Nixed by the FDA, Ther­a­peu­tic­sMD is now plan­ning a long-shot bid to re­verse the de­ci­sion

Robert G. Finizio

Ther­a­peu­tic­sMD al­ready flagged that the FDA was about to re­ject its ap­pli­ca­tion for TX-004HR, a pro­posed new ther­a­py to treat vagi­nal pain dur­ing in­ter­course for women with vul­var and vagi­nal at­ro­phy trig­gered by menopause. To­day it con­firmed that the agency has in­deed nixed the treat­ment, and it’s prepar­ing a re­but­tal case to see if they can turn it around.

That’s doubt­ful, as the FDA rarely re­vers­es course once reg­u­la­tors have made up their minds — though it can hap­pen.

In this case, though, Ther­a­peu­tic­sMD {TXMD} al­ready faces ri­vals on the mar­ket, un­der­cut­ting any ar­gu­ment it could make on un­met med­ical need. We don’t have the CRL, as the FDA doesn’t pub­lish those, but the Bo­ca Ra­ton, FL-based com­pa­ny says that the reg­u­la­tors’ sole con­cern was lack of longterm da­ta on en­dome­tri­al safe­ty be­yond the 12 weeks it has in the Phase III.

They don’t agree.

The Com­pa­ny be­lieves that the NDA was ap­prov­able as filed and in­tends to meet with the FDA as soon as pos­si­ble to ad­dress the con­cerns raised by the FDA.

If the FDA sticks with its de­mand, Ther­a­peu­tic­sMD would have to launch a new tri­al which would sig­nif­i­cant­ly de­lay any re­turn to the agency.

Ther­a­peu­tic­sMD start­ed the day with a mar­ket cap at $952 mil­lion. The stock dropped about 10% as in­vestors got a bet­ter idea of what was trip­ping the com­pa­ny up.

“While we are dis­ap­point­ed that the NDA for TX-004HR was not ap­proved at this time and re­spect­ful­ly dis­agree with the FDA’s de­ci­sion, we be­lieve there are mul­ti­ple paths for­ward to ad­dress the con­cerns raised by the FDA. The FDA has en­cour­aged us to re­quest a meet­ing to dis­cuss our path for­ward and we in­tend to meet with the FDA as quick­ly as pos­si­ble,” said Ther­a­peu­tic­sMD CEO Robert G. Finizio. “We are al­so con­tin­u­ing to pre­pare the NDA for TX-001HR, our in­ves­ti­ga­tion­al oral bio-iden­ti­cal com­bi­na­tion of estra­di­ol and prog­es­terone, and an­tic­i­pate that we will sub­mit that NDA to the FDA as ear­ly as the third quar­ter of 2017.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.

Martin Shkreli (AP Images)

Mar­tin Shkre­li's in­fa­mous Dara­prim falls off top 20 most ex­pen­sive drugs list

Martin Shkreli incited a national uproar five years ago when he raised the price of Daraprim by a factor of 56 essentially overnight from $13.50 to $750 per pill. Now that the “Pharma Bro’s” high-priced project has received a generic, it no longer places among the most expensive drugs in the world.

GoodRx is back with the latest update of the top 20 most expensive drugs and Daraprim’s exclusion marks the biggest change. The drug had previously ranked seventh on the list’s last iteration, which came in February before the world went into quarantine. Another of Shkreli’s former companies, Retrophin, saw its Chenodal drug place in the top 10 again.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.