Stick­er shock: No­var­tis says its top drug prospect is worth $4M-$5M for a once-and-done use

Val­ue and pric­ing in life-long rare dis­ease treat­ment. (No­var­tis, page 48)

AveX­is man­aged to grab the in­dus­try’s at­ten­tion when re­searchers post­ed ear­ly and stun­ning re­sults for their life-sav­ing gene ther­a­py to treat rare cas­es of spinal mus­cu­lar at­ro­phy. They were back in the spot­light when No­var­tis reached out to buy the com­pa­ny for $8.7 bil­lion. And now they’re mak­ing a re­turn trip to cen­ter stage with an ar­gu­ment that their once-and-done ap­proach to SMA — AVXS-101 — could jus­ti­fi­ably be priced at $4 mil­lion to $5 mil­lion.

Dave Lennon (LinkedIn)

“Four mil­lion dol­lars is a sig­nif­i­cant amount of mon­ey, but we be­lieve this is a cost-ef­fec­tive point,” Dave Lennon, pres­i­dent of AveX­is told in­vestors ear­li­er to­day, ac­cord­ing to a re­port from Reuters. “We’ve shown through oth­er stud­ies we are cost ef­fec­tive in the range of $4-$5 mil­lion. And ul­ti­mate­ly, this is im­por­tant con­text as we con­sid­er how we’re go­ing to eval­u­ate val­ue for (the ther­a­py called) AVXS-101.”

No­var­tis out­lined their ar­gu­ment in a slide that used price guide­lines from the cost watch­dogs at ICER and NICE to make their case. ICER, for ex­am­ple, backed Roche’s $482,000 first-year price for Hem­li­bra, with a 10-year cost of $4.5 mil­lion. But it’s hard to see ei­ther group go­ing for $4 mil­lion worth of stick­er shock for a one-time ther­a­py. ICER re­ject­ed Spark’s $850,000 price tag for its gene ther­a­py for a rare form of blind­ness as ex­treme and need­ed to be cut by at least 50% to re­flect its true val­ue to pa­tients. And NICE has had no prob­lem re­ject­ing oth­er rare dis­ease drugs with much small­er prices at­tached.

No­var­tis may not ac­tu­al­ly be plan­ning to try and col­lect $4 mil­lion for their drug, and ex­ecs de­murred on pro­ject­ing the cost to­day. But they seem in­tent on soft­en­ing up the pay­er mar­ket for some­thing sig­nif­i­cant­ly high­er than $1 mil­lion — once con­sid­ered a jaw-drop­ping, break-the-bank sum.

And that would put them at the very top of the heap of the most ex­pen­sive drugs on the plan­et.

In their fa­vor: Ri­vals at Bio­gen charge $750,000 for the first year of Spin­raza, then $375,000 a year af­ter that — for life. That’s $1.87 mil­lion over 4 years. And they don’t get much grief for it.

At the very least, you can ex­pect a whole new dis­cus­sion around the pric­ing of gene ther­a­pies, as de­vel­op­ers dis­cuss dif­fer­ent pay plans to spread out the cost or of­fer var­i­ous mon­ey-back guar­an­tees.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

Amgen VP of R&D David Reese

Am­gen rolls out da­ta for KRAS in­hibitor com­bo study in col­orec­tal can­cer, hop­ing to move on from ug­ly ear­ly re­sults

With the first win for its KRAS inhibitor sotorasib in hand, Amgen is pushing ahead with an aggressive clinical plan to capitalize on its first-to-market standing. The drugmaker thinks combinations — in-house or otherwise — could offer a path forward, and one early readout from that strategy is bearing fruit.

A combination of Amgen’s sotorasib and its EGFR inhibitor Vectibix posted an overall response rate of 27% in 26 patients with advanced colorectal cancer (CRC) with the KRAS-G12C mutation, according to data from the larger Phase Ib/II CODEBREAK 101 study set to present at this weekend’s virtual ESMO Congress.

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Boston skyline (Shutterstock)

Boston, the Bay Area and San Diego dom­i­nate the life sci­ences re­al es­tate mar­ket. Where to next?

With strong competition for life sciences real estate in key clusters — greater Boston, San Francisco and San Diego — where will the industry look to expand next? That’s the question that real estate company JLL sought to answer in its latest report, released on Wednesday.

JLL scored US biotech hubs on a variety of criteria to come up with this year’s ranking, including talent, industry depth, innovation and lab real estate dynamics. Unsurprisingly, they found that last year’s top three clusters remain unchanged. JLL predicts that these core clusters will be “immovable” for the foreseeable future, comparing them to the Silicon Valley of biotech.

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Multiple antibiotic resistant Pseudomonas aeruginosa bacterium

A new way to in­fil­trate (and de­stroy) some of the dead­liest drug-re­sis­tant bugs

About four years ago, Ruben Tommasi, the gregarious scientific chief of antibiotics startup Entasis, walked into a meeting with his top chemist and top biologist to chew over another batch of unchanging results.

“It felt like we were running the same experiment over and over,” Tommasi told Endpoints News. “We had all sort of come to that point in time where we felt like we were banging our heads against the wall.”

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Take­da scores a win for a rare type of lung can­cer, gear­ing up for a show­down with J&J

Four months after J&J’s infused drug Rybrevant scored the industry’s first win in a rare type of non-small cell lung cancer (NSCLC), Takeda is following up with an oral option for the small but desperate patient population.

The FDA granted an accelerated approval to Takeda’s oral TKI inhibitor Exkivity (mobocertinib) in metastatic NSCLC patients with EGFR exon 20 gene mutations who had previously undergone platinum-based chemotherapy, the company announced on Wednesday.

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Ali Tehrani, Zymeworks CEO

Zymeworks squares up with Her­ceptin af­ter HER2 bis­pe­cif­ic aces mid-stage test in esophageal can­cer

Roche’s Herceptin has long stood as standard of care across multiple advanced cancers, but a suite of next-gen players are looking to beat the aging giant at its own game. In HER2-expressing esophageal cancer, BeiGene partner Zymeworks thinks its bispecific antibody could have the juice to get it done.

Zymeworks’ bispecific antibody zanidatamab, combined with one of two chemotherapy regimens, posted an overall response rate of 75% in patients with advanced gastroesophageal adenocarcinoma (GEA) who had not previously received a HER2-targeted cancer therapy, the Vancouver-based biotech said Thursday.

UP­DAT­ED: Fourth boy dies in Astel­las-Au­dentes gene ther­a­py tri­al, rais­ing fresh fears for the field

A fourth boy has died in the Astellas-Audentes gene therapy trial for a rare muscle-wasting disease, the company announced Tuesday morning.

The death comes a little over a year after the first of three boys with myotubular myopathy, or XLMTM, died after receiving the therapy and suffering liver failure. The company was cleared to resume studies in December after laying out a plan to give patients a much lower dose and enrolled their first new volunteer over the summer.

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Patricio Massera, AGC Biologics CEO

AGC un­veils Ger­man ex­pan­sion plans as gene and cell ther­a­py man­u­fac­tur­ing com­pe­ti­tion heats up

AGC Biologics is significantly expanding a German site focusing on cell and gene therapies.

The Seattle company will add another cGMP manufacturing line at its Heidelberg facilities, which already feature 100-liter and 1,000-liter manufacturing capacities for microbial protein and cell and gene therapy. The site will add more warehouse capacity too, which AGC says will help the company prepare for the rapid increase in demand.

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Jonny Ohlson, Touchlight Genetics

DNA play­er Touch­light dou­bles fundrais­ing this year with lat­est round of pri­vate back­ers on board

When the news got out about Touchlight’s $60 million financing round in March, the most promising part of it all was the suggestion that the company could triple the production of its “doggybone” DNA and supply up to 1 billion Covid-19 shot doses per month.

Now, the company will have more than double that amount of money to work with.

The UK-based biotech that makes enzymatic DNA announced the extension of its fundraising round to a total of $125 million, the company announced. This comes at a time when demand for synthetic DNA has grown in the last year. Touchlight says the funding will help it become the largest synthetic DNA manufacturer in the world.

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