Phase I

Aeglea Biotherapeutics Inc., of Austin, Texas


Arginase-I stimulator

Arginase 1 deficiency

Following 20 doses, data on 14 participants from completed phase I/II and ongoing phase II open-label extension trials showed sustained reductions in plasma arginine; 79% (11 of 14) were clinical responders, using mobility assessment components that correspond with secondary endpoint of ongoing pivotal Peace trial

Autolus Therapeutics plc, of London


B-lymphocyte antigen CD19 modulator

Pediatric acute lymphoblastic leukemia

Nature Medicine published data from ongoing Carpall trial showing the CAT chimeric antigen receptor T cells showed increased proliferation and cytotoxicity in vitro and enhanced proliferative capacity and antitumor activity compared to FMC63 CAR T therapies in vivo; 86% (n=14) of recurrent/refractory patients achieved molecular complete remission after single dose, with median duration of remission of 7.4 months and no severe cytokine release syndrome; event-free survival based on morphological relapse was 67% and 46% and overall survival was 84% and 63% at 6 and 12 months, respectively

Biolinerx Ltd., of Tel Aviv, Israel


Alpha-gal glycolipid immunotherapy

Solid tumors

Dose-escalation portion of phase I/IIa study completed; maximal tolerated dose was not reached, and recommended dose was determined for part 2

Hutchison China Meditech Ltd., of Hong Kong


Phosphoinositide-3 kinase delta inhibitor


Global open-label, 2-stage phase I/Ib study initiated in individuals with relapsed/refractory disease; primary outcome measures are safety and tolerability; secondary outcomes include pharmacokinetics and preliminary efficacy measures, such as objective response rate

Inhibrx Inc., of San Diego


Multivalent agonist of death receptor 5

Solid tumors

Dose-escalation portion in 20 participants completed, with maximum tolerated dose not reached; pharmacokinetic profile was shown to be similar to conventional antibodies, and initial signs of disease control were shown in 5 of 8 with tumor types chosen for inclusion in expansion cohorts; dose-expansion portion set to begin enrollment of 20-patient cohorts in each of gastric and colorectal adenocarcinomas and mesothelioma

Kaleido Biosciences Inc., of Lexington, Mass.


Oral prebiotic gluco-oligosaccharide microbiome metabolic therapy

Urea cycle disorders

Data from open-label, single-arm study evaluating safety, tolerability and effect on measures of nitrogen metabolism in 4 participants with well-controlled UCD showed no clinically significant safety signals or serious treatment-emergent adverse events, and study drug was generally well-tolerated; in evaluable patients, KB-195 was associated with general decrease in urinary 15N excretion and increase in stool 15N excretion, measured by lactose-15N-ureide tracer to evaluate changes in nitrogen metabolism in the gut

Phase II

Aeglea Biotherapeutics Inc., of Austin, Texas


Arginase-I stimulator

Arginase 1 deficiency

10 participants dosed subcutaneously in ongoing phase II open-label extension trial; subcutaneous administration shown to control plasma arginine similarly to intravenous administration and be well-tolerated, with no patient discontinuations

Apogenix AG, of Heidelberg, Germany


CD95 antagonist

Solid tumors

Review in Cancer Management and Research suggested study drug may offer treatment option for individuals with malignancies beyond glioblastoma, where proof-of-concept study showed 5-year overall survival rate of 7% for those dosed with asunercept in combination with radiotherapy compared to 0% for those treated with radiotherapy alone

Aridis Pharmaceuticals Inc., of San Jose, Calif.


Fully human IgG1 monoclonal antibody

Pseudomonas aeruginosa-related ventilator-associated pneumonia

Study missed primary endpoint of superiority in clinical cure rates on day 21 compared to placebo and showed statistically significant imbalance in all-cause mortality and in serious adverse event rates that favored placebo, prompting company to halt development

Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Zealand Pharma A/S, of Copenhagen, Denmark


Dual-acting glucagon-like peptide 1 GLP-1/glucagon agonist

Type 2 diabetes, obesity

BI will initiate phase II development of agent in-licensed from Zealand, initially in dose-finding, placebo and active comparator proof-of-concept trial

Concert Pharmaceuticals Inc., of Lexington, Mass.


Dual JAK1/JAK2 kinase inhibitor

Alopecia areata

Randomized, double-blind, sequential dose trial of participants with moderate to severe disease treated with 8 mg or 12 mg twice daily met primary endpoint of percentage of patients achieving ≥ 50% relative change from baseline at 24 weeks using Severity of Alopecia Tool (SALT), with statistically significant differences of 47% and 58%, respectively, compared to placebo response of 9% (p<0.001); these dose cohorts also differed from placebo in number of participants achieving ≥ 75% and ≥ 90% relative change in SALT from baseline at 24 weeks and in rating significantly greater improvement on Patient Global Impression of Improvement Scale

Debiopharm International SA, of Lausanne, Switzerland


Apoptosis protein inhibitor

Head and neck cancer

Following completion of 2-year follow-up, randomized, double-blind study in high-risk, previously untreated patients with locally advanced squamous cell carcinoma of the head and neck met primary endpoint of locoregional control along with prolonged progression-free survival

Idorsia Ltd., of Allschwil, Switzerland


Dual endothelin receptor antagonist


In parallel-group study that randomized 490 individuals, where 430 completed double-blind treatment period, decreases in sitting systolic/diastolic automated office blood pressure from baseline to week 8 were 10.3/6.3, 15/9.9, 18.5/12 and 15.1/10 mmHg for aprocitentan 5, 10, 25 and 50 mg, respectively, vs. 7.7/4.9 mmHg for placebo and 12.8/8.4 mmHg for lisinopril; no changes in heart rate or body weight were seen for any dose of aprocitentan

Inventiva SA, of Daix, France


Galactosyltransferase modulator

Mucopolysaccharidosis type VI

Initiated biomarker study examining leukocyte glycosaminoglycan levels in 3 children and skin glycosaminoglycan levels in 3 adults with MPS VI before and following enzyme replacement therapy and in 6 age-matched control subjects to inform ongoing phase IIa Improves study

Kura Oncology Inc., of San Diego


Protein farnesyltransferase inhibitor

Urothelial carcinoma

Top-line data from ongoing investigator-sponsored single-arm trial designed to enroll at least 18 participants with relapsed/refractory HRAS mutant disease at single site in Korea showed that 5 of 13 evaluable participants had confirmed objective responses, according to RECIST 1.1 criteria, for overall response rate of 38%; 4 experienced progression-free survival of > 6 months

Myokardia Inc., of South San Francisco


Myosin inhibitor

Hypertrophic cardiomyopathy

Data on 12 participants evaluable at 36 weeks from Pioneer-OLE study that enrolled 13 individuals with symptomatic, obstructive disease from phase II Pioneer-HCM study showed treatment resulted in reductions in resting and provoked left ventricular outflow tract gradient while maintaining left ventricular ejection fraction above 55% at all times of assessment; NT-proBNP, an established circulating blood marker of cardiac wall stress, decreased about 10-fold to mean value of 186 pg/mL (normal considered < 125 pg/mL); E/e', a non-invasive marker of left ventricular filling pressure, decreased from 12.8 to 8.5 (normal range is < 8)

Valbiotis SA, of La Rochelle, France


5 plant extracts


In addition to previously announced reductions in hyperglycemia, body weight and waist size, Valedia reduced triglycerides by 32.2% (p<0.01), fatty liver index by 18.7%(p<0.001) and LDL cholesterol by 11.7% (p<0.05), all compared to placebo; systolic blood pressure was reduced by 10.57 mmHg (p<0.01) compared to placebo and by 18.86 mmHg (p <0.001) relative to placebo in patients with systolic blood pressure levels higher than 130 mmHg at the start of the study

Phase III

Akebia Therapeutics Inc., of Cambridge, Mass.


Hypoxia-inducible factor prolyl hydroxylase inhibitor

Anemia due to chronic kidney disease

Pro2tect studies are fully enrolled with 3,513 non-dialysis-dependent patients; data expected in mid-2020

Alnylam Pharmaceuticals Inc., of Cambridge, Mass.

Onpattro (patisiran)

RNAi targeting transthyretin

Polyneuropathy of hereditary ATTR amyloidosis

Biomarker analysis of the Apollo study showed 66 proteins with changes after treatment with Onpattro; neuroaxonal damage marker neurofilament light chain (NfL) had the greatest statistical significance (p=3.95×10^-21) for change relative to placebo over the 18-month study period; there was a correlation between NfL reduction and improvement in mNIS+7 score

Apellis Pharmaceuticals Inc., of Crestwood, Ky.


Inhibits C3 and C3b complement proteins

Treatment-naïve paroxysmal nocturnal hemoglobinuria

Treated the first of 54 patients in the Prince (APL2-308) trial to measure hemoglobin stabilization from baseline in the absence of transfusion through week 26 and reduction in lactate dehydrogenase levels from baseline to week 26

Ardelyx Inc., of Fremont, Calif.


Inhibits sodium hydrogen exchanger 3

Chronic kidney disease on dialysis with hyperphosphatemia not controlled by phosphate binders

In the 235-patient Amplify study, tenapanor plus phosphate binders reduced serum phosphorus from baseline to the end of week 4 by 0.84 mg/dL, compared to a mean reduction of 0.19 mg/dL for phosphate binders alone (p=0.0004); up to 49.1% of patients treated with tenapanor plus a binder achieved a serum phosphorus of <5.5 mg/dL, compared to 23.5% of patients treated with a binder alone (p≤0.0097); FGF23 levels were reduced by 22% to 24% (p≤0.0027) compared to binder alone

Astrazeneca plc, of Cambridge, U.K.

Brilinta (ticagrelor)

P2Y12 receptor antagonist

Coronary artery disease and type 2 diabetes with no prior heart attack or stroke

In the Themis study, Brilinta plus aspirin reduced the relative risk for the composite of cardiovascular death, heart attack or stroke by 10% compared with aspirin alone; in patients who had undergone a percutaneous coronary intervention, Brilinta plus aspirin reduced the risk by 15% compared to aspirin alone

Astrazeneca plc, of Cambridge, U.K.

Farxiga (dapagliflozin)

SGLT2 inhibitor

Heart failure and reduced ejection fraction

In the DAPA-HF study, Farxiga reduced the composite of cardiovascular (CV) death or worsening of heart failure by 26% (p<0.0001); the risk of experiencing a first episode of worsening heart failure was reduced by 30% (p<0.0001) and the risk of CV death was reduced by 18% (p=0.0294)

Genentech, a unit of Basel, Switzerland-based Roche Holding AG

Xofluza (baloxavir marboxil)

Inhibits cap-dependent endonuclease

Influenza treatment

In the Ministrone-2 study of children, drug was deemed safe; time to alleviation of influenza signs and symptoms was 138.1 hours for Xofluza compared to 150 hours for Tamiflu (oseltamivir, Gilead Sciences Inc.); median time to stop viral shedding was 24.2 hours for Xofluza compared to 75.8 hours for Tamiflu

Genentech, a unit of Basel, Switzerland-based Roche Holding AG

Xofluza (baloxavir marboxil)

Inhibits cap-dependent endonuclease

Influenza prevention

In the Bockstone study of people living with an infected household member, 1.9% of Xofluza-treated household contacts developed the flu compared with 13.6% of household contacts treated with placebo (p<0.0001); data were similar regardless of influenza A subtype and for household contacts who are at high risk of flu-associated complications and children under 12 years of age

The Medicines Co., of Parsippany, N.J.


siRNA targeting PCSK9

Atherosclerotic cardiovascular disease and familial hypercholesterolemia

In the Orion-11 study, inclisiran produced placebo-adjusted LDL-C reductions of 54% at day 510 and time-averaged placebo-adjusted LDL-C reductions of 50% from days 90 through 540 (p<0.0001 for both)

Novartis AG, of Basel, Switzerland

Entresto (sacubitril/valsartan)

Neprilysin inhibitor and an angiotensin II receptor blocker

Heart failure patients with preserved ejection fraction

In the 4,822-patient Paragon-HF study, Entresto reduced the composite primary endpoint of total (first and recurrent) heart failure hospitalizations and cardiovascular death by 13% compared to valsartan (p=0.059); total heart failure hospitalizations were reduced by 15% (p=0.056)

Phase IV

Novartis AG, of Basel, Switzerland

Entresto (sacubitril/valsartan)

Neprilysin inhibitor and an angiotensin II receptor blocker

Heart failure with reduced ejection fraction

In the 794-patient Prove-HF study, NT-proBNP was reduced by 30% from baseline to day 14 and by 37% at 12 months; LVEF, LAVi, LVEDVi, LVESVi and E/e all improved at 12 months


For more information about individual companies and/or products, see Cortellis.


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