Jon P. Stonehouse, President, Chief Executive Officer of BioCryst

BioCryst bags $250M in a debt and roy­al­ty deal as the ex­ec­u­tive team preps a new drug launch for HAE

Three days af­ter land­ing an FDA OK for their HAE drug bero­tral­stat, BioCryst $BCRX now has some cash to see how far they can go in com­mer­cial­iz­ing the ther­a­py as Or­ladeyo.

The biotech re­port­ed Mon­day morn­ing that they’ve inked a deal with Roy­al­ty Phar­ma that trades a chunk of their rev­enue from the drug for cash in hand. Roy­al­ty is pay­ing out $125 mil­lion up­front, with a $200 mil­lion cred­it fa­cil­i­ty from Athyri­um Cap­i­tal Man­age­ment that is be­ing tapped for $125 mil­lion right off the bat. The rest will be held in re­serve.

8.75% on di­rect an­nu­al net sales of OR­LADEYO up to $350 mil­lion, 2.75% on sales be­tween $350 mil­lion and $550 mil­lion, no roy­al­ty on sales over $550 mil­lion

Roy­al­ty Phar­ma chief Pablo Legor­re­ta, who took the com­pa­ny pub­lic this year, gets  8.75% on di­rect an­nu­al net sales of Or­ladeyo up to $350 mil­lion, 2.75% on sales be­tween $350 mil­lion and $550 mil­lion, with no roy­al­ties on sales over $550 mil­lion. There’s al­so a 1% roy­al­ty take for one of the biotech’s ex­per­i­men­tal drugs, if it makes it to the mar­ket.

Legor­re­ta of­fered an en­dorse­ment with his mon­ey, call­ing their new­ly ap­proved ther­a­py “trans­for­ma­tive.”

But no mat­ter how much the ex­ec­u­tive crew at BioCryst have in the bank, mar­ket­ing this drug won’t be easy.

BioCryst has an ad­van­tage mar­ket­ing a new oral ther­a­py for HAE, but it comes with marked­ly low­er ef­fi­ca­cy rates, which an­a­lysts be­lieve could well keep pa­tients go­ing back to the cur­rent main­stays in the mar­ket.

An­oth­er lim­it­ing fac­tor is price. The biotech priced their drug at $485,000, which is in line with the mar­ket lead­ers, and way ahead what some an­a­lysts had pro­ject­ed.

BioCryst al­so plans to de­vote a por­tion of the new mon­ey to “ad­vance the de­vel­op­ment of BCX9930 in­to clin­i­cal tri­als in mul­ti­ple com­ple­ment-me­di­at­ed dis­eases.”

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.

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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.

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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.

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?

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Hedge fund jumps in with Avoro ac­tivists in an at­tempt to de­rail Mer­ck­'s $11B Ac­celeron buy­out

Avoro Capital, which made its bones blowing up the Seagen-Immunomedics deal and then selling the smaller biotech for $21 billion, is getting an assist in its quest to derail Merck’s $11 billion buyout of Acceleron $XLRN.

Wednesday morning one of Acceleron’s biggest investors joined the opposition. Darwin Global Management, a hedge fund which owns about 4% of Acceleron, blasted the Merck deal, saying the Big Pharma is getting the company for billions less than what it’s worth. Earlier, Holocene Advisers, reportedly a top-20 investor in Acceleron, said it would not tender its stock after criticizing the $180-per-share deal.

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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.”

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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.

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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.

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