PharmaShots' Key Highlights of Second Quarter 2022
- The second quarter of 2022 highlights major acquisitions in the pharma and biotech industry along with multiple approvals. Meanwhile, AstraZeneca’s Farxiga (dapagliflozin) meet its primary endpoints for heart failure with a preserved ejection fraction
- Starting with the latest acquisitions, Pfizer acquire Biohaven for ~$11.6B, GSK acquire Affinivax for ~$3.3B. The second quarter of the year also showcases multiple approvals of the therapies which include Daiichi Sankyo and AstraZeneca’ Enhertu US FDA’s BTD for HER2 low metastatic breast cancer, Amarin’s Vazkepa (icosapent ethyl) NICE recommendation for Cardiovascular Disease
- Our team at PharmaShots has summarized and complied with the insights of Q2'22
Date- Apr 27, 2022
Product- Cyclasol
- The P-III (ESSENCE-2) trial evaluating CyclASol vs vehicle in 834 patients with DED not responding to artificial tears
- The results showed that CyclASol has consistent & superior therapeutic effects on the ocular surface in patients with DED, 71.6% had a ≥ 3-grade improvement in tCFS @4wks. & the proportion of responders was higher compared to vehicles with superior relief of blurred vision in patients with high central staining scores
- The therapy showed that 78.6% rated their satisfaction with CyclASol & has excellent safety & tolerability profile, improvement in tear production & variety of symptoms over non-responders @Day 29, change from baseline in tCFS was significant in CyclASol @Day 15 & 29
Date- Apr 27, 2022
Product- Enhertu
- The designation was based on the P-III (DESTINY-Breast04) trial evaluating Enhertu (5.4mg/kg) vs CT in a ratio (2:1) in 540 patients with HER2 low unresectable or metastatic breast cancer prior treated with 1 or 2 prior lines of CT at multiple sites in Asia, EU & North America
- The results showed an improvement in PFS & OS in patients with HR+ or HR- disease. The safety profile was consistent with previous trials with no new safety concerns, ILD rates were consistent with late-line HER2+ breast cancer trials with a lower rate of grade 5 ILD
- Enhertu (6.4mg/kg) was approved in multiple countries for LA or metastatic HER2+ gastric or GEJ adenocarcinoma, based on (DESTINY-Gastric01) trial results
Date- Apr 21, 2022
Product- Tibsovo (ivosidenib)
- The P-III (AGILE) trial evaluates Tibsovo + azacitidine vs PBO + azacitidine in adults with prior untreated IDH1-mutated AML who are not candidates for intensive CT or who have comorbidities that preclude the use of intensive induction CT
- The study met its 1EPs & 2EPs i.e., improvement in EFS & OS with a m-OS of 24.0mos. vs 7.9mos., CR rate (47.2% vs 14.9%), CR + CRh rate (52.8% vs 17.6%), ORR (62.5% vs 18.9%)
- Tibsovo is the 1st approved therapy for patients with prior treated IDH1-mutated cholangiocarcinoma & has been approved in the US as monothx. for IDH1-mutant r/r AML & IDH1-mutant AML. The company works with the US FDA & health authorities globally to bring the therapies to market
Date- Apr 15, 2022
Product- Bempegaldesleukin + Opdivo
- BMS and Nektar Therapeutics are terminating their cancer drug research alliance after the failure of Bempeg + Opdivo in RCC and bladder cancers. The companies have decided to discontinue both the studies and all other ongoing studies in the program
- The P-III PIVOT-09 study showed that the combination therapy vs TKI did not meet its prespecified boundary for statistical significance in RCC patients. Additionally, P-II PIVOT-10 study also failed to reach an efficacy threshold in UC
- These disappointing results come one month after the drug combo failed a pivotal melanoma clinical trial
GSK to Acquire Sierra Oncology for ~$1.9B
Date- Apr 13, 2022
Product- momelotinib
- GSK to acquire Sierra for $55/share of common stock in cash making a total deal value of ~$1.9B. It represents a 39% premium to Sierra Oncology’s closing stock price on 1 day prior & the transaction is expected to be close in Q3’22 or before
- Following the completion of the transaction & the approval of momelotinib for myelofibrosis patients with anaemia, the therapy will contribute to GSK's growing business with expected sales in 2023. The stockholders of Sierra hold ~28% of outstanding shares
- The acquisition complements GSK’s expertise in haematology with the addition of Momelotinib & supports the development of a strong portfolio for new therapies & vaccines. Sierra plans for regulatory submission in the US in Q2’22 & EU submission in H2’22
Date- Apr 13, 2022
Product- Jardiance (empagliflozin)
- The approval was based on the P-III (EMPEROR-Preserved) trial evaluating the safety & efficacy of Jardiance (10mg, qd) vs PBO in 5988 adults with chronic HFpEF
- The results showed a 21% & 3.3% relative & absolute risk reduction for the composite 1EPs of CV death or hospitalization for HF & the benefit was generally consistent across LVEF subgroups based on the pooled EMPEROR studies. In key 2EPs analysis, 27% reduction in relative risk of first & recurrent hospitalizations for HF, breakthrough benefits for a widely underserved group
- The approval marks the 1st approved treatment to improve outcomes of HF patients regardless of ejection fraction including HFrEF & HFpEF
Date- Apr 08, 2022
Product- Xarelto (rivaroxaban) + Aspirin
- The P-III (VOYAGER PAD) trial evaluates Xarelto (2.5mg, bid) + aspirin (100mg, qd) vs aspirin alone in a ratio (1:1) in 6564 patients with PAD with/out CKD & with/out the history of statin therapy
- The results showed a reduction in thrombotic hospitalizations due to thrombotic events (8.7% vs 12.1%) for PAD patients with/out CKD, 4.7% ARR in the subgroup with CKD (7.9% vs 12.6%)
- The analysis showed that patients who received Xarelto + aspirin in addition to statin therapy had a 19% reduction in 1EPs composite of ALI, 26% of MALE & 32% in acute limb injury & reduced the composite of MACE or MALE after revascularization for PAD. Both analyses showed a numerical increase in TIMI major bleeding
Pfizer to Acquire ReViral for ~$525M
Date- Apr 07, 2022
Product- Sisunatovir
- Pfizer to acquire ReViral for a total consideration of ~$525M including upfront and development milestones. The program is expected to generate ~$1.5B in revenue, if successful
- The acquisition strengthens Pfizer’s capabilities in R&D for infectious disease with a complementary strategy & also expands its anti-infective pipeline to improve patient outcomes for RSV infections and prevent illness through vaccination
- Pfizer will use its R&D, manufacturing, and commercialization expertise for the clinical development of a therapy to treat RSV disease. Sisunatovir is currently in P-II clinical development in infants & has received FTD from the US FDA
Date- Apr 05, 2022
Product- Vyvgart (efgartigimod alfa-fcab)
- The ongoing P-III (ADAPT+) 3yr. extension study evaluates Vyvgart in adults patients with gMG. Additionally, 106 AChR-Ab+ & 33 AChR-Ab– received 1 dose of Vyvgart
- The results showed that the long-term treatment with Vyvgart provides a consistent & clinical improvement on MG-ADL & QMG scales that remains consistent over multiple treatment cycles, 54.6% received ≤5.5 treatment cycles/yr. in patients who completed 1yr. of treatment at the time of interim analysis, well-tolerated with a consistent safety profile to the (ADAPT) trial
- Vyvgart is a human IgG1 Ab fragment that binds to FcRN resulting in the reduction of circulating IgG auto Abs. The therapy has been approved in the US & Japan for gMG
Date- Apr 01, 2022
Product- VX-548
- The 2 P-II POC studies evaluate VX-548 (selective NaV1.8 inhibitor) vs PBO in 274 & 303 patients with acute pain, following abdominoplasty surgery or bunionectomy surge. The study also included HB/APAP (5mg/325mg, PO, q6h over 42hrs.) to support the evaluation of the VX-548 treatment effect
- The trials met its 1EPs in both acute pain studies i.e., improvement in SPID48 at the high dose @48hrs. was well tolerated in both studies, fewer patients discontinued treatment in the mid & high-dose VX-548 or HB/APAP reference arm, no patients discontinued treatment due to AEs & no patients had SAEs
- The company plans to advance VX-548 into pivotal development in H2’22, following discussions with regulators
GSK to Acquire Affinivax for ~$3.3B
Date- May 31, 2022
Product- AFX3772
- GSK to acquire 100% of the outstanding shares of Affinivax with a total consideration of ~$3.3B incl. $2.1B up front & $0.6B upon achievement of milestones. The transaction is expected to close in Q3’22
- The acquisition bolsters GSK’s vaccines R&D pipeline, provides access to Affinivax’s MAPS technology for pneumococcal diseases & expands its footprint in the Boston area. The acquisition will establish a strong portfolio of innovative vaccines & therapy, providing access to a 24-valent pneumococcal vaccine candidate currently in P-II trial
- In the P-I/II trials, AFX3772 was well tolerated & showed good immune responses. The vaccine's use in paediatrics will initiate in 2022 & the P-III is expected to initiate shortly
Date- May 17, 2022
Product- Opdivo (nivolumab) + Yervoy (ipilimumab)
- The P-III (CheckMate -901) trial evaluates Opdivo (1mg/kg) + Yervoy (3mg/kg, q3w for 4 cycles) or Opdivo (480mg, q4w) + CT vs CT alone in 707 patients with untreated unresectable or metastatic UC who are ineligible for cisplatin-based CT
- The trial failed to meet the 2EPs of OS in patients whose tumor cells express PD-L1 ≥1% at the final analysis, no new safety signals were identified. Following the IDMC’s recommendation, the trial continues to evaluate other 1EPs & 2EPs
- The primary & sub-studies for the (CheckMate -901) trial are still ongoing & the company will provide additional results shortly. Opdivo + Yervoy showed improvements in OS in 6 P-III trials for NSCLC, MM, RCC, MPM & ESCC
Date- May 16, 2022
Product- Sarclisa (isatuximab)
- The P-III (IKEMA) trial evaluating Sarclisa + carfilzomib & dexamethasone vs Kd in 302 patients with relapsed MM across 69 centers spanning 16 countries
- The results showed an m-PFS of 35.7mos. vs 19.2mos., m-PFS (41.7mos. vs 20.8mos.), time to next treatment (44.9mos.) @25mos., safety & tolerability were consistent with the safety profile of Sarclisa in other clinical trials with no new safety signals, treatment exposure was 30wks. longer, TEAEs of ≥ grade 3 (83.6% vs 73%), serious TEAEs (70.1% vs 59.8%) with no difference after exposure adjustment
- The results will also be presented at ESMO 2022. Sarclisa was approved in multiple countries in combination with carfilzomib & dexamethasone including in the US & EU for MM
Date- May 13, 2022
Product- Valbenazine
- The US FDA has granted ODD to valbenazine for the treatment of HD. The company reported the results from the P-III (KINECT-HD) study that evaluates valbenazine (qd) vs PBO in 128 adults aged 18 to 75yrs. with chorea associated with HD
- The study met the 1EPs i.e., reduction in chorea movements as measured by a change in UHDRS TMC score from baseline to avg. score @10 & 12wks. & a mean reduction in TMC score while 2EPs of CGI-C response status & PGI-C response were also in favor of valbenazine treatment
- Additionally, patient enrollment is ongoing in the (KINECT-HD2) study to evaluate the safety & tolerability of valbenazine for chorea in HD. The company is planning to submit sNDA to the US FDA in 2022
Date- May 12, 2022
Product- Tiragolumab + Tecentriq (atezolizumab)
- The P-III (SKYSCRAPER-01) study evaluating tiragolumab + Tecentriq vs Tecentriq alone in a ratio (1:1) in 534 patients with PD-L1-high LA or metastatic NSCLC
- The study failed to meet its co-primary EPs of PFS & the other co-primary EPs of OS were immature, numerical improvement was identified in both co-primary EPs. The results demonstrated that the therapy was well-tolerated and no new safety signals were reported
- The program continues to advance the therapy for multiple clinical trials & expand into earlier stages of the disease. The company focuses to develop novel therapeutic options for malignancies that are advanced and difficult to treat in patients with a high unmet medical needs
Date- May 11, 2022
Product- Nirsevimab
- The results from a prespecified pooled analysis of the P-III (MELODY) & P-IIb trials evaluates nirsevimab (50mg, IM) vs PBO in a ratio (2:1) in 1490 & 1453 infants against medically attended LRTI due to RSV through 150 days post-dose
- The results from both trials showed an efficacy of 79.5% in infants who received the optimized dosing regimen of 50mg. The safety profile was similar to prior trials & the regulatory submissions are expected to be initiated in 2022
- Under the terms of the 2017 agreement, Sanofi & AstraZeneca collaborated to develop & commercialize nirsevimab. AstraZeneca will lead development & manufacturing activities while Sanofi will lead commercialization activities & record revenues
Pfizer to Acquire Biohaven for ~$11.6B
Date- May 10, 2022
Product- rimegepant & zavegepant
- Pfizer to acquire all outstanding shares of Biohaven for $148.50/share in cash making a total deal value of ~$11.6B, representing a premium of 78.6% to Biohaven's last closing price of $83.14. The transaction is expected to be close in early 2023
- Biohaven shareholders including Pfizer to receive 0.5 of a share of a new publicly-traded company (New Biohaven) that retains Biohaven’s non-CGRP pipeline compounds. The acquisition expands Pfizer’s pipeline for further growth through 2030 & beyond
- Biohaven’s CGRP programs include rimegepant & zavegepant for migraine. Additionally, New Biohaven to receive royalties from Pfizer for rimegepant & zavegepant in the US, if sales exceed ~$5.25B
Date- May 06, 2022
Product- MR-004
- MedRhythms to receive $3M up front & is eligible to receive ~$117.5M upon achievement of development & commercial milestones along with royalties
- The collaboration will combine MedRhythms’ digital expertise with Biogen’s leadership & global footprint to advance novel therapies for MS & address the patient's unmet needs. MR-004 is the prescription digital therapeutic that uses a combination of sensors, software & music based on rhythmic auditory stimulation
- If approved, MR-004 will become the 1st prescription digital therapeutic for gait deficit in MS. MedRhythms conducts 2 ongoing feasibility studies for MR-004 & also plans to initiate a registrational trial, based on 1st study results
Date- May 05, 2022
Product- Farxiga (dapagliflozin)
- The P-III (DELIVER) trial evaluates the efficacy of Farxiga (qd) vs PBO in patients with HFpEF with LVEF ≥40% with/out T2D
- The results showed a reduction in the primary composite EPs of CV death or worsening HF, safety & tolerability were consistent with the well-established safety profile of the therapy. The results will be submitted for presentation at a medical meeting and regulatory submissions are expected in the coming mos.
- Farxiga was effective in treating HF regardless of ejection fraction. The 2EPs include total number of HF events & CV death, change from baseline in the total symptom score of KCCQ @8mos., time to the occurrence of CV death & death from any cause
Date- May 02, 2022
Product- Zuranolone
- The company has initiated a rolling submission of NDA to the US FDA for zuranolone to treat MDD. The NDA submission for MDD is expected to complete the remaining component in H2’22 & for PPD in H1’23
- The NDA was based on the (LANDSCAPE) & (NEST) programs, including the (SHORELINE) study & data from the completed clinical pharmacology studies. The results from the (LANDSCAPE) & (NEST) programs showed rapid & sustained effects along with a well-tolerated safety profile in clinical trials
- The companies have submitted the nonclinical module of the NDA to the US FDA. Zuranolone is an investigational oral NAS GABA-A receptor PAM & has received the FTD from the US FDA in 2017 in MDD & BTD in 2018
Date- June 24, 2022
Product- Fezolinetant
- The company has submitted an NDA to the US FDA for fezolinetant in patients with mod. to sev. VMS associated with menopause
- The NDA submission was based on the P-III (SKYLIGHT 1 & 2) in 1000 women for the first 12wks. followed by a 40wk. treatment extension period along with the P-III (SKYLIGHT 4) study in 1800 women for 52wk. evaluating fezolinetant to treat mod. to sev. VMS at ~180 sites within the US, Canada & EU
- Fezolinetant is an oral, nonhormonal compound indicated for VMS associated with menopause & blocks NKB binding on the KNDy neuron to mod. neuronal activity in the thermoregulatory center of the brain to reduce the frequency & severity. The financial effects of this submission for Mar 2023 are being assessed by Astellas
Date- June 22, 2022
Product- Vaxneuvance (pneumococcal 15-valent conjugate vaccine)
- The approval is based on seven P-III clinical studies assessing the safety, tolerability & immunogenicity of Vaxneuvance vs PCV13 in infants, children, and adolescents aged 6wks. through 17yrs. with IPD caused by 15 serotypes
- Results: Vaxneuvance showed a non-inferior immune response to PCV13 for 13 shared serotypes based on serotype-specific IgG GMCs, following four-dose pediatric series & superior immune responses for shared serotypes 3 and unique serotypes 22F and 33F
- The approval follows the FDA’s Priority Review of Merck’s application. Vaxneuvance is the first PCV to be approved in almost a decade to protect pediatric populations against IPD & has also received BTD from the US FDA for the pediatric indication
Date- June 22, 2022
Product- Nemolizumab
- The P-III (OLYMPIA 2) trial evaluating the efficacy, safety, PK & immunogenicity of nemolizumab as monothx. vs PBO in 540 patients aged 18yrs. with PN after 16wk. treatment period
- The trial met all 1EPs & 2EPs i.e., 38% vs 11% of patients showed clearance or almost-clearance of skin lesions as assessed by using the IGA score, 56% vs 21% achieved a 4-point reduction in itch as measured by PP-NRS score. The safety profile was consistent with the P-II trial results
- Nemolizumab also showed a positive benefit-risk balance & has been approved in Japan for pruritus associated with AD and is under clinical development in many countries globally. The therapy has received BTD from the US FDA for pruritus associated PN
Date- June 21, 2022
Product- EDG-5506
- The ongoing P-Ib (ARCH) study evaluates the safety, tolerability, impact on muscle damage biomarkers & PK of EDG-5506 (10mg, qd) in 12 adult males with BMD
- The results showed a lowered muscle damage biomarker, reduction of CK & fast skeletal muscle troponin I (TNNI2) by 30% & 68% after 2mos., reduced exposure for BMD patients which was consistent with the lower dose of 10mg, shorter half-life associated with a decreased overall muscle mass. The therapy was transient & typically observed in the first few days of dosing
- Additionally, patients were more active during 2mos. of dosing with EDG-5506 relative to activity measured in the P-I study, well-tolerated with no discontinuations or dose reductions
Date- June 17, 2022
Product- Imcivree (setmelanotide)
- The approval was based on the P-III trial to evaluate Imcivree vs PBO in adult & pediatric patients aged ≥6yrs. with monogenic or syndromic obesity due to POMC, PCSK1, LEPR deficiency, or BBS
- The trial met its 1EPs & 2EPs i.e., reductions in weight & hunger @52wks., mean percent change in BMI was -7.9% without requirements for diet & exercise; PBO-adjusted change in BMI was -4.5% @14wk. (-4.6% vs -0.1%); mean change in hunger score was -2.1 @52wks. in patients aged ≥12yrs., were generally well-tolerated
- The US FDA has issued a CRL for setmelanotide’ sNDA in ALMS & the company plans to advance the therapy for ALMS in the US along with a clinical research program for setmelanotide in patients with obesity
Date- June 14, 2022
Product- DES-7114
- DESRES to receive an upfront of $60M & is eligible to receive development and commercial milestones of ~$475M along with royalties
- DES-7114 is a highly selective small-molecule inhibitor of the ion channel protein Kv1.3 & has recently completed P-I clinical trials in healthy volunteers. The therapy showed efficacy in preclinical models of multiple chronic inflammatory and autoimmune disease, including UC, CD, & AD
- The utilization of special-purpose supercomputers that have been developed by DESRES to undertake ultra-high-speed, atomically detailed simulations of the three-dimensional motion of physiologically and pharmaceutically relevant molecules enabled the construction of DES-7114
Date- June 10, 2022
Product- Vazkepa (icosapent ethyl)
- The NICE has issued FAD recommending the use of Vazkepa as a treatment option for adult statin-treated patients & for reimbursement and routine use in the NHS in England & Wales to reduce the high risk who have elevated triglycerides & eCVD. The final guidance is expected to be published in July 2022 & the product should be available on all local NHS formularies within 3mos.
- The P-III (REDUCE-IT) trial showed a 25% relative reduction & 4.8% absolute risk reduction in 1st occurrence of MACE, well-managed LDL-C levels are still at risk of a CV event & other factors
- Vazkepa is expected to be available in ~6 EU countries in 2022. The product has also won reimbursement approval in Sweden & is under review in other EU markets
Date- June 8, 2022
Product- Lagevrio (molnupiravir)
- The P-III (MOVe-OUT) trial evaluates molnupiravir (800mg, bid) vs PBO in non-hospitalized, unvaccinated adult patients with COVID-19 who had 1 risk factor associated with poor disease outcomes
- In a pre-specified exploratory EPs i.e., a lower proportion of patients in the MITT population had an acute care visit & COVID-19-related acute care visit (7.2% vs 10.6%) @29 days & (6.6% vs 10.0%) with RRR (32.1% & 33.8%), fewer patients required respiratory interventions with a RRR of 34.3%. The results were published in the Annals of Internal Medicine
- In post hoc analyses, participants in the safety population showed reductions in mean CRP values, improvements in mean change from baseline SpO2 values & patients required respiratory interventions with a RRR of 21.3%
Date- June 6, 2022
Product- Keytruda (pembrolizumab)
- The P-III (KEYNOTE-716) trial evaluated Keytruda vs PBO in 976 adult and pediatric patients aged ≥12yrs. with resected stage IIB or IIC melanoma
- The results showed a significant clinical improvement in DMFS at a median follow-up of 27.4mos., m- DMFS was not reached. Additionally at the median 27.4mos. follow-up, the therapy showed a reduction in the risk of recurrence, 81.2% vs 72.8% were recurrence-free @2yrs
- The safety profile was consistent with prior reported studies & no new safety signals were observed at the time of DMFS analysis. TRAEs grade ≥3 (17% vs 5%), immune-mediated events and infusion reactions (38% vs 9%). The prespecified exploratory analysis showed that HRQoL was similar b/w Keytruda & PBO
BMS to Acquire Turning Point for ~$4.1B
Date- June 3, 2022
Product- N/A
- BMS commences a tender offer to acquire all outstanding shares of Turning Point’s common stock at $76.00/share in an all-cash transaction making a total deal value of $4.1B. It represents a 122.5% premium to its last closing price & the transaction is expected to close in Q3’22
- The transaction is expected to be ~$0.08/share dilutive to non-GAAP EPS in 2022. The acquisition enables BMS to broaden its oncology franchise by adding precision oncology asset
- Turning Point’s repotrectinib has received 3 BTD from the US FDA & is expected to be approved in the US in H2’23. In the P-I/II (TRIDENT-1) trial, longer DoR has been observed in the landmark analysis with repotrectinib over existing ROS1 agents in 1L NSCLC
Related Post: PharmaShots' Key Highlights of First Quarter 2022
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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
