News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months af­ter Rea­ta Phar­ma­ceu­ti­cals sug­gest­ed its Friedre­ich’s atax­ia pro­gram omavelox­olone could be de­layed, the com­pa­ny re­vealed that is in­deed go­ing to be the case.

Rea­ta $RE­TA shares took a nose­dive Wednes­day af­ter the biotech re­vealed that the FDA said sup­ple­men­tal da­ta for its piv­otal tri­al did not strength­en the case for ap­proval. As a re­sult, the drug is like­ly to need an­oth­er study be­fore the FDA takes up the case.

In ear­ly Wednes­day morn­ing trad­ing, Rea­ta stock was down rough­ly 8% off the news. It’s a small­er dive than when the com­pa­ny first said the pro­gram could be set back, though, as shares lost about a third of their val­ue at the time.

As such, this news was not en­tire­ly un­ex­pect­ed but still proved dis­ap­point­ing to an­a­lysts.

“While the path for­ward for omavelox­olone in the US re­mains to be de­ter­mined, we be­lieve it is large­ly de­risked by the pos­i­tive re­sults seen” in the study, SVB Leer­link’s Joseph Schwartz wrote to in­vestors.

Baird’s Bri­an Sko­r­ney took a more bull­ish view, de­scrib­ing the up­date as mere­ly an “in­cre­men­tal neg­a­tive.” Sko­r­ney said he has been op­er­at­ing with a de­lay be­ing the “base-case as­sump­tion” and that the news doesn’t af­fect Rea­ta’s oth­er pro­gram, bar­dox­olone for rare kid­ney dis­eases, which is ex­pect­ed to be filed in the first quar­ter of 2021.

The FDA did not rule out re­con­sid­er­ing omavelox­olone’s ap­pli­ca­tion once the new study has been com­plet­ed, Rea­ta not­ed.

J&J’s Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Four months af­ter nab­bing an ex­pand­ed la­bel in the US for ac­tive pso­ri­at­ic arthri­tis, J&J’s Trem­fya has got­ten the OK for the in­di­ca­tion in Eu­rope as well.

Trem­fya, al­so known as guselkum­ab, had pre­vi­ous­ly been ap­proved on the con­ti­nent for mod­er­ate to se­vere plaque pso­ri­a­sis. The drug is a mon­o­clon­al an­ti­body tar­get­ing p19 in the IL-23 and in­hibits its in­ter­ac­tion with the IL-23 re­cep­tor.

Wednes­day’s ap­proval comes on the ba­sis of two Phase III stud­ies look­ing at adults who had ac­tive PsA and those who were bi­o­log­ic-naïve. Both stud­ies looked at pa­tients who had pre­vi­ous­ly failed stan­dard ther­a­pies.

Back when it first got US ap­proval in 2017, an­a­lysts pegged peak an­nu­al sales for the drug at $1.6 bil­lion, which would make it a top 20 drug. Through the first three quar­ters of 2020, Trem­fya had net­ted J&J and Janssen $965 mil­lion world­wide.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

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.

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What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

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.

News brief­ing: Five pub­lic biotechs, over 2 days, raise $883M from fresh of­fer­ings; Bel­gian biotech ex­pands Se­ries B fund­ing

The wave of biotech IPOs we’ve been seeing in the last few days underscores that the public markets remain one of the key channels for fresh investments in drug R&D. And that trend was in full view this week as a slate of biotechs nailed down hundreds of millions of dollars in fresh funds.

One of the big winners of the week is Editas $EDIT, which nailed $231 million to back its pioneering work on a gene editing platform. The biotech sold 3.5 million shares at $66 each.

Northway Biotech's new manufacturing facility in Greater Boston

North­way Biotech sets up shop in Boston hub, look­ing to court more cus­tomers with bi­o­log­ics-fo­cused plant

Getting a foot in the door in Boston’s bustling biopharma hub is a rite of passage for many companies, but it comes with a steep price tag. Lithuanian CDMO Northway — now with a new moniker — will set up a new plant in close proximity, and it’s hoping its biologics focus will find a willing customer base.

Northway Biotech (formerly Northway Biotechpharma) on Wednesday held a virtual grand opening ceremony for its $40 million Waltham, MA facility — a 30,000 square-foot cGMP manufacturing and process development plant that will widely expand on the company’s previous capabilities.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

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.

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Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

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.

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Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

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.

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Covid-19 roundup: Italy won­ders aloud if it can sue Pfiz­er for vac­cine short­falls; Flood, dead­ly fire threat­en As­traZeneca vac­cine plants

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.