High Point, N.C.-based Vtv Therapeutics Inc. shares (NASDAQ:VTVT) shares were trading midday at $3.38, up $1.28, or 60%, on positive results from the phase II study called Simpliciti-T1 testing TTP-399 as an oral adjunctive therapy to insulin in adults with type 1 diabetes (T1D). The 12-week trial, conducted with support from Juvenile Diabetes Research Foundation International, explored the efficacy and safety of 800 mg of liver-selective glucokinase activator TTP-399 compared with placebo in 85 people with T1D on optimized insulin therapy. Simpliciti-T1 achieved its primary objective analyzed using two statistical approaches to evaluate the effect of the drug. The primary statistical analysis evaluated the effect on HbA1c regardless of treatment adherence or notable changes in insulin administration. That measure turned up statistically significant improvements in HbA1c with TTP-399 vs. placebo (p=0.03).

Roche, Lilly drugs fail to slow decline in early onset AD

A high-profile test of two experimental medicines in people with autosomal dominant Alzheimer’s disease (ADAD), a rare inherited form of the disease, found neither drug significantly slowed the rate of cognitive decline vs. placebo, the primary endpoint. Both, Roche Holding AG's gantenerumab and Eli Lilly and Co.'s solanezumab, target amyloid beta and remain in late-stage tests for broader populations of people with more common forms of AD. While lessons to be learned from studying ADAD were initially framed as an important path to learning about more common types of AD, on Monday Roche and Lilly discouraged read-through to their ongoing studies of the drugs.

Akeso refiles IPO with HKEX amid coronavirus outbreak

BEIJING – Zhongshan-based Akeso Inc., which focuses on bispecific antibodies, is again seeking pre-revenue listing on the Hong Kong Stock Exchange, after its first IPO attempt failed to go through in December. The move comes at a time when analysts believe the coronavirus outbreak is weighing on the city’s IPO market.

Oculis’ topical DME drug hits phase II endpoints, validates nanoparticle delivery solution

LONDON – Oculis SA reported positive results for its topical nanoparticle formulation of dexamethasone in the treatment of diabetic macular edema, showing the eye drops reduced central macular thickness in the phase II proof-of-concept trial. There also was an improvement in best corrected visual acuity, with the “p” value 0.125 meeting the prespecified conditions in the protocol for statistical superiority of the product, OCS-01, in the 144-patient randomized study.

EEG signature can predict response to SSRIs

Large-scale trials show little to no statistical benefit of antidepressant drugs over placebo, and “we’ve had this perspective as a field for the past 10 years at least, that antidepressants don’t work very well,” Amit Etkin told BioWorld. But another way to understand the underwhelming trial results is that “antidepressants are just fine, but our diagnoses are not.” In the Feb. 10, 2020, issue of Nature Biotechnology, Etkin and his colleagues described an EEG signature that could predict which patients would respond well to the antidepressant Zoloft (sertraline).

Budget draft for 2021 eyes NIH cuts; stakeholders wary of treatment of FDA numbers

The general outlines of the Trump administration’s budget proposal for fiscal 2021 has been released, and the Alliance for a Stronger FDA said in a Feb. 10 statement that multiple administrations have exaggerated the proposed increases to the FDA budget, and thus those numbers should be eyed with skepticism. The proposed budget for the FDA was omitted in the general budget framework released shortly after noon ET Feb. 10, although the White House proposed a budget of $38 million for the NIH. That figure comes in at more than $3 billion less than congressionally authorized for the current fiscal year, and thus is sure to be overridden by the House and Senate.

Anxious results: Biohaven’s troriluzole misses its primary endpoint

Top-line results from Biohaven Pharmaceutical Holding Co.’s phase III trial of troriluzole against placebo in treating generalized anxiety disorder (GAD) failed to hit its primary endpoint, prompting the company CEO to say the poor results support halting development plans for the glutamate modulator as a monotherapy in GAD. Over eight weeks, patients treated with troriluzole had a mean improvement change from baseline of -9.28 points on the HAM-A total score vs. -9.35 points on placebo, with a “p” value of 0.917. The company stock (NYSE:BHVN) suffered at midday, dropping 10%. Biohaven’s rimegepant for acute migraine treatment, however, was just named to the Cortellis Drugs to Watch list identifying rimegepant with the potential to hit at least $1 billion in annual sales by 2024.

2020 Cortellis Drugs to Watch: 11 potential blockbuster drugs offer hope, threaten budgets

Crowned by a potential cure for severe hemophilia A, that could become the most expensive drug ever, a new list of 11 medicines expected to generate $1 billion-plus in annual sales by the end of 2024 or earlier throws into stark relief the growing tension between medical innovation and society's ability to pay for it. The 2020 Cortellis Drugs to Watch list, including medicines both approved and likely to be, points to a future of ongoing conflict between payers and industry spurred by fundamental disagreements.

Pricing could assume a larger role for would-be blockbusters

No matter how effective it is, a drug is worthless if the people who need it can’t afford it. That’s been almost an anthem for patients and policy wonks testifying before U.S. Congress on drug prices. With the increased public focus and scrutiny on the subject, pricing is likely to play an outsized role in how some of the 11 medicines on this year’s Cortellis Drugs to Watch list perform on the market. It especially will be true for those entering crowded therapeutic spaces where they will face not only competition from other brand drugs but also from generic or biosimilar versions of those drugs.

CNS drugs provide new delivery options, cleaner safety for MS and migraine

While the efficacy of three central nervous system (CNS) drugs awaiting regulatory approvals is not vastly different from currently marketed products, their formulations and methods of delivery, combined with what payers will support, make them formidable players in the multiple sclerosis (MS) and migraine markets. Two of these drugs on the Cortellis Drugs to Watch list are for relapsing MS and include ozanimod(RPC-1063) from Bristol-Myers Squibb and Co., of Princeton, N.J., and ofatumumab (OMB-157) from Novartis AG, of Basel, Switzerland. For migraine, rimegepant from New Haven, Conn.-based Biohaven Pharmaceutical Holding Co. Ltd. could become a new oral option for patients with acute, or episodic, attacks.

A patient’s perspective: Jaime Sanders finds relief with CGRP therapies

Jaime Sanders was just a child, barely 8 years old, when a debilitating condition kept her inside from recess and home from school. A neurologist diagnosed her with migraines, and the condition worsened from three to five attacks per month in high school to intractable migraines every day for three months during a pregnancy with her third child. Over the years, she tried propranolol, Esgic-Plus, Tylenol, ibuprofen and a cup of coffee or caffeinated soda, but the attacks continued and became progressively more severe, lasting longer each time.

A patient’s perspective: Karen Jury’s MS progression halted, but fatigue, drug cost concerns persist

At 26 years old, Karen Jury stood before a class of elementary students as her right arm tingled before falling completely numb. Lhermitte’s phenomenon, she would later discover, is a classic indicator of relapsing-remitting multiple sclerosis. An MRI confirmed the diagnosis when it lit up with active plaque lesions on her brain in 2008. Next began the parade of trial-and-error therapies.

Therapies poised to reshape the treatment landscape for hematologic disorders

Millions of people are affected by blood disorders, and the prevalence is expected to grow as our population ages. In the wings and poised to reshape the treatment landscape for hematologic disorders are two potential blockbuster drugs - Valrox (valoctocogene roxaparvovec) for hemophilia A and vadadustat for anemia related to chronic kidney disease - that are among the 11 included in the Cortellis Drugs to Watch analysis predicted to achieve annual sales of $1 billion by 2024.

Out of basic science, a blockbuster: Vadadustat

Vadadustat and other drugs in its class work by inhibiting prolyl hydroxylase-hypoxia induced factor 1 (PH-HIF1). And their discovery was driven by scientific curiosity about how cells sense and cope with changing oxygen levels.

Filgotinib looks to JAK up sales with multiple indications

Filgotinib (GLPG-0634) has the potential to be a blockbuster drug, according to Cortellis Drugs to Watch analysis, but it may take a few approvals in various indications to reach the coveted mark of $1 billion in annual sales. The drug is an oral inhibitor of JAK1; overactivation/dysregulation of JAK1 can lead to autoimmune responses. Filgotinib is currently up for approval as a treatment for rheumatoid arthritis, with marketing applications for the initial indication filed in the EU in August 2019, in Japan in October 2019 and in the U.S. in December 2019.

Novartis’ high ‘steaks’ with inclisiran in FH; optimism for Rybelsus in diabetes

The less-frequent dosing regimen of Basel, Switzerland-based Novartis AG’s cholesterol therapy, inclisiran, under development in the hands of subsidiary The Medicines Co., positions the small interfering RNA (siRNA) drug to take on marketed proprotein convertase subtilisin/kexin type 9 (PCSK9)-targeting antibodies as well as statins in the busy therapeutic space. Also in the metabolic class in the Cortellis Drugs to Watch analysis is Bagsvaerd, Denmark-based Novo Nordisk A/S' oral once-daily Rybelsus (semaglutide) for type 2 diabetes, approved in September 2019 thanks to a priority review voucher that guaranteed a greased path for the first non-injected GLP-1 receptor protein therapy. Cardiovascular safety data were added to the drug’s label in January 2020. Additional filings for adults with type 2 diabetes have also been submitted in the EU and Canada.

Inclusive research brings scientific, monetary riches: Inclisiran

Inclisiran’s inclusion on the 2020 Cortellis Drugs to Watch list is an example of target discovery possibilities hiding in plain sight – if companies and institutions are willing to put effort into increasing sample diversity in genomic research.

Is it finally their time? Two ADCs for cancer make the blockbuster list

Two of three oncology drugs selected for blockbuster status in the Cortellis Drugs to Watch analysis are antibody-drug conjugates (ADCs), highly targeted cancer therapies designed to leave the healthy cells be and zap the bad ones. Astrazeneca plc’s and Daiichi Sankyo Co. Ltd.’s Enhertu (fam-trastuzumab deruxtecan-nxki) was an FDA approval preemie: the ADC arrived months ahead of schedule in December 2019 and launched in the U.S. in January. Immunomedics Inc.’s most advanced product candidate is sacituzumab govitecan, an ADC targeting the Trop-2 receptor expressed by many solid cancers and delivering the moderately toxic drug, SN-38, directly to the tumor. The non-ADC cancer drug on the Drugs to Watch list is lisocabtagene maraleucel, also known as liso-cel or JCAR-017, from Bristol-Myers Squibb Co. It’s an intravenous anti-CD19 CAR T-cell therapy targeting large B-cell lymphoma.

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