FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednes­day, in part due to pan­dem­ic-re­lat­ed trav­el re­stric­tions.

North Car­oli­na-based Liq­uidia Tech­nolo­gies was hand­ed a CRL for its lead pul­monary ar­te­r­i­al hy­per­ten­sion drug, cit­ing the need for more CMC da­ta and on-site pre-ap­proval in­spec­tions, which the FDA hasn’t been able to con­duct due to trav­el re­stric­tions. The agency al­so de­ferred its de­ci­sion on Re­vance Ther­a­peu­tics’ BLA for its frown line treat­ment, be­cause it needs to in­spect the com­pa­ny’s north­ern Cal­i­for­nia man­u­fac­tur­ing fa­cil­i­ty. The ac­tion, Re­vance em­pha­sized, was not a CRL.

The FDA said it’s “ac­tive­ly work­ing to de­fine an ap­proach for out­stand­ing in­spec­tions,” ac­cord­ing to Re­vance.

Neal Fowler

“With the pan­dem­ic, as you know, they’ve been very chal­lenged to do these,” Liq­uidia CMO Tushar Shah said dur­ing a call with in­vestors on Wednes­day morn­ing. “We an­tic­i­pate a lot of com­pa­nies are strug­gling, and in the best case sce­nario, we would have an­tic­i­pat­ed some­time mid-next year would have been the ear­li­est they would prob­a­bly get to in­spec­tion,” he con­tin­ued.

De­spite the set­back, Liq­uidia is still eye­ing a 2022 launch for LIQ861 — if it gets ap­proval. The tre­pros­tinil in­hala­tion pow­der was the fo­cal point of Liq­uidia’s 2018 IPO fil­ing, which priced at $11 per share, the mid­point of a $10 to $12 range. The biotech raked in $50 mil­lion, $30 to $32 mil­lion of which was tagged for LIQ861.

On Wednes­day, Liq­uidia’s $LQ­DA stock was down 6.13%,with shares pric­ing at $2.91 apiece. The biotech says the ad­di­tion­al CMC da­ta sought by the FDA per­tain to the “drug prod­uct and de­vice bio­com­pat­i­bil­i­ty,” and that the agency didn’t ask for fur­ther clin­i­cal stud­ies, or stud­ies re­lat­ed to tox­i­col­o­gy or clin­i­cal phar­ma­col­o­gy.

Liq­uidia has a tech­nol­o­gy col­lab­o­ra­tion go­ing with Glax­o­SmithK­line, which it ex­pand­ed to in­clude three ad­di­tion­al pro­grams last year. The biotech is on a mis­sion to un­seat Unit­ed Ther­a­peu­tics’ Ty­va­so and its neb­u­liz­er, which net­ted $415.6 mil­lion last year. John­son & John­son al­so en­tered the PAH space when it bought Acte­lion for $30 bil­lion back in 2017.

If Re­vance’s in­jectable Dax­i­bot­u­linum­tox­i­nA gets ap­proved, it’ll go head-to-head with Al­ler­gan’s Botox. The drug was test­ed against mod­er­ate to se­vere glabel­lar lines, more com­mon­ly known as frown lines. It met its pri­ma­ry and sec­ondary end­points in two Phase III tri­als and was shown to last for six months, ac­cord­ing to Al­ler­gan, while Botox gen­er­al­ly lasts for three to four.

Mark Fo­ley

Al­ler­gan com­ment­ed back in 2017, though, that it doesn’t be­lieve Re­vance’s da­ta “will sup­port a longer du­ra­tion claim as the on­ly com­pos­ite da­ta (2-point im­prove­ment, none or mild, and both in­ves­ti­ga­tor and pa­tient) is at 30 days not at 6 months.”

The FDA ac­cept­ed Re­vance’s BLA back in Q1. Its stock $RVNC was up 4.02% on Wednes­day, sell­ing at $24.35 a share. News of the FDA’s de­fer­ral comes just over a year af­ter co-founder Dan Browne stepped down as CEO “due to mis­judg­ment in han­dling an em­ploy­ee mat­ter,” leav­ing board mem­ber Mark Fo­ley, for­mer chief of Zel­tiq Aes­thet­ics, to fill his shoes.

“We look for­ward to con­tin­ued in­ter­ac­tion with the Agency and re­main ready to sup­port FDA’s pre-ap­proval in­spec­tion as soon as pos­si­ble. We are for­tu­nate that we man­u­fac­ture our prod­uct at a sin­gle lo­ca­tion in the U.S., which should put us at an ad­van­tage com­pared to in­ter­na­tion­al man­u­fac­tur­ing lo­ca­tions once trav­el re­sumes,” Fo­ley said in a state­ment.

He lat­er added that he be­lieves Re­vance “is in an ex­cel­lent po­si­tion, both com­mer­cial­ly and fi­nan­cial­ly, to weath­er a change to the tim­ing of this po­ten­tial ap­proval.”

A pre­vi­ous ver­sion of this ar­ti­cle in­cor­rect­ly at­trib­uted a quote by Liq­uidia CMO Tushar Shah to CEO Neal Fowler. 

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

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.

An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

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.

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.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 98,200+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Mike Grey, Plexium chairman (Horizon Therapeutics)

Plex­i­um adds in­dus­try vet Mike Grey to the brain trust with new in­vestor cash fund­ing its pro­tein degra­da­tion play

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.