­tase (re­cAP). Re­sults from Ph 2 trial in 300+ pts, de­mon­s­trat­ed rel­a­tive im­prove­ment of 40%+ OS in re­cAP group. Ini­ti­at­ing Ph 3 in Q3 2020 – in­ter­im look (n=400) exp Q3 2021 (P end­point: Day 28 all-cause mor­tal­i­ty). [more in­for­ma­tion]

Amarin Corporation [AMRN] US$2,560 MM MCap

Com­mer­cial­iz­ing Vas­ce­pa, the first 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. 2019 gui­dance of $380-420M, or 66-83% high­er y/y. [more in­for­ma­tion]

Antev

Phase 3 ready, Tevere­lix, a long act­ing in­jectable pep­tide GnRH an­ta­g­on­ist. Lead in­di­ca­tion for pros­tate can­cer, fol­lowed by be­nign pro­stat­ic hy­per­pla­sia (BPH), acute uri­nary re­ten­tion (AUR), uterine fi­broids and en­dometrio­sis. Over 450 sub­jects have been dosed over 850 times to date with no se­ri­ous ad­verse re­ac­tions and good lo­cal tol­er­ance. [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$105 MM MCap

Ap­to­rum is Hong Kong based, US list­ed com­pany (Mcap $100m) fo­cused on gen­er­at­ing late stage clin­i­cal can­di­dates by de­vel­op­ing al­ready ap­proved drugs for new or­phan in­di­ca­tions, as well as in­fec­tious dis­eas­es. Pipe­line – • SACT-1 for Neu­roblas­to­ma – ph2/3 trial planned for 2H’20 • SACT-COV­19 – COVID-19 treat­ment - pre­clin­i­cal [more in­for­ma­tion]

Arcellx

Clin­i­cal stage de­vel­op­ment of Anti­gen Re­cep­tor Com­plex T-cells (ARC-T) that are read­i­ly si­lenced, ac­ti­vat­ed and re­pro­grammed in vi­vo by ad­min­is­tra­tion of a tu­mo-tar­get­ing anti­gen pro­tean called a sparX. Backed by NEA, No­vo, SR One, Take­da, So­las­ta, Quan, Clough, Mi­rae, JVC and LG. [more in­for­ma­tion]

AsclepiX Therapeutics

AX­T107 de­mon­s­trat­ed ef­fi­ca­cy and dura­bil­i­ty in rab­bit and mouse mod­els that is su­pe­ri­or to stan­dard of care Eylea. [more in­for­ma­tion]

Assembly Biosciences, Inc. [ASMB] US$753 MM MCap

Tar­get­ing HBV w/ oral core in­hibi­tors - po­ten­tial to in­crease cure rates. Lead can­di­date 731 in Ph 2: +ive safe­ty and de­creas­es in cc­cD­NA pop with pts ex­pect­ed to be with­drawn from treat­ment in 2020 and fol­lowed for sus­tained vi­ral DNA sup­pres­sion off ther­a­py. [more in­for­ma­tion]

AxoGen [AXGN] US$434 MM MCap

Da­m­age or tran­sec­tion to pe­ripher­al nerves. Q1 revs $23.3M, 35% growth YoY; exp 2019 rev bt $109-$114M [more in­for­ma­tion]

Bavarian Nordic A/S [BVNRY] US$1,506 MM MCap

Vaccines for in­fec­tious dis­eas­es and on­col­o­gy. [more in­for­ma­tion]

Bellicum Pharmaceuticals Inc. [BLCM] US$37 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]

Bellus Health [BLU] US$186 MM MCap

BEL­LUS' lead prod­uct can­di­date, BLU-5937, is be­ing de­vel­oped for the treat­ment of chron­ic cough (Phase II an­nounced Ju­ly 2020) and chron­ic pru­ri­tus (Phase II planned to be­gin 1H20). [more in­for­ma­tion]

BerGenBio [BGBIO:OS] NKr3,413 MM MCap

De­vel­op­ing first-in-class se­lec­tive Axl ki­nase in­hibi­tors for liquid and solid can­cers, lead can­di­date is bem­cen­tinib (BG­B324) in four com­pany-spon­sored trials. [more in­for­ma­tion]

Biohaven Pharmaceuticals, Inc [BHVN] US$4,340 MM MCap

NURTEC® ODT ap­proved in 1Q20. To­p­line re­sults ex­pect­ed 1Q20 in Ph 3 study of tro­r­ilu­zole for mi­graine pre­ven­tion. To­p­line re­sults ex­pect­ed 2Q20 in Ph 2/3 study of tro­r­ilu­zole for OCD. ~$600M to­tal in cash on hand, in­clud­ing re­cent $282.3M raise to sup­port NURTEC ODT launch. [more in­for­ma­tion]

BiomX [PHGE] US$122 MM MCap

The com­pany's ther­a­peu­tics dis­cov­er and vali­date pro­pri­e­tary bac­te­rial tar­gets, de­vel­op ra­tio­n­al­ly de­signed phage ther­a­pies that seek and de­stroy harm­ful bac­te­ria in mi­cro­biome-re­lat­ed dis­eas­es, en­abling med­i­cal prac­ti­tion­ers to erad­i­cate harm­ful bac­te­ria in chron­ic dis­eas­es. [more in­for­ma­tion]

BioXcel Therapeutics, Inc. [BTAI] US$1,053 MM MCap

BX­CL501 (sublin­gual dexmede­to­mi­dine): Phase 3 trials on­go­ing for the acute treat­ment of ag­i­ta­tion in pa­tients with schi­zophre­nia (SERENI­TY I) and bipo­lar di­s­or­der (SERENI­TY II). Phase 1b/2 trial (TRAN­QUIL­I­TY) on­go­ing for the acute treat­ment of ag­i­ta­tion as­so­ci­at­ed with ge­ri­a­tric de­men­tia. [more in­for­ma­tion]

CASI Pharmaceuticals Inc. [CASI] US$235 MM MCap

Launched EVOMELA® (mel­pha­lan for in­jec­tion, mul­ti­ple myelo­ma) in Au­gust 2019, on­ly mel­pha­lan com­mer­cial­ly avai­l­able in Chi­na; ad­vanc­ing de­vel­op­ment of an­ti-CD19 T-cell ther­a­py (CNC­T19) in B-NHL and B-ALL and an­ti-CD38 mAb (CID-103) in mul­ti­ple myelo­ma, fo­cused on be­com­ing Chi­na’s ma­jor he­ma­tol­o­gy on­col­o­gy com­pany; [more in­for­ma­tion]

Catabasis Pharmaceuticals Inc [CATB] US$115 MM MCap

Rare dis­ease pipe­line, with lead prod­uct Edasa­lonex­ent in Ph 3 trial in Duchenne mus­cu­lar dys­tro­phy re­gard­less of mu­ta­tion type. North Star Am­bu­la­to­ry As­sess­ment as the pri­mary end­point based on Phase 2 re­sults and FDA in­put. Top-line re­sults ex­pect­ed in 2020. [more in­for­ma­tion]

Catalyst Pharmaceuticals, Inc. [CPRX] US$511 MM MCap

Fir­dapse ap­proved for Lam­bert-Ea­ton Myas­thenic Syn­drome; 3Q19 rev of $30.9M, FY19 gui­dance of ~$102M and FY20 gui­dance of $135M-$155M. Mul­ti­ple key Fir­dapse trial read­outs in 1H20. [more in­for­ma­tion]

Celldex Therapeutics, Inc. [CLDX] US$448 MM MCap

I-O pipe­line of Abs, ADCs, im­mune sys­tem mo­d­u­la­tors and vaccines tar­get­ed to spe­cif­ic pa­tient pop­u­la­tions. STIC da­ta on an­ti-CD40; col­lab with Mer­ck. [more in­for­ma­tion]

Cellular Biomedicine Group, Inc. [CBMG] US$289 MM MCap

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]

CerSci Therapeutics

De­vel­op­ing a new gen­er­a­tion of non-opi­oid medicines to treat acute post-op­er­a­tive and chron­ic neu­ro­path­ic pain. Lead pro­gram in Phase 1 (MAD/SAD/Food Ef­fect), CT-044, tar­get­ing re­ac­tive spe­cies that cause neu­ro­n­al ion chan­nel hy­per-sen­si­ti­za­tion, known to trig­ger pain. [more in­for­ma­tion]

CureVac AG

Cure­vac is a pri­vate Ger­man com­pany with mR­NA plat­form, di­ver­si­fied pipe­line, and three lead pro­grams in ph1: CV8102 (vari­ous can­cer in­di­ca­tions), CV9202 (NS­CLC) and CV7202 (Ra­bies). Last fi­nanc­ing round done at over $1bn. Other com­pa­nies in the space: MR­NA, TBIO, BN­TX. [more in­for­ma­tion]

Enzo Biochem, Inc. [ENZ] US$137 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]

ESSA Pharma Inc. [EPIX] US$129 MM MCap

1Q20 IND sub­mis­sion for lead as­set, EPI-7386, a small molecule tar­get­ing n-ter­mi­nal do­main of an­dro­gen re­cep­tor for mCR­PC. First pa­tient dosed in EPI-7386 Phase 1 study in mCR­PC pa­tients fail­ing se­cond gen­er­a­tion an­tian­dro­gens by 2Q20. [more in­for­ma­tion]

Exicure [XCUR] US$207 MM MCap

Ex­i­cure en­tered in­to a col­lab­o­ra­tion with Al­ler­gan Phar­ma­ceu­ti­cals In­ter­na­tio­n­al Limit­ed in late 2019 and is now ac­tive­ly en­gaged in pre­clin­i­cal re­search and dis­cov­ery in two clin­i­cal pro­grams re­lat­ed to the treat­ment of hair loss di­s­or­ders. [more in­for­ma­tion]

Faraday Pharmaceuticals

El­e­men­tal re­duc­ing agents for crit­i­cal care treat­ment to pre­vent car­di­ac and skele­tal mus­cle loss: lead can­di­date FDY-5301 Ph 3 (piv­o­tal) to ini­ti­ate mid-2020 for reper­fu­sion in­jury fol­low­ing heart at­tack (AMI); da­ta ear­ly 2023. SPA sub­mis­sion re­ceived by FDA Jan 22, 2020 and ini­tial feed­back re­ceived Feb 2020 - Type A meet­ing re­quest, like­ly in April 2020. Sig­ni­f­i­cant ad­di­tio­n­al treat­ment ap­pli­ca­tions with limit­ed to no com­pe­ti­tion. $57M raised to date from Arch Ven­ture Part­n­ers, Po­laris Part­n­ers, WRF, Osa [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]

Freenome Holdings, Inc.

Biotech­nol­o­gy di­ag­nos­tics com­pany uti­l­iz­ing its deep ex­per­tise in molec­u­lar bi­ol­o­gy with ad­vanced com­pu­ta­tio­n­al bi­ol­o­gy and ma­chine learn­ing tech­niques; the com­pany pi­oneered a com­pre­hen­sive mul­ti­omics plat­form for ear­ly can­cer de­tec­tion through a rou­tine blood draw. At AS­CO GI 2020, de­mon­s­trat­ed a sen­si­tiv­i­ty of 94% and spe­ci­fic­i­ty of 94% for ear­ly-stage (I/II) col­orec­tal ade­no­car­ci­no­ma. [more in­for­ma­tion]

Galera Therapeutics [GRTX] US$178 MM MCap

Clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pany fo­cus­ing on trans­form­ing ra­dio­ther­a­py in can­cer. Lead as­set GC4419 is a po­tent, high­ly se­lec­tive small molecule dis­mu­tase mimet­ic be­ing de­vel­oped for se­vere oral mu­cosi­tis, cur­rent­ly in Phase 3 with Break­through Ther­a­py Desig­na­tion. To­p­line re­sults ex­pect­ed in 1H21. Top line re­sults in Phase 1b/2a in se­cond as­set, GC4419 in pan­cre­at­ic can­cer ex­pect­ed in 2H20. GC4419 ex­pect­ed to ini­ti­ate in Phase 1b/2a in lung can­cer in 2H20. [more in­for­ma­tion]

Genfit SA [GNFTF] US$213 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]

HaemaLogiX

Pro­pri­e­tary, dif­fer­en­ti­at­ed B cell tar­gets en­gaged via anti­body and CAR-T pro­grams Kap­paMab & Lamb­daMab tar­get ac­ti­vat­ed plas­ma cell anti­gens (KMA/LMA); Kap­paMab plus RevDex vs. RevDex Ph 2b in MM, da­ta 1H20; Kap­pa CAR-T Ph 1 Q120 in MM; Ca­pac­i­ty for du­al-tar­get­ing via CD-19, BC­MA, etc; Lamb­daMab in pre­clin­i­cal for amy­loi­do­sis [more in­for­ma­tion]

Heidelberg Pharma AG [HPHA:DB] €139 MM MCap

Hei­del­berg Phar­ma AG (WL6, Frank­furt Stock Exchange), based in La­den­burg, Ger­many, is a lead­ing biotech com­pany pi­oneer­ing the use of its AT­AC (Anti­body Tar­get­ed Amanitin Con­ju­gate) plat­form to de­vel­op nov­el can­cer treat­ments with a unique mode of ac­tion ef­fec­tive at spe­cif­i­cal­ly killing both in­ac­tive and di­vid­ing can­cers cells and over­com­ing tu­mour re­sis­tance mech­anisms. [more in­for­ma­tion]

Hutchison China MediTech Ltd ("Chi-Med") [HCM] US$4,045 MM MCap

Chi-Med has a port­fo­lio of 8 drugs in clin­i­cal de­vel­op­ment from own dis­cov­ery en­gine in its In­no­va­tion plat­form and a cash gen­er­at­ing Com­mer­cial plat­form with 2,500+ sales reps. Glob­al col­lab­o­ra­tion with As­traZene­ca in two reg­is­tra­tion en­abling studies with savol­i­tinib in Ta­gris­so re­frac­to­ry lung can­cer that is MET+ and EGFRm+ (glob­al) and MET Ex­on 14 dele­tion NS­CLC (Chi­na). [more in­for­ma­tion]

Impact Therapeutics Inc

Based in Chi­na, IM­PACT is com­mitt­ed to the dis­cov­ery and de­vel­op­ment of best-in-class ther­a­peu­tics to treat can­cer and other life-threat­en­ing dis­eas­es. IM­PACT is a clin­i­cal-stage com­pany with a unique small-molecule drug dis­cov­ery and de­vel­op­ment plat­form tar­get­ing DDR. IM­PACT's lead­ing pro­gram, PARP in­hibi­tor IM­P4297, is cur­rent­ly un­der­go­ing phase I trials in Aus­tralia and Chi­na. [more in­for­ma­tion]

Inspirion Delivery Sciences LLC

Mor­phaBond ER™ US com­mer­cial launch Nov 2017 (FDA-ap­proved, first sin­gle-en­ti­ty abuse de­ter­rent ER mor­phine prod­uct) and Rox­y­Bond™ (FDA ap­proved, first and on­ly IR abuse-de­ter­rent opi­oid prod­uct) both li­censed to Daiichi Sankyo, Inc. Nov­el ex­tend­ed and im­me­di­ate re­lease tech­nolo­gies en­able pain med­i­ca­tions with abuse de­ter­rent fea­tures, ad­dress­ing the Opi­oid epi­dem­ic. [more in­for­ma­tion]

Iovance Biotherapeutics [IOVA] US$4,318 MM MCap

Late-stage cell ther­a­py com­pany uti­l­iz­ing pro­pri­e­tary au­tol­o­gous tu­mor in­fil­trat­ing lym­pho­cytes (TILs) to at­tack solid tu­mors. The com­pany is con­duct­ing piv­o­tal stage trials in me­tastat­ic me­lano­ma and ad­vanced cer­vi­cal can­cer with BLAs ex­pect­ed 2020. [more in­for­ma­tion]

ISA Pharmaceuticals BV

is an im­munother­a­py com­pany de­vel­op­ing ra­tio­n­al­ly de­signed, ful­ly syn­thet­ic im­munother­a­peu­tics against can­cer and per­sis­tent vi­ral in­fec­tions. The Com­pany has built a pro­pri­e­tary im­munother­a­py plat­form based on the Syn­thet­ic Long Pep­tide (SLP®) con­cept and AM­PLI­VANT®tech­nol­o­gy. [more in­for­ma­tion]

Junshi Biosciences [1877:HK]

Fo­cused on de­vel­op­ing ther­a­pies in I-O, au­toim­mune and metabolic dis­eas­es. 13 pipe­line can­di­dates (7 in I-O, 2 in metabolic, 3 in au­toim­mune and 1 in neu­ro­log­i­cal dis­eas­es). JS001, or tori­pal­imab, is ap­proved by the NM­PA and the first mar­ket­ed an­ti-PD-1 mAb anti­body self-de­vel­oped by a PRC com­pany. For full year 2019, tori­pal­imab sales were ap­prox­i­mate­ly $11.1M / 135,525 bot­tles. [more in­for­ma­tion]

Kadmon Holdings, Inc. [KDMN] US$674 MM MCap

An­nounced pos. pri­mary anal­y­sis re­sults (May 2020) in piv­o­tal trial of ROCK in­hibi­tor-KD025 (Belu­mo­sudil) in cGVHD pts re­ceiv­ing 2+ pri­or ther­a­py lines; ORRs of 73% and 74% in 200mg QD and 200mg BID arms, re­spec­tive­ly. Belu­mo­sudil is be­ing re­viewed un­der Re­al-Time On­col­o­gy Re­view (RTOR) Pi­lot Pro­gram by the FDA; NDA sub­mis­sion exp Q4 2020. En­rolling pts in Ph 2 trial (sys. scle­ro­sis). [more in­for­ma­tion]

KAHR medical

KAHR Med­i­cal de­vel­ops nov­el fu­sion pro­tein phar­ma­ceu­ti­cals for the treat­ment of can­cer and au­toim­mune dis­eas­es. [more in­for­ma­tion]

Kaleido BioSciences [KLDO] US$243 MM MCap

Har­ness­ing pro­pri­e­tary small molecule chem­istry plat­form to de­vel­op mi­cro­biome metabolic ther­a­pies (MMT) in in­di­ca­tions span­n­ing I-O, ni­tro­gen metabolism, metabolic dis­eas­es, and CNS. Da­ta ex­pect­ed in 4Q20 from Ph 2 trial (UN­LOCKED) of KB195 in pa­tients with urea cy­cle di­s­or­der (UCD) in­ad­e­qu­ate­ly con­trolled on SOC, study of KB109 (VI­TO­RA) in pa­tients col­onized with mul­ti-drug re­sis­tant patho­gens, and in vi­vo find­ings from on­go­ing pro­grams in I-O, car­diometabolic and liv­er dis­eas­es. [more in­for­ma­tion]

Keros Therapeutics [KROS] US$660 MM MCap

Our lead pro­tein ther­a­peu­tic prod­uct can­di­date, KER-050, is be­ing de­vel­oped for the treat­ment of cy­tope­nias, in­clud­ing ane­mia and throm­bo­cy­tope­nia, in pa­tients with myelodys­plas­tic syn­drome and myelo­fi­bro­sis. [more in­for­ma­tion]

Kineta Inc.

Kine­ta (Pri­vate) - whol­ly owned pre­clin­i­cal an­ti-VIS­TA (in­nate im­mune tar­get) anti­body re­pro­grams the tu­mor mi­croen­vi­ron­ment and is dif­fer­en­ti­at­ed from other im­munother­a­pies by block­ing sup­pres­sive cells in cold tu­mors. $520M deal with Pfiz­er for its small molecule RIG-I im­munother­a­py and $360M+ deal with Ge­nen­tech for KCP506, pre­clin­i­cal, non-opi­oid for chron­ic pain. [more in­for­ma­tion]

Landos Biopharma

Based on ex­ten­sive pre­clin­i­cal studies, Lan­dos has vali­dat­ed a unique mech­anism of ac­tion in­volv­ing LAN­CL2 that ex­erts po­tent an­ti-in­flam­ma­to­ry ef­fects with an out­s­tand­ing safe­ty pro­file. Lead as­set BT-11 Phase 2 in­ter­im read out in mid-2020 in UC. [more in­for­ma­tion]

Lineage Cell Therapeutics [LCTX] US$116 MM MCap

Cell ther­a­py com­pany with 3 cl­ni­cal pro­grams 1) OpRe­gen, an RPE tran­s­plant ther­a­py in Ph1/2a de­vel­op­ment for the treat­ment of dry-AMD. 2) OPC1, an oli­go­den­dro­cyte pro­gen­i­tor cell ther­a­py for acute spi­nal cord in­juries with Or­phan and RMAT desig­na­tions. 3) VAC2, an al­lo­gene­ic can­cer im­munother­a­py of anti­gen-pre­sent­ing den­drit­ic cells cur­rent­ly in a Ph 1 for NS­CLC con­duct­ed by CRUK. [more in­for­ma­tion]

Marker Therapeutics [MRKR] US$89 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 2020 with en­roll­ment com­ple­tion an­ti­ci­pat­ed in 2H20. The com­pany al­so has a T-cell vaccine pro­gram, cur­rent­ly be­ing studied in breast can­cer. [more in­for­ma­tion]

Maxcyte [MXCT:LN] £162 MM MCap

Har­ness­ing cell-en­gi­neer­ing de­liv­ery plat­form to de­vel­op small molecules, vaccines, and cell and gene ther­a­pies. Plat­form based on Flow Elec­tro­po­ra­tion™ Tech­nol­o­gy, with abil­i­ty to trans­fect pri­mary cells, stem cells and cell lines with min­i­mal cell dis­tur­bance and trans­fec­tion ef­fi­cien­cies routine­ly >90% for a va­ri­e­ty of cell types. [more in­for­ma­tion]

Minoryx Therapeutics

Oral PPAR&gam­ma; ag­on­ist, MIN-102, in Piv­o­tal Ph 2/3 for X-linked ALD and Ph 2 Frie­drich’s Ataxia; da­ta for both ex­pect­ed by end of 2020. VC in­ves­tors in­clude Kur­ma, Ysios, Fund+, Roche, Id­in­vest, etc [more in­for­ma­tion]

Neoleukin Therapeutics [NLTX] US$467 MM MCap

Lead pro­gram NL-201: cy­tokine mimet­ic (ne­oleukin) to ac­ti­vate im­mune cells for can­cer im­munother­a­py; first de no­vo pro­tein ther­a­peu­tic ev­er cre­at­ed. IND planned for 2020. Po­ten­tial for best-in-class IL-2/IL-15. [more in­for­ma­tion]

NeuroRx

De­vel­op­ing the first Glx tar­get­ed an­tide­pres­sant to ad­dress sui­ci­dal­i­ty in bipo­lar de­pres­sion. Lead can­di­date, NRX-101 is in Phase 2b/3 de­vel­op­ment, ini­ti­at­ed in Ju­ly 2019 (Break­through, SPA and Fast Track by FDA) for Se­vere Bipo­lar De­pres­sion in pts w Acute Sui­ci­dal Idea­tion and Be­havior. Re­port­ed Phase 2 ef­fi­ca­cy and safe­ty da­ta for NRX-011 in De­cem­ber 2018. [more in­for­ma­tion]

NexImmune, Inc.

Mul­ti-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. Two Ph 1's on­go­ing. [more in­for­ma­tion]

NorthSea Therapeutics

First-in class, oral, struc­tu­ral­ly en­gi­neered fat­ty acids [SE­FAs] ad­dress­ing NASH and as­so­ci­at­ed metabolic di­s­or­ders via unique tar­get­ing of path­ways reg­u­lat­ing metabolism, oxida­tive stress, in­flam­ma­tion, and fi­bro­sis. $40m Se­ries B (Jan 2020) with ven­Bio Part­n­ers (lead) and Sofin­no­va In­vest­ments. [more in­for­ma­tion]

Oncopeptides AB [ONCO:ST] SKr8,007 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%. Fil­ing for AA ex­pect­ed in H1'20. [more in­for­ma­tion]

OncoResponse

The com­pany's tech­nol­o­gy lev­er­ages the hu­man im­mune sys­tem to iden­ti­fy ful­ly hu­man mon­o­clo­n­al anti­bodies and dis­cov­er nov­el tar­gets that will lead to the de­vel­op­ment of anti­body-de­rived ther­a­peu­tics for the treat­ment of can­cer, pro­vid­ing a larg­er num­ber of can­cer pa­tients with the ben­e­fit of im­munother­a­py. [more in­for­ma­tion]

PharmaMar SA [PHM:ES]

Yon­delis (soft tis­sue sar­co­ma and ovarian can­cer) com­mer­cial­ized with di­rect sales-force in EU and part­n­er­ship in US with Janssen. Signed li­cens­ing agree­ment with Jazz Phar­ma for Lur­binecte­din in the US - up­front pay­ment of $200M, $100mm on ap­pro­va­land up to $550M sales mile­s­tones. Jazz to launch Zepzel­ca in US Ju­ly 2020. [more in­for­ma­tion]

Pharming Group NV [PHARM:AS] €727 MM MCap

RU­CON­EST (rhC1INH), the on­ly re­com­bi­nant en­zyme re­place­ment ther­a­py mar­ket­ed for acute Hered­i­tary An­gioede­ma (HAE) at­tacks and be­ing de­vel­oped for large un­met in­di­ca­tions. [more in­for­ma­tion]

Pharvaris

Phar­varis is 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. Phar­varis us­es small molecules to ad­vance po­tent, oral­ly avai­l­able com­pounds tar­get­ing HAE. [more in­for­ma­tion]

Pieris Pharmaceuticals, Inc. [PIRS] US$160 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. [PLRX] US$861 MM MCap

Pliant has de­vel­oped a port­fo­lio of ful­ly-owned de­vel­op­ment can­di­dates fo­cused on in­hibit­ing the TGFb path­way through in­te­grin in­hi­bi­tion. Pliant has a strate­g­ic li­cens­ing and col­lab­o­ra­tion agree­ment with No­var­tis cov­er­ing the de­vel­op­ment and com­mer­cial­iza­tion of our pre­clin­i­cal liv­er fi­bro­sis pro­gram, PLN-1474 and up to three ad­di­tio­n­al ear­ly stage in­te­grin tar­gets. [more in­for­ma­tion]

Protagonist Therapeutics [PTGX] US$581 MM MCap

PTG-300 in­jectable hep­cidin mimet­ic Ph 2 trials on­go­ing in be­ta-tha­lassemia, hered­i­tary he­mochro­ma­to­sis, po­ly­cythemia ve­ra, and MDS; Phase 2 re­sults and first in­di­ca­tion choice for piv­o­tal study ex­pect­ed 2020. PN-943 oral GI-re­strict­ed al­pha-4-be­ta-7 an­ta­g­on­ist Ph 2 in ul­cer­a­tive coli­tis to be­gin ear­ly 2020; PTG-200, part­nered w/ Janssen, oral GI-re­strict­ed IL-23R an­ta­g­on­ist Ph 2 Crohn’s dis­ease study on­go­ing. [more in­for­ma­tion]

R2 Dermatology

Di­rec­tor of the Well­man Cen­ter of Pho­tomedicine at Mas­sachusetts Gen­er­al Hos­pi­tal in Bos­ton, a teach­ing af­fili­ate of Har­vard Med­i­cal School; of the Cu­ta­neous Bi­ol­o­gy Re­search Cen­ter in the De­part­ment of Der­ma­tol­o­gy at Mas­sachusetts Gen­er­al Hos­pi­tal; [more in­for­ma­tion]

Regent Pacific [575:HK]

Re­gent Pa­cif­ic is a di­ver­si­fied in­vest­ment group based in Hong Kong cur­rent­ly hold­ing vari­ous cor­po­rate and strate­g­ic in­vest­ments fo­cus­ing on the health­care, well­ness and life sci­ences sec­tors. The Group has a strong track re­cord of in­vest­ments and has re­turned ap­prox­i­mate­ly US$298 mil­lion to share­hold­ers in the 21 years of fi­nan­cial re­port­ing since its ini­tial public of­fer­ing. [more in­for­ma­tion]

Ryvu Therapeutics [RVU:WAR]

Po­ten­tial first in class CD­K8 in­hibi­tor for leukemias, lym­pho­mas, and solid tu­mors in Ph1b in AML and high-risk MDS, PIM/FLT3 ki­nase in­hibi­tor in Ph 1/2 in AML (part­nered w Me­nari­ni). Da­ta in 2020. Ear­ly stage pipe­line ad­dress­ing emerg­ing solid tu­mor tar­gets. [more in­for­ma­tion]

Savara Inc. [SVRA] US$110 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]

Secarna Pharmaceuticals GmbH

Se­car­na's unique­ly ef­fi­cient LNA­plus™ dis­cov­ery pro­cess is fu­eled by their pro­pri­e­tary and stream­lined Oli­go­fy­er™ bioin­for­mat­ics sys­tems large in-house cell li­brary and own in vit­ro screen­ing as­says. [more in­for­ma­tion]

Selecta Biosciences, Inc. [SELB] US$229 MM MCap

Im­mune tol­er­ance plat­form (Imm­TOR) de­vel­oped in com­bo with rare dis­ease ther­a­pies. SEL-212 (chron­ic re­frac­to­ry gout) in COM­PARE Ph 2 trial vs KRYS­TEXXA®; to­p­line da­ta mid-2020. Gene tx pipe­line in pre­clin­i­cal de­vel­op­ment with Imm­TOR where re-dos­ing re­quire. Ask­Bio col­lab. [more in­for­ma­tion]

Seneca Biopharma [SNCA] US$14 MM MCap

Stream­lined rab­bit anti­body plat­form fo­cused on im­muno­log­i­cal dis­eas­es. Lead pro­gram is a fast fol­low­er to Dupix­ent in Asth­ma and Atopic Der­mati­tis, en­ter­ing the clin­ic late 2020 in Chi­na and ear­ly 2021 in the US. [more in­for­ma­tion]

Sol-Gel Technologies Ltd. [SLGL] US$188 MM MCap

Com­mer­cial­iz­ing brand­ed gener­ic top­i­cal drug prod­ucts (skin dis­eas­es). Two pos­i­tive Phase 3 pro­grams in rosacea and acne, with NDAs ex­pect­ed in 1H20 and 2H20, re­spec­tive­ly. Ad­di­tio­n­al brand­ed pipe­line as­sets in­clude SGT-210, er­lo­tinib, for the treat­ment of punc­tu­ate pal­mo­plan­tar ker­a­to­der­ma, and pre­clin­i­cal as­set tap­i­narof and rof­lu­mi­last. Sol-Gel al­so has rev­enue from sev­er­al gener­ic col­lab­o­ra­tions. [more in­for­ma­tion]

Sonnet Biotherapeutics, Inc. [SONN] US$31 MM MCap

Son­net's in­ter­leukin-based can­di­date has been shown to en­hance pK (up to 10 fold) and im­prove tu­mor de­liv­ery with an in­crease in in vi­vo ef­fi­ca­cy (30 fold) as de­mon­s­trat­ed in a mice tu­mor mod­el. The Son­net plat­form de-risks the use of in­ter­leukins by great­ly ex­tend­ing their in vi­vo half-life, while al­so im­prov­ing their spe­ci­fic­i­ty to tu­mor tis­sue. [more in­for­ma­tion]

Stemline Therapeutics, Inc. [STML] US$621 MM MCap

EL­ZON­RIS, a com­mer­cial tar­get­ed ther­a­py di­rect­ed to IL-3 re­cep­tor (CD123), ap­proved for BPD­CN and on­go­ing trials in other in­di­ca­tions; 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]

sterna biologicals

By tar­get­ing tran­scrip­tion fac­tors that play a cen­tral role in reg­u­lat­ing Th1- and Th2-driv­en in­flam­ma­to­ry mech­anisms, the Com­pany’s pro­pri­e­tary DNAzyme-based drug can­di­dates can in­ter­vene with up­stream in­flam­ma­to­ry pro­cess­es to ad­dress re­lat­ed dis­eas­es more ef­fec­tive­ly. Ster­na cur­rent­ly has four pro­grams in phase 2 de­vel­op­ment. [more in­for­ma­tion]

Strongbridge Biopharma [SBBP] US$190 MM MCap

Rare dis­eas­es; Keveyis for PPP revs ~21.7M for 2019 and $26 - $27M for 2020. Re­cor­lev for en­doge­nous Cush­ing's syn­drome Ph 3 pub­lished with to­p­line place­bo-con­trolled Ph 3 3Q20. [more in­for­ma­tion]

Sutro Biopharma [STRO] US$324 MM MCap

FolR&al­pha;-tar­get­ing ADC (STRO-002) Phase 1, ad­di­tio­n­al dose es­ca­la­tion da­ta in 1H 2020. CD74-tar­get­ing ADC (STRO-001) Phase 1, ad­di­tio­n­al dose es­ca­la­tion da­ta in 2H 2020. BMS col­lab­o­ra­tion Phase 1 clin­i­cal de­vel­op­ment up­date for CC-99712 (BC­MA-tar­get­ing ADC). Mer­ck col­lab­o­ra­tion up­date in 2020 on first cy­tokine deri­va­tive pro­gram. [more in­for­ma­tion]

Syneos Health [SYNH] US$5,873 MM MCap

Sy­neos Health® is an end-to-end, ful­ly in­te­grat­ed bio­phar­ma­ceu­ti­cal so­lu­tions com­pany that works dif­fer­ent­ly. At Sy­neos Health, all the dis­ci­p­lines in­volved in bring­ing new ther­a­pies to mar­ket, from clin­i­cal to com­mer­cial, work to­gether to cre­ate cus­tomer suc­cess. Our unique Bio­phar­ma­ceu­ti­cal Ac­cel­er­a­tion Mod­el de­liv­ers val­ue across the small to mid-size to large cus­tomer cont­in­u­um. [more in­for­ma­tion]

Targovax [TRVX:OS] NKr497 MM MCap

Pre­lim­i­nary Ph1b da­ta of OV ON­COS-102+che­mo in me­sothe­lio­ma re­sults: com­bo mPFS=8.4 mos. vs. con­trol mPFS=6.8 mos.; 1L pts mPFS-8.9 mos. Up­com­ing mile­s­tones: up­dat­ed clin­i­cal & im­mune da­ta in Ph2 me­sothe­lio­ma (1H20); pre­clin­i­cal da­ta on next-gen OV w/ dou­ble trans­genes (1H20); and clin­i­cal da­ta from Ph1 me­lano­ma study, part 2 with ex­tend­ed dos­ing (2H20). [more in­for­ma­tion]

Teon Therapeutics

Pipe­line of next-gen­er­a­tion can­cer ther­a­peu­tics tar­get­ing GPCRs in­clud­ing a high­ly-spe­cif­ic A2B an­ta­g­on­ist and EP4 an­ta­g­on­ist, both slat­ed to en­ter Phase 1b trials in 2021. [more in­for­ma­tion]

Tessa Therapeutics

On­go­ing Ph III (na­sopha­ryn­geal can­cer), up­com­ing piv­o­tal Ph II (Hodgkin lym­pho­ma), and mul­ti­ple other clin­i­cal-stage pro­grams. GMP fa­cil­i­ty un­der con­struc­tion. Key col­lab­o­ra­tions: Bay­lor, Park­er Inst., St. Jude Chil­dren’s, and JV in Chi­na. [more in­for­ma­tion]

Vaccitech Limited

Pro­pri­e­tary ChA­dOx (prime) and MVA (boost) tech­nol­o­gy plat­form in­duces and main­tains CD8+ T-cells at the max­i­mum lev­els pub­lished to date. Ph1/2 for HPV and HBV im­munother­a­pies ini­ti­at­ing 1H20. Two can­cer im­munother­a­py pro­grams (per­so­n­al­ized vaccine and MAGE-NYE­SO ther­a­peu­tics) en­ter clin­ic in 2020. Two ChA­dOx1 vaccines for coro­n­avirus­es with Ox­ford Univ fund­ed by CEPI – COVID-19 (an­nounced 3/10/20) and MERS (Ph.1 com­plete). $150M fund­ing at Jen­n­er In­sti­tute and com­pany raised $48M since for­ma­tion in 2016 [more in­for­ma­tion]

Vanda Pharmaceuticals Inc. [VNDA] US$627 MM MCap

The com­pany has as­sem­bled an ex­pe­ri­enced team along with a port­fo­lio of clin­i­cal and com­mer­cial stage pro­grams with the goal of bring­ing im­por­tant new treat­ment op­tions to pa­tients. Com­mer­cial­iz­ing Het­lioz for the treat­ment of Non-24-Hour Sleep-Wake Di­s­or­der. [more in­for­ma­tion]

Vericel Corporation [VCEL] US$701 MM MCap

Mar­ket­ing MA­CI (car­ti­lage re­pair) and Epi­cel (skin re­place­ment) in TAMs of $2b+. FY19 rev­enue of $117.9M in­creased 30% over 2018; FY20 gui­dance of $141M-$146M. Exp mid-20 sub­mis­sion of Nexo­Brid BLA. [more in­for­ma­tion]

Verrica Pharmaceuticals [VRCA] US$246 MM MCap

Ver­ri­ca Phar­ma­ceu­ti­cals is a der­ma­tol­o­gy ther­a­peu­tics com­pany de­vel­op­ing med­i­ca­tions for vi­ral skin dis­eas­es re­quir­ing med­i­cal at­ten­tion. Lead prod­uct, VP-102 for mol­lus­cum con­ta­gio­sum has a PD­U­FA date of Ju­ly 13, 2020. Phase 2 da­ta in ex­ter­nal geni­tal warts ex­pect­ed in 2H20. Ini­ti­a­tion of Phase 3 in com­mon warts in 1H20 and ini­ti­a­tion of Phase 2 in plan­tar warts in mid-2020. [more in­for­ma­tion]

Viela Bio [VIE] US$1,982 MM MCap

BLA for lead can­di­date, ine­bi­l­izumab (an­ti-CD19 mAb), is un­der FDA re­view for the treat­ment of neu­romyeli­tis op­ti­ca spec­trum di­s­or­der (NMOSD); PD­U­FA date June 11, 2020. VIB7734, an­ti-ILT7 mAb, Phase 1b in­ter­im da­ta 1H20. Ad­di­tio­n­al trials with ine­bi­l­izumab ex­pect­ed to ini­ti­ate in 2020, in­clud­ing myas­the­nia gravis and Ig­G4-re­lat­ed dis­ease. [more in­for­ma­tion]

Vyome Therapeutics Inc.

$22M fund raise Jan '19. Ad­vanced lead as­set VB-1953 to Ph 2 for mod­er­ate to se­vere acne. Pipe­line DART - du­al ac­tion over­com­ing an­timi­cro­bial re­sis­tance - and MRT - pa­tent­ed an­ti­fun­gal plat­form. Treat drug-re­sis­tant skin patho­gens. [more in­for­ma­tion]

Zentalis [ZNTL] US$1,435 MM MCap

In ad­di­tion to its lead pro­gram, ZN-c5, an oral SERD for es­tro­gen re­cep­tor pos­i­tive, HER2-neg­a­tive breast can­cer, the Com­pany is de­vel­op­ing a broad pipe­line of on­col­o­gy can­di­dates, tar­get­ing ar­eas of ma­jor un­met med­i­cal need. [more in­for­ma­tion]