PharmaShots' Key Highlights of First Quarter 2020
The first quarter of 2020 includes big news regarding global companies. The highlight of the quarter is the acquisition of QIAGEN by ThermoFisher for ~$11.5B, Danaher's acquisition of the biopharma business of GE Life Sciences in an all-cash transaction for ~$21.4B. Numerous alliances are there which include Fosun's agreement with BioNTech to develop and commercialize BNT162 to combat COVID-19. The first quarter of the year also showcases regulatory events which include RG6042's (ONIS-HTTRx) ODD in Japan and Abbott's HeartMate 3 Heart Pump FDA's approval to avoid open-heart surgery. Our team at PharmaShots has summarized and complied with the insights of Q1'20.
Date - Mar 31, 2020
Product - Lokelma
- The EMA's CHMP has adopted a positive opinion on a dosing and administration label update for AstraZeneca's Lokelma to include patients with hyperkalemia on stable hemodialysis
- The recommendation is based on P-IIIb DIALIZE study assessing Lokelma (qd) vs PBO in 196 patients on non-dialysis days for 8wks. The study demonstrated that patients-maintained pre-dialysis potassium levels on at least 3 out of 4 dialysis treatments, following the long interdialytic interval and did not require urgent rescue therapy (41% vs 1%)
- Lokelma is an insoluble, non-absorbed sodium zirconium silicate, formulated as a powder for oral suspension and acts as a highly selective potassium-removing medicine and is an approved therapy for the treatment of hyperkalemia in the US, EU, Canada, Hong Kong, China, Russia and Japan
Date - Mar 30, 2020
Product - Ozanimod
- The approval is based on P-III SUNBEAM & RADIANCE Part B studies assessing Zeposia (0.92 mg and 0.46 mg, equivalent to 1 mg and 0.5 mg ozanimod HCI) vs Avonex (qw, IM) in 1,346 & 1,320 patients with RMS for at least 12 & 24mos. treatment period respectively
- The results will further be reviewed by the European Commission, which has the authority to approve medicines for the European Union
- Zeposia (ozanimod) is an oral S1P receptor modulator involved in binding with S1P receptors 1 and 5. On Mar 25, 2020 the US FDA approved Zeposia for the treatment of adults with relapsing forms of multiple sclerosis (RMS)
Medopad Provided its Remote Patient Monitoring Platform to Support Global COVID-19 Pandemic
Date - Mar 27, 2020
Product - RPM Platform
- Medopod's RPM platform will be available for NHS Trusts across the UK as well as global healthcare providers that allow doctors to track patient's data, symptom progression and flag patients with worsening symptoms, both inside and outside of clinical settings
- Patients will use the app to share their personal data such as temperature, respiration rate and heart rate. Medopad has worked with Imperial College, Guys and St Thomas NHS Foundation Trust, as well as Johns Hopkins University to create the specialist RPM platform for tracking COVID-19
- The Medopod's RPM software is CE marked and under FDA enforcement discretion and is geared up to be deployed across the EU, US, China, Singapore, Hong Kong and Saudi Arabia
Date - Mar 25, 2020
Product - Tepmetko
- The approval is based on ongoing P-II VISION study assessing Tepmetko in 99 patients (including 15 Japanese patients) with NSCLC and METex14 skipping alterations
- The P-II VISION study results: ORR as assessed by IRC (42.4%) in patients identified by LBx/ TBx; mDOR (12.4mos) for both LBx/TBx identified. Additionally, in the safety analysis of 130 patients, Tepmetko was well tolerated
- Tepmetko (500mg, qd) is an oral MET inhibitor in Japan to be approved for the line-agnostic treatment of advanced NSCLC with MET exon 14 (METex14) skipping alterations and has received FDA's BT designation in Sept'2019 with its anticipated regulatory filing to the US FDA in 2020
The US FDA Approved a New Pathway for Biosimilars
Date - Mar 23, 2020
Product- Nepexto
- The EMA's CHMP has adopted a positive opinion recommending the approval of Nepexto, a biosimilar referencing Enbrel (etanercept), for all indications of the reference product including RA, JIA, PsA, axSpA (including AS and nr-axSpA), PsO and pediatric PsO
- The positive opinion is based on biosimilarity assessment which included pre/ clinical studies demonstrating bioequivalence to the reference product. Additionally, the P-III clinical study demonstrated the equivalence of Nepexto to the Enbrel in patients with mod. to sev. RA
- The EC will review the CHMP's positive opinion with its anticipated approval in May'2020. In Jun'2018, the companies collaborated to commercialize Nepexton in multiple global markets
Date - Mar 19, 2020
Product - Lynparza
- The ODD is based on P-III POLO study assessing Lynparza (300mg, bid) as maintenance monothx. vs PBO in 154 patients in a ratio (3:2) with gBRCAm metastatic PC without disease progression on 1L Pt. based CT
- The P-III POLO study results: improvement in PFS (7.4 vs 3.8mos.); 47% reduction in disease progression; patients with measurable disease at baseline, responded patients (23% vs 12%); median duration of treatment in excess of 2yrs. (24.9 vs 3.7mos.); median OS (18.9 vs 18.1mos.)
- Lynparza is a first-in-class PARP inhibitor acts by targeting DDR in cells/tumors harboring a deficiency in HRR (BRCA1& 2 mutation) and has received the US FDA's approval in Dec'2019 with its ongoing regulatory reviews in the EU and other regulatory bodies
Moleculin Collaborated with UTMB to Evaluate WP1122 for Multiple Viruses Including Coronavirus
Date - Mar 18, 2020
Product - WP1122
- Moleculin will provide its lead candidate, WP1122, and related inhibitors along with technical support while UTMB will investigate candidates against multiple viruses including COVID-19, in connection with UTMB's CBEID
- UTMB's CBEID collaborates with the Galveston National Laboratory, which is funded by NIAID the US Department of Defense, the US Centers for Disease Control & Prevention and other federal agencies along with academic partners, private foundations, and the biopharmaceutical industry
- WP1122 is a prodrug of 2-DG (2-deoxy-D-glucose) that overcome 2-DG's lack of drug-like properties and demonstrated increment in the tissue/organ concentration in its preclinical study
Date - Mar 17, 2020
Product - Nucala
- Nucala (mepolizumab) has received Health Canada's approval as an add-on maintenance therapy for adolescents & children aged =6yrs. with SEA
- The extension of NUCALA's indication for adolescents and children aged =6yrs. allow the company to bring the therapy to patients and their families with limited treatment options
- Nucala is the first IL5 biologic to be approved for SAE in a pediatric population (as young as 6 yrs. old) in Canada. The therapy has received approval in 20+ markets, including the US & EU for adult patients with SAE and indicated as an approved therapy for pediatric use in patients aged 6-17yrs. as an add-on treatment for SAE
Fosun Pharma Signed an Agreement with BioNTech to Develop and Commercialize BNT162 for COVID-19
Date - Mar 16, 2020
Deal Value - ~ $ 135M
- BioNTech to receive $135M as up front, future investment & milestone payments and $50M as an equity investment for 1,580,777 ordinary shares in BioNTech. Fosun to get the rights to commercialize BNT162 in China while BioNTech retain all rights of the vaccine in ROW
- The companies will jointly conduct clinical studies of BNT162 in China, leveraging BioNTech's mRNA vaccine technology and Fosun's clinical development and commercialization capabilities in China
- The companies will share profits from sales in China. Additionally, BioNtech with its partner Polymun will supply the mRNA vaccine for clinical studies from GMP manufacturing facilities in the EU
Date - Mar 10, 2020
Product - Fiasp
- Health Canada has expanded the approval of Fiasp (10mL vials) to include use in insulin infusion pumps for the improvement of glycemic control in pediatric patients aged =2yrs with diabetes (T1D/T2D both)
- The approval is based on data from 7 clinical studies that verified the safety & efficacy of Fiasp in children. The label change for use in insulin infusion pumps is based on Health Canada's review of data from 5 clinical studies which demonstrated the efficacy of Fiasp used in insulin infusion pumps in adults with diabetes
- Fiasp is the first and only fast-acting mealtime insulin injection, administered at the beginning of a meal or within 20 minutes after starting a meal and has received FDA's approval as an IV infusion or SC MDI in adults with diabetes
Date - Mar 04, 2020
Product - Givlaari
- The EC has granted MAA which is based on P-III ENVISION study assessing Givlaari (2.5 mg/kg monthly, SC) vs PBO in 94 patients aged =12yrs. in a ratio (1:1) with a documented diagnosis of AHP
- The P-III ENVISION study results: 74% reduction in the annualized composite rate of porphyria attacks in AIP patients; attack-free patients during 6mos. treatment (50% vs 16.3%); improvement in pain; reduction in the use of hemin, ALA & PBG
- Givlaari is an RNAi therapy targeting ALAS1 and has received EMA's ODD and PRIME designation. The EC approval follows the US FDA approval of the therapy received in Nov'2019 and is under priority review in Brazil
Thermo Fisher Scientific Acquired QIAGEN for ~$11.5B
Date - Mar 3, 2020
Deal Value - ~$11.5 B
- Thermo Fisher to acquire QIAGEN in an all-cash transaction for $43.3/ share, making a total deal value ~$11.5B with a premium of 23% to the closing price of QIAGEN's common stock on the Frankfurt Prime Standard on Mar 02, 2020. The transaction is expected to be completed in H1'21
- The focus of the acquisition is to expand Thermo Fisher's specialty diagnostics portfolio with attractive molecular diagnostics capabilities, complements its life sciences offering with innovative sample preparation, assay and bioinformatics technologies and expand its customer access
- The acquisition provides Thermo Fisher an opportunity to leverage its capabilities and R&D expertise to accelerate innovation, addressing the emerging healthcare needs
Fujifilm's CAD EYE Received CE Mark to Support the Detection of Colonic Polyp Utilizing AI in the EU
Date - Feb 28, 2020
Product - CAD EYE
- Fujifilm's CAD EYE receives CE mark, backing the real-time detection of colonic polyps during colonoscopy utilizing AI, will be available with software EW10-EC01 and the compatible expansion unit EX-1 in combination with the ELUXEO 7000 system
- CAD EYE utilizes FUJIFILM's REiLi AI technology and can perform complex segmentation of 2D/3D images, spot lesions & is compatible with various imaging modalities. It automatically engages with white light or LCI mode to improve the accuracy of lesion detection
- CAD EYE is customized detection support used with the ELUXEO system, aimed to improve lesion detection in the colon at the expert level and is expected to be available with EX-1 in Mar'2020
Date - Feb 27, 2020
Product - Zejula
- The EMA has accepted Type II Variation (T2V) for Zejula as maintenance therapy in a 1L setting for women with advanced platinum-responsive advanced OC, regardless of biomarker status. The validation indicates the acceptance of MAA and the initiation of CHMP's formal review process
- The submission is based on P-III PRIMA study assessing Zejula vs PBO in women in a ratio (2:1) as 1L therapy for stage III/ IV platinum-responsive advanced OC. The study demonstrated clinical outcomes of Zejula
- Zejula (PO, qd) is a PARP inhibitor, indicated as a monothx. for the maintenance treatment of patients with platinum-sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response to platinum-based CT
Date - Feb 26, 2020
Product - Nexlizet
- The approval is based on P-III trial Fixed Combination Drug Product LDL-C Lowering program, involves assessing of Nexlizet vs PBO when added on to maximally tolerated statins
- The study resulted in well-tolerated data and lowered LDL-C by 38%, when added on to maximally tolerated statins. Also, the results are published in The European Journal of Preventative Cardiology
- Nexlizet is an oral qd, non-statin LDL-cholesterol lowering medicine approved by the US FDA on Feb 21, 2020 and will be available in Jul 2020. Nexletol (bempedoic acid) is a novel ATP Citrate Lyase inhibitor involves lowering of LDL-C by cholesterol biosynthesis and up-regulating the LDL receptors, will be available from Mar 30, 2020
WuXi Vaccines Signed Twenty Years Manufacturing Agreement with Global Vaccine Leaders for $3B
Date - Feb 19, 2020
Deal Value -~ $3B
- Wuxi Vaccines to establish new manufacturing facility which includes drug substance manufacturing (DS), drug product manufacturing (DP), manufacture science and technology labs (MS&T) as well as quality control labs (QC)
- The facility will develop and manufacture partner's vaccine therapy for the global market. The new facility is expected to be operational in 2022
- Additionally, in Nov'2019, Wuxi invests $240M to establish a new vaccine manufacturing facility in Ireland. Wuxi biologics will provide its global partners with a robust and premier-quality supply chain network to develop high-quality vaccines protecting humans globally
Chugai's RG6042 Received MHLW's Orphan Drug Designation for Huntingto's Disease in Japan
Date - Feb 18, 2020
Product - RG6042
- The MHLW has granted Chugai's RG6042 an ODD for the expected treatment of Huntington's disease
- The RG6042 is currently being evaluated in P-III GENERATION HD1 study and is expected to delay the progression of Huntington's disease as the first disease-modifying therapy
- RG6042 (ONIS-HTTRx) is an antisense oligonucleotide targeting HTT mRNA and has received FDA & EMA's ODD for the Huntington's disease in May & Dec'2015 with EMA's PRIME designation in 2018
Date - Feb 17, 2020
Product - Remsima SC
- The clinical study involves assessing the PK, efficacy, and safety of CT-P13 SC vs CT-P13 IV in 131 patients with active CD & UC for 1yr. Following loading doses of IV 5mg/kg @0 & 2wks., patients were randomized @6wks. to receive either SC 120/240 mg q2w or continued IV 5 mg/kg q8w, @30wks. IV arm switched to SC arm based on body weight
- The clinical study results demonstrated that Remsima SC is comparable to its IV formulation @1yr., improvement in clinical activity, rate of clinical response and remission are maintained, improvement in the rate of clinical healing
- Remsima SC (CT-P13 SC) is the first & only SC infliximab to be available in EU with its expected approval in EU for IBD in mid-2020 and has received EMA's MAA for RA in H2'19
Date - Feb 14, 2020
Product - Tazverik
- The US FDA has accepted NDA for the accelerated approval of Tazverik for patients with relapsed/refractory FL prior treated with 2L+ systemic therapies
- The US FDA has granted PR and designated its NDA as sNDA. The sNDA is based on P-II study that demonstrated clinical benefits and was well tolerated in FL patients with EZH2 activating mutations & with wild type EZH2
- Tazverik is a methyltransferase inhibitor indicated to treat patients aged =16yrs. with metastatic or LA epithelioid sarcoma not eligible for complete resection with its anticipated PDUFA date as Jun 18, 2020
Date - Feb 14, 2020
Product - Tecentriq
- The NMPA's approval is based on P-III IMpower133 study assessing Tecentriq + CT vs PBO + CT as monothx. in 403 CT naïve patients in a ratio (1:1) with ES-SCLC
- The P-III IMpower study results: OS (12.3 vs 10.3 mos.); mPFS (5.2 vs 4.3 mos.); follow up analysis @18mos. OS rate (34% vs 21%); safety profile is consistent with the previous profile
- Tecentriq is a mAb targeting PD-L1, blocking its interaction with both PD-1 and B7.1 receptor. In Jan'2020, NMPA has accepted sBLA for Tecentriq + Avastin (bevacizumab) for unresectable HCC, prior not treated with systemic therapy
Date - Feb 12, 2020
Product - Keytruda
- The P-III KEYNOTE-355 (part 1 & 2) study involves assessing of Keytruda (200mg, IV, on day 1 of each 21-day cycle) + CT vs PBO + CT in 30 & 847 patients with LA inoperable or mTNBC prior not treated with CT in the metastatic setting
- The P-III KEYNOTE-355 study resulted in meeting one of its dual 1EPs i.e, improvement in PFS, the safety profile is consistent with the previous studies with no new safety signals. Following the recommendation of DMC, the study will continue its other 1EPs of OS
- Keytruda is an anti-PD-1 therapy, act by blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2 thus activating T lymphocytes affecting both tumor and healthy cells
Date - Feb 11, 2020
Product - Capmatinib
- The US FDA has accepted NDA and granted PR designation to Capmatinib as a 1L treatment for LA/m- MET exon 14 skipping (METex14) mutated NSCLC
- The NDA submission is based on GEOMETRY mono-1 P-II study that demonstrated ORR (67.9%) assessed by BIRC assessment per RECIST v1.1, mDOR (11.4 and 9.72 mos.) in treatment naïve and prior treated patients respectively
- Capmatinib (INC280) is an investigational, oral, potent and selective MET inhibitor with its expected review to be completed within 6mos. The therapy is licensed from Incyte in 2009, under which Novartis got WW exclusive development and commercialization rights for the therapy and certain back-up compounds in all indications
Collegium Acquired US Rights of Assertio's Nucynta Franchise for $375M
Date - Feb 07, 2020
Deal Value - ~ $375 M
- Assertio to receive $375M upfront in cash and fewer royalties in 2020, subject to certain adjustments. Collegium to get the US license or the Nucynta Franchise and will not pay royalties to Assertio with the anticipated completion of the transaction on Feb 14, 2020
- Additionally, Collegium will continue to pay 14% royalties on net sales on Nucynta Franchise to Grunenthal and will not pay royalties on sales >$180M
- In 2017, Collegium signed a commercialization agreement with Depomed (now Assertio) for the Nucynta franchise in exchange for royalties with an annual commitment of $135M for the first 4yrs and after 4yrs. the royalty mechanism remains the same but without a minimum
ProQR's QR-421a Received FDA's Rare Pediatric Disease Designation for Retinitis Pigmentosa
Date - Jan 31, 2020
Product - QR-421a
- The US FDA has granted RPD to the QR-421a for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene
- The RPD designation provides PR voucher by the FDA to the sponsor, that voucher can be redeemed to receive FDA's PR designation for any subsequent MAA for a different product
- QR-421a (IVT) is a first-in-class investigational RNA-based oligonucleotide addressing the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic RP due to mutations in exon 13 of the USH2A gene and has received FDA's ODD and EU's FT designation
Date - Jan 29, 2020
Product - Selpercatinib
- The NDA is based on P-I/II LIBRETTO-001 study assessing selpercatinib in patients with advanced RET fusion+ NSCLC & thyroid cancer and RET-mutant MTC. The study included P-I as dose-escalation phase and P-II dose-expansion phase
- Selpercatinib has received FDA's BT and ODD for the above mention indications and is currently being evaluated in two P-III studies LIBRETTO-431 for patients with treatment-naïve RET fusion+ NSCLC, and LIBRETTO-531 for patients with treatment-naïve RET-mutant MTC
- Selpercatinib is a highly selective and potent investigational therapy for multiple cancer indications harboring abnormalities in the RET kinase with its anticipated PDUFA date in Q3'2020
Bausch + Lomb Collaborated with TerraCycle to Launch First Contact Lens Recycling Program in Canada
Date - Jan 28, 2020
Product - Contact Lens
- The companies will collaboratively launch Bausch + Lomb Every Contact Counts recycling program in eyecare offices across Canada- allowing people to recycle their disposable contact lenses and blister pack packaging
- The goal of the initiative is to provide an opportunity to eye care professionals and patients to collect waste with an aim to increase the number of recycled contact lenses and their associated packaging thus reducing their overall impact on the environment
- Contact lenses and blister packs are collected at Every Contact Counts- shipped to TerraCycle recycling facility to get recycled into plastic that can be remolded to create new products. Additionally- Bausch + Lomb has similar recycling programs in the Netherlands- the US- and Australia
Related Post: Insights+: The US FDA New Drug Approvals in December 2019 and January 2020
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
