On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Mer­ck has notched an ap­proval for us­ing Keytru­da to treat a bio­mark­er-based sub­set of first-line col­orec­tal can­cer pa­tients with un­re­sectable or metasta­t­ic tu­mors, as the phar­ma gi­ant con­tin­ues to find new nich­es for its block­buster PD-1 star.

The OK is sig­nif­i­cant in a num­ber of ways. Not on­ly does it build on an ac­cel­er­at­ed ap­proval for all tu­mors char­ac­ter­ized as mi­crosatel­lite in­sta­bil­i­ty-high (MSI-H) or mis­match re­pair de­fi­cient (dMMR); it al­so marks the first sin­gle treat­ment for col­orec­tal can­cer that doesn’t con­tain chemother­a­py.

Richard Paz­dur

“Metasta­t­ic col­orec­tal can­cer is a se­ri­ous and life-threat­en­ing dis­ease with a poor prog­no­sis. Avail­able cur­rent ther­a­py with chemother­a­py com­bi­na­tions and oth­er bi­o­log­ics are as­so­ci­at­ed with sub­stan­tial tox­i­c­i­ty,” Richard Paz­dur, the face of the FDA’s fast-mov­ing on­col­o­gy unit, said in a state­ment. “Hav­ing a non-chemother­a­py op­tion avail­able for se­lect­ed pa­tients is a note­wor­thy par­a­digm shift in treat­ment.”

Keytru­da beat out a num­ber of chemo-based reg­i­mens in a head-to-head study read out re­cent­ly, al­most dou­bling the me­di­an pro­gres­sion-free sur­vival. Me­di­an PFS in the Keytru­da group was 16.5 months while the stan­dard-of-care arm — fea­tur­ing pair­ings like mFOL­FOX6 plus be­va­cizum­ab — lived 8.2 months be­fore pass­ing away or see­ing the can­cer re­turn.

The KEYNOTE-177 study is still on­go­ing to mea­sure over­all sur­vival.

The FDA grant­ed pri­or­i­ty re­view for the in­di­ca­tion just days af­ter Mer­ck tout­ed the topline win in ear­ly April. It al­so ush­ered the com­pa­ny down the re­al-time on­col­o­gy re­view (RTOR) pi­lot pro­gram, com­ing up with a thumbs up less than a month af­ter the of­fi­cial sBLA sub­mis­sion.

Around 5% of all metasta­t­ic col­orec­tal can­cer have MSI-H or dMMR tu­mors, which con­tain ab­nor­mal­i­ties that af­fect the prop­er re­pair of DNA in­side the cell. In 2017 Mer­ck made its first for­ay in­to the field with an ac­cel­er­at­ed OK for all can­cers bear­ing this fea­ture re­gard­less of where they orig­i­nate in the body.

Roy Baynes

More re­cent­ly Mer­ck moved in­to the more well-known space of tu­mor mu­ta­tion­al bur­den, notch­ing an ac­cel­er­at­ed ap­proval for un­re­sectable or metasta­t­ic TMB-high sol­id tu­mors.

The re­search team is com­mit­ted to un­earth yet more ways to iden­ti­fy pa­tients most like­ly to ben­e­fit from their check­point in­hibitor, “par­tic­u­lar­ly for those who have few avail­able op­tions,” said CMO Roy Baynes.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Eiger nabs the first FDA ap­proval for Prog­e­ria, an ul­tra-rare pre­ma­ture ag­ing dis­ease, with an old Mer­ck drug

Eiger BioPharmaceuticals $EIGR has received an FDA OK for a drug Merck licensed to them at no cost — and now reportedly plans to charge a level consistent with other ultra-rare disease medicines.

The biotech announced Friday evening that regulators had approved lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome, also known as Progeria, as well as some progeroid laminopathies in children older than one year. It’s the first approval granted for the condition, and the drug will be marketed and sold as Zokinvy.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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