Phase I

Ampliphi Biosciences Corp., of San Diego



Staphylococcal sepsis

Clinical case series data from ongoing expanded access program showed that 13 patients treated with AB-SA01 had severe S. aureus sepsis and/or bacteremia; about 290 doses of AB-SA01 were administered intravenously and were well-tolerated with no adverse events; 83% of patients (10 of 12) in the modified intent-to-treat population achieved treatment success at the end of therapy, defined as a complete resolution or significant improvement of baseline signs and symptoms; initial gene expression data indicate bacteriophage treatment may down-regulate pro-inflammatory genes and up-regulate anti-inflammatory genes

International Stem Cell Corp., of Carlsbad, Calif.


Stem cells

Parkinson's disease

Second patient of third cohort was successfully transplanted with 70M ISC-hpNSC cells; 6 patients have now fully completed the study and entered the follow-up phase, while 2 others have completed the 6-month evaluation; treatment phase will conclude following dosing of the last 2 patients of the third cohort, which ISCO plans to complete soon

Loxo Oncology Inc., of Stamford, Conn.


RET inhibitor

Medullary thyroid cancer

Data from LIBRETTO-001 trial in 38 patients with RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer showed 16 of 17 (94%) responding RET-mutant MTC patients remained on therapy, with median follow-up of 8.4 months; 7 of 7 (100%) responding RET fusion-positive thyroid cancer patients remained on therapy, with median follow-up of 8.5 months; inclusion of data from recently enrolled patients resulted in a 59% overall response rate (56% confirmed ORR) in the presented subset of RET-mutant MTC patients, and a 78% confirmed ORR in the presented subset of RET fusion-positive thyroid cancer patients

Lyra Therapeutics Inc., of Watertown, Mass.


Steroid, mometasone furoate

Chronic rhinosinusitis

20 patients who have not had sinus surgery received in-office bilateral administration of LYR 210, and results showed drug was safe and well-tolerated; patients achieved significant improvement in the sinonasal outcome test (SNOT-22) after single administration; a phase II study is planned to begin in the first half of 2019

Tetraphase Pharmaceuticals Inc., of Watertown, Mass.


Tetracycline-derived compound

Gram-negative bacterium infection

Generally safe and well-tolerated; advancing to bronchopulmonary disposition study in Q1 2019

Phase II

Biogen Inc., of Cambridge, Mass.

Spinraza (nusisersen)

Antisense oligonucleotide

Spinal muscular atrophy

Interim results from NURTURE in 25 presymptomatic infants showed that, as of May, all patients were alive and none required tracheostomy or permanent ventilation; 22 of the 25 participants were able to walk either with assistance or independently, according to the motor milestone standard of the World Health Organization, and all 25 were able to sit without support; using the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders, the mean scores were 62.6 for study participants with 3 copies of the SMN2 gene and 61 for those with 2 copies

Cavion Inc., of Charlottesville, Va.


Calcium channel modulator

Essential tremor

Results of the T-CALM trial showed treatment at 10 mg twice daily resulted in statistically significant improvement vs. placebo in activities of daily living and patient-reported and clinical outcome measures, despite the study's primary endpoint, remote measurement of Tremor Research Group Essential Tremor Rating Assessment Scale – Performance Subscale, being missed (p=0.696); when rated by study investigators who observed patients in person, there was significant improvement in tremor severity at day 28 vs. placebo (p=0.027)

Cocrystal Pharma Inc., of Atlanta


Non-nucleoside inhibitor

Hepatitis C

Agreement for investigator-initiated study with the Humanity & Health Research Centre in Hong Kong

Menlo Therapeutics Inc., of Redwood City, Calif.


NK1 receptor antagonist

Refractory chronic cough

Top-line results showed treatment failed to demonstrate benefit vs. placebo on the primary and key secondary endpoints; in the primary endpoint analysis of change from baseline in 24-hour cough frequency after 12 weeks of treatment, the serlopitant group had 31% less reduction than the placebo group; in a key secondary analysis of response rates, 54% of placebo-treated patients and 44% of serlopitant-treated patients experienced a 30% or greater reduction in 24-hour cough frequency at week 12 compared to baseline; serlopitant was well-tolerated

Phase III

Adamas Pharmaceuticals Inc., of Emeryville, Calif.



Parkinson's disease

Overall, final results from 2-year, open-label study demonstrated the product was generally well-tolerated; treatment effect on motor complications (dyskinesia and OFF), as measured by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale, Part IV, was maintained for up to 2 years, and that effect was seen in all subgroups, including those switched to Gocovri from placebo (n=78) or amantadine immediate release ( n=32) and those with uncontrollable dyskinesia after deep brain stimulation (n=61) treatment

Ardelyx Inc., of Fremont, Calif.


Sodium transporter NHE3 inhibitor

Irritable bowel syndrome with constipation

T3MPO-1 and T3MPO-2 trials achieved statistical significance for their primary endpoint and demonstrated the ability of tenapanor to have a durable effect on reducing the constipation and abdominal pain

Diurnal Group plc, of Cardiff, U.K.


Cortisol replacement product

Congenital adrenal hyperplasia

Achieved control on a lower overall dose of glucocorticoid with fewer patients requiring rescue therapy (sick day rules); eliminated the early morning peak in androgen levels seen using conventional therapy

Exelixis Inc., of Alameda, Calif.


Multi-tyrosine kinase inhibitor

Radioiodine-refractory differentiated thyroid cancer

Trial begins in patients who have progressed after up to 2 prior VEGFR-targeted therapies; co-primary endpoints are progression-free survival and objective response rate

Poxel SA, of Lyon, France


Glimin agent that targets mitochondrial bioenergetics

Type 2 diabetes

Patient enrollment completed in the Times 2 trial

Synergy Pharmaceuticals Inc., of New York

Trulance (plecanatide)

Analogue of uroguanylin and oral guanylate cyclase-C receptor

Constipation and irritable bowel syndrome

Post-hoc analysis of CIC and IBS-C patients showed safety and tolerability of Trulance in adults ≥65 similar to younger cohort; second analysis found efficacy and safety in patients on concomitant acid suppression therapy similar to overall population

Tetraphase Pharmaceuticals Inc., of Watertown, Mass.

Xerava (eravacycline)

Synthetic fluorocycline antibiotic

Bacterial infection

Pooled analysis of data from 2 phase III trials in complicated intra-abdominal infection found high clinical cure rates and microbiological eradication among patients with concurrent bacteremia

Phase IV

Takeda Pharmaceuticals USA Inc., of Deerfield, Ill.

Entyvio (vedolizumab)

Anti-alpha-4/beta-7 integrin receptor antibody

Ulcerative colitis and Crohn's disease

New long-term efficacy real-world data analyses found 78.6% of ulcerative colitis and 74.8% of Crohn's disease patients persisted on Entyvio treatment, and at 24 months, 76.9% of UC and 74.8% of CD patients persisted

Theravance Biopharma Inc., of Dublin

Vibativ (telavancin)

Semisynthetic lipoglycopeptide antibiotic

Staphylococcus aureus infection

Post-approval observational study found positive clinical response after treatment in 77.1% of registry patients diagnosed with monomicrobial infections caused by S. aureus with vancomycin minimum inhibitory concentration ≥ 1 µg/mL; prolonged treatment led to no change in renal function for 68.7% of very sick patients


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


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