AveX­is shares soar af­ter it out­lines FDA’s sup­port for a loom­ing piv­otal study on SMA

AveX­is CEO Sean Nolan

Shares of AveX­is $AVXS ripped high­er af­ter the mar­ket close on Tues­day af­ter the gene ther­a­py biotech out­lined the FDA’s ac­cep­tance of a small, sin­gle arm piv­otal study for rare cas­es of spinal mus­cu­lar at­ro­phy Type 1 (SMA).

Ac­cord­ing to the Chica­go-based biotech, reg­u­la­tors signed off on a 20-pa­tient study that would use a his­tor­i­cal com­par­i­son for eval­u­at­ing ef­fi­ca­cy. And that lays a straight path to a piv­otal study now slat­ed to be­gin in the first half of a loom­ing 2017.

AveX­is shares shot up 25% on the tri­al plans.

Here’s the sum­ma­ry:

At the Type B meet­ing and in the meet­ing min­utes, the FDA ac­knowl­edged the com­pa­ny’s ra­tio­nale for a sin­gle-arm piv­otal study and pro­vid­ed a num­ber of con­struc­tive sug­ges­tions to help op­ti­mize such a tri­al de­sign. The FDA al­so in­di­cat­ed its pref­er­ence for a de­sign with co-pri­ma­ry end­points con­sist­ing of a mea­sure of de­vel­op­men­tal mile­stone achieve­ment (such as sit­ting unas­sist­ed) along with a clin­i­cal­ly mean­ing­ful mea­sure of sur­vival (such as time to an “event” as de­scribed above). Based on FDA’s sug­ges­tions as well as oth­er ex­pert in­put, AveX­is con­tin­ues to eval­u­ate a num­ber of the de­tails of the tri­al de­sign. More spe­cif­ic in­for­ma­tion will be made avail­able at the time the study is ini­ti­at­ed, which is ex­pect­ed in the first half of 2017.

“We be­lieve the Type B meet­ing had a pos­i­tive tone, with FDA of­fer­ing a num­ber of con­struc­tive sug­ges­tions which we be­lieve will bet­ter en­able im­ple­men­ta­tion of a piv­otal study de­sign that is most ap­pro­pri­ate for the pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease,” said AveX­is CEO Sean Nolan in a state­ment. “With the feed­back need­ed from the FDA to move for­ward with our piv­otal tri­al, we plan to pro­ceed as ex­pe­di­tious­ly as pos­si­ble to be­gin the study in the first half of 2017.”

Not on­ly that, the FDA — which ap­pears to be quite ac­com­mo­dat­ing in AveX­is’s sum­ma­ry — is al­so will­ing to con­sid­er an ap­proval on the Phase I study. At least one an­a­lyst says these de­vel­op­ments could pose a prob­lem for Bio­gen and Io­n­is, which are part­nered on nusin­ersen for SMA. Notes Jef­feries’ Bri­an Abra­hams:

While ear­ly da­ta for AVXS’s AVXS-101 has looked promis­ing in SMA, it is hard to com­pare be­tween treat­ments and BI­IB re­tains a first mover ad­van­tage that could po­ten­tial­ly lim­it up­take of ‘101. Ad­di­tion­al­ly, nusin­ersen is al­so be­ing ex­plored in stud­ies of Type 2/3 SMA, which rep­re­sent ad­di­tion­al sig­nif­i­cant mar­ket op­por­tu­ni­ties, and the pos­si­bil­i­ty of broad ini­tial ap­proval across all sub­types re­mains; in con­trast, AVXS is just be­gin­ning to ex­plore such ad­di­tion­al SMA pop­u­la­tions. Sys­temic ad­min­is­tra­tion of AVXS-101 could, how­ev­er, of­fer greater con­ve­nience, at least in Type 1 pts. We would not rule out the pos­si­bil­i­ty the ther­a­pies could be used in the same pts, though pric­ing could be an is­sue. We cur­rent­ly mod­el a 2017 nusin­ersen launch with prob­a­bil­i­ty-ad­just­ed rev­enues po­ten­tial­ly reach­ing $1.5B by 2025.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty


I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pascal Soriot, AstraZeneca CEO (via Getty images)

UP­DAT­ED: FDA slaps As­traZeneca's MCL-1 can­cer drug with a hold af­ter safe­ty is­sue — 2 years af­ter Am­gen axed a trou­bled ri­val

There are new questions being posed about a class of cancer drugs in the wake of the second FDA-enforced clinical hold in the field.

Two years after the FDA hit Amgen with a clinical hold on its MCL-1 inhibitor AMG 397 following signs of cardiac toxicity, AstraZeneca says that regulators hit them with a hold on their rival therapy of the same class.

The pharma giant noted on clinicaltrials.gov that its Phase I/II study for the MCL-1 drug AZD5991 “has been put on hold to allow further evaluation of safety related information.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

Sur­geons suc­cess­ful­ly at­tach pig kid­ney to a hu­man for the first time, us­ing tech from Unit­ed's Re­vivi­cor

In a first, researchers reportedly successfully transplanted a pig kidney into a human without triggering an immediate immune response this week. And the technology came from the biotech United Therapeutics.

Surgeons spent three days attaching the kidney to the patient’s blood vessels, but when all was said and done, the kidney appeared to be functioning normally in early testing, Reuters and the New York Times were among those to report. The kidney came from a genetically altered pig developed through United’s Revivicor unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,300+ biopharma pros reading Endpoints daily — and it's free.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.