PharmaShot's key highlights of Q4 2020 (2023)

• The fourth quarter of 2020 includes multiple clinical trial initiations, major approvals and numerous deals. News related to COVID-19 continues to be at an all-time high this quarter
• Several companies have received EUAs from regulatory authorities for their vaccines and treatments against COVID-19. Regeneron initiated and applied for the US FDA EUA for the REGN-COV2-Ab combination, while Health Canada accelerated the review of the COVID-19 vaccine from AZ. Meanwhile, AZ's AZD1222 met its primary endpoint in preventing COVID-19
• Our team at PharmaShots has summarized and incorporated the insights from Q4'20

Sanofi launches Tetraxim (DTaP-IPV) for preschool children in India

Date: 01.10.2020

Product :Tetraxim

• Sanofi's Tetraxim combines four vaccines in one and provides protection against the four diseases diphtheria, whooping cough, tetanus and polio. Launch reduces the number of injections, increases convenience and improves vaccine compliance for children
• Booster vaccines are designed to boost immunity acquired during a previous vaccination. With the launch, Sanofi is currently protecting school children in over 100 countries and distributing 63 million doses worldwide
• Tetraxim (DTaP-IPV) is a 4-in-1 booster vaccine and the only inactivated poliomyelitis-containing combination vaccine in India that has full antigenic potency of diphtheria, tetanus and acellular pertussis (whooping cough) for preschool children

BioMarin receives US FDA approval allowing a maximum dose of 60 mg with Palynziq (pegvaliase-pqpz) for PKU

Date - 8 October 2020

Produkt: Palynziq (pegvaliase-pqpz)

• The US FDA has approved the sBLA to increase the maximum permitted dose of 60 mg with Palynziq for PKU. Previously, the maximum dose was 40 mg
• The label extension is based on a 3-year OLE study. This shows that 66% had a Phe blood level of 360 µmol/L, which was consistent with the ACMG's recommended 2-year Phe target guidelines. treatment and 50% had blood Phe levels of 120 µmol/L @ 2 years. 75%, 66% and 48% had a blood pH of 600, 360 and 120 µmol/L respectively after 3 years. the treatment
• Additional safety data over 6 years. follow-up remains consistent with the previous safety profile of Palynziq, regardless of dose. In addition, BioMarin administered BMN 307 for PKU to the first participant in the global Phearless P-I/II study

AbbVie Announces Results of Skyrizi (risankizumab) in P-III LIMMidless Study in Patients with Moderate to Severe Plaque Psoriasis

Date - 8 October 2020

Product: Skyrizi (Risankizumab)

• The P-III LIMMitless study was designed to evaluate the long-term safety and efficacy of risankizumab (150 mg every 12 weeks) continuously with a starting dose in adults with moderate to severe plaque psoriasis. The analysis includes integrated data from five P-II and III studies (ultIMMa-1, ultIMMa-2, SustaIMM, IMMvent and NCT03255382) and the LIMMitless study
• Results: ~63% of patients with moderate to severe plaque psoriasis treated with SKYRIZI achieved completely clear skin as measured by a 100% improvement from baseline in PASI 100 at 172 weeks. New results from the P-III study LIMMitless were presented at the 29th virtual EADV congress
• Risankizumab is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. It is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization worldwide

Regeneron applies for US FDA EUA for REGN-COV2 antibody combination for treatment of COVID-19

Date - 9 October 2020

product -RAIN-COV2

• Regeneron has submitted an application to the US FDA for an EUA for their REGN-COV2 investigational Ab regimen for the treatment of COVID-19. If the EUA is granted, the US government will committed to making REGN-COV2 available free of charge in the United States and will be responsible for distribution
• The company reported that doses are available to approximately 50,000 patients and expects to have doses available to a total of 300,000 patients within the next few months
• REGN-COV2 is a combination of two mAbs, REGN10933 and REGN10987, designed to block SARS-CoV-2 infectivity. The company reported that President Donald Trump was given a single dose of 8 g of REGN-COV2 at the request of doctors as part of a treatment regimen

Boehringer Ingelheim Initiates P-II Study of BI 764198 in Patients with Severe Respiratory Disease Due to COVID-19

Date - 29 October 2020

Product - BI 764198

• The P-II study will evaluate BI 764198 (qd for ~4 weeks) in patients hospitalized for COVID-19, with an expected start of enrollment in October 2020. The 1EPs are the percentage of patients who were on day 29 of the treatment. alive and not receiving mechanical ventilation, while other EPs include clinical improvement, oxygen saturation, and ICU admission
• The treatment has shown a reduction in cellular damage and pulmonary edema in preclinical studies and may provide similar benefits in patients with severe SARS-CoV-2 infection. BI 764198 was well tolerated in P-I studies in healthy adults
• BI 764198 is a potent and selective inhibitor of TRPC6 that aims to reduce the need for ventilatory support and improve the rate of patient recovery

Roche's Tecentriq in combination with Avastin receives approval from the Chinese NMPA for the treatment of unresectable hepatocellular carcinoma

Date - 30 October 2020

product -Tecentriq + Avastin

• The approval was based on the P-III study IMbrave150 (n=501), in which the combination of Tecentriq (1200 mg, IV) and Avastin (15 mg/kg, IV) or sorafenib (400 mg, bite) in inoperable HCC patients had not received prior systemic therapy, which included analyzes of a cohort of Chinese patients (n=194) from the same study
• Results: Tecentriq in combination with Avastin reduced the risk of OS by 56% (in Chinese patients) and 42% (global results) and the risk of PFS by 40% (in Chinese patients) and 41% (global results) compared to Sorafenib
• IMbrave150 is the first P-III cancer immunotherapy trial to demonstrate improvement in overall survival (OS) and PFS in patients with unresectable or metastatic HCC compared to sorafenib. Other than that. In May 2020, the US FDA approved Tecentriq in combination with Avastin for the treatment of patients with unresectable or metastatic HCC who have not received prior systemic therapy

Sanofi has entered into an agreement with Merck to conduct a P-II study of THOR-707 in sequential dosing with MSD's Keytruda (pembrolizumab) in patients with multiple cancers

Date - 30 October 2020

Product - Keytruda

• Sanofi will sponsor the clinical trials, while MSD will supply KEYTRUDA. In addition, Sanofi is separately investigating the activity of THOR-707 in combination with other anti-PD-1 antibodies, including Libtayo (cemiplimab-rwlc), and with anti-EGFR and anti-CD38 antibodies against different types of cancer tumors
• In preclinical studies, THOR-707 demonstrated the ability to induce CD8+ T cell expansion, resulting in antitumor effects both as a single agent and in combination with an anti-PD1 mAb
• THOR-707 is currently being evaluated by Sanofi in an ongoing P-I dose escalation and extension study that will evaluate THOR-707 and determine the recommended P-II dose alone and in combination with anti-PD-1 and anti-EGFR antibodies will be

Janssen Announces Tremfya (guselkumab) Results in Interim Analysis of P-II GALAXI 1 Study in Moderately to Severely Active Crohn's Disease

Date - 30 October 2020

Product - Tremfya

• The P-II GALAXI 1 study includes evaluation of Tremfya vs. PBO in patients with mod. for severe active CD with intolerance to conventional therapies. In interim analyses, patients were randomized equally into 5 arms with Tremfya (200/600/1200 mg (IV) at 0, 4 and 8 weeks or with ustekinumab dosed at ~6 mg/kg (IV) at 0 weeks). dosed 90 mg (SC) @ 8 weeks or PBO
• @12 weeks. (Tremfya vs PBO), reduction in CDAI (154.1, -144.3, -149.5 vs -36.0); Patients in clinical remission vs. PBO (CDAI < 150): (54.0%, 56.0%, 50.0% vs. 15.7%); clinical remission in failure of conventional treatment and in patients who had previously failed biological treatment (61.6% vs. 18.5% and 45.5% vs. 12.5%, respectively).
• Within 12 weeks, Tremfya resulted in a major improvement in key clinical and endoscopic outcome parameters with a safety profile consistent with approved indications. Tremfya is a mAb that selectively binds to the p19 subunit of (IL)-23 and blocks its interaction with the IL-23 receptor

Given -02.11.2020

Product BNT162b2

• Pfizer & BioNTech report that the UK MHRA has granted EU provisional approval for BNT162b2 against COVID-19. Distribution of the vaccine will be prioritized according to the populations identified in the JCVI guidelines
• The MHRA's decision is based on a rolling filing, including data from the P-III study showing 95% efficacy in participants without or prior SARS-CoV-2 infection, each measured from 7 days after the second dose
• This is the first EUA following a WW P-III study of a vaccine to combat the pandemic. The companies expect further regulatory decisions around the world in December 2020

AstraZeneca to sell European rights to Crestor (rosuvastatin) to Grünenthal for ~$350M.

Given -02.11.2020

Product- Crestor

• Grünenthal acquires the EU rights (excl. Spain and UK) to Crestor and its associated brands for ~$350 million and will handle mass production and packaging until 2025. The deal is expected to close in 1Q21
• Payments will be made in two tranches: $320 million to be paid at closing and $30 million as additional milestones
• Crestor is a statin, a lipid-lowering drug used to treat blood lipid disorders and prevent cardiovascular events such as heart attack and stroke, and is approved in over 100 countries as a lipid-regulating drug.

Genentech and Novartis' Xolair (omalizumab) receives FDA approval for adults with nasal polyps

Given -02.11.2020

Product- Xolair

• The approval is based on studies P-III POLYP 1 & 2, which evaluated Xolair vs. PBO in 138 and 127 adult patients with nasal polyps who had an inadequate response to nasal corticosteroids, respectively
• Results: @24wks. Improvement in NPS (-1.1 vs. 0.1 & -0.9 vs. -0.3); improvement in NCS (-0.9 vs. -0.4 & -0.7 vs. -0.2); no new or unexpected safety signals were identified
• Xolair is the first biologic drug to treat nasal polyps that targets and blocks IgE. In the US, Novartis and Genentech are collaborating to develop and co-promote Xolair

Celltrion presents results of CT-P17 (Biosimilar, Adalimumab) in P-III study in RA at ACR 2020

Date - November 3, 2020

product -CT-P17 (Biosimilar, Adalimumab)

• The P-III study will evaluate CT-P17 (40 mg, q2w) versus reference adalimumab for up to 24 weeks. in 648 patients with active moderate to severe RA despite MTX treatment
• The results showed that CT-P17 had the same effect as the reference adalimumab, i.e. H. ACR20 is 82.7% for both, 2EPs include ACR20/50/70 response rates, mean DAS28, CDAI, SDAI and EULAR (CRP) responses, Ctrough for adalimumab is higher for CT -P17 and lower in ADA positive subgroup than in the ADA-negative subgroup in both treatment groups, the safety profile is comparable
• In addition, comparative pharmacokinetic and safety data for CT-P17 compared with EU-approved and US-approved adalimumab in 312 healthy subjects are presented. Celltrion also presented PK and safety data for two delivery methods for CT-P17, the auto-injector (AI) and the prefilled syringe (PFS).

AstraZeneca and Fusion collaborate to develop and commercialize radiopharmaceuticals and combination therapies for cancer

Date - November 3, 2020

product -FPI-1434

• Fusion receives upfront payments, as well as development milestones and other payments. The companies will jointly discover, develop and have the opportunity to co-commercialize new TATs in the US, while AstraZeneca will lead the commercialization in the ROW with global equal profit and loss sharing
• The collaboration leverages Fusion's TAT ​​platform and radiopharmaceutical expertise with AstraZeneca's leading portfolio of abs and cancer therapies, including DDRis
• In addition, the companies will exclusively investigate certain specific combination strategies between TATs (including Fusion's FPI-1434) and AstraZeneca's therapies for the treatment of multiple cancers. Both companies retain full rights to their respective assets

Janssen acquires rights to Hemera's HMR59 for late age-related macular degeneration

Given -03.11.2020

Product HMR59

• Janssen acquires rights to Hemera's HMR59, which is administered as a single-dose, ambulatory IVT injection. to preserve vision in patients with geographic atrophy
• The acquisition will strengthen Janssen's eye disease portfolio and enhance its gene therapy capabilities
• HMR59 was designed to increase the ability of retinal cells to make a soluble form of CD59, preventing further damage to the retina and preserving vision. The P-I study of the therapy in patients with geographic atrophy has been completed, while the P-I study investigating HMR59 in patients with wet AMD is in follow-up to assess long-term safety

BMSDeucravacitinib (BMS-986165) Demonstrates Superiority to Amgen Otezla (Apremilast) in P-III POETYK PSO-1 Trial in Plaque Psoriasis

Date - November 4, 2020

Product-Deucravacitinib

• The P-III POETYK PSO-1 trial is evaluating deucravacitinib (6 mg, qd) versus PBO and Otezla (apremilast) in 666 patients with moderate to severe plaque psoriasis
• The study met its Co-1EPs and 2EPs and showed that deucravacitinib was superior to Otezla (apremilast) in those patients who achieved a PASI 75 and an sPGA 0/1 at 16 weeks. The overall safety profile of deucravacitinib was consistent with previously reported P-II results
• Deucravacitinib (BMS-986165, PO) is the first and only novel selective TYK2 inhibitor currently being investigated in psoriasis, PsA, lupus and IBD

Janssen Announces US FDA BLA Submission of Amivantamab for Metastatic NSCLC with EGFR Exon 20 Insertion Mutations

Given -04.11.2020

Product – Amivantamab

• The BLA is based on the P-I CHRYSALIS study, which evaluated amivantamab as a monothx. and in combination with lazertinib for adult patients with advanced NSCLC
• The company has established an EAP for patients in the US who may be eligible for access to mivantamab during the review of the BLA
• Amivantamab is a bispecific EGFR and MET Ab with immune cell-directing activity that targets tumors with activation and resistance to EGFR and MET mutations and amplifications. Amivantamab received BTD from the US FDA in March 2020

Merck buys VelosBio for $2.75 billion

Given -05.11.2020

Product- VLS-101

• Merck will acquire all of VelosBio's outstanding shares for $2.75 billion in cash. The deal is expected to close by the end of 2020
• The acquisition will strengthen Merck's oncology pipeline with the addition of VelosBios VLS-101, an investigational ADC targeting ROR1 for the treatment of hematologic malignancies and solid tumors
• In October 2020, VelosBio initiated a P-II study evaluating VLS-101 for the treatment of patients with solid tumors, including patients with TNBC, HR+/HER2+ BC and NSCLC. In addition, VelosBio is developing a preclinical pipeline of ADCs and bispecific Abs targeting ROR1 to complement VLS-101 by providing alternative methods to kill tumor cells

Merck KGaA collaborates with Iktos to use AI in new drug design

Given -05.11.2020

Produkt- N/A

• Iktos will leverage its de novo generative design technology to be implemented in a structure-based context, facilitating the rapid and cost-effective design of Merck KGaA's drug discovery program
• The collaboration follows the companies' previous agreement, signed in 2019. Merck KGaA uses the Iktos de novo design software platform Makya for MPO
• Ikto's AI technology is based on deep generative models that help speed up the drug discovery process and make it more efficient by automatically designing virtual new molecules with desired activities to treat a disease

Novavax partners with Commonwealth of Australia to provide 40 million doses of NVX-CoV2373 for COVID-19

Date - November 5, 2020

Product NVX-CoV2373

• Novavax has signed a non-binding Heads of Terms document with the Australian Government to provide 40 million doses of NVX-CoV2373 to the Australian community
• Delivery will begin as early as H1'21 following completion of the P-III study and approval of the vaccine by the TGA. The vaccination schedule is expected to require two doses per person at 21-day intervals.
• NVX-CoV2373 is being evaluated in a P-II study in the UK and in two ongoing P-II studies that started in August 2020, a P-llb study in SA and a P-II/II continuation in the US and Australia, evaluated. In addition, Novavax has several agreements to supply NVX-CoV2373 directly to the US, UK and Canada and through partnerships to supply to Japan, South Korea and India

Roche Presents Hemlibra Results Reinforcing Long-Term Benefits in Hemophilia A at ASH 2020

Given -07.11.2020

Product - Hemlibra

• The 4 pivotal HAVEN studies (HAVEN-1, 2, 3, 4) included pooled data from 401 subjects with haemophilia A with/without factor VIII inhibitors with a median duration of action of 120.4 weeks.
• Hemlibra maintained low treated bleeding rates, with the ABR remaining low at 1.4 throughout the evaluation period. The proportion of participants experiencing 0 treated bleeding (70.8–83.7%) increased with each consecutive 24-week week. Period
• Roche also presented the first preliminary analysis of the EUHASS database, which indicates that the real-world safety profile of Hemlibra is consistent with the clinical studies and shows no new/new safety signals. Hemlibra is a bispecific anti-factor IXa and factor X Ab

Celltrion Announces Results of CT-P59 in P-I Study for COVID-19

Date - November 9, 2020

product -CT-P59

• The clinical P-I study includes the evaluation of CT-P59 (20/40/80 mg/kg) versus PBO in 18 patients with mild symptoms of SARS-COV-2 infection, randomized in 3 cohorts
• Results: Patients showed a 44 percent reduction in mean clinical recovery time, while no patient required hospitalization or antiviral therapy
• Celltrion has submitted the IND for the clinical study worldwide and plans to conduct global P-II and P-III studies, including Korea. In addition, Celltrion has also initiated a post-exposure prophylaxis study evaluating CT-P59 as a protective treatment by examining the efficacy of the treatment in patients who have been in contact with confirmed SARS-CoV-2 infected patients

AstraZeneca's Calquence (acalabrutinib) receives EU approval for chronic lymphocytic leukemia

Given -10. november 2020

Produkt- Calquence (Obinutuzumab)

• The approval is based on the P-III ELEVATE-TN study evaluating Calquence + obinutuzumab or Calquence alone versus obinutuzumab + CT in patients with previously untreated CLL and the ASCEND study evaluating Calquence versus rituximab + idelalisib/bendamustine in patients with r/ r CLL
• ELEVATE-TN results: a reduction in the risk of disease progression or death (90% and 80%). ASCEND Results: Patients remained alive and showed no disease progression @12 months. (88% vs. 68%)
• Calquence is a selective inhibitor of BTK that covalently binds to BTK and thereby inhibits its activity. The approval follows the CHMP recommendation received in July 2020

Pfizer reports results of abrocitinib in the fifth P-III JADE REGIMENT across different dosing regimens

Given -12. november 2020

Product- Abrocitinib

• The P-III JADE REGIMEN trial is evaluating abrocitinib (100/200 mg) versus PBO in 1233 patients aged =12 years. in the ratio (1:1:1) with mod. to seven. AD after response to initial open-label induction treatment with abrocitinib (200 mg)
• Outcome: The study achieved its 1EPs ie. H. the probability of no flare-up for 52 weeks. (81.1%, 57.4% vs. 19.1%) and 2 EPs, i.e. H. Patients maintain an IGA response of significant or near-significant relative status
• After the first 12 weeks. Induction treatment phase, fewer patients flared at any point in the study @40 weeks. Abrocitinib is a JAK inhibitor that modulates several cytokines involved in AD pathophysiology, including IL-4, IL-13, IL-31, IL-22, and TSLP

UCB acquires Handl Therapeutics to expand its gene therapy portfolio

Date – 13. November 2020

product -Handl-Therapeutics

• The acquisition will strengthen UCB's gene therapy pipeline program, capabilities and platforms. Handl Therapeutics will remain based in Leuven, Belgium and will work closely with UCB's international research team
• In addition, UCB has worked with Lacerta to focus on CNS disorders, where Lacera will lead research, preclinical activities and early manufacturing development, while UCB will conduct IND-enabling studies, manufacturing and clinical development
• The collaboration will provide UCB with access to Lacerta's expertise in AAV-based CNS-targeted gene therapies, enhancing UCB's ability to produce effective treatments for neurodegenerative diseases

Henlius reports NMPA approval of HLX15 (biosimilar, daratumumab) for treatment of multiple myeloma

Given -16. november 2020

Product HLX15

• The NMPA has accepted the IND of HLX15 for use in the treatment of multiple myeloma. HLX15 is the second product developed by Henlius for the treatment of blood tumors
• The company has evaluated the biosimilar in comparative clinical studies showing that HLX15 is very similar to its reference product daratumumab, while the safety profiles are also similar.
• The company developed HLX15 in accordance with the technical guidelines for the development and evaluation of biosimilars and the EMA guidance on similar biological medicinal products

Pfizer launches US pilot program to supply and distribute COVID-19 vaccine BNT162b2

Given -20. november 2020

Product BNT162b2

• collaboration to focus on the development and commercialization of transformative therapies in Greater China. Both companies will use their know-how in clinical development, approval and sales
• Pfizer will contribute approximately $70 million in non-dilutive capital for in-licensing and joint development. At LianBio's discretion, products will be sent to Pfizer for joint development
• Pfizer will have first-negotiation rights to commercial rights to jointly developed assets, and each company will have separate financial considerations. In addition, Pfizer may make in-kind contributions to marketing, development and regulatory activities

AstraZeneca's AZD1222 met its primary endpoint in the prevention of COVID-19

Date – 23. November 2020

product -AZD1222

• Preliminary analysis results from the COV002 and COV003 studies of AZD1222 in the UK and Brazil showed that the vaccine was effective in preventing COVID-19 and no hospitalizations/serious cases of the disease were reported
• One dosing regimen demonstrated 90% efficacy when AZD1222 was administered as a half dose followed by a full dose for at least 1 month. separate and a different dosing regimen showed 62% efficacy when administered as 2 full doses for at least 1 month. part. Combined analysis of both dosing regimens showed ∼70% efficacy
• AZ will plan regulatory submissions of data around the world and seek an emergency list from the WHO for an accelerated path to vaccine availability in low-income countries.

Roche launches Coba's PIK3CA mutation test for patients with advanced or metastatic breast cancer

Given -15. december 2020

Product-Cobas PIK3CA-Mutationstest

• Roche launches cobas PIK3CA mutation test for patients with advanced/m-BC in countries accepting the CE mark
• The IVT test is a real-time PCR test intended for the qualitative detection and identification of 17 mutations in exons 2, 5, 8, 10 and 21 of the gene encoding the catalytic subunit of PIK3CA in DNA isolated from FFPET, identification of patients with m-BC harbor mutations
• This test provides automated results with flexible throughput processing of 30 samples/run on the widely used cobas z 480 analyzers. The test can detect 17 mutations in the PIK3CA gene and help clinicians identify patients who may benefit from targeted PI3K therapy

Abbott's COVID-19 IgG Antibody Quantitative Blood Test Receives CE Mark

Given -16. december 2020

Product-SARS-CoV-2 IgG II Quanttest

• Abbott received CE mark approval for its new quantitative SARS-CoV-2 IgG laboratory serology test. The test measures levels of IgG-Abs to help evaluate a person's immune response
• The study to determine the clinical performance of Abbott's SARS-CoV-2 IgG II Quant test on its Alinity i instrument found it had a specificity of 99 in patients tested 15 days or more after symptom onset, 60% and a sensitivity of 99.35%
• The test will be available on the Abbott ARCHITECT and Alinity i platforms. Abbott plans to submit its test for the US FDA EUA shortly

Health Canada speeds up review of AstraZeneca's COVID-19 vaccine

Given -15. december 2020

Product-Covid-19 vaccination

• Health Canada accelerates review of AstraZeneca's COVID-19 vaccine after vaccine receives UK MHRA emergency use approval
• Following an agreement to supply 20 million doses to the Canadian government, AstraZeneca is seeking approval from Health Canada in October 2020, resulting in ongoing data sharing to expedite the review process
• Health Canada is committed to providing Canadians with access to COVID-19 vaccines as quickly as possible without compromising safety, efficacy and quality standards

Related post:The key highlights from PharmaShots in the third quarter of 2020