Is bio­phar­ma’s first $1.5M ther­a­py now in Phase III?

Key­ing off of Spark Ther­a­peu­tics’ roll­out of Lux­tur­na as the first ap­proved gene ther­a­py in the US, the an­a­lysts at Leerink have been crunch­ing the num­bers on what the first he­mo­phil­ia gene ther­a­pies could fetch. And by rais­ing their es­ti­mate to $1.5 mil­lion, they may well be tempt­ing fate at a time the de­bate over drug pric­ing is reach­ing the boil­ing point in the US.

Spark sat­is­fied most an­a­lysts with its $425,000 price per eye for Lux­tur­na. And Leerink says that the pos­i­tive re­sponse by Har­vard Pil­grim to a 30-month dura­bil­i­ty start­ing point for a gene ther­a­py pric­ing agree­ment set the stage for many of the rest of the field to fol­low suit. And that will jus­ti­fy a $1.5 mil­lion-plus price for a ther­a­py that could re­place cur­rent stan­dards in he­mo­phil­ia with a sim­i­lar track record on dura­bil­i­ty.

Af­ter check­ing with some KOLs in the field, Leerink of­fered:

As a re­sult of this work, we are rais­ing our as­sumed price for he­mo­phil­ia gene ther­a­pies in our cov­er­age uni­verse to $1.5M per pa­tient in the US ($1M in the EU), an in­crease from pri­or as­sump­tions of $350,000-$500,000 (he­mo­phil­ia A) and $500,000 (he­mo­phil­ia B) per pa­tient. Our checks in­di­cate that there may be some head­room above this price, po­ten­tial­ly up to $2M per pa­tient on a gross ba­sis, but giv­en the un­cer­tain­ty about re­spon­sive­ness, re­bates and oth­er fac­tors, we be­lieve $1.5M is a jus­ti­fi­able es­ti­mate for net price per pa­tient at this junc­ture….

We be­lieve the man­u­fac­tur­ers are test­ing the wa­ters cur­rent­ly as they em­bark on Phase 3 tri­als in or­der to de­ter­mine where pay­ors push back sig­nif­i­cant­ly. Based on our checks, a price some­where in the range of $1-2M per pa­tient seems most like­ly.

The big rea­son for that 7-fig­ure price tag is based on pay­ers’ will­ing­ness to cov­er the $580,000 to $800,000 price per pa­tient for fac­tor re­place­ment ther­a­py, giv­ing them some up­side even with a $1 mil­lion-plus stick­er for a once-and-done gene ther­a­py. And that could have a pro­found ef­fect on Spark $ONCE, Bio­Marin $BM­RN and uniQure $QURE, which are all en­gaged in de­vel­op­ment ef­forts.

As long as the av­er­age pa­tient can re­ly on fac­tor re­place­ment ther­a­py, Leerink adds, the low hang­ing fruit for the de­vel­op­ers ear­ly on will be among the most se­vere pa­tients, and stick­ing with that small pa­tient pop­u­la­tion al­so helps jus­ti­fy the big num­ber.

At this stage of the game, the biotechs lead­ing this field will like­ly hedge their bets, steer­ing clear of of­fer­ing any num­bers they may have in mind as they test the wa­ters on the fi­nal price tag. But Leerink just gave them a big op­por­tu­ni­ty to con­tin­ue their pri­vate pay­er talks with some big num­bers in mind.

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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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.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

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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