PharmaShots' Most Read News of 2022
The year 2022 has been a busy year for global pharma and biotech companies involved in M&A, option & licensing agreements, clinical trial results and gaining approvals. Our team has compiled a list of 30 most read life sciences news on PharmaShots in 2022
- The companies collaborated to evaluate ficlatuzumab + Erbitux in registrations study in patients with recurrent or metastatic HNSCC. The study is expected to initiate in H1’23
- Merck KGaA will provide cetuximab clinical drug supply in all countries outside of the US & Canada for AVEO’s registrational study to evaluate ficlatuzumab + cetuximab for HPV negative R/M HNSCC. AVEO will be the study sponsor & will be responsible for costs associated with trial execution
- The company is planning to initiate the manufacturing of ficlatuzumab clinical supply in Q2'22. Ficlatuzumab is being evaluated in HNSCC & metastatic PDAC & has received FTD from the US FDA for recurrent or metastatic HNSCC
- Exscientia to receive $100M up front & is eligible to receive ~$5.2B upon achievement of research, translational, clinical development, regulatory & commercial milestones & also get an option for clinical co-investment to increase the royalty ~21% on net sales of co-funded products
- The agreement will use Exscientia’s AI-based capabilities & personalized medicine platform to identify & select target projects along with the development of 15 new small molecule therapies for cancer & immune-mediated diseases
- Exscientia will lead the small molecule drug design & optimization activities. Sanofi will lead the preclinical/ clinical development, manufacturing & commercialization of the product
- The P-III (U-EXCEL) induction study to evaluate upadacitinib (45mg) in adults with mod. to sev. CD who had inadequately responded or intolerant to one or more conventional & biological therapies
- The trial met its 1EPs & 2EPs i.e., patients achieved clinical remission @12wks. (49% vs 29%), clinical remission (51% vs 22%), endoscopic response (46% vs 13%), steroid-free clinical remission, symptom improvement & the safety profile was consistent with the safety profile observed in previous studies with no new safety risks, SAEs (6.9% vs 6.8%)
- RINVOQ (15/30mg) is a selective & reversible JAK inhibitor & is approved in the US & EU for AD while 15 mg is also approved in the US & EU for RA
4. Biohaven to Acquire Channel Biosciences for ~$3B
- Biohaven acquired Knopp Biosciences' Channel Biosciences for its Kv7 channel targeting platform to advance BHV-7000 (formerly known as KB-3061)
- Knopp Biosciences to receive $100M up front including $35M in cash and $65M in Biohaven's common shares and is eligible to receive ~$3B including up to $325M in development and regulatory milestones for approvals in the US, EU and Japan & up to $250M for the Kv7 pipeline in other indications and additional country approvals, and up to $562.5M in commercial milestones along with royalties
- Channel Biosciences’s Kv7 channel targeting platform will support the advancement in ion-channel modulation to BHV-7000 (a potent activator of Kv7.2/Kv7.3). Biohaven additionally plans to bring BHV-7000 into the clinic in 2022 to treat focal epilepsy
- Positive detailed results from a second P-III trial of Dupixent (dupilumab-300 mg, Q1W), showed the significant improvement in signs and symptoms of EoE at 24 wks. vs. to PBO in patients aged ≥12 yrs. In 2022, regulatory filings are planned in the US and other countries
- Results showed the reduction of 64% vs. 41% in disease symptoms from baseline, DSQ improvement is 23.78 vs. 13.86 point & 59% vs. 6% of patients achieved histological disease remission
- In Sep 2020, Dupixent received BTD from the U.S. FDA for the treatment of patients aged ≥12yrs. with EoE and ODD in 2017 for the treatment of EoE
- The P-III (C.L.E.A.R.) program evaluates the safety & analgesic effects of a single & repeat transforaminal inj. of SP-102 vs PBO in 401 patients with lumbosacral radicular pain or sciatica
- The results showed that patients experienced a rapid onset of pain relief as measured by NPRS of avg. daily pain in affected leg @ first 4wks., following a single transforaminal inj. The 2EPs of ODI showed a 28% improvement @4wks. on SP-102 over baseline
- The company plans to discuss with the US FDA in 2022 for licensure application & BTD. If SP-102 will be approved by the US FDA, the product will be available in a pre-filled syringe formulation & will be given as an epidural inj. for sciatica
7. Tonix Reports Results of TNX-102 SL in P-III (RALLY) Study for the Treatment of Fibromyalgia
- The P-III (RALLY) study evaluates TNX-102 SL (5.6mg) vs PBO in a ratio (1:1) in 514 patients with FM across 36 US sites. The trail failed to achieve statistical significance on the 1EPs of reducing FM daily pain @14wk., based on interim analysis as reported in July 2021
- The results also showed nominal significance on the PROMIS sleep disturbance measure & proportion of responders (29.7% vs 21.7%) while other key 2EPs did not achieve nominal significance, was well tolerated with no new safety signals
- Additionally, 73.8% & 81.4% has completed 14wk. dosing period & study had an unexpected increase of AEs-related discontinuations. The company plans to initiate P-III (RESILIENT) study in H1’22
- The P-III (PSOARING 3) LTE study evaluates tapinarof (1%) vs vehicle-controlled cream in adults patients with PsO for ~40wks. & a 4wks. safety follow-up period. The patients had completed the treatment in the P-III (PSOARING 1 & 2) studies
- The results showed continued improvements for ~52wks. in efficacy outcomes, QoL measures & tolerability scores with no associated tachyphylaxis, were well-tolerated in sensitive skin & intertriginous areas with a majority of patients experiencing no irritation for ~52wks.
- The therapy showed a continued & durable improvement in QoL. The US FDA has accepted an application for tapinarof to treat PsO in Aug 2021 & assigned a PDUFA date in Q2’22
- The P-I/II (CodeBreaK 100) trial evaluates Lumakras in patients with KRAS G12C-mutated advanced NSCLC
- In the 2yr. analysis from 174 heavily pre-treated patients, Lumakras showed a long-term clinical benefit & prolonged tumor response with ORR of 40.7%, DCR (83.7%) & m-DoR (12.3mos.), 5 patients achieved CR & 65 patients achieved PR, m-PFS of 6.3mos. & OS (12.5mos.) with 32.5% of patients still alive @2yr. with no new safety signals during long-term follow-up
- Lumakras is marketed as Lumykras (sotorasib) in the EU, UK & Switzerland. In May 2021, Lumakras was the 1st KRASG12C inhibitor to receive regulatory approval in the US under accelerated approval & has been approved in 39 countries
- The P-I/II (SYMPHONY) trial evaluates BLU-945 in patients with EGFR-mutant NSCLC. The results showed early evidence of safety & clinical activity consistent with preclinical data
- The P-I (SYMPHONY) trial showed dose-dependent reductions in ctDNA & radiographic tumor including PR & PK results showed BLU-945 exposures at higher doses associated with broad EGFR mutation coverage, were well-tolerated with no significant AEs
- The company initiates (SYMPHONY) trial of BLU-945 + osimertinib in patients with 2L or later EGFR-mutant NSCLC. Under the clinical trial supply agreement with AstraZeneca, Blueprint will evaluate BLU-945 & BLU-701 in combination with osimertinib in (SYMPHONY) & (HARMONY) trials
- The P-III (RATIONALE 306) trial evaluates tislelizumab + CT vs PBO + CT in a ratio (1:1) in 649 patients with advanced or metastatic ESCC across Asia-Pacific, EU & North America
- The study had met its 1EPs of OS in patients with prior untreated advanced or metastatic ESCC. The safety & tolerability profile was consistent with previous trials with no new safety signals
- Tislelizumab is currently under the US FDA & EMA’s review for advanced or metastatic ESCC. The EMA also reviews tislelizumab for advanced or metastatic NSCLC after prior CT. In Jan 2021, BeiGene collaborated with Novartis to accelerate the clinical development and marketing of tislelizumab in North America, the EU, and Japan
- Byondis to receive an up front & will also be eligible to receive an additional fee upon achievement of development and sales milestones along with sales royalties
- Under the terms of license, collaboration & supply agreement, medac gets an exclusive license to commercialize SYD985 in the EU, UK, and EU countries in all approved indications
- The results from the P-III (TULIP/SYD985.002) study of SYD985 in HER2-positive unresectable LA or metastatic breast cancer showed a significant PFS & the company plans to submit SYD985’ MAA to the EMA shortly. The therapy is also being evaluated in a P-II trial for HER2-expressing recurrent, advanced or metastatic endometrial cancer
- 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
- 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
- The P-II (APEX) trial evaluated bezuclastinib in patients with AdvS. The updated data will be expected at the end of 2022
- As of the data cutoff date of May 2022, 11 patients were evaluated for signs of clinical activity, all patients achieved a ≥50% reduction in serum tryptase levels with a median reduction of 89% & 6 achieved a reduction to <20 ng/mL. All 8 bone marrow biopsy-assessed patients achieved ≥50% bone marrow mast cells reduction & 6 with complete clearance
- 8 patients showed a reduction in KIT D816V VAF, 3 discontinued avapritinib for toxicity reasons, clinical outcomes consistent with avapritinib-naïve patients, were well-tolerated with no reported periorbital or peripheral edema, cognitive effects, or intracranial bleeding events
- DESRES to receive an up front 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
- The P-II/III (EPIC-SR) trial evaluated Paxlovid (BID for 5 days) vs PBO in a ratio (1:1) in 1153 patients who are at standard risk for developing sev. COVID-19
- The updated analysis showed a non-significant 51% relative risk reduction; 57% in hospitalization or death in a sub-group analysis of vaccinated patients. In an additional analysis of 2EPs, 62% reduction in COVID-19-related medical visits/day
- In pre-specified analysis, 72% reduction on avg. no. of days in hospital & the results were consistent with (EPIC-HR1) study, discontinuation due to AEs (1.7% vs 1%). The company cease enrollment in the (EPIC-SR) trial due to the low rate of hospitalization or death & will continue to access treatment in patients with high unmet need
18. Medtronic to Acquire CathWorks for ~$585M and Entered into a Co-Promotion for FFRangio System
- Medtronic will invest ~$75M in CathWorks & begin marketing CathWork’s FFRangio catheter system in the US, EU & Japan. The collaboration highlights Medtronic's dedication to providing new, innovative technologies and solutions from diagnosis to treatment
- Under the separate agreement, Medtronic gets an option to acquire CathWorks if undisclosed milestones are met. CathWorks get the rights to compel Medtronic to acquire the company, if Medtronic does not exercise the option which expires in July 2027 with an estimated value of $585M & earn-out payments post-acquisition
- FFRangio system provides diagnostic & physiologic inf. through AI to disrupt traditional FFR & revolutionize the management of CAD. The system showed diagnostic accuracy (93%), sensitivity (91%) & specificity (94%)
19. Labcorp Launched Neurofilament Light Chain Blood Test to Identify Neurodegenerative Disease
- The company launched the new NfL blood test that allows doctors to identify and verify signs of neurodegenerative disease along with diagnosing and treat patients more quickly & effectively
- The test measures key biomarkers associated with neuronal damage; assists in the diagnosis of diseases incl. MS, AD Parkinson’s, or brain injury. It is anticipated that having a wide range of diagnostic tools available to doctors will have a positive impact on the ability to diagnose & treat numerous conditions more successfully
- The test's launch and availability showed Labcorp's dedication to neurology and to enhance the health and lives of people suffering from neurodegenerative and related disorders
- The approval was based on the results from the P-II (DESTINY-Lung02) trial evaluating the safety & efficacy of Enhertu (5.4mg/kg & 6.4mg/kg) in patients (n=101) with HER2 mutant mNSCLC. The US FDA has also approved companion diagnostic tests to detect HER2 mutation in lung tumor tissue & plasma
- The results demonstrated an ORR of 57.7% in patients who previously received a systemic therapy as assessed by BICR along with a CR of 1.9%, PR of 55.8% & a mDoR of 8.7mos.
- Enhertu is a HER2-directed ADC developed using Daiichi Sankyo’s proprietary DXd ADC technology. Daiichi Sankyo & AstraZeneca will ensure access to medication and necessary financial support to the US patients who are prescribed Enhertu
- The company discontinued the P-III (AMEERA-5) trial evaluating the efficacy and safety of amcenestrant + palbociclib vs letrozole + palbociclib in a ratio (1:1) in 1068 patients with ER+/HER2- advanced breast cancer
- The decision was based on the prespecified interim analysis which showed that P-III (AMEERA-5) trial failed to meet the prespecified boundary over the control arm while no new safety signals were observed
- The company will continue to review the data and intends to eventually inform the scientific community of its findings. All additional amcenestrant studies will discontinue, incl. the (AMEERA-6) study in early-stage breast cancer
- Jemincare to receive $60M up front & is also eligible to receive ~$590M in additional fees upon achievement of development, regulatory and sales-based milestones along with royalties on net sales. The collaboration focuses to develop new treatment options for patients with advanced prostate cancer
- Jemincare to get an exclusive license to develop and commercialize JMKX002992 globally Genentech will be fully responsible for the development and commercialization costs
- JMKX002992 is a novel oral degrader of the androgen receptor & is indicated to treat patients with prostate cancer who have developed resistance to current therapies
- Zimmer Biomet & Surgical Planning Associates collaborated to commercialize HipInsight, a US FDA-cleared mixed reality navigation system for total hip replacement
- This collaboration enables to offer surgeons for the exclusive use of Microsoft HoloLens 2 glasses to visualize a hologram of a patient's pelvic anatomy during surgery with the addition of HipInsight in Zimmer Biomet's hip implant portfolio
- The system also improves accuracy for better outcomes and optimizes efficiency during total hip arthroplasty. The system enhances the capabilities of the ZBEdge suite of integrated smart, digital, and robotic technologies to deliver game-changing data-driven insights to optimize patient outcomes
- The (XALOC) RWE program consists of (XALOC-1 & 2) study evaluating Fasenra in 750+ & 400+ patients along with the (ImPROve Asthma) study in Germany with SEA
- The (XALOC-1) study results showed an AAER reduction of 84% @48wk. over baseline in an overall patient population, reduction in AAER of 79–91% across patient subgroups regardless of atopic status, CRSwNP comorbidity, maintenance OCS use, FeNO & blood eosinophil cut-offs, improvements in asthma control
- The (ImPROve Asthma) study, part of the (XALOC-2) study showed improvements across PROs measures in the total SEA cohort & CRSwNP, 35% achieved clinical remission @6mos. in a real-world setting & 44% with SEA & comorbid CRSwNP, and no decrease of forced expiratory volume in FEV1 ≥200 mL vs baseline
25. Eyenovia Reports Results of MicroLine in P-III Trial for the Treatment of Presbyopia
- The P-III (VISION-2) study evaluates the safety & efficacy of MicroLine vs PBO in patients (n=140) with presbyopia randomly assigned in groups receiving a 2% dosing of MicroLine vs PBO
- The results demonstrated that the study met its 1EP depicting a ≥15-letter improvement in distance corrected near visual acuity with ≤5-letter loss in distance acuity vs PBO in low light conditions @2hrs. post-treatment. The study also met all 2EPs at a statistically significant level
- MicroLine is a pilocarpine ophthalmic solution developed for the treatment of presbyopia. Additionally, Eyenovia expects to submit an NDA application for the approval of MicroLine to the US FDA within its planned commercialization timeline
- The P-II (SUMMIT) basket trial evaluating neratinib in 582 patients with EGFR exon 18-mutant NSCLC. 79% of patients prior treated with an EGFR-targeted TKI
- In an interim efficacy, the therapy showed ORR (35%), 30% of patients pretreated with TKIs, and 50% not pretreated with TKIs, response or SD lasting for ≥ 48wks. was observed in 7 patients (6 PR, 1 SD), 3% permanently discontinued the treatment due to diarrhea
- Neratinib was approved in the US for early-stage HER2-overexpressed/amplified breast cancer & is marketed as Nerlynx. The therapy was also approved in the US in combination with capecitabine for advanced or metastatic HER2+ breast cancer & has received EC's marketing authorization for early-stage HR+ HER2-overexpressed/amplified breast cancer
- Ocuphire will receive $35M up front, $10M milestones upon FDA approval & is eligible to receive additional regulatory & sales milestones along with royalties. The companies collaborated for Nyxol across 3 indications in the US, EU, Japan, India, China & other markets globally
- Famy will be responsible to fund & commercialize the development of Nyxol eye drops in the US incl. clinical, manufacturing & regulatory activities required for FDA approval of all 3 Nyxol indications, incl. Nyxol+Low-Dose Pilocarpine
- The activities will be managed by Ocuphire. Famy will lead the development in non-US markets. Nyxol is currently being studied in the P-III trial for presbyopia and NVD while NDA submission for RM is expected in Q4’22
- Minoryx to receive up front & is eligible to receive milestones and development funding of ~$256M along with royalties
- Neuraxpharm gets an exclusive right to commercialize leriglitazone (selective PPAR gamma agonist) in the EU and continues to collaborate with Minoryx to advance the development of leriglitazone while Minoryx holds the full rights to leriglitazone in the US and globally (ex- China)
- If Leriglitazone is approved in the EU, it will be the first approved treatment for adult male patients with X-ALD. Both companies continue to advance the therapy for additional X-ALD patient populations and other orphan indications
- Tonix & Boston Children's Hospital collaborated to study TNX-1500 (Fc-modified anti-CD40L mAb) in GvHD after HCT in animals
- The primary objective of the preclinical research is to study the activity of TNX-1500 administered prophylactically to modify GvHD progression after HCT & support an IND application for human studies. The P-I study of TNX-1500 is expected to be initiated in H1’23
- In animal studies, TNX-1500 showed activity in preventing organ rejection & was well tolerated in non-human primates. Non-human primate models of cardiac & kidney allograft transplantation successfully and safely prevented pathologic alloimmunity by blocking CD40L with TNX-1500 monotx. without clinical thrombosis
- The P-III (DINAMO) trial evaluating the efficacy & safety of Jardiance (10/25mg) and Tradjenta (5mg) vs PBO in 175 children and adolescents aged 10-17yrs. with T2D
- The trial met its 1EPs i.e., Jardiance (SGLT2 inhibitor) showed a reduction in HbA1c while 0.84% reduction @26wk. with the addition of Jardiance to other baseline treatments (diet, exercise, metformin & insulin). Additionally, the overall safety data were consistent with prior results with a well-established safety profile
- The 2EPs also showed a reduction in fasting plasma glucose @26wk., a numerical reduction of 0.34% were seen. The results were submitted to a peer-reviewed journal
Related Post: PharmaShots' Most Read News of 2020
Tags
AVEO
Gilead
AbbVie
Biohaven
Sanofi
Channel Biosciences
Scilex
Tonix
Amgen
Dermavant
Blueprint
BeiGene
Byondis
Biogen
MedRhythms
Cogent
Lilly
Labcorp
Pfizer
Medtronic
Daiichi Sankyo
AstraZeneca
Genentech
Jemincare
Zimmer Biomet
Puma Biotechnology
Ocuphire Pharma
Minoryx
Eyenovia
Neuraxpharm
Boehringer Ingelheim
Eli Lilly
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.
