Seven years after the FDA first approved Esbriet, the blockbuster Roche IPF drug is getting an expedited review for a second indication.
On Thursday, the agency gave Esbriet priority review for unclassified interstitial lung diseases, or forms of pulmonary inflammation and scarring that don’t fit easily into the over 200 known types of ILD. The move comes 10 months after Esbriet received breakthrough designation and sets Roche up for a decision by May.
The Swiss pharma is bidding for a new indication at an acute period for their longtime blockbuster. One of two major idiopathic pulmonary fibrosis drugs alongside Boehringer Ingelheim’s Ofev, Esbriet continues to bring over $1 billion per year. But sales growth has slowed, particularly in the US, and the company’s orphan designation protections in IPF begin to expire this year.
With that deadline looming, Roche has sought to guard against would-be generics, filing around a dozen lawsuits to stop Teva, Sandoz and others from releasing cheaper, copycat versions of the drug. Esbriet’s annual wholesale cost is around $100,000.
Roche acquired Esbriet in their $8.3 billion buyout of the California biotech InterMune, two months before the FDA would give the drug a green light. The agency had initially rejected the drug in 2010, demanding InterMune run a second Phase III trial.
The Big Pharma, though, would not put the drug into a pivotal trial for unclassifiable interstitial lung disease until 2017. In 2019, investigators published Phase II results from 253 unclassifiable ILD patients, showing patients who took the drug had significantly more stable lung function, as measured by an endpoint called forced vital capacity.
The new indication is just one route Roche is using to try to keep its fibrosis franchise alive. The company has also invested in finding a new blockbuster for IPF, acquiring Promedior and its pipeline of anti-fibrotic molecules for up to $1.4 billon in 2019. The lead drug, which has received breakthrough designation, went into Phase III earlier this year.
So far, Roche has stayed ahead of Boehringer in that search; two months after Roche started their Phase III, the German pharma dropped out of its own $1 billion fibrosis pact, citing toxicity with their partner’s drug.
By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures
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Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.
But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.
It’s official: Janet Woodcock is now the acting chief of the FDA.
And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.
To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.
The firehose of biopharma news is gushing these days.
That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.
Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.
But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.
Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.
GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game.
The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen.
Several months after a name change and major restructuring, Kuur Therapeutics has offered an early glimpse at results from a small number of patients treated with their CAR-NKT cell therapies developed with the Baylor College of Medicine.
Out of 10 evaluable neuroblastoma patients dosed with Kuur’s lead candidate, an autologous GD2-targeting CAR-NKT therapy, researchers noted one complete response, one partial response, and three patients with stable disease, according to the Houston-based biotech.
Tony Coles’ team at Cerevel Therapeutics is adding two high-profile board members, including an ex-governor that has lots of connections to the Boston area where the biotech is based.
Former Massachusetts Gov. Deval Patrick is hopping on Cerevel’s board of directors, the company announced Thursday, joining less than three months after Cerevel went public on the backs of Perceptive’s ARYA II SPAC. And in a twist, Pfizer’s new business development chief Deborah Baron is joining the board as well, about three years after the Big Pharma shuttered the neuroscience pipeline that Cerevel is seeking to revive.
The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.
BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.
根据Houston生物技术中心的研究，在10名接受Kuurs lead候选药物治疗的神经母细胞瘤患者中，研究人员注意到1例完全应答，1例部分应答，3例病情稳定。Kuurs lead候选药物是一种自体靶向GD2的CAR-NKT疗法。
Cerevel Therapeutics的Tony Colesteam将增加两名高知名度的董事会成员，其中包括一名前州长，他与该生物技术公司所在的波士顿地区有很多联系。