PharmaShots' Key Highlights of Second Quarter 2020

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PharmaShots' Key Highlights of Second Quarter 2020

The second quarter of 2020 has ended with big approvals in pharma and biotech industry. Moving with the latest approval of Roche's Phesgo and Enspryng & Zogenix's Fintepla. Highlights of the quarter is COVID-19 as the development of vaccines and drugs were on peak. Celltrion evaluated Remsima (biosimilar, infliximab) against COVID-19 in the UK. The quarter also showcase multiple biosimilar news which includes the news of major global companies. Our team at PharmaShots has summarized and complied the insights of Q2'20.

Roche's Phesgo (fixed-dose combination of Perjeta and Herceptin) Receives the US FDA's Approval for Early and Metastatic HER2-Positive Breast Cancer

Date - Jun 30, 2020

Product - Phesgo

  • The approval is based on P-III FeDeriCa study which involves assessing of Phesgo (SC) + CT vs Perjeta (pertuzumab, IV) & Herceptin (trastuzumab, IV) + CT in 500 patients with HER2+ve early breast cancer treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings evaluated PK, efficacy, and safety
  • The study met its 1EPs showing non-inferior levels of Phesgo (SC) vs Perjeta (IV) in the blood plus no cardiac toxicity was demonstrated also the safety profile was comparable including no new safety signals. Additionally, in the P-II PHranceSCa study, 85% (136/160) patients preferred SC vs IV administration involving less time in the clinic with more comfort
  • Phesgo is an (FD, SC) formulation which involves a combination of pertuzumab + trastuzumab developed using Halozyme Therapeutics Enhanze drug delivery technology and administered within 8 mins for initial dosing and 5 mins for maintenance dosing

Roche's Enspryng (satralizumab) Receives MHLW's Approval for Neuromyelitis Optica Spectrum Disorder in Japan

Date - Jun 29, 2020

Product - Enspryng

  • The approval is based on two P-III SAkuraStar & SAkuraSky studies involve assessing Enspryng (120mg, SC, q4w) as a monothx & as an add-on therapy to baseline IST vs PBO in 95 & 83 patients aged 20-70 & 13-73yrs. in a ratio (2:1) & (1:1) administered at week 0,2 & 4 in patients with NMOSD respectively
  • In overall population: reduction in the risk of relapse (62% & 55%); In the pre-specified subgroup of AQP4-IgG seropositive patients: reduction in the risk of relapse (79% & 74%) respectively
  • Enspryng is a mAb targeting IL-6 and is under PR in Canada for NMOSD patients who are AQP4-IgG seropositive. In Oct'2019, the FDA & EMA has accepted the MAA for the therapy with expected CHMP & FDA's decision in 2020

Zogenix's Fintepla (fenfluramine) Receives the US FDA's Approval for the Treatment of Seizures Associated with Dravet Syndrome

Date - Jun 26, 2020

Product - Fintepla

  • The US FDA's approval is based on two P-III studies assessing Fintepla vs PBO in patients aged =2yrs. with Dravet syndrome and a safety data from an OLE trial in which many patients received Fintepla for up to 3yrs.
  • In addition to the existing treatment regimens, the therapy demonstrated a reduction in the frequency of monthly convulsive seizure in patients whose seizures were not controlled on one or more antiepileptic drugs
  • The company will launch the therapy through restricted distribution program, called the Fintepla REMS Program, and is expected to be available through Zogenix's specialty pharmacy partner by July'20

Gilead Holds an Exclusive Option to Acquire Pionyr Immunotherapeutics for ~$1.75B

Date - Jun 24, 2020

Deal Value - ~$1.75B

  • Gilead to acquire 49.9% stakes in Pionyr for $275M and get an exclusive option to acquire the remaining shares. Gilead may exercise its option upon completion of P-Ib studies for PY314 and PY159 or can exercise before the completion by paying $315M as option exercise fee
  • Additionally, Pionyr will receive up to $1.15B as milestones. Meanwhile, Gilead will provide Pionyr with additional funding for the PY314 and PY159 clinical programs, as well as ongoing research and development programs
  • Pionyr's Myeloid Tuning therapies (PY314 & PY159) showed preclinical efficacy, suggesting potential in solid tumors in combination with anti-PD(L)-1 agents. Pionyr anticipates IND filing with the US FDA for both PY314 and PY159 in Q3'20

Cipla Launches Cipremi (remdesivir lyophilized powder for injection 100 mg) to Treat Patients with Severe COVID-19

Date - Jun 22, 2020

Product - Cipremi

  • The US FDA has issued a EUA to Gilead for emergency use of remdesivir to treat hospitalized COVID-19 patients. In May, Gilead has extended a voluntary non-exclusive license to Cipla to manufacture and market Cipla's Remedisvir called CIPREMI
  • Cipla has received DCGI's approval for restricted emergency use in India as part of the accelerated approval process. Cipla will provide training on the use of the drug, informed patient consent documents, conduct post-marketing surveillance as well as to conduct a P-IV clinical trial on Indian patients
  • As per ACTT-1 study, 1063 patients were treated with Remdesivir vs PBO over 60 centers across the US, EU and Asia demonstrated faster time to clinical recovery in hospitalized patients with the mortality rate as (7.1% vs 11.9%)

Merck's Keytruda (pembrolizumab) Receives the US FDA's Approval as Monotherapy for Patients with Unresectable or Metastatic Tumor Mutational Burden-High Solid Tumors

Date - Jun 18, 2020

Product - Keytruda

  • The accelerated approval is based on KEYNOTE-158 study assessing Keytruda (200mg, q3w). In a study, 1,050 patients were included in the efficacy analysis population in which TMB was analyzed in the subset of 790 patients with sufficient tissue for testing based on protocol-specified testing requirements, out of 790, 102 had tumors identified as TMB-H, defined as TMB =10 mut/Mb
  • Results: In patients whose tumors were TMB-H, ORR (29%); CR (4%); PR (25%); after a median follow-up time of 11.1 mos. mDOR (not reached). In a pre-specified analysis of patients with TMB =13 mut/Mb: ORR (37%); CR (3%); PR (34%), In an exploratory analysis in 32 patients whose cancer had TMB =10 mut/Mb & <13 mut/Mb, the ORR (13%); median duration of exposure (4.9mos.)
  • The FDA also approved FoundationOne CDx test as the companion diagnostic to identify patients with solid tumors that are TMB-H (=10 mutations/ megabase) who may benefit from immunotherapy treatment with Keytruda monotherapy

Sanofi Report Results of Avalglucosidase alfa in P-III COMET Study for Late Onset Pompe Disease

Date - Jun 17, 2020

Product - Avalglucosidase

  • The P-III COMET study involves assessing avalglucosidase alfa (20 mg/kg) vs alglucosidase alfa (SOC, 20 mg/kg) IV infusion q2w for 49 wks. After 49 wks., patients previously receiving SOC switched to avalglucosidase alfa for the ongoing open-label treatment portion of the study
  • Results: met its 1EPs i.e., demonstrating non-inferiority in improving respiratory function, FVC (% predicted) (2.89 vs 0.46); 2EPs is measured mobility with the 6-minute walk test (6MWT) (32.21 vs 2.19 mts.); pre-specified preliminary analysis evaluating percent-predicted FVC and 6MWT in those patients who switched (switch patients) @49 wks. for the OLE period of the trial showed 0.15-point improvement in FVC and 23.32-meter improvement in 6MWT
  • Avalglucosidase alfa is an investigational ERT for Pompe disease designed to improve the delivery of enzyme to the cells in the muscles and is evaluated in ongoing P-II NEO-EXT study in patients with LOPD for a duration of up to ~ 8 yrs. in patients from the P-I /II NEO1 study. The regulatory submissions to be anticipated in H2'20

Celltrion Report Results of Truxima (biosimilar, rituximab) Based Regimen in P-II Study for R/R Aggressive B-cell Lymphoma #EHA25

Date - Jun 16, 2020

Product - Truxima

  • Celltrion has presented the result of P-II study that showed that regimen of Truxima (375mg/m2) + lenalidomide (20mg, day 1-21, qd) + acalabrutinib (100mg, day 1-28, bid) (R2A) is well-tolerated and effective in relapsed/refractory aggressive B-cell lymphoma
  • In the 13 patients who underwent disease assessment following the R2A regimen, ORR (69%) and CR (31%), @6-mos. PFS (83%) and only one patient experienced disease progression after the initial objective response
  • Throughout the study, out of 22 patients, dose reduction was performed in 3 and 1 patients for lenalidomide and acalabrutinib respectively due to hematologic toxicities. The data indicate that the R2A regimen was well tolerated in Korean r/r BCL patients, with initial analysis in non-GCB DLBCL patients showing a promising response

Mylan and Biocon Receive the US FDA's Approval for Semglee (biosimilar, insulin glargine)

Date - Jun 12, 2020

Product - Semglee

  • The US FDA's approval is based on comprehensive analytical, preclinical, and clinical programs (including the INSTRIDE (1&2) studies assessing MYL-1501D (qd) vs Lantus in 558 T1DM & 560 T2DM patients for 52wks. & 24wks. respectively. The 1EPS is changed from baseline in HbA1c after 24 wks.
  • The INSTRIDE demonstrated no difference in safety, efficacy, and immunogenicity of Semglee in comparison to Lantus in T1D & T2D. The drug is approved under the 505(b) NDA pathway and is a deemed a biologic under section 351(a) in accordance with the Biologics Price Competition and Innovation Act in line with other insulin products
  • Semglee has an identical amino acid sequence to Sanofi's Lantus and is approved in vials and pre-filled pen presentations, to control high blood sugar in adults with T2D and adult and pediatric patients with T1D. Semglee has received regulatory approval in 45+ countries across the world and is the third product approved by the FDA through the Mylan-Biocon biologics collaboration

Celltrion to Evaluate Remsima (biosimilar, infliximab) Against COVID-19 in the UK

Date - Jun 11, 2020

Product - Remsima

  • Celltrion collaborates with the University of Oxford and University Hospitals Birmingham to study the efficacy of Remsima (CT-P13) as a potential treatment for COVID-19
  • UHB will test a series of new drugs in a CATALYST trial. If the CATALYST study indicates benefit CT-P13 it would then be assessed in larger-scale studies in one of the UK national platform trials RECOVERY or REMAP-CAP
  • Additionally, UHB will evaluate Izana Bioscience's Namilumab (IZN-101) which is a mAb in a late-stage studies for RA and ankylosing spondylitis

RedHill Biopharma Reports the Submission of CTA for P-II/III Study Evaluating Opaganib Against COVID-19 in Russia

Date - Jun 11, 2020

Product - Opaganib

  • RedHill has submitted CTA with the Ministry of Health of the Russian Federation for a P-II/III clinical study evaluating Yeliva (opaganib, ABC294640)1 in patients hospitalized with severe COVID-19 and pneumonia
  • The P-II/III study will assess opaganib vs placebo, on top of SOC and plans to enroll 270 patients with severe COVID-19 pneumonia requiring hospitalization and treatment with supplemental oxygenation. The 1EPs of study will be the proportion of patients requiring intubation and mechanical ventilation by day 14
  • An interim analysis will be conducted when ~100 patients have been evaluated for 1EPs. The study will be conducted in clinical sites across Russia and other EU countries. Additionally, RedHill is initiating P-IIa study with opaganib in the US

Sandoz Receives Health Canada's Approval to Launch Ziextenzo (biosimilar, pegfilgrastim) and Riximyo (biosimilar, rituximab) in Canada

Date - Jun 10, 2020

Product - Ziextenzo

  • Health Canada has approved Ziextenzo (a biosimilar referencing Neulasta) and Riximyo (a biosimilar referencing Rituxan) for marketing in Canada
  • Sandoz has received Health Canada's Notice of Compliance on Apr 21, 2020, for the use of Ziextenzo to reduce the incidence of febrile neutropenia while Riximyo has received approval on Apr 28, 2020 to treat NHL, CLL and RA
  • The approval of Ziextenzo and Riximyo expands Sandoz's oncology biosimilar portfolio. The approval reinforces Sandoz’ status as the leading biosimilar and generic pharmaceutical company in Canada

Inovio to Initiate P-I/II Study of INO-4800 Against COVID-19 in South Korea

Date - Jun 4, 2020

Product - INO-4800

  • Inovio and IVI collaborate with Seoul National University Hospital to begin P-I/II clinical study of INO-4800 in South Korea
  • The P-II study will assess the safety, tolerability, and immunogenicity of the INO-4800 vaccine in 40 adults aged 19-50 yrs. and will expand to enroll an additional 120 people aged 19-64 yrs. the companies expect the trial to be initiate in 2020
  • The companies plan to initiate the clinical study in Jun'2020. The study will follow the study conducted in the US, began in Apr'2020. Earlier, the two companies collaborated to conduct P-I/IIa trials for a MERS vaccine (INO-4700/GLS-5300) developed by Inovio and GeneOne, evaluated in a clinical trial and has achieved promising results

Seattle Genetics Report Updated Results of Tukysa (tucatinib) in HER2CLIMB Study for Patients with HER2-Positive Breast Cancer with Stable or Active Brain Metastases #ASCO20

Date - Jun 1, 2020

Product - Tukysa

  • HER2CLIMB study involves assessing of Tukysa (tucatinib) + trastuzumab + capecitabine vs trastuzumab + capecitabine alone in 612 patients in a ratio (2:1) with LA unresectable/m-HER2+ BC prior treated with trastuzumab, pertuzumab, and T-DM1
  • Updated results: 42% reduction in the risk of death, 68% reduction in the risk of CNS disease; intracranial response rate (47% vs 20%); mOS (18.1 vs 12mos.); m-CNS-PFS (9.9 vs 4.2mos); DOR (6.8 vs 3.0mos.)
  • The additional analyses along with the primary analysis of HER2CLIMB, shows Tukysa is active for patients with/ out a disease that has spread to the brain. Tukysa (PO) is a TKI inhibitor of HER2 that improves OS and PFS in patients with metastatic HER2+ BC with/ out brain metastases

Eli Lilly's Tauvid (flortaucipir F 18) Receives the US FDA's Approval as the First Drug to Image Tau Pathology in Patients Being Evaluated for Alzheimer's Disease

Date - May 29, 2020

Product - Tauvid

  • The safety and effectiveness of Tauvid imaging is evaluated in two clinical studies. In study 1, reader interpretations of premortem TAUVID scans from 64 cognitively normal & terminally ill patients agreed to undergo Tauvid imaging and participate in a post-mortem brain donation program were compared to tau pathology at autopsy. The study met its 1EPs as with reader sensitivity ranging from 92%-100% & specificity from 52%- 92%
  • In study 2, images from the same terminally ill patients as in Study 1 and 159 patients with cognitive impairment being evaluated for AD were evaluated by 5 new readers. The study met criteria for comparison of Tauvid reads to NFT pathology, additionally, inter-reader agreement was evaluated using Fleiss kappa statistic and found to be 0.87 across 241 patients in Study 2
  • The availability of Tauvid will initially be limited and will expand in response to commercial demand and payor reimbursement. Tauvid is the first and only approved diagnostic agent to image tau NFTs in the brain

Fresenius Kabi Reports the US FDA's Acceptance of BLA for its MSB11455 (biosimilar, pegfilgrastim)

Date - May 28, 2020

Product - MSB11455

  • The BLA submission is based on analytical, PK/ PD, safety and immunogenicity data from two clinical studies that demonstrated equivalent PK/ PD profile to Neulasta, similar immunogenicity & comparable safety profile in healthy volunteers
  • The BLA represents Fresenius Kabi's first biosimilar candidate submitted to the FDA. Additionally, Fresenius Kabi also received EMA's acceptance for review MAA of MSB11455 on May 22, 2020
  • MSB11455 is a biosimilar referencing Amgen's Neulasta, acts by stimulating the growth of WBCs. Neulasta is indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with febrile neutropenia in the US

Algernon Reports Submission of IND to the US FDA for Ifenprodil to Treat COVID-19

Date - May 26, 2020

Product - Ifenprodil

  • The company has submitted the IND to the US FDA for evaluating its re-purposed drug NP-120 (Ifenprodil) in P-IIb/III study as a potential therapeutic treatment against COVID-19. The study will begin as a P-IIb study in 100 patients and with positive preliminary data, the clinical trial will move directly from a P-IIb into P-III study
  • The study will evaluate the safety and efficacy of NP-120 (20mg, q3w for 2wks.) + SOC vs SOC in confirmed COVID-19 infected hospitalized patients. The company has received clearance in Canada and has also filed for ethics approval in Australia
  • NP-120 is an NMDA receptor antagonist specifically targeting the NMDA-type subunit 2B (Glu2NB), acts by preventing glutamate signaling. Algernon has filed new IP rights globally for NP-120 to treat respiratory diseases and is working to develop an injectable and slow-release formulation

Daewon Pharmaceutical Launches its Terrosa (biosimilar, teriparatide) to Treat Osteoporosis

Date - May 26, 2020

Product - Terrosa

  • Terrosa is a parathyroid hormone (PTH) gene recombination candidate originally developed by Richter-Helm Biotec to treat osteoporosis in men and postmenopausal women including those at high risk of fracture. Additionally, Terrosa has completed its P-I and P-III trials in EU and Japan has shown an increase in bone density in the spine, femur, and hip joint
  • The 4-year post marketing surveillance study is conducted as the drug is listed in equivalent biologic treatment to the reference product. The launch was successful as the patent for 'stable teriparatide solution and 'increasing bone toughness and rigidity and fracture reduction method' expired in Dec 2018 and Aug 2019 respectively
  • In 2017, EMA approved Terrosa and was launched in EU and the US but not as biosimilar in Aug 2019 in 2019 respectively

Daiichi Sankyo and AstraZeneca's Enhertu (fam-trastuzumab deruxtecan-nxki) Receive the US FDA’s Orphan Drug Designation for Gastric Cancer

Date -  May 22, 2020

Product -  Enhertu

  • The US FDA's ODD is based on P-II DESTINY-Gastric01 study assessing Enhertu (6.4 mg/kg, q3w) vs CT in 188 patients in a ratio (2:1) with HER2-expressing, advanced gastric cancer, progressed with 2+ prior treatment regimens including 5-FU, Pt. CT and trastuzumab
  • The study demonstrated improvement in ORR & OS and will be presented at ASCO20. The designation follows US FDA's BT designation of Enhertu for HER2+ metastatic gastric cancer and HER2 mutant m-NSCLC
  • Enhertu is a HER2 directed ADC, designed utilizing Daiichi Sankyo's DXd ADC technology and has received MHLW’s SAKIGAKE designation in Mar'2018 for HER2 positive gastric cancer with recently submitted sNDA to MHLW

Samsung Bioepis Reports Results of SB11 (proposed biosimilar ranibizumab) in P-III Study for Neovascular Age-Related Macular Degeneration

Date - May 19, 2020

Product - SB11

  • The P-III study involves assessing of SB11 vs reference product, LUCENTIS in monthly injections (0.5mg) in 705 patients in a ratio (1:1) with nAMD
  • The P-III study results demonstrated equivalent efficacy in terms of change in BCVA @8wk. and CST @4wk., LS change in BCVA (6.2 vs 7.0 letters), LS change in CST ( -108.4µm vs -100.1µm), AEs (66.0% vs 66.9%). The data was to be presented at ARVO 2020 which has been cancelled due to COVID-19
  • Additionally, in Nov'2019, Samsung collaborated with Biogen for two ophthalmology biosimilar candidates, SB11 (ranibizumab) and SB15 (aflibercept), in the US, Canada, EU, Japan, and Australia

AstraZeneca's Bevespi Aerosphere (glycopyrronium/formoterol fumarate) Receives NMPA’s Approval for Patients with COPD

Date - May 18, 2020

Product - Bevespi Aerosphere

  • The NMPA's approval is based on P-III PINNACLE 4 study assessing Bevespi Aerosphere (bid via a pMDI) vs its monotherapy components (glycopyrronium and formoterol fumarate) and PBO in patients with moderate to very severe COPD
  • The P-III PINNACLE 4 study demonstrated improvement in lung function as measured by trough forced expiratory volume in one second (FEV1), published in the International Journal of Chronic Obstructive Pulmonary Disease
  • The approval follows the approval of AstraZeneca's triple-combination therapy, Breztri Aerosphere (budesonide/glycopyrronium/formoterol fumarate) for the maintenance treatment of COPD in China. Bevespi Aerosphere (FD) is a dual bronchodilator, administered in a pressurized metered-dose inhaler utilizing Aerosphere delivery technology

Samsung Bioepis Reports Results of Ontruzant (biosimilar, trastuzumab) in P-III Follow-up Study (SB3-G31-BC-E) for Early or Locally Advanced HER2-Positive Breast Cancer

Date - May 15, 2020

Product - Ontruzant

  • The four-year follow-up data is part of the ongoing follow-up study, assessing the cardiac safety and survival outcome of Ontruzant vs Herceptin in a subgroup of patients from P-III study with completed neoadjuvant and adjuvant therapy for 1yr. 367 out of 875 patients were enrolled in the extension study, with a median follow-up of 53mos.
  • Four-year follow-up data: EFS (83.4% vs 80.7%); OS (94.4% vs 89.6%); no occurrence of symptomatic CHF with a very low incidence of asymptomatic significant LVEF decrease. The data supports comparable safety and efficacy profiles of Ontruzant to the Herceptin
  • Ontruzant showed high bio-similarity to Herceptin in terms of safety, purity and potency of therapy and has received the FDA's approval in Jan'2019. The company will present the four-year follow-up data in ASCO

BMS and bluebird bio to Resubmit the BLA for Idecabtagene Vicleucel (ide-cel, bb2121) to Treat Patients with Multiple Myeloma

Date - May 14, 2020

Product - Idecabtagene Vicleucel

  • The companies receive the US FDA's Refusal to File letter regarding the BLA for idecabtagene vicleucel for patients with heavily pre-treated r/r MM, which was submitted in Mar'2020
  • Upon preliminary review, the US FDA determined that the Chemistry, Manufacturing and Control module of the BLA requires further detail to complete the review
  • Ide-cel is a BCMA-directed genetically modified autologous CAR T cell immunotherapy and has received the US FDA's BT designation and EMA's PRIME designation for r/r MM. Additionally, BMS to resubmit the BLA no later than the end of July 2020

Neurocrine Biosciences Exercises its Option with Idorsia for ACT-709478

Date - May 12, 2020

Product - ACT-709478

  • Idorsia to receive $45M upfront in cash- $365M for development & regulatory milestone- one-time sales threshold and royalties on sales. Additionally- will receive $7M in funding to discover- identify and develop additional novel T-type calcium channel blockers
  • Neurocrine exercises its option to license rights for ACT-709478 (post IND acceptance from the US FDA on Apr 30- 2020) for rare pediatric epilepsy. In 2019- Neurocrine and Idorsia signed a preclinical research collaboraion for ACT-709478 to treat rare pediatric epilepsy
  • ACT-709478 is an selective- orally-active and brain penetrating T-type calcium channel blocker also received the US FDA's Rare Pediatric Disease designation for rare pediatric epilepsy with completion of P-I in 2019 and expected P-II initiation in in H2'20

Related Post: PharmaShots' Key Highlights of Second Quarter 2020

Senior Editor

This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com

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