Once re­ject­ed, Kala's dry eye drug now gains en­try to a field where No­var­tis is groom­ing its own block­buster

When the FDA slapped a re­jec­tion on Kala Phar­ma’s dry eye drug last Au­gust, the biotech’s ex­ecs promised in­vestors that a third Phase III study — they had al­ready start­ed at that point — would re­verse their for­tune.

Mark Iwic­ki

To­day they made good on that promise, clinch­ing an ap­proval for Ey­su­vis, an oc­u­lar cor­ti­cos­teroid be­ing po­si­tioned as a first-line, short term treat­ment of dry eye dis­ease.

Boast­ing a tech­nol­o­gy in­vent­ed by Bob Langer out of MIT, Ey­su­vis is a cor­ti­cos­teroid, lotepred­nol etabon­ate, de­liv­ered by mu­cus-pen­e­trat­ing par­ti­cles. It promis­es to en­hance pen­e­tra­tion in­to tar­get tis­sue on the oc­u­lar sur­face, achiev­ing an ef­fect quick­er than sys­temic cor­ti­cos­teroids and stronger than over-the-counter eye drops.

Kala, with $159.1 mil­lion in cash, cash equiv­a­lents and short-term in­vest­ments that it ex­pects to last un­til Q3 of 2022, ex­pects to launch the drug by year’s end pend­ing some fi­nal touch­es on the sales­force (125 reps to start with) and man­u­fac­tur­ing.

The top team, led by CEO Mark Iwic­ki, has re­peat­ed­ly em­pha­sized that the mar­ket is huge yet un­tapped, with just a few pre­scrip­tion drugs avail­able in the cat­e­go­ry. One of them, Shire’s Xi­idra, was bought by No­var­tis for $3.4 bil­lion up­front.

Here’s Iwic­ki from an earn­ings call in Au­gust:

Over 30 mil­lion peo­ple in the Unit­ed States live with dry eye dis­ease, of which over 17 mil­lion have al­ready been di­ag­nosed by an eye care pro­fes­sion­al.

Our mar­ket re­search sug­gests that 80% to 90% of these pa­tients suf­fer from episod­ic flares rather than con­tin­u­ous symp­toms, and feed­back from eye care pro­fes­sion­als sug­gest that these flares are – will be un­der-man­aged by cur­rent ther­a­pies.

In a note from Sep­tem­ber, Jef­feries an­a­lyst Biren Amin wrote that steroids are com­mon­ly pre­scribed off-la­bel for flares but risks of el­e­vat­ed in­traoc­u­lar pres­sure dis­cour­age non-corneal spe­cial­ists from do­ing so.

Da­ta from STRIDE3, the fi­nal Phase III study that fi­nal­ly sat­is­fied reg­u­la­tors, sug­gest­ed that Ey­su­vis in­duced a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in oc­u­lar dis­com­fort sever­i­ty over place­bo.

Doc­tors Amin spoke to “see the op­por­tu­ni­ty for it to be adopt­ed by corneal spe­cial­ists and should re­move the bar­ri­er for non-corneal spe­cial­ists to pre­scribe a steroid type treat­ment for DED flares.”

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.

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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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.