Phil Gomez, CEO of Siga Tech­nolo­gies, is hav­ing a busy sum­mer.

His com­pa­ny, which makes the on­ly avail­able mon­key­pox treat­ment, has been prepar­ing for this mo­ment for the last decade, work­ing with a team of sup­pli­ers and ramp­ing up just ahead of the out­break — hop­ing that it would nev­er have to de­ploy its treat­ment.

First ap­proved in 2018 for small­pox, and known as Tpoxx or tecovir­i­mat, the FDA didn’t ini­tial­ly ex­tend the ap­proval to mon­key­pox be­cause the com­pa­ny didn’t run a tri­al pri­or to the cur­rent out­break even though a tri­al could’ve the­o­ret­i­cal­ly been done.

Still, next week, the US plans to ramp up ship­ments of the drug and send out 55,000 dos­es to states and oth­er ju­ris­dic­tions, which is more than dou­ble the cur­rent sup­ply.

So what should peo­ple ex­pect in terms of ac­cess­ing this treat­ment? Is the clin­i­cal tri­al da­ta com­ing? And who’s mak­ing the de­ci­sions around the cur­rent dis­tri­b­u­tion? Read be­low a light­ly edit­ed phone in­ter­view with Gomez from Fri­day morn­ing.

End­points: Can you help me un­der­stand the math bet­ter — if there are about 13,000 con­firmed cas­es by the CDC, and the US says it’s ship­ping out 55,000 dos­es of Tpoxx be­gin­ning next week — shouldn’t every­one who wants the an­tivi­ral get ac­cess to it?

Gomez: I’ll have to de­fer to the CDC and FDA on how they’re do­ing it. It wasn’t clear to me that they’ve changed the cri­te­ria on who would re­ceive the dos­es, and it sound­ed like [they] said they will still fo­cus on pro­vid­ing Tpoxx to high-risk pa­tients. But by pre-po­si­tion­ing more cours­es, they will be able to more rapid­ly get cours­es to pa­tients.

End­points: Is the plan still to ramp up to about 600,000 cours­es of Tpoxx per year, and are you work­ing with con­tract man­u­fac­tur­ers to ramp up pro­duc­tion?

Gomez: There are 1.7 mil­lion dos­es of Tpoxx in the Strate­gic Na­tion­al Stock­pile, so we de­liv­ered those be­gin­ning in 2013, and start­ed de­liv­er­ing more in 2020 — so there is still a sub­stan­tial amount in the SNS. In terms of the CMOs, Siga works with WR Grace and Co. on the ac­tive in­gre­di­ent, Catal­ent for en­cap­su­la­tion, PCI for pack­ag­ing, and Pow­der­size [a Lon­za sub­sidiary] mi­cronizes and tests API for use.

End­points: The NIH next month is about to kick off a place­bo-con­trolled mon­key­pox tri­al in the US, fund­ed and spon­sored by the NIH. Can you talk about Siga’s in­volve­ment in the tri­al?

Gomez: We’re sup­port­ing 10 ran­dom­ized-con­trolled tri­als around the world via gov­ern­ment pub­lic health agen­cies, and we pro­vide drug, place­bo and ex­per­tise, and the good news is these pro­to­cols have been shared broad­ly across the US, Cana­da, UK and EU. So we’re hope­ful wher­ev­er the out­break goes, we’ll have an op­por­tu­ni­ty to rapid­ly col­lect da­ta and file that in the US and Cana­da.

We have ap­proval in the UK and Eu­rope for mon­key­pox, but the NI­AID tri­al would ul­ti­mate­ly al­low us to sup­port a US ap­proval for Tpoxx in mon­key­pox. From a high-lev­el per­spec­tive, these out­pa­tient tri­al pro­to­cols will look at the res­o­lu­tion of symp­toms, with a sec­ondary end­point of re­duc­ing vi­ral load. If any­one pro­gress­es to se­vere dis­ease, they would be un­blind­ed and switched to ac­tive drug.

End­points: How would you com­pare this tri­al to the Covid-19 vac­cine and drug tri­als from ear­li­er in the pan­dem­ic?

Gomez: It’s very dif­fer­ent than a Covid vac­cine tri­al be­cause it’s just look­ing at symp­toms re­solv­ing, which usu­al­ly on­ly last three to four weeks and Tpoxx will po­ten­tial­ly short­en that. With hope­ful­ly hun­dreds en­rolled, these tri­als should be much more rapid than the vac­cine tri­al, but it will de­pend on the out­break and where peo­ple are en­rolled.

End­points: US gov­ern­ment of­fi­cials have dis­trib­uted about 22,000 dos­es of Tpoxx so far. Any idea what the da­ta have shown in terms of the treat­ment work­ing?

Gomez: We haven’t got­ten back any re­al-world ev­i­dence from the CDC yet, so I de­fer to them. Broad­ly, we’ve been ac­tive­ly col­lect­ing da­ta on mon­key­pox, there were da­ta from the UK pub­lished last year, there’s been com­pas­sion­ate use treat­ment in the Cen­tral African Re­pub­lic, and there’s an­oth­er RCT that we’ve set up along­side NI­AID in the De­mo­c­ra­t­ic Re­pub­lic of Con­go be­fore the cur­rent out­break.

The FDA said in 2018 that when we won ap­proval for small­pox, that we couldn’t win one for mon­key­pox per the An­i­mal Rule be­cause we could’ve run a tri­al in the DRC. But the FDA has al­so said the DRC clade is dif­fer­ent from the cur­rent one, so now, with the broad out­break, un­for­tu­nate­ly, the op­por­tu­ni­ty is to col­lect the re­al world da­ta.

End­points: The de­ci­sion to use an ex­pand­ed ac­cess pro­gram to dis­trib­ute Tpoxx — was that a de­ci­sion Siga made? And any plans to move Tpoxx in­to the com­mer­cial mar­ket?

Gomez: The CDC made that de­ci­sion. The drug was de­vel­oped for small­pox and we’ve on­ly ever sold it to gov­ern­ments. So we have not looked at the com­mer­cial sales chan­nels, but we cer­tain­ly now are work­ing to eval­u­ate and plan to put it through the com­mer­cial mar­ket, but that will take time. We’re look­ing at the RCT da­ta as an in­flec­tion point for that.

End­points: Did you push back against the idea of us­ing an ex­pand­ed ac­cess pro­gram to dis­trib­ute Tpoxx?

Gomez: They [the CDC] have the prod­uct in the SNS so they con­trol it. We don’t have a lot of in­put on that.

End­points: And HHS yes­ter­day an­nounced the for­mu­la by which states and ju­ris­dic­tions would be al­lo­cat­ed Tpoxx — did you work with HHS on that for­mu­la?

Gomez: Yes­ter­day was the first time I saw it. The drug is in the SNS. I think we’re viewed more as a sup­pli­er.

End­points: What can you say about the sup­ply and de­mand of Tpoxx world­wide — giv­en that it’s so dif­fi­cult to find in the US, is there a short­age right now? And can you meet the grow­ing de­mand?

Gomez: This is a ques­tion we look at every day. I think most pub­lic health or­ga­ni­za­tions saw the ini­tial out­break and thought it wouldn’t be so dead­ly, so they took a let’s wait-and-see ap­proach. Then in the next phase, as cas­es start­ed to grow, coun­tries bought small sup­plies to treat the cur­rent out­break, and we’ve been work­ing pre­dom­i­nant­ly in that space, and have $60 mil­lion in in­ter­na­tion­al or­ders of prod­uct.

We’re now in the next phase which is where can see this out­break go, and what might we need over the next year or two, and that’s evolv­ing and we have prod­uct avail­able but we don’t know where it will go.

If mil­lions and mil­lions of cours­es are need­ed, we will need to rad­i­cal­ly re­think our sup­ply net­work and ex­pand it. But we just don’t know where it goes.

To pro­vide con­text: In 2020 and 2021, we de­liv­ered about 363,000 cours­es to the US gov­ern­ment, and we an­tic­i­pat­ed de­liv­er­ing that next year and the year af­ter, so we an­tic­i­pat­ed a lot of de­liv­er­ies over the next few years, and we ad­vanced pro­duc­tion of Tpoxx to make sure API was avail­able, so we, for­tu­nate­ly, were in a good place when the out­break hit.

In the fu­ture, there isn’t an ac­tive or­der for oral Tpoxx from BAR­DA but there are two re­main­ing or­ders from a 2018 con­tract and sev­er­al oth­ers for in­tra­venous ver­sions of the drug.

End­points: So if there isn’t a short­age of treat­ments right now, why is it still so dif­fi­cult to get? And why is the gov­ern­ment ret­i­cent to ship out more?

Gomez: That’s a ques­tion for CDC. Dis­tri­b­u­tion is their purview, and there are a lot of com­pli­cat­ed reg­u­la­tions be­cause of the SNS, and the fact that it’s ap­proved for small­pox and not mon­key­pox. We get a lot of ques­tions about use and dos­ing of the drug, and how to give it to chil­dren. For any­one un­der 13 kilo­grams [about 29 pounds], the tablet has to be frac­tion­at­ed, which has been done so far for some chil­dren, and we’re work­ing on a liq­uid re­sus­pen­sion dose that hasn’t been used yet.

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

James Sabry’s BD team at Roche has a long track record in hunting the globe for new biotech deals. But they’ve never journeyed into China before to ink a worldwide development and commercialization pact with a China-based biotech on an experimental med.

As Max Gelman reported yesterday, Roche fronted a new alliance with China’s Jemincare with $60 million in cash and $590 million in milestones for worldwide commercial rights to an oral androgen receptor degrader. The deal itself is fairly typical of an early-stage alliance around a promising treatment. The Shanghai-based biotech is largely unknown outside China, but this is a classic high-risk, modest upfront pact that Roche routinely inks.

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

Roche’s Genentech is going high style next month for New York Fashion Week. The pharma is hosting its first-ever runway fashion show to raise disability visibility, featuring models from the spinal muscular atrophy (SMA) community.

“Double Take” will be held Sept. 8, the day before the official New York event begins, with models walking and rolling across the stage wearing stylish and functional adaptive clothing. Eleven people living with SMA and four advocates will show off the custom fashions created by Open Style Lab, a Brooklyn nonprofit and accessible clothing design collaborative.

Unlock this story instantly and join 148,500+ biopharma pros reading Endpoints daily — and it's free.

It’s been just two days since Endo International filed for bankruptcy in an attempt to dig itself out of thousands of opioid lawsuits. Now one of its top sellers is in trouble.

A federal appeals court on Thursday affirmed a Delaware judge’s decision that Eagle Pharmaceuticals’ generic version of Endo’s vasopressin injection Vasostrict does not infringe on the company’s patents. Eagle’s version won approval back in December, and already, the generic and others like it have driven down Vasostrict sales.

After a nearly 365-day delay, Axsome Therapeutics has secured its first drug approval with an FDA green light for Auvelity as a treatment for adults with major depressive disorder.

The biotech is keeping shy on the pricing for now and, on an investor call, CEO Herriot Tabuteau attributed the FDA’s yearlong delay mainly to the Covid-19 pandemic. The rapid-acting NMDA receptor antagonist is not a scheduled drug under the DEA, the CEO confirmed on the call.

Unlock this story instantly and join 148,500+ biopharma pros reading Endpoints daily — and it's free.

Martin Landray knows what controversy in clinical drug development feels like, from first-hand experience.

Landray was the chief architect of RECOVERY, a study that pitted a variety of drugs against Covid-19. And he offered some landmark data that would help push dexamethasone out into broader use as a cheap treatment, while helping ice hydroxy’s reputation as a clear misfire.

“Lots of people told us we shouldn’t use it,” Landray says about dexamethasone and Covid-19. “It was dangerous. We shouldn’t even do a trial. They also cared about hydroxychloroquine and lots of people said we shouldn’t do a trial because it must be used. I’ve got the letters from both sets of people.”

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

ALS is well-known as one of the most difficult drug targets, and the US cost-effectiveness watchdog ICER isn’t quite inching closer to endorsing the two candidates.

ICER’s Midwest CEPAC (Comparative Effectiveness Public Advisory Council) met Friday to discuss clinical evidence on Amylyx’s AMX0035 and Mitsubishi Tanabe Pharma America’s intravenous version of an older ALS medicine, edaravone.

Unlock this story instantly and join 148,500+ biopharma pros reading Endpoints daily — and it's free.

As Congress continues to mull whether and how to reform the FDA’s accelerated approval pathway, new research on the pathway continues to crop up, attempting to guide the way for new reforms.

Earlier this week, several prominent researchers from Harvard, UPenn and the Brookings Institution called for new financial incentives to encourage companies to finish the trials necessary to convert accelerated approvals to full approvals, or at least reform how companies are paid after winning an accelerated approval.

Unlock this story instantly and join 148,500+ biopharma pros reading Endpoints daily — and it's free.

Two drug manufacturers on opposite sides of the globe are facing the heat from the FDA over several quality control issues.

India-based manufacturer Sun Pharma was hit with a Form 483 following an FDA inspection of its Mohali facility from Aug. 3-12.

The FDA found failures when reviewing unexplained discrepancies regarding whether a batch already had been distributed at the location.

The investigation found that there were instances of backdating, but Sun’s report was not thorough enough for the FDA to evaluate the full scope of the offense. Sun did not interview all quality assurance reviewers, but it did confirm backdating by employees who had denied the practice initially. Sun also did not have a thorough review of other work performed by the employees, according to the FDA report.

Bioscience & Technology Business Center The University of Kansas Lawrence, Kansas

If you're already an Endpoints subscriber, enter your email below for a magic link that lets you log in quickly without using a password. Please note the magic link is one-time use only and expires after 24 hours.

We'll e-mail you a link to set a new password. Please note this link is one-time use only and is valid for only 24 hours.

ENDPOINTS NEWS Daily at 11:30 AM ET

EARLY EDITION Daily at 7:15 AM ET

ENDPOINTS PHARMA Daily at 2 PM ET

ENDPOINTS MARKETING RX Tue at 2 PM ET

ENDPOINTS FDA+ Wed at 2 PM ET

ENDPOINTS MANUFACTURING Thu at 2 PM ET

ENDPOINTS WEEKLY Sat at 6 AM ET