New Pipeline Updates from Seattle Genetics, Novartis, Vybion and Notal Vision

December 10, 2018

Company Drug/Device Medical Condition Status
Innovent Biologics, Inc. IBI101, a recombinant fully human anti-OX40 monoclonal antibody (mAb) drug candidate Advanced solid tumors FDA approved Investigational New Drug (IND) application and plans to initiate Phase I clinical trial based on results from China Phase I trial
Sunny Pharmtech, Inc. and Vitruvius Therapeutics, Inc. Aminocaproic Acid Tablets 500 mg and 1000 mg tablets Fibrinolysis FDA approved Abbreviated New Drug Application (ANDA)
Vybion, Inc. INT41 Huntington’s disease FDA granted Orphan Drug designation
Ocular Therapeutix™, Inc. RP-L201 DEXTENZA® (dexamethasone ophthalmic insert) 0.4mg Occular pain following eye surgery
Notal Vision, Ltd. Notal Vision Home-based Optical Coherence Tomography (OCT) System Home testing between doctor ntra- and/or subretinal fluid in the central 10 degrees of eyes diagnosed with exudative age-related macular degeneration (eAMD) FDA granted Breakthrough Device designation
Mirati Therapeutics, Inc. MRTX849 Cancers driven by KRAS G12C mutations FDA has cleared the company’s Investigational New Drug (IND) application to initiate Phase I/II Trial
AbbVie Rovalpituzumab Tesirine (Rova-T) Second-line therapy for advanced small-cell lung cancer Halted Phase III trial due to shorter overall survival in the Rova-T arm
Genentech, Inc. Kadcyla® (ado-trastuzumab emtansine) HER2-positive early Breast cancer (EBC) Phase III KATHERINE trial met its primary end-point, significantly reducing risk of recurrence or death compared to Herceptin® (trastuzumab) as an adjuvant treatment
Global Blood Therapeutics Voxelotor Sickle cell disease in patients 12 and older Interim analysis of its Phase III HOPE study showed patients experienced “rapid, robust and sustained improvements in hemoglobin levels and measures of hemolysis with a favorable safety and tolerability profile”
Seattle Genetics Adcetris Previously untreated systemic anaplastic large cell lymphoma Granted approved by FDA
Novartis ligelizumab (QGE031) Chronic spontaneous urticartia (CSU) whose symptoms are inadequately controlled by H1-antihistamines Taking it into Phase III trials after a mid-stage trial showed out-performed Xolair
Janssen Pharmaceutical IMBRUVICA® (ibrutinib) plus rituximab Chronic lymphocytic leukemia (CLL); small lymphocytic lymphoma (SLL) Phase III trial results: significantly prolonged PFS compared to FCR in previously untreated patients aged 70 years or younger with CLL/SLL
Exelixis, Inc. and IPSEN Cabozantinib (CABOMETYX®) in combination with atezolizumab (TECENTRIQ®) previously untreated advanced HCC Previously untreated advanced hepatocellular carcinoma (HCC) initiation of COSMIC-312, a Phase III pivotal trial of this med versus sorafenib


New Pipeline Updates from Allergan, Bayer, Genentech and Pfizer

December 3, 2018

Company Drug/Device Medical Condition Status
GlycoMimetics Uproleselan (GMI-1271) Relapsed/refractory acute myeloid leukemia First patient enrolled in global Phase III trial
Sollis Therapeutics Clonidine micropellet Sciatica pain First patient enrolled in the RePRIEVE-CM trial
Stallergenes Greer Sublingual allergy immunotherapy tablet STAGR320 House dust mite (HDM)-induced allergic rhinitis Phase III trial met its primary endpoint with statistical significance in patients treated with STAGR320 compared to patients on placebo
Rocket Pharmaceuticals RP-L201 Severe Leukocyte Adhesion Deficiency-I FDA approved IND for Phase I trial
Pliant Therapeutics PLN-74809 Primary sclerosing cholangitis Granted Orphan Drug designation by FDA
Allergan Avycaz Complicated urinary tract infections and complicated intra-abdominal infections FDA accepted for review sNDA to expand label indication
NovImmune S.A. Gamifant® (emapalumab-lzsg) Primary hemophagocytic lymphohistiocytosis Granted approval by FDA
Pfizer Daurismo™ (glasdegib), to be used in combination with low-dose cytarabine (LDAC) Newly diagnosed acute myeloid leukemia in adults 75 years or older Granted approval by FDA
AbbVie and Genentech Venclexta® (venetoclax) Newly diagnosed acute myeloid leukemia in adults 75 years or older who cannot tolerate intensive chemotherapy Granted accelerated approval by FDA
Synthetic Biologics Synthetic Biologics Antibiotic-mediated Clostridium difficile infection Successfully completed End-of-Phase II meeting; Phase III go-ahead from FDA
Bayer and Loxo Oncology Vitrakvi (larotrectinib) Solid tumors with NTRK gene fusion Granted approval by FDA
HighTide Therapeutics HTD1801 Nonalcoholic steatohepatitis Fast Track designation granted by FDA


New Pipeline Updates from Eli Lilly, Merck, AbbVie and Lumendi

November 19, 2018

Company Drug/Device Medical Condition Status
Stallergenes Greer Oralair®, an allergy immunotherapy sublingual tablet Grass pollen-induced allergic rhinitis Received FDA approval for the extension of the indication to treat patients ages five to nine
REGENXBIO RGX-181, a one-time treatment candidate Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease Granted Orphan Drug Designation by the FDA
Eli Lilly and Company Lasmiditan Migraine with or without aura in adults Submitted a New Drug Application (NDA) to the FDA
Eli Lilly and Company Emgality Episodic cluster headache Granted Breakthrough Therapy Designation by the FDA
Lumendi DiLumen™ EIP Easier dissection and resection of polyps without the need for surgical intervention Received 510(k) clearance from the FDA
Theravance Biopharma, Inc. and Mylan N.V. YUPELRITM (revefenacin), once-daily, nebulized bronchodilator COPD Granted approval by the FDA
Biohaven Pharmaceutical Holding Company Ltd Rimegepant, oral calcitonin gene-related peptide (CGRP) receptor antagonist Migraine Enrolled first patient in Phase III clinical trial
AbbVie Mavyret (glecaprevir/pibrentasvir) Hepatitis C (HCV) Results from Phase IIIb EXPEDITION-8 trial: patients receiving the drug for 8 weeks with genotype 1, 2, 4, 5 and 6 had a 100-percent sustained virologic response 12 weeks after treatment
Merck KEYTRUDA® (PEMBROLIZUMAB) Advanced or metastatic esophageal or esophagogastric junction carcinoma in patients whose tumors expressed PD-L1 Significantly improved Overall Survival (OS) compared to chemotherapy in Phase III KEYNOTE-181 clinical trial
AbbVie Elagolix, in combination with add-back therapy Heavy menstrual bleeding associated with uterine fibroids Achieved statistically significant reduction in two replicate pivotal Phase III clinical trials
Calliditas Therapeutics NEFIGARD IgA nephropathy (IgAN) First patient enrolled in pivotal Phase III clinical trial
Palvella Therapeutics, Inc. PTX-022 (novel, high-strength rapamycin topical formulation Pachyonychia congenita (PC) Granted Fast Track Designation by the FDA
TWi Biotechnology, Inc. AC-201CR Hemophilic arthropathy Completed patient enrollment in Phase II clinical trial

New Pipeline Updates from Eli Lilly, Bone Therapeutics, Shoulder Innovations and BD

November 12, 2018

Company Drug/Device Medical Condition Status
AVEO Oncology Tivozanib Highly refractory advanced or metastatic renal cell carcinoma (RCC) Met its primary endpoint of demonstrating a statistically significant benefit in
progression-free survival (PFS) in Phase III clinical trial
Fortis Therapeutics, Inc. FOR46 Metastatic castration-resistant prostate cancer and late-stage multiple myeloma The FDA has cleared two Investigational New Drug (IND) applications
Shoulder Innovations InSet Humeral Short Stem System Degenerative, rheumatoid or traumatic arthritis in the shoulder Received 510(k) clearance from the FDA
Eli Lilly Trulicity (dulaglutide) Heart attacks and stroke in type 2 diabetes Reduced major adverse cardiovascular events (MACE) in REWIND Phase III clinical trial
Themis Bioscience MV-CHIK Chikungunya fever Primary endpoint —neutralizing antibodies after one or two injections — was met in Phase II clinical trial
Bristol-Myers Squibb Empliciti (elotuzumab) Multiple myeloma in adults
who have received at least
two prior therapies
Granted approval by the FDA
Bone Therapeutics PREOB Hip osteonecrosis (ON) Halted Phase III trial after interim analysis showed it would not meet endpoints
Endo International plc Collagenase clostridium histolyticum (CCH) Cellulite Positive results announced from two identical Phase III RELEASE* clinical trials
Mallinckrodt plc H.P. Acthar Gel Severe Noninfectious Intermediate Uveitis, Posterior Uveitis, or Panuveitis (NIPPU) First patient screened in company’s Phase IV, multi-center, multiple-dose, open label trial to assess effect
OncoSec Medical TAVO™ (in combination with Merck’s KEYTRUDA®) Late-stage triple negative breast cancer (TNBC) First patient treated in KEYNOTE-890 Phase II clinical trial
Glenmark Pharmaceuticals CD38xCD3 bispecific antibody GBR 1342 Solid tumors Launching a Phase I
clinical trial
Drug-Coated Balloon (DCB) IDE
Narrowed or obstructed
arteries below the knee
Primary safety and efficacy endpoints were met in
Phase I clinical trial
Urovant Sciences, Inc. Vibegron Overactive bladder Completed enrollment in international Phase III clinical trial, EMPOWUR to evaluating safety and efficacy

New Pipeline Updates from Merck, Sandoz, Genentech and Gilead

November 5, 2018

Company Drug/Device Medical Condition Status
Aquestive Therapeutics, Inc. SYMPAZAN™ (clobazam) Seizures associated with Lennox-Gastaut syndrome (LGS) in
patients 2 years of age or older
Granted approval by the FDA
Sandoz Hyrimoz (Adalimumab), biosimilar to AbbVie’s Humira Metastatic squamous non-small cell lung cancer (NSCLC) Granted approval by the FDA
Merck Keytruda Metastatic squamous non-small cell lung cancer (NSCLC) FDA has approved, in combination with chemotherapy, as a first-line treatment
Cerus Corporation Pathogen-reduced cryoprecipitate Massive hemorrhage Granted Breakthrough Device designation by the FDA
Alkermes ALKS 5461 Depression FDA advisory committee rejected New Drug Application
Genentech Venclexta® (venetoclax) in combination with Gazyva® (obinutuzumab) Previously untreated chronic lymphocytic leukemia (CLL) and
co-existing medical conditions
Randomized Phase III CLL14 trial met its primary endpoint; showed a statistically significant reduction in risk of disease worsening or death compared to standard-of-care Gazyva plus chlorambucil
Genmab and Johnson & Johnson’s Janssen Darzalex (daratumumab) Multiple myeloma In Phase III MAIA trial, met its primary endpoint of improved progression-free survival (PFS) at a pre-planned interim analysis
Bio-Thera Solutions, Ltd. HER2 Antibody-Drug Conjugate (ADC), BAT8001 HER-2 positive metastatic
breast cancer
Initiated Phase III clinical trial evaluating the efficacy and safety
Gilead Sciences, Inc. Biktarvy®
(bicte-gravir 50 mg/emtricitabine 200 mg/tenofovir alafen-amide 25 mg tab-lets, BIC/FTC/TAF)
HIV-1 infection Results from Phase III randomized, double-blinded trial; found to be statistically non-inferior to a regimen of dolutegravir and emtricitabine/tenofovir alafenamide (50 mg) (DTG+FTC/TAF) through 96 weeks of therapy

New Pipeline Updates from Abbott, Sanofi, Roche and Seqirus

October 29, 2018

Company Drug/Device Medical Condition Status
Regeneron Pharmaceuticals and Sanofi Dupixent (dupilumab) as an add-on maintenance therapy Moderate-to-severe asthma Granted approval by the FDA
Eiger BioPharmaceuticals, Inc. lonafarnib Hutchinson-Gilford Progeria Syndrome (HGPS or progeria) and progeroid laminopathies Granted Rare Pediatric Disease (RPD) designation by the FDA
Abbott HeartMate 3™ Left Ventricular Assist Device (LVAD) Advanced heart failure Granted approval by the FDA
Bristol-Myers Squibb Combination of Opdivo and Yervoy Peviously untreated advanced or metastatic renal cell carcinoma (RCC) Yielded a significantly longer treatment-free survival period than sunitinib, the standard of care, in Phase III clinical trial
Takeda Pharmaceutical Company Investigational subcutaneous formulation vedolizumab Moderately to severely active ulcerative colitis Significant portion of patients in Phase III VISIBLE 1 clinical trial show achieved clinical remission
Roche Xofluza (baloxavir marboxil) Flu in patients 12 years of age and older Granted approval by the FDA
Incyte Corporation ruxolitinib (Jakafi®) Acute graft-versus-host-disease (GVHD) inpatients with inadequate response to corticosteroids FDA accepted its supplemental New Drug Application (sNDA) for priority review
Scarless Laboratories, Inc. Novel therapeutic peptide, SLI-F06 Scar reduction Granted clearance by the FDA to enter a Phase I/IIa clinical trial
CStone Pharmaceuticals Monoclonal antibody (mAb) CS1003 Cancer FDA approved company’s Investigational New Drug (IND) application
Avita Medical RECELL® Autologous Cell Harvesting Device (RECELL® System) Superficial partial- and mid-thickness pediatric burns, including scald injuries Beginning of a randomized, controlled clinical trial
Seqirus AFLURIA® QUADRIVALENT (Influenza Vaccine) Flu in patients six months and older Granted approval by the FDA

New Pipeline Updates from Pfizer, Immusoft, Allergan and UCB

October 22, 2018

Company Drug/Device Medical Condition Status
Viseon, Inc. Voyant System, featuring proprietary HD imaging sensor and illumination technology Minimally invasive spine surgery Granted 510(k) clearance by the FDA
Centric Medical SATURN External Fixation System which consists of rings, struts, threaded rods, pins, wires and connectors To stabilize the foot, ankle and long bone segments Granted 510(k) clearance by the FDA
Pfizer PARP inhibitor, Talzenna (talazoparib) BRCA-mutated (gBRCAm), HER2-negative breast cancer Granted approval by the FDA
Abireo Pharma, Inc. A4250 Progressive familial intrahepatic cholestasis (PFIC) Granted Fast Track designation by the FDA
Immusoft Corp. Iduronicrin genleukocel-T Mucopolysaccharidosis type I (MPS I) Granted Rare Pediatric Disease designation (RPDD) by the FDA
Cellenkos, Inc. CK0801 Treatment-resistant Guillain-Barré Syndrome (GBS) FDA has cleared its Investigational New Drug (IND) application to proceed with a Phase I clinical trial
Allergan, plc and Medicines360 LILETTA® (levonorgestrel-releasing intrauterine system) 52 mg Prevention of pregnancy for up to five years FDA approved Supplemental New Drug Application (sNDA) to extend the duration of use
AstraZeneca and Merck LYNPARZA Pancreatic cancer Granted Orphan Drug designation (ODD) by the FDA
X4 Pharmaceuticals X4P-001-RD WHIM syndrome, a primary immunodeficiency disease Granted Orphan Drug designation by the FDA
AcelRx Pharmaceuticals, Inc. DSUVIA™ Moderate-to-severe acute pain in medically supervised settings for adult patients FDA Anesthetic and Analgesic Drug Products Advisory Committee voted in favor of recommending approval
Sutro Biopharma, Inc. STRO-001 multiple myeloma Granted Orphan Drug designation by the FDA
Grifols ID CORE XT molecular diagnostic test Molecular red blood cell typing Granted approval by the FDA for use in the U.S.
UCB CIMZIA® (certolizumab pegol) Axial spondyloarthritis (axSpA) spectrum In Phase III trial, showed clinically relevant, statistically significant improvements in patients with active disease and signs of inflammation, as measured by major improvement in Ankylosing Spondylitis Disease Activity Score (ASDAS-MI)

New Pipeline Updates from Akcea Therapeutics, Genentech, Eximo Medical and Valneva USA

October 15, 2018

Company Drug/Device Medical Condition Status
Akcea Therapeutics TEGSEDITM (inotersen) Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults Granted approval by the FDA
Akcea Therapeutics AKCEA-APO(a)-LRx Cardiovascular disease (CVD) and elevated levels of lipoprotein(a), or Lp(a) Positive topline results from a Phase II clinical trial
Bausch Health Companies, Inc. BRYHALI™ (halobetasol propionate) Lotion, 0.01% Plaque psoriasis in adults FDA tentatively approved New Drug Application; final OK pending expiration of exclusivity for related product
Eximo Medical Ltd B-Laser™ Atherectomy System Peripheral artery disease (PAD) Granted 510(k) clearance by the FDA
YiSheng BioPharma Co. PIKA® rabies vaccine Rabies Granted clearance by the China FDA to proceed with a clinical trial
Genentech Hemlibra Hemophilia A patients without factor VIII inhibitors Granted approval by the FDA
Leadiant Biosciences, Inc. Revcovi™ (elapegademase-lvlr) injection Adenosine deaminase severe combined immune deficiency (ADA-SCID) in children and adults Granted approval by the FDA
Breckenridge Pharmaceutical, Inc. Roflumilast Tablets, 500mcg (generic for Daliresp® Tablets) COPD Abbreviated New Drug Application approved by the FDA
Valneva USA XIARO® (Japanese Encephalitis Vaccine, Inactivated, Adsorbed) Japanese encephalitis Accelerated dosing regimen approved by the FDA
Eidos Therapeutics, Inc. AG10 amyloidosis (ATTR) Granted Orphan Drug designation by the FDA
Paratek Pharmaceuticals, Inc. NUZYRA™ (omadacycline) Community-acquired bacterial pneumonia; acute skin infections (ABSSSI) in adults Granted approval by the FDA
Poxel SA Imeglimin, an investigational therapeutic agent Type 2 diabetes Patient enrollment completed in the TIMES 2 trial of the Phase III registration program
Chugai Pharmaceutical Co., Ltd HEMLIBRA® (US generic name: emicizumab-kxwh) Hemophilia A without factor VIII inhibitors in newborns, children and adults Granted approval by the FDA
Genentech baloxavir marboxil Influenza type A/H3N2 and type B Phase III CAPSTONE-2 trial showed symptoms improved significantly faster in patients at high risk of serious flu who took the drug v. a placebo
Zealand Pharma A/S Glepaglutide Short bowel syndrome (SBS) First patient enrolled in a global Phase III trial

New Pipeline Updates from Amgen, Eli Lilly, Applied BioCode and Ocugen

October 8, 2018

Company Drug/Device Medical Condition Status
Paratek Pharmaceuticals, Inc. SEYSARA™ (sarecycline) Inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older Granted approval by the FDA
Clovis Oncology, Inc. Rubraca® (rucaparib) BRCA1/2-mutated mCRPC after androgen receptor (AR)-directed therapy and taxane-based chemotherapy Granted Breakthrough Therapy designation by the FDA
PharmAbcine, Inc. Bevacizumab (Avastin®) Recurrent glioblastoma Received “Study May Proceed Letter “ for IND application from the FDA; can initiate Phase II trials
Amgen KYPROLIS® (carfilzomib) Relapsed or refractory multiple myeloma FDA approved supplemental New Drug Application to expand prescribing information
Applied BioCode BioCode® Gastrointestinal Pathogen Panel on BioCode® MDx-3000 molecular system Tests for 17 most common bacteria, viruses and parasites that cause infectious diarrhea Granted 510 (k) clearance by the FDA
Eisai Co., Ltd. Fycompa (perampanel) Partial-onset seizures in pediatric patients with epilepsy 4 years of age and older Granted approval by the FDA for an indication expansion
Eli Lilly and Company Ultra Rapid Lispro (URLi) Type 1 and Type 2 diabetes Phase III clinical trials show met primary efficacy endpoint
Seattle Genetics, Inc. and Takeda Pharmaceutical Company Limited ADCETRIS (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) CD30-expressing peripheral T-cell lymphoma (PTCL), also known as mature T-cell lymphoma (MTCL) Phase III ECHELON-2 clinical trial met its primary endpoint
Acorda Therapeutics, Inc. INBRIJA, investigational inhaled levodopa OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen Releasing efficacy analysis from SPAN-PD, Phase III clinical trial
Five Prime Therapeutics, Inc. Bemarituzumab (FPA144), an isoform-selective FGF receptor 2b (FGFR2b) antibody, with chemotherapy Advanced gastric cancer (GC) or gastroesophageal junction (GEJ) cancer First patient dosed in Phase III FIGHT pivotal trial
Ocugen, Inc. OCU310 Dry eye Initiated Phase III clinical trial
PTC Therapeutics Risdiplam (RG7916) Type 1, 2 and 3 spinal muscular atrophy (SMA) Interim data from open-label clinical trials show increases in developmental motor milestones
Veloce BioPharma, LLC VBP-926 (Povidone-Iodine) topical treatment Chemo-associated paronychia, or painful, inflamed nails, a chemo side effect Positive results of Phase IIb trial

New Pipeline Updates from AstraZeneca, Genentech, Cook Medical and Avita Medical

October 1, 2018

Company Drug/Device Medical Condition Status
Verastem Copiktra Leukemia Granted approval by the FDA
MT.DERM GmbH Exceed, microneedling device Facial wrinkles Granted clearance by the FDA
Cook Medical 5 mm diameter version of Zilver® PTX® Peripheral artery disease (PAD) Granted clearance by the FDA
Avita Medical RECELL® Autologous Cell Harvesting Device (RECELL® System) Severe thermal burns in patients 18 and older Granted clearance by the FDA
Cardurion Pharmaceuticals CRD-733, a Phosphodiesterase-9 (PDE-9) inhibitor Heart failure FDA has accepted Investigational New Drug Application (IND)
OBI Pharma, Inc. OBI-3424 Acute Lymphoblastic Leukemia (ALL) Granted Orphan Drug Designation by FDA
Alnylam Pharmaceuticals Givosiran Acute hepatic porphyrias (AHPs) Successful early Phase III clinical trial results announced
AstraZeneca Imfinzi (durvalumab) Non-small cell lung cancer (NSCLC) Phase III PACIFIC clinical trial found cut deaths by a third
Genentech Tecentriq plus chemotherapy Extensive-stage small cell lung cancer (ES-SCLC) Phase III trial showed treatment helped patients live significantly longer than chemo alone
Daiichi Sankyo Company, Ltd Trastuzumab deruxtecan (DS-8201) Previously-treated HER2 positive unresectable and/or metastatic breast cancer Initiated two Phase III trials
Aurinia Pharmaceuticals, Inc. Voclosporin added to background therapy of mycophenolate mofetil (MMF)/CellCept® Lupus nephritis (LN) Completed enrollment in AURORA Phase III clinical trial
Catabasis Pharmaceuticals, Inc. Edasalonexent Duchenne muscular dystrophy (DMD) Initiated Polaris DMD Phase III clinical trial
AstraZeneca Farxiga (dapagliflozin) Cardiovascular disease Positive Phase III clinical trial results announced
Alexion Pharmaceuticals, Inc. Soliris® (eculizumab) Anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD) Positive topline results from the Phase III PREVENT announced