End­points News re­cent­ly re­port­ed on a new pa­per in JA­MA that pur­ports to show that there is no cor­re­la­tion be­tween the price of a drug and its R&D costs. If the JA­MA ed­i­tors were to ap­ply sim­i­lar stan­dards of ev­i­dence to a pa­per about a drug’s clin­i­cal tri­al, they would be jus­ti­fi­ably crit­i­cized.

The pa­per cher­ry picks 60 out of hun­dreds of drugs, with the ex­cuse that the rel­e­vant da­ta for oth­er drugs ap­proved over the se­lect­ed 10-year pe­ri­od were dif­fi­cult to source. Imag­ine if a bio­phar­ma com­pa­ny on­ly pub­lished da­ta on 60 out of sev­er­al hun­dred par­tic­i­pants in a tri­al, claim­ing that the da­ta from the oth­ers were “dif­fi­cult to ac­cess.” The au­thors them­selves note an­oth­er sig­nif­i­cant weak­ness: that they did not have ac­cess to the net prices of many of the drugs in their sam­ple. This re­flects our sys­tem of drug re­bates, in which phar­ma­cy ben­e­fit man­agers, or PBMs, can re­ceive 30%-50% or more of a drug’s list price, leav­ing the drug de­vel­op­er with a sub­stan­tial­ly low­er ac­tu­al, or “net,” price.

More con­cern­ing, the au­thors’ study de­sign sim­ply ig­nored the fact that for every FDA-ap­proved drug, there are on av­er­age about nine oth­ers that made it in­to clin­i­cal tri­als, but failed to reach the mar­ket. Yet drug com­pa­nies must in­vest huge sums de­vel­op­ing the 90% of drug can­di­dates that ul­ti­mate­ly fail. The re­turn on in­vest­ment for each suc­cess­ful drug must take in­to ac­count the ex­pen­di­tures on all of them, in­clud­ing the in­evitable fail­ures, or in­vestors will not con­tin­ue to sup­port the de­vel­op­ment of new med­i­cines. There­fore, one should not ex­pect there to be a cor­re­la­tion be­tween an in­di­vid­ual drug’s de­vel­op­ment costs and its price.

Ron Co­hen, M.D.
Pres­i­dent and CEO
Acor­da Ther­a­peu­tics

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.