Busy IPO week winds down as two more biotechs — and one look­ing to im­prove clin­i­cal tri­als — file for pub­lic de­buts

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Two more biotech com­pa­nies — and one that’s com­mit­ted to sup­port­ing clin­i­cal re­search — filed for IPOs just be­fore the long week­end.

No­bel lau­re­ate Jen­nifer Doud­na’s Cari­bou Bio­sciences is pen­cil­ing in a $100 mil­lion raise to sup­port its off-the-shelf CAR-T ther­a­pies, one of which is mak­ing its way through a Phase I study in B-cell non-Hodgkin lym­phoma. Al­so look­ing for a $100 mil­lion raise is WCG Clin­i­cal Ser­vices, which es­tab­lished it­self as an al­ly to bio­phar­ma com­pa­nies and CROs back in 2012. The com­pa­ny of­fers plan­ning, pa­tient en­gage­ment, and eth­i­cal re­view ser­vices, and says it was be­hind more than 723 Covid-19 tri­als. Mean­while, transpa­cif­ic biotech Brii Bio­sciences is look­ing for its own tick­er on the Hong Kong Stock Ex­change, and says it’ll use the funds across its broad in­fec­tious dis­ease and CNS pipeline.

Here’s what you need to know about the lat­est set of com­pa­nies gear­ing up for a pub­lic de­but:

Jen­nifer Doud­na-launched Cari­bou Bio­sciences says it’s ready for Wall Street

When Cari­bou Bio­sciences nabbed a $115 mil­lion Se­ries C round back in March, CEO Rachel Hau­r­witz told End­points News an IPO was “con­ceiv­able one day.” Now, that day has come.

Rachel Hau­r­witz

Hau­r­witz sub­mit­ted Cari­bou’s S-1 pa­pers on Thurs­day, pen­cil­ing in a $100 mil­lion raise — though, in this mar­ket, it wouldn’t be sur­pris­ing if the com­pa­ny ends up with more. The funds will give a boost to the com­pa­ny’s three off-the-shelf CAR-T ther­a­pies, the most ad­vanced of which is an an­ti-CD19 can­di­date in Phase I for B-cell non-Hodgkin lym­phoma.

“It’s been in par­tic­u­lar over the past few years that we’ve re­al­ly fo­cused on us­ing this next-gen­er­a­tion CRISPR tech­nol­o­gy plat­form to de­vel­op our own pipeline of off-the-shelf cell ther­a­pies for on­col­o­gy,” Hau­r­witz said in March.

The com­pa­ny got its start in Jen­nifer Doud­na’s lab at Cal-Berke­ley, where Hau­r­witz was Doud­na’s first grad­u­ate stu­dent to work on CRISPR. In 2011, they launched the com­pa­ny with James Berg­er (who’s now at the Johns Hop­kins School of Med­i­cine) and Mar­tin Jinek.

What sets Cari­bou apart is its hy­brid DNA/RNA edit­ing plat­form, which Hau­r­witz calls chRD­NA (pro­nounced “chardon­nay,” like the wine). The biotech be­lieves the hy­brid guides can pre­vent off-tar­get edit­ing com­mon to cur­rent CRISPR ap­pli­ca­tions.

Some of the IPO pro­ceeds will go to IND-en­abling ac­tiv­i­ties for Cari­bou’s oth­er two can­di­dates, CB-011 and CB-012. The for­mer tar­gets BC­MA for the treat­ment of re­lapsed/re­frac­to­ry mul­ti­ple myelo­ma and us­es “im­mune cloak­ing” to avoid the im­mune sys­tem. That can­di­date is ex­pect­ed to hit the clin­ic next year. CB-012 goes af­ter CD371, which Cari­bou says is an at­trac­tive tar­get for acute myeloid leukemia.

The com­pa­ny’s al­so look­ing to go be­yond on­col­o­gy, and they’re ex­plor­ing the ar­eas of im­mune cell ther­a­py, iP­SC, and in vi­vo-gene edit­ing. In Feb­ru­ary, Ab­b­Vie shelled out $40 mil­lion up­front with an­oth­er $300 mil­lion in biobucks for two of the biotech’s al­lo­gene­ic CAR-Ts.

“We view this col­lab­o­ra­tion as an ex­ter­nal recog­ni­tion of the po­ten­tial for our chRD­NA genome-edit­ing tech­nol­o­gy to sig­nif­i­cant­ly im­prove genome-edit­ing speci­fici­ty and ef­fi­cien­cy,” Cari­bou’s S-1 states.

Months af­ter a $155M raise, trans-Pa­cif­ic Brii Bio­sciences looks to make its pub­lic de­but

Word spread last Au­gust that Brii Bio­sciences was con­sid­er­ing a Hong Kong IPO. Now, al­most a year lat­er, the com­pa­ny has filed its pa­pers.

Zhi Hong

Zhi Hong launched Brii back in 2018 to bridge the gap in in­no­va­tion be­tween the US and Chi­na. The trans-Pa­cif­ic biotech hooked a $155 mil­lion Se­ries C round in March to fund its broad in­fec­tious dis­ease pipeline and CNS pro­gram. And now, it plans to of­fer about 111.6 mil­lion shares glob­al­ly, ac­cord­ing to the fil­ing. 

“Since our found­ing in 2018, Brii Bio has made tremen­dous progress to­ward our mis­sion to ac­cel­er­ate the de­vel­op­ment and de­liv­ery of break­through med­i­cines through part­ner­ships and our own in­sight in high­ly dif­fer­en­ti­at­ed med­i­cine dis­cov­ery,” Hong said in a March state­ment.

A ma­jor­i­ty of the IPO pro­ceeds would fund Brii’s he­pati­tis B pro­grams, in­clud­ing its core prod­uct BRII-179 which Brii in-li­censed from VBI Vac­cines short­ly af­ter its launch. Last week, Brii said the can­di­date was “well-tol­er­at­ed” in a Phase Ib/IIa study for chron­ic HBV pa­tients, and in­duced B and T cell re­spons­es when ad­min­is­tered alone or in com­bi­na­tion with in­ter­fer­on-al­pha.

Brii kicked off an­oth­er Phase II chron­ic HBV study as­sess­ing BRII-179 along with BRII-835, its can­di­date li­censed from Vir, back in April. The com­pa­ny plans to launch two more Phase II stud­ies this year test­ing BRII-835 in com­bo with oth­er agents.

Brii’s oth­er in­fec­tious dis­ease pro­grams range from HIV to Covid-19. Back in March, the NIH eject­ed Brii’s Covid-19 an­ti­body com­bo from its AC­TIV-3 tri­al, af­ter the cock­tail failed to show a trend to­ward clin­i­cal ben­e­fit. The NIH found no safe­ty is­sues with the treat­ment — which con­tained two Brii an­ti­bod­ies dubbed BRII-196 and BRII-198 — and said the ther­a­py would con­tin­ue in the AC­TIV-2 tri­al in mild-to-mod­er­ate Covid-19 pa­tients who don’t need to be hos­pi­tal­ized.

WCG Clin­i­cal Ser­vices seeks to solve ‘pain points’ in clin­i­cal tri­als

WCG Clin­i­cal Ser­vices launched back in 2012 as an al­ly to bio­phar­ma com­pa­nies and con­tract re­search or­ga­ni­za­tions, seek­ing to ad­dress what it calls “crit­i­cal pain points” in clin­i­cal tri­al per­for­mance. Now, it’s look­ing for its own stock tick­er.

Don­ald Deieso

The Prince­ton, NJ-based com­pa­ny filed an S-1 on Thurs­day, pen­cil­ing in a $100 mil­lion raise. It claims to have sup­port­ed around 90% of glob­al clin­i­cal tri­als that took place over the last two years, pro­vid­ing ser­vices like study plan­ning, pa­tient en­gage­ment, and eth­i­cal re­view. The team par­tic­i­pat­ed in more than 723 Covid-19 tri­als, ac­cord­ing to CEO Don­ald Deieso.

The com­pa­ny aims to tack­le is­sues like slow en­roll­ment, “bur­den­some ad­min­is­tra­tive process­es,” and the un­der-rep­re­sen­ta­tion of mi­nor­i­ty pa­tients.

“We firm­ly be­lieve that we must have the clin­i­cal in­sight to de­vel­op, the courage to ad­vance, and the per­sis­tence to trans­form a change-re­sis­tant in­dus­try, while nev­er com­pro­mis­ing the high­est lev­el of eth­i­cal stan­dards,” Deieso said in the S-1.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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