Scrip: industry news and insights
By Kevin Grogan 05 Aug 2021
Just approved In US for chronic kidney disease in patients with type 2 diabetes, Kerendia is going to be an...
US specialty firm AMAG Pharmaceuticals Inc. is viewed as an acquisition target for companies established in women’s health – specifically Novartis AG – due to the potential of bremelanotide in hypoactive sexual desire disorder (HSDD). Licensed from Palatin Technologies Inc., the first-in-class melanocortin-4 receptor agonist is thought to offer a better safety and efficacy profile than Sprout Pharmaceuticals Inc.’s Addyi (flibanserin), which was returned to Sprout by Bausch Health Companies Inc. last November. (Also see "Valeant Returns $1bn Female Libido Drug For Free" - Scrip, 7 Nov, 2017.) The US FDA is currently reviewing bremelanotide with a March 2019 user fee date; an FDA advisory committee is expected in Q4 2018. (Also see "BIO Notebook Day 4: Gottlieb Seeks Early Engagement On Gene Therapy; Ireland’s Brexit Opportunities; AMAG’s Bremelanotide Strategy; Alzheimer’s ‘Learnings’ " - Scrip, 7 Jun, 2018.)
Germany’s MediGene AG is partnered with bluebird bio Inc. on TCR-modified T-cell programs for multiple indications. Its platform targets intracellular tumor antigens that aren’t addressable with CAR-T therapeutics. The deal was expanded in May, adding another $500m in earn-outs on top of the $1bn possible under the original agreement.[See Deal] (Also see "Medigene's bluebird Deal Validates TCR Technology And Immune-Oncology Strategy" - Scrip, 4 Oct, 2016.) The expanded deal and complementary tech make Medigene a logical acquisition target for bluebird.
UK specialty firm Allergy Therapeutics PLC has a short-course vaccine candidate for allergic rhinitis – Pollinex Quattro Grass– that should begin Phase III in 2019. (Also see "Phase III Beckons For A Short-Course Seasonal Allergic Rhinitis Jab" - Scrip, 21 May, 2018.) PharmaVitae says the product has $400m peak earnings potential. Allergy Therapeutics might make good takeout candidate for a company established in the grass allergy space, such as Merck & Co. Inc.
With financing in place, Verona will next start a Phase II study of nebulized RPL554 added onto LABA/LAMA or triple therapy as a maintenance treatment for COPD patients - and then move to the crucial Phase III program.
UK-based Verona Pharma PLC's first-in-class, inhaled PDE3/4 inhibitor RPL554 could be first new bronchodilator in 40 years. It is expected to enter Phase III in 2019 as maintenance therapy for COPD and could eliminate need for adjunctive therapy with inhaled corticosteroids. Verona, which netted $81.1m in its 2017 US IPO, could be targeted by AstraZeneca PLC or GlaxoSmithKline PLC to enhance their respiratory portfolios.
The US biotech’s severe peanut allergy oral immunotherapy AR101 has FDA breakthrough therapy status and has shown strong efficacy versus placebo in Phase III, though there are still safety concerns; additional safety study is due in Q3. BLA filing is expected before end of 2018 and approval second half 2019. PharmaVitae predicts $1.2bn in peak sales potential. AR101 is in a Phase II combination study for peanut allergy with Sanofi/Regeneron Pharmaceuticals Inc.’s Dupixent. Sanofi might see Aimmune Therapeutics Inc. as an attractive takeout target. Aimmune is well underway with launch planning and intends to target 5,000 allergy specialists. (Also see "Aimmune Accelerates Commercial Planning For Peanut Allergy Drug " - Scrip, 20 Feb, 2018.)
Pfizer's Ibrance has a strong lead in the CDK4/6 space, but Novartis' Kisqali and Lilly's Verzenio are looking to carve out their own niches, expand the category and move into earlier treatment settings. Another scramble is on as well, to answer the question: what comes next?
G1 Therapeutics Inc. is a US oncology-focused biotech specializing in CDK 4/6 inhibitors. It has two Phase Ib/IIa candidates: trilaciclib for small-cell lung cancer, which may reduce myelosuppression, and G1T38, which in combination with AstraZeneca’sFaslodex showed best-in-class tolerability in breast cancer at ASCO 2018.[See Deal] ( (Also see "Other ASCO 2018 Highlights – It’s Not All IO " - Scrip, 17 May, 2018.)) Although these candidates are behind approved CDK inhibitors, PharmaVitae perceives market opportunity based on differentiation. Merck & Co. could see G1 as an acquisition target to strengthen its cancer portfolio, after suspending development of its CDK inhibitor dinaciclib.
The amount of venture capital that Denali Therapeutics Inc. has raised and the number of collaborations it's negotiated in less than two years is pretty staggering for a startup biopharmaceutical company, not to mention a startup focused on the central nervous system and neurodegeneration.
Still early stage, theneurodegenerative disease-focused US biotech Denali Therapeutics Inc. raised the largest Series A round of 2015 at $217m, followed by a $265m IPO in 2017.[See Deal] Denali has two Phase I Parkinson’s candidates that target LRRK2, DNL151 and DNL201. Its platform technology offers potential to create first-in-class candidates focused on lysosomal function, glial biology and cellular homeostasis. Denali’sAntibody Transport Vehicle platform also can generate antibodies that can permeate the blood-brain barrier. Denali could be attractive buyout target for Takeda Pharmaceutical Co. Ltd., its partner in a 2018 neurodegenerative disease collaboration.[See Deal] Also, companies in the neurology space, such as Biogen Inc. and Celgene Corp., that might have appetite for risk in areas of high unmet medical need could be interested.
The US biotech’s CGRP candidate eptinezumab, given as a quarterly I.V., has shown numerically stronger reduction in migraine days than monthly CGRP drugs.Alder Biopharmaceuticals Inc.expects to file a BLA first quarter 2019. PharmaVitae thinks the market potential of CGRP class in migraine is underestimated due to too small patient population estimates and the belief that payers will be reluctant to reimburse these products long-term. With Amgen Inc./Novartis’Aimovig priced lower than expected in hopes of increased access, Alder could be an attractive target for neurology-focused Biogen if payer response to the class is more positive than expected, even though Alder’s late entry to market and I.V. formulation will prevent it from being top seller in the class. (Also see "Amgen's Aimovig Aims To Capture As Many Migraine Patients As Possible With $6,900 Price" - Scrip, 17 May, 2018.) and (Also see "Best-In-Class Or First-In-Class: CGRP Inhibitors Line Up To Win The Migraine Market" - Scrip, 8 May, 2017.)
The US rare disease specialist Amicus Therapeutics Inc.'s Galafold was cleared by the FDA Aug. 10 for Fabry disease, an oral therapy compared to Sanofi’s injectableFabrazyme. Galafold already approved in the EU and Japan. Amicus also has a Phase I/II Pompe disease candidate. The company might be a logical acquisition target for Sanofi, which has the only current marketed therapies for both diseases. PharmaVitae expects big market impact in the US for Galafold, and quick sales growth globally. Amicus announced $16.7m in net product sales for Q1 2018. Amicus has brought in more than $1.2bn in follow-on offerings since its 2007 IPO.[See Deal]
Company reported only $33,000 in sales for late anticoagulant market entrant Bevyxxa and $2.2m for the antidote Andexxa, leaving a lot of challenges ahead after the retirement of CEO Bill Lis, effective Aug. 1.
US-based Portola Pharmaceuticals Inc. has the oral anticoagulantBevyxxa and Factor Xa inhibitor antidoteAndexXa on the market, with a third pipeline candidate (cerdulatinib for hematologic malignancies) slated to enter pivotal trials in 2019. Bevyxxa sales have been non-material to date, and the EMA issued a negative review this past March. Andexxa obtained US approved in May but Portola still needs FDA approval for a manufacturing process that will facilitate production sufficient to grow sales. (Also see "Manufacturing Snag May Stall Portola's Bevyxxa Launch" - Scrip, 5 Sep, 2017.) With Portola’s CEO retiring in August, these issues indicate a big pharma buyout might be beneficial.Bristol-Myers Squibb Co.,Pfizer Inc.,Bayer AG and Johnson & Johnson are likely suitors due to their stakes in the factor Xa anticoagulant market; acquiring Portola could increase uptake of their growing brands by using their manufacturing expertise to ensure the Andexxa launch goes smoothly, PharmaVitae notes.
Scrip: industry news and insights
05 Aug 2021
Sarepta believes it is still very much in the running with its gene therapy but is playing down talk of...
Scrip: industry news and insights
05 Aug 2021
New chief medical officers at IGM Biosciences, Compass Pathways, Passage Bio and more.
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