Jeffrey Nau, Oyster Point CEO (Oyster Point Pharma)

Small biotech Oys­ter Point sets up pitch to dis­rupt dry eye mar­ket with nasal spray

Oys­ter Point Phar­ma — a small Prince­ton, NJ-based biotech look­ing to dis­rupt a mas­sive eye drug mar­ket large­ly mo­nop­o­lized by two ther­a­pies — has pos­i­tive piv­otal tri­al da­ta as it march­es on a path to­wards US ap­proval.

Oys­ter, which went pub­lic in an $80 mil­lion IPO last year, has a nasal for­mu­la­tion of vareni­cline —a for­mu­la­tion of Pfiz­er’s smok­ing-ces­sa­tion drug Chan­tix — it is de­vel­op­ing for dry eye dis­ease, a chron­ic, episod­ic dis­ease af­fect­ing the tears and oc­u­lar sur­face that af­fects some 33 mil­lion Amer­i­cans.

The con­di­tion cur­rent­ly rep­re­sents a glob­al mar­ket of more than $5 bil­lion and is ex­pect­ed to grow to about $8 bil­lion by 2025, ac­cord­ing to Need­ham an­a­lysts. The ther­a­peu­tic mar­ket is dom­i­nat­ed by an­ti-in­flam­ma­to­ry eye drops: Al­ler­gan’s Resta­sis — al­though gener­ic com­pe­ti­tion is eat­ing in­to sales as Ab­b­Vie buys the large drug­mak­er — and Shire’s Xi­idra (pur­chased last year by No­var­tis in a $5.3 bil­lion deal), as well as over-the-counter ar­ti­fi­cial tears.

Two dos­es of Oys­ter’s for­mu­la­tion, called OC-01, (0.6 mg/ml and 1.2 mg/ml) were test­ed against a place­bo in 758 pa­tients with mild, mod­er­ate, and se­vere dry eye dis­ease in the ON­SET-2 study.

Both dos­es met the main goal of the study — a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in sub­jects gain­ing > 10 mm in Schirmer’s score (a test de­signed to as­sess a per­son’s eyes make enough tears to keep the eyes moist) at week 4, ver­sus the con­trol arm. In the small­er dose group, the per­cent­age of pa­tients hit­ting that score was 44%, and on the high­er group the per­cent­age of pa­tients hit­ting the goal was 47% — in the con­trol group, the per­cent­age was 26%.

In ad­di­tion, in the small­er dose group, the mean change in Schirmer’s score was 11.0 mm. In the high­er dose group the mean change in score was 11.2 mm — in the con­trol group, the change was 5.9 mm. Key sec­ondary goals, in­clud­ing an im­prove­ment in eye dry­ness, were al­so met. The most com­mon side ef­fect, ex­pe­ri­enced by half the pa­tients, was sneez­ing.

The ex­ist­ing crop of drugs such as Xi­idra and Resta­sis can take weeks or months to take ef­fect. Eye con­sul­tants in­di­cate that Resta­sis and Xi­idra have fail­ure rates of 50%+ and tol­er­a­bil­i­ty is­sues which fur­ther lim­it their use, and nei­ther prod­uct is com­plete­ly ef­fec­tive for all pa­tients, Cowen’s Ken Cac­cia­tore wrote in a note in late March, es­ti­mat­ing OC-01 rep­re­sents a $1 bil­lion-plus op­por­tu­ni­ty giv­en the need for new treat­ment al­ter­na­tives.

The ON­SET-2 study adds to mixed but large­ly pos­i­tive mid-stage tri­al da­ta on OC-01 — and Oys­ter is prep­ping to sub­mit a mar­ket­ing ap­pli­ca­tion in the sec­ond quar­ter.

“The abil­i­ty to show sta­tis­ti­cal­ly sig­nif­i­cant sign and symp­tom end­points with­in the same clin­i­cal tri­al has been elu­sive in dry eye dis­ease,” said com­pa­ny chief Jef­frey Nau in a state­ment. “The abil­i­ty to meet this high bar in the ON­SET-2 pop­u­la­tion con­sist­ing of mild, mod­er­ate, and se­vere sub­jects is even more no­table and speaks to the broad ap­plic­a­bil­i­ty of OC-01 to treat dry eye pa­tients.”

For ex­am­ple, mixed tri­al re­sults prompt­ed the FDA to ask for ad­di­tion­al ef­fi­ca­cy da­ta on Xi­idra, which were even­tu­al­ly sup­plied. On­ly then did the agency sanc­tion ap­proval in 2016.

An­oth­er po­ten­tial dry eye dis­ease drug, Kala Phar­ma­ceu­ti­cals’ Ey­su­vis, which is be­ing de­vel­oped for acute use, al­so had a mixed dataset, which prompt­ed a re­jec­tion last Au­gust. In March, the com­pa­ny — one of the pletho­ra of biotechs that trace their roots to the pro­lif­ic lab at MIT led by Bob Langer — post­ed da­ta from an­oth­er late-stage study as it looks to re­sub­mit its mar­ket­ing ap­pli­ca­tion.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Al Sandrock, Voyager Therapeutics CEO

Al San­drock prunes his post-Bio­gen voy­age with sim­i­lar fo­cus on ALS

Al Sandrock is narrowing the focus of Voyager Therapeutics, concentrating on CNS diseases that were the hallmark of his time leading R&D at Biogen, including an emphasis on a familial form of ALS for which his former employer is getting a speedy review at the FDA.

Less than six months into his journey as CEO at Voyager, Sandrock is focusing the preclinical pipeline on Alzheimer’s disease, GBA1 Parkinson’s disease and SOD1 amyotrophic lateral sclerosis, the rare form of ALS for which the FDA will decide whether to approve Biogen’s tofersen by Jan. 25, 2023.

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Pfiz­er ter­mi­nates PhI­II study of rare car­dio­vas­cu­lar drug picked up in $11.4B Ar­ray ac­qui­si­tion

While Pfizer’s $11.4 billion acquisition of Array BioPharma in the summer of 2019 was mainly focused on oncology, namely Braftovi and Mektovi, there were a few non-cancer assets, including a Phase III drug being tested in a rare cardiovascular disease.

The late-stage trial is now being axed, alongside any further development of the oral small molecule, the pharma giant disclosed after the closing bell on Wednesday. Based on an interim futility analysis of the global Phase III REALM-DCM trial, Pfizer determined a path forward was not in its best interest. Pfizer no longer expected the study would meet its primary goal.

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