PharmaShots' Key Highlights of Third Quarter 2023
Shots:
- Third quarter of 2023 highlights major acquisitions in the pharma and biotech industry along with clinical trial results and approvals. Starting with the latest acquisitions, Biogen acquired Reata Pharmaceuticals for ~$7.3B and Danaher acquired Abcam for ~$5.7B
- The third quarter of the year also showcases multiple clinical trial results which include Janssen reported P-IIb trial (FRONTIER 1) results of JNJ-2113 for Plaque Psoriasis, Novartis highlighted P-III trial (NETTER-2) results of Lutathera as 1L advanced Gastroenteropancreatic Neuroendocrine Tumors
- Our team at PharmaShots has summarized and complied with the insights of Q3'2023
Date - July 03, 2023
Product – Cyltezo
- The company launch Cyltezo, a US FDA-approved interchangeable biosimilar to Humira in the US for multiple chronic inflammatory diseases. Cyltezo is available as a prefilled syringe or an autoinjector pen
- The efficacy & safety of Cyltezo were based on the P-III randomized (VOLTAIRE-X) trial evaluating the effect of multiple switches b/w Humira & Cyltezo vs continuous treatment with Humira. The results showed PK equivalence with highly similar efficacy & immunogenicity, and comparable safety was observed in patients who received Humira continuously or who switched b/w Humira & Cyltezo
- Cyltezo was approved as a biosimilar in 2017 for multiple chronic inflammatory diseases & was approved as an interchangeable biosimilar in 2021
Janssen Reports P-IIb Trial (FRONTIER 1) Results of JNJ-2113 for Moderate-to-Severe Plaque Psoriasis
Date - July 04, 2023
Product – JNJ-2113
- The P-IIb trial evaluating three (25/50/100, qd) & two (25 & 100mg, BID) dosages of JNJ-2113 (IL-23R antagonist peptide) vs PBO in 255 adult patients
- The trial achieved all primary & secondary efficacy EPs i.e., patients who received JNJ-2113 achieved PASI 75 (37.2% at 25mg, qd)/(51.2% at 25mg, BID)/(58.1% at 50mg, qd)/(65.1% at 100mg, qd)/(78.6% at 100mg, BID) over 9.3% in PBO, PASI 90 (25.6%/26.8%/51.2%/46.5%/59.5% vs 2.3%) and PASI 100 (11.6%/9.8%/25.6%/23.3%/40.5% vs 0%), respectively, 100% improvement in skin clearance at highest dose level at 16wk.
- The therapy was well tolerated, patients experienced one or more AEs was 52.4% vs 51.2%, and no evidence of a dose-dependent increase in the occurrence of specific AEs was reported
Kashiv Biosciences Reports P-I Clinical Study Results of ADL018 (biosimilar, omalizumab)
Date - July 05, 2023
Product – ADL018 (biosimilar, omalizumab)
- The P-I study has been completed evaluating ADL018, a biosimilar candidate to Xolair, EU-approved omalizumab, or US-licensed omalizumab via SC inj. in healthy volunteers
- The primary objective of the study was to demonstrate PK similarity by evaluating the area under the concentration-time curve from time zero to infinity (AUCinf) and Cmax and compare the safety & tolerability while the secondary objectives were to determine the PD activity & immunogenicity b/w three treatment groups
- The company plans to initiate the patients dosing in the P-III study shortly for CSU. Omalizumab was approved to treat persistent asthma in patients aged ≥6yrs. and chronic rhinosinusitis with nasal polyps in patients aged ≥18yrs.
Date - July 12, 2023
Product – Opzelura
- The P-III study (TRuE-AD3) evaluating Opzelura (selective JAK1/JAK2 inhibitor) vs vehicle in 300 patients aged ≥2-<12yrs. with AD
- The study met its 1EPs i.e., patients achieved IGA-TS (0.75% vs 1.5%). The overall safety profile was consistent with previous data with no new safety signals while the long-term safety portion of the study will continue as planned
- The therapy was approved in the US for the topical treatment of nonsegmental vitiligo in patients aged ≥12yrs. & for the topical short-term and non-continuous chronic treatment of mild to mod. AD in non-immunocompromised patients & is also approved in the EU for non-segmental vitiligo with facial involvement in adults and adolescents
Eli Lilly to Acquire Versanis for ~$1.92B
Date - July 17, 2023
Product – Bimagrumab
- Versanis shareholders will receive ~$1.925B in cash incl. up front & subsequent fees upon achievement of development & sales milestones
- Versanis' bimagrumab, a mAb that binds activin type II A & B receptors to block activin & myostatin signaling is currently being evaluated in the P-IIb trial (BELIEVE) as monotx. & in combination with semaglutide for adults who are overweight or obese. The results are expected in the middle of 2024
- The acquisition focuses to expand Eli Lilly’s weight loss treatment portfolio & improves patient outcomes in cardiometabolic diseases. Bimagrumab & incretins have the potential to reduce fat mass while maintaining muscle mass which improves outcomes for persons with obesity & obesity-related complications
Date - July 25, 2023
Product – EryDex
- EryDel stockholders will receive ~$485M in total downstream cash fees incl. ~ $5M in development milestones, $25M at NDA acceptance, $60M in approval milestones & $395M in market & sales milestones with no royalties. The transaction is expected to close in Q3’23 & will incl. the assumption of EryDel’s $13M EIB loan with scheduled payments beginning in H2’26
- The acquisition will advance EryDel’s AIDE technology & development of EryDex to deliver innovative treatments to patients. EryDel stockholders will own 16.7% of the combined company & will operate as a wholly owned subsidiary of Quince with plans to retain EryDel’s corporate & manufacturing presence in Italy
- EryDex received ODD from the US FDA & EMA for Ataxia-Telangiectasia (A-T) & has completed a pilot P-II trial in A-T patients. The first patient is expected to enroll in the P-III trial for EryDex in Q2’24 with NDA submission at the end of 2025
Date - July 26, 2023
Product – V940 + Keytruda
- The companies initiate the P-III (V940-001) clinical trial evaluating the safety and efficacy of V940 (mRNA-4157) + Keytruda (anti-PD-1 therapy) vs Keytruda alone in a ratio (2:1) in 1089 patients with resected high-risk (Stage IIB-IV) melanoma at 165+ sites in ~25 countries globally
- The 1EPs of the study is RFS while the 2EPs incl. distant metastasis-free survival (DMFS), OS, and safety
- V940 (mRNA-based individualized neoantigen therapy) + Keytruda received the BTD & PRIME scheme from the US FDA and EMA for the adjuvant treatment of patients with high-risk melanoma, based on the results from the P-IIb study (KEYNOTE-942/mRNA-4157-P201) study
Teleflex to Acquire Palette Life Sciences for ~$650M
Date - July 26, 2023
Product – N/A
- Teleflex to acquire Palette Life Sciences AB for an up front cash fee of $600M & an additional ~$50M upon achievement of commercial milestones. The acquisition is expected to be close in Q4’23
- The acquisition will expand the Teleflex Interventional Urology portfolio incl. UroLift System with the addition of non-animal stabilized hyaluronic acid (NASHA) spacer & tissue bulking products improves patient outcomes in urology & urogynecology disorders, colorectal conditions & radiation oncology procedures
- The Palette Life Sciences portfolio also includes Deflux & Solesta designed to treat pediatric vesicoureteral reflux and fecal incontinence, respectively. Barrigel was cleared for marketing in the US, and Australia, and is CE Marked
Date - July 27, 2023
Product – Enhertu
- The P-II trial (DESTINY-PanTumor02) evaluating Enhertu (5.4mg/kg) in 268 patients with prior-treated LA, unresectable or metastatic HER2 expressing solid tumors not eligible for curative therapy, incl. biliary tract, bladder, cervical, endometrial, ovarian, pancreatic & rare cancers
- The trial met the pre-specified target for ORR & showed a durable response. The safety profile was consistent with that observed in other trials with no new safety signals
- The results will be presented at an upcoming medical meeting & shared with global regulatory authorities. Enhertu (5.4mg/kg) was approved in 40+ countries for unresectable or metastatic HER2+ breast cancer, based on (DESTINY-Breast03) trial results
Date - July 28, 2023
Product – UGN-102
- The company highlighted the P-III trials (ATLAS) at clinical sites in the US, EU & Israel, and (ENVISION) across 56 sites evaluating UGN-102 in 282 & 240 patients
- The (ATLAS) study met its 1EPs of DFS, 55% reduction in risk of recurrence, progression, or death, CR rate at 3mos. (64.8% for UGN-102 vs 63.6% for TURBT) & demonstrated superiority to TURBT. The (ENVISION) trial also met its 1EPs & showed a 79.2% CR rate at 3mos., following the initial treatment while additional data evaluates the EPs of DoR
- The results showed a robust & consistent therapeutic profile across multiple clinical trials, was well tolerated with a side effect profile similar to prior trials. The company is expected to submit an NDA to the FDA in 2024, based on the P-III study (ENVISION) results
Date - July 28, 2023
Product – AL102
- Under the merger agreement, the combined company will operate under Ayala Pharmaceuticals, Inc., and trade on the OTCQX under Ayala’s ticker symbol (“ADXS”). The transaction is expected to close by the end of Q3’23
- The combined company focuses on accelerating a portfolio of oncology assets with the addition of Ayala’s AL102 currently in the registrational (RINGSIDE) study for desmoid tumors and Biosight’s Aspacytarabine (BST-236). After merger completion, the combined company's ownership will be divided with 55% going to Biosight stockholders and 45% to Ayala stockholders
- The results from the P-II portion of the (RINGSIDE) study showed activity of AL102 against progressing desmoid tumors, 50% PR, and 100% DCR in evaluable desmoid tumor patients treated with AL102 (1.2mg, qd). The updated data are expected in 2023
Biogen to Acquire Reata Pharmaceuticals for ~$7.3B
Date - July 31, 2023
Product – Skyclarys
- Biogen to acquire Reata for $172.50/share in cash representing a 60% premium from Reata’s share price with a total enterprise value of ~$7.3B. The transaction is expected to close in Q4’23
- The acquisition will add Skyclarys (omaveloxolone), the first and only US FDA-approved treatment for Friedreich’s ataxia (FA) to Biogen’s portfolio of drugs. Additionally, Reata is developing a portfolio of innovative products for a range of neurological diseases
- Additionally, the MAA for omaveloxolone is under EMA’s review. Omaveloxolone also received ODD from EC for the treatment of FA
Date - August 01, 2023
Product – Jemperli
- The approval of Jemperli (dostarlimab-gxly) + carboplatin + paclitaxel, followed by Jemperli as monotx. for the treatment of adult patients with dMMR primary advanced or recurrent endometrial cancer
- This approval is based on the interim analysis results from Part 1 of the P-III study (RUBY), which demonstrated a median duration of follow-up of ≥25mos. A significant and clinically meaningful benefit in PFS and a 71% reduction in the risk of disease progression or death. The trial continues to evaluate OS
- Consistent safety and tolerability were seen over the monotherapies. The most common TEAEs were ≥20%. Also, the data were presented at ESMO and SGO 2023 and published in The New England Journal of Medicine.
Date - August 02, 2023
Product – Talicia
- RedHill's partner Gaelan Medical, part of the Ghassan Aboud Group (GAG) receive marketing approval from the UAE Ministry of Health for Talicia (omeprazole magnesium, amoxicillin & rifabutin)
- The P-III study results showed that patients treated with Talicia achieved 84% eradication of H. pylori inf. in the ITT group vs 58% in active comparator arm, minimal to zero resistance to rifabutin was detected, patients had response rates of 90.3% vs 64.7%
- Talicia, a novel rifabutin-containing all-in-one oral capsule designed to treat H. pylori & was approved in the US for H. pylori inf. in adults along with eligible for 8yrs. of US market exclusivity under QIDP designation
Cybin Initiates Dosing of CYB003 in Final Cohort of the P-II Trial for Major Depressive Disorder
Date - August 02, 2023
Product – CYB003
- The company initiated the dosing in cohort 6, the final cohort of the P-II study evaluating CYB003 in patients with major depressive disorder (MDD). The P-II efficacy results are expected in Q3/Q4’23 & dosing is expected to be completed in Q3’23
- The results showed that patients treated with CYB003 showed a robust psychedelic response and a favorable safety and tolerability profile at doses of up to 12mg
- Additionally, the first five cohorts of CYB003 administered at doses of 1/3/8/10/12mg have completed dosing with no serious AEs, and no patients discontinued due to AEs. CYB003, an investigational deuterated analog of psilocybin for the treatment of MDD
Date - August 03, 2023
Product – UV1
- The patient’s enrolment has been completed & the last patient received the first dose in the P-II study evaluating UV1 + pembrolizumab vs pembrolizumab alone at 10 sites across Germany. The trial was sponsored by Martin-Luther-University Halle-Wittenberg with support from Ultimovacs
- The study focuses to determine the clinical performance of UV1 vaccination as an add-on to standard pembrolizumab treatment. The 1EPs is PFS rate at 6mos. after 1st administration & 2EPs incl. PFS, OS, ORR, DoR & safety with a minimum of 12mos. follow-up, results are expected in H2’24
- UV1 is being evaluated in a broad clinical development program across multiple cancer indications with different biologics & disease stages in combination with different checkpoint inhibitors
Addimmune to go Public via 10x Capital Venture Acquisition Corp. III SPAC Merger for ~$500M
Date - August 11, 2023
Product – AGT103-T
- The proposed transaction represents a pre-money enterprise value of $500M for Addimmune incl. earnout of ~$300M in various clinical and priced-based milestones. The transaction is expected to close in Q1’24
- The combined company is expected to trade under the ticker symbol “HIV”. Existing Addimmune stockholders will roll 100% of their equity into the combined company
- Additionally, 10X III and Addimmune signed a non-binding LOI with Cantor for a $50M committed equity facility. The P-I trial results of AGT103-T showed positive safety results in 7 patients while the P-I trial along with another sponsor-initiated follow-on study showed active immune responses to HIV ~6mos. after dosing
Date - August 16, 2023
Product – Senvelgo
- The US FDA has approved Senvelgo (velagliflozin oral solution), the first liquid once-daily, orally administered prescription medication to improve glycemic control in cats with diabetes mellitus
- Senvelgo oral solution, a highly selective inhibitor of the sodium-glucose co-transporter 2 (SGLT2) will be available in veterinary clinics in the US in mid-October. The product is also expected to be commercially available in countries globally
- By lowering elevated blood glucose levels and reducing the risk of clinical hypoglycemia events, Senvelgo oral solution improves the clinical signs of diabetes that cats suffer as soon as one week after starting treatment
Harmony Biosciences to Acquire Zynerba for ~$200M
Date - August 17, 2023
Product – Zynerba
- Harmony to acquire all outstanding shares of Zynerba for a purchase price of $1.1059/ share in cash, $60M in the aggregate at the closing of the transaction. The transaction is expected to close in Q4'23
- Zynerba shareholders are eligible for ~$2.5444 in CVRs per share upon completion of milestones for a total value of $140M incl. ~$15M/ (~$0.2747/share) upon completion of P-III trial for FXS, additionally $30M/ (~$0.5494/share) on positive P-III data readout before Dec 2024 or $20M/ (~$0.3663/share) before June 2025 or $10M/ (~$0.1831/share) after June 2025
- For regulatory milestones, $35M/ (~$0.6389/share) upon the US FDA approval in FXS; $15M/ (~$0.2707/share) for 2nd indication while $15M/ ($0.2702/share) & $30M/ ($0.5405/share) upon $250M & $500M aggregate net sales respectively. Zygel is being studied in a P-III for FXS while open-label P-II results of Zygel showed positive signals for 22q11.2 deletion syndrome
Date - August 22, 2023
Product – Magrolimab
- The US FDA has placed a partial clinical hold on the initiation of new patients into the US studies as well as for the expanded access program evaluating magrolimab, an anti-CD47 immunotherapy in patients with AML
- The decision was based on the previous announcement on the discontinuation of the P-III study (ENHANCE) for magrolimab + azacytidine in higher-risk myelodysplastic syndromes due to futility observed during a planned analysis. No new patients can be screened or enrolled at this time due to a partial clinical hold
- Enrolled patients may continue to receive treatment & monitored. The company is working with regulators to determine the next steps to release the hold and resume enrolment while studies of magrolimab in solid tumors continue without any impact action
Danaher to Acquire Abcam for ~$5.7B
Date - August 29, 2023
Product – N/A
- Danaher will acquire all of the outstanding shares of Abcam for $24.00 per share in cash representing a 2.7% premium to the stock's last closing price making a total enterprise value of ~$5.7B incl. assumed indebtedness and net of acquired cash. The deal is expected to close in mid-2024
- Additionally, Abcam will operate as a standalone company within the Life Sciences segment of Danaher while mapping complex diseases and accelerating the drug-discovery process.
- Abcam focuses on delivering antibodies, reagents, biomarkers, and assays to address targets in biological pathways vital for progressing drug development and research in the life sciences sector
Date - August 29, 2023
Product – N/A
- Cybin will acquire all of Small Pharma’s issued and outstanding securities in an all-share transaction. Small Pharma' shareholders will receive 0.2409 common shares of Cybin for each common share of Small Pharma implying ~$0.10 per Small Pharma share representing a 43.64% premium based on recent avg. prices. The transaction is expected to be close in late Oct 2023
- Cybin shareholders will own 74.5% of the combined company & Small Pharma shareholders will possess the remaining 25.5%. Both companies possess a comprehensive IP portfolio in the psychedelic drug development sector & combined dataset that covers a variety of clinical programs
- Small Pharma’s two late-stage clinical programs are currently in development for depression & anxiety disorders. The results from both companies’ P-I deuterated programs, CYB004 and SPL028 are expected in late 2023
Date – September 01, 2023
Product – Enhertu
- The US FDA has granted 2 additional BTDs to Enhertu for adult patients with unresectable or metastatic HER2+ solid tumors and HER2+ (IHC 3+) metastatic colorectal cancer. The therapy is being jointly developed & commercialized by Daiichi Sankyo & AstraZeneca
- The BTD for metastatic HER2+ solid tumors was based on a P-II study (DESTINY-PanTumor02) in 268 patients with supporting data from other trials in the Enhertu clinical development program demonstrated durable responses, ORR (37.1%), CR (5.6%), PR (31.5%) & SD (46.1%) while DCR in the overall trial population was 68.2%, presented at ASCO 2023
- The BTD for HER2+ metastatic colorectal cancer was based on final results from the P-II trial (DESTINY-CRC01) in 86 patients showed promising activity & durability with longer-term follow-up. The safety profile was consistent with prior results, presented at ASCO GI 2022
Date – September 01, 2023
Product – SER-252
- Serina will merge with a wholly-owned subsidiary of AgeX, and stockholders of Serina will receive shares of AgeX common stock, the transaction is expected to close in Q1’24. Pre-merger AgeX stockholders are expected to own ~25% of the newly combined company while pre-merger Serina stockholders are expected to own ~75%
- The combined company will operate under Serina Therapeutics & common stock is expected to trade on NYSE American under the "SER" ticker. The combined company focuses on advancing Serina’s pipeline of small molecule drug candidates targeting CNS using the POZ Platform
- Advancing pre-clinical studies of Serina’s SER-252 for advanced Parkinson’s disease with an expected submission of IND application to the US FDA to initiate the P-I trial in Q4’24. The combined company will advance Serina’s lipid nanoparticles & ADC by collaborating with other partners
Date – September 04, 2023
Product – Alecensa
- The P-III study evaluating adjuvant Alecensa (ALK inhibitor) vs Pt-based CT in 257 patients with completely resected stage IB (tumor≥4cm) to IIIA anaplastic lymphoma kinase (ALK)-positive NSCLC
- The trial met its 1EPs of DFS at a prespecified interim analysis & showed a significant and clinical improvement in DFS while the OS were immature at the time of this analysis & no unexpected safety findings were reported
- The results will be submitted to health authorities globally incl. the US FDA and EMA and presented at an upcoming medical meeting. Alecensa was approved in ~100 countries as a 1L treatment for ALK+, metastatic NSCLC, incl. in the US, EU, Japan, and China
- through preclinical, clinical development & commercialization
Date – September 12, 2023
Product – Rybrevant
- The P-Ib/II study cohort evaluating Rybrevant (a bispecific Ab targeting EGFR & MET) + lazertinib (EGFR TKI) and Pt-based CT in 460 patients with r/r NSCLC & EGFR mutations
- The results showed that patients treated with the combination therapy achieved an ORR (50%) with 11 out of 20 patients remaining on treatment, m-DoR (not reached) after a median follow-up of 13.1mos., m-PFS was 14mos., 8 out of 10 responders had a response duration of 6mos., 5 were treated beyond progression with a median incremental treatment duration of 4.2mos. & showed durable PFS
- Rybrevant received accelerated approval in the US for adult patients with LA or metastatic NSCLC with EGFR exon 20 insertion mutations & received approval from health authorities in the EU & other markets globally
Date – September 14, 2023
Product – Tyvalzi
- Under the terms of the agreement, Sun Pharma received commercialization rights of sovateltide in India under the brand name Tyvalzi, and in exchange, Pharmazz will receive upfront and milestone payments along with royalties
- Tyvalzi (sovateltide), is a selective endothelin B receptor agonist, neuronal progenitor cell therapy available for the treatment of acute cerebral ischemic stroke by inducing neurovascular remodeling
- It can be administered for up to 24hrs. after the onset of symptoms and produced clinical improvement in neurological outcomes at 90days post-treatment as measured by the NIHSS, mRS, and B.I. scales
Date – September 25, 2023
Product – Lutathera
- The P-III trial (NETTER-2) evaluating Lutathera + long-acting octreotide in 222 patients with newly diagnosed somatostatin receptor (SSTR)-positive, grade 2 & 3, advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs)
- The trial met its 1EPs & demonstrated a significant improvement in PFS over high-dose long-acting octreotide alone while no new or unexpected safety findings were seen and the results were consistent with the well-established safety profile of Lutathera
- The results will be presented at an upcoming medical meeting and discussed with regulatory authorities. Lutathera, an advanced accelerator applications RLT was approved in the US for SSTR+ GEP-NETs
Date – September 26, 2023
Product – N&A
- Valo will receive an up front & near-term milestone fee of ~$60M and is eligible to receive ~$2.7B as an additional milestone for up to 11 programs along with R&D funding & royalty
- The collaboration will use Valo’s Opal Computational Platform, real-world patient data set, AI-enabled small molecule discovery & Biowire human tissue modeling platform to discover & develop novel cardiometabolic drug programs
- Novo Nordisk will license three of Valo’s preclinical cardiovascular drug programs using the Opal Computational Platform. Valo's preclinical capabilities allow the discovery and validation of novel druggable targets, the development of drug candidates against targets & the prediction of compound safety and efficacy
Date – September 27, 2023
Product – Rystiggo and Zilbrysq
- The approval of rozanolixizumab was based on the P-III study (MycarinG) in 200 adult patients with gMG, which showed a reduction in MG-ADL score from baseline to Day 43 was greater in rozanolixizumab (7mg/kg) group and (10mg/kg group) vs PBO while secondary efficacy EPs incl. change from baseline to Day 43 in the QMG
- The approval of zilucoplan was based on the P-III study (RAISE) in 174 adult patients with anti-AChR Ab+ gMG, which showed a significant & clinical difference favoring zilucoplan over PBO in the MG-ADL & QMG total score change from baseline while 2EPs incl. change from baseline to 12wk. in QMG, MGC & MG-QoL15r
- Improvements in fatigue were reported as an exploratory EP. Both studies' results were published in The Lancet Neurology
Related Post: PharmaShots' Key Highlights of Second Quarter 2023
Tags
Boehringer Ingelheim
Janssen
Kashiv Biosciences
Incyte
Eli Lilly
Versanis
Quince Therapeutics
EryDel SpA
Merck
Moderna
Teleflex
Palette Life Sciences
Daiichi Sankyo
AstraZeneca
UroGen Pharma
Ayala Pharmaceuticals
Biosight
Biogen
Reata Pharmaceuticals
GSK
RedHill Biopharma
Cybin
Ultimovacs
Addimmune
Harmony Biosciences
Zynerba
Danaher
Abcam
Serina Therapeutics
AgeX Therapeutics
Roche
Pharmazz
Sun Pharma
Novartis
Valo Health
Novo Nordisk
UC
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.
