June 2, 2020

Impact: High

Gilead and Galapagos have announced positive topline results from the phase 2b/3 randomized, double-blind, placebo-controlled of the oral selective JAK1 inhibitor given once-daily. The results were particularly favorable for biologic-naive patients.

The study evaluated filgotinib 200mg, filgotinib 100mg and placebo in 1,348 biologic-naïve or biologic-experienced adult patients with moderate to severe active ulcerative colitis.

Read more

June 2, 2020

Impact: High

During the virtual AAN 2020 conference, Biogen presented the baseline characteristics of participants in its phase 2 SPARK study of cinpanemab in PD. On May 20th, a month after the virtual conference, AAN opened its presentations to be viewed by the public without need for AAN registration, allowing us to view the slides on the SPARK study.

The study design for SPARK includes 3 treatment arms with cinpanemab doses of 250mg, 1250mg, and 3500mg. 357 patients are enrolled, split into two cohorts, the first consisting of 29 patients undergoing an intensive PK evaluation:

Read more

May 7, 2020

Impact: Moderate

Cortexyme today announced the publication of research further documenting the ability of the pathogen Porphyromonas gingivalis to invade neurons and trigger Alzheimer’s-like neuropathology. This paper was published in the Journal of Alzheimer’s disease. Although P. Gingivalis is best known as a primary driver of chronic periodontal disease, it is also known that it can infiltrate the brain, where it can cause characteristics similar to AD. P. gingivalis breaks down proteins for its primary source of energy by utilizing toxic virulence factor proteases known as gingipains.

More than 90% of post-mortem brains of patients with AD showed a presence of gingipains. Further, in animal models, P. gingivalis results in brain infection and classic AD pathology. In this paper, Cortexyme proofs for the first time that P. gingivalis can indeed invade the human brain and can persist in mature human neurons expressing active gingipains and that these infected neurons show signs of AD-like neuropathology, such as synapse loss. Read more

May 7, 2020

Impact: Moderate

Supernus Pharmaceuticals announced that it will acquire the CNS portfolio of US WorldMeds, including Apokyn, Myobloc, and Xadago, as well as the phase 3 Apomorphine Infusion Pump, which is expected to be filed in 2H 2020. For Supernus, the acquisition aligns with its corporate strategy to diversify its product portfolio into PD and other movements disorders, building on its marketed products in epilepsy and pipeline in psychiatric disease. The acquisition expands Supernus’ commercial platform to include sales and marketing capabilities, which will work toward achieving potential peak sales of $100M-$175M for Apomorphine Infusion Pump.

An upfront cash payment of $300M will be made plus regulatory and commercial milestones up to $230M. The transaction is anticipated to close in 2Q 2020. Read more

March 24, 2020

Impact: Moderate

FDA has announced that it will postpone or cancel all non-essential meetings through April 30, 2020 due to COVID-19. The following meetings are affected:

  • April 21, 2020: FDA Pulmonary-Allergy Drugs AdCom to discuss Trelegy Ellipta sNDA for IMPACT trial results (GlaxoSmithKline).
  • April 22, 2020: FDA AdCom (undetermined) to discuss sNDA for OCA (Ocaliva) in liver fibrosis due to NASH (Intercept).
  • April 23, 2020: FDA imaging Drugs AdCom to discuss NDA for flortaucipir F18, radioactive diagnostic agent for Alzheimer’s disease (Ely Lilly).

Read more

March 20, 2020

Impact: High

Regeneron and Sanofi have announced the initiation of a U.S. phase 2/3 trial evaluating anti-IL6 mAb Kevzara (sarilumab) in patients hospitalized with severe COVID-19 infection, to begin at medical centers in New York.

The adaptive design trial is expected to enroll up to 400 patients. The first part will evaluate the impact of Kevzara on fever and need for supplemental oxygen and the second part will evaluate the improvement in longer-term outcomes (preventing death, reducing the need for medical ventilation, supplemental oxygen and/or hospitalization).

Patients will be randomized 2:2:1 into Kevzara high dose, Kevzara low dose and placebo. The primary endpoint is reduction of fever and the secondary endpoint is decreased need for supplemental oxygen.

Trials outside the U.S. are expected to rapidly initiate, including in most affected areas such as Italy.

The restructuring of the Kevzara collaboration between the two companies is expected to be finalized in 1Q2020 as they continue to collaborate on COVID-19, with Regeneron leading U.S. work and Sanofi leading ex-U.S. work.

IL6 may play a role in driving the overactive inflammatory response in the lungs of COVID-19 patients and this is the first controlled U.S. trial to evaluate the effect of this cytokine inhibition.

Read more

February 7, 2020

Impact: Moderate

  • Innovent Biologics and Eli Lilly have announced that the phase 3 ORIENT-11 trial (NCT03607539) in China has met its primary endpoint of progression-free survival (PFS) for the use of Tyvyt (sintilimab) in combination with ALTIMA (pemetrexed) and platinum-based chemotherapy for patients with 1L advanced or recurrent nonsquamous NSCLC without EGFR mutations or ALK rearrangements tumors, when compared to placebo.
  • The safety profile of Tyvyt was consistent with previous studies, and no new safety signals emerged.
  • Based on this data, Innovent and Eli Lilly plan to initiate regulatory discussions with China’s National Medical Products Administration (NMPA) in the near future.
  • Data will be presented at an upcoming medical conference.

Read more

December 18, 2019

Impact: Moderate

  • Gilead’s Kite subsidiary has announced that it has submitted a BLA for the investigational CAR-T KTE-X19 for the treatment of patients with relapsed or refractory mantle cell lymphoma (R/R MCL).
  • The BLA is based on data from the Phase 2 ZUMA-2 trial which demonstrated an ORR of 93% (CR rate = 67%) in 60 evaluable patients. Enrolled patients had MCL that was refractory to or had relapsed following up to 5 prior lines of therapy.
  • Grade 3 or higher cytokine release syndrome (CRS) and neurotoxicity were seen in 15% and 31% of patients, respectively. No Grade 5 CRS or neurological events were observed.
  • Detailed findings from ZUMA-2 were presented Monday at ASH 2019.
  • KTE-X19 has been granted FDA Breakthrough Therapy and EMA PRIME status for the MCL indication.
  • Kite anticipates submitting a European Marketing Authorization Application in early 2020.

Read more

December 5, 2019

Impact: High

  • CymaBay Therapeutics has terminated its phase 2b study of PPARδ agonist, seladelpar, in patients with NASH due to atypical histological findings.
  • Planned, blinded histological assessments of liver biopsies from trial participants revealed histology characterized as an interface hepatitis presentation, with or without biliary injury.
  • Notably, the atypical histological findings were observed in patients who demonstrated on-study improvement or stabilization of their biochemical measures of inflammation and liver injury and none of these patients experienced liver-related adverse events after 52 weeks of treatment. Evaluation is ongoing to better understand these findings.
  • The company also terminated its recently initiated phase 2 study of seladelpar in primary sclerosing cholangitis (PSC), and all studies in primary biliary cholangitis (PBC) have been placed on clinical hold.

Read more

October 31, 2019

Impact: High

  • China regulators have cleared the IND for the fully human BCMA CAR-T, CT-103A, in development by Nanjing Iaso and Innovent Biologics.
  • In the recent issue of BioWorld (pages 1 & 6), Brian Hall, VP of Business Development at Iaso, stated that a phase 1b/2 trial to confirm the recommended phase 2 dose is planned to start 1Q20 and will enroll 60-70 patients. Hall also notes a CT-103A approval is planned in 2H21.
  • In the investigator-initiated trial at Tongji Hospital, CT-103A treatment led to an ORR of 100% (sCR: 71%, CR: 88%) and four patients who relapsed on a previous murine BCMA CAR-T therapy have all achieved > VGPR. Onset of CRS occurred within 2-5 days and resolved within two weeks.

Read more