Am­i­cus eyes Pompe ap­proval; Atea ends Dengue work; 2sev­en­ty, Tra­vere line up of­fer­ings

Am­i­cus Ther­a­peu­tics be­gan March with word that the FDA has sched­uled a pre-ap­proval in­spec­tion of its po­ten­tial treat­ment for Pompe dis­ease. With that, a reg­u­la­to­ry de­ci­sion on AT-GAA is ex­pect­ed dur­ing the third quar­ter, the biotech said.

Am­i­cus said last Oc­to­ber the FDA wasn’t able to do the re­quired in­spec­tion of its man­u­fac­tur­ing part­ner, WuXi Bi­o­log­ics in Chi­na, dur­ing the re­view time­frame be­cause of “re­stric­tions on trav­el re­lat­ed” to the pan­dem­ic. AT-GAA con­sists of bi­o­log­ic cipaglu­cosi­dase al­fa and miglu­s­tat, a sta­bi­liz­er of the bi­o­log­ic. The ap­proval re­quest in­cludes both a BLA and NDA.

AT-GAA has break­through ther­a­py des­ig­na­tion for late-on­set Pompe dis­ease. Reg­u­la­to­ry re­views are on track in the EU and UK, Am­i­cus said, with ap­proval de­ci­sions al­so ex­pect­ed in the third quar­ter. — Kyle LaHu­cik

2sev­en­ty looks to raise $125M in pub­lic of­fer­ing for R&D

2sev­en­ty bio has priced more shares to work on R&D along with its Bris­tol My­ers-part­nered ther­a­py.

The blue­bird bio on­col­o­gy spin­out said late Tues­day night that it will be of­fer­ing more than 10.8 mil­lion shares at $11.50 each, seek­ing to raise ap­prox­i­mate­ly $125 mil­lion. The com­pa­ny said that un­der­writ­ers would al­so get ac­cess to pur­chase an ad­di­tion­al 1.6 mil­lion shares as a 30-day op­tion.

2sev­en­ty said in its an­nounce­ment that it plans to close the of­fer­ing on or around March 3.

Shares of $TVST closed at $13.49 Tues­day af­ter­noon.

2sev­en­ty wrote in an SEC fil­ing that it plans to use the mon­ey raised for R&D to ad­vance prod­uct can­di­dates and ex­pand oth­er de­vel­op­ment pro­grams. Ad­di­tion­al­ly, the biotech said it would be in­vest­ing to ad­vance Kar­M­MA tri­als in a bid to test Abec­ma in ear­li­er lines of ther­a­py — along with in­vest­ing in­to Abec­ma’s com­mer­cial ac­tiv­i­ties as part of 2sev­en­ty’s cost-shar­ing agree­ment with Bris­tol My­ers Squibb.

The pair rolled out ear­li­er-line da­ta in Abec­ma last month, show­ing the tri­al met the pri­ma­ry end­point of pro­gres­sion-free sur­vival in pa­tients with sec­ond to fourth line mul­ti­ple myelo­ma. Abec­ma is cur­rent­ly on­ly in­di­cat­ed in the fifth-line and lat­er set­ting. — Paul Schloess­er

Tra­vere takes ap­proval to fundrais­ing route in bid for $200M

Af­ter the FDA green­lit Tra­vere Ther­a­peu­tics’ IgAN treat­ment sparsen­tan less than two weeks ago, the biotech is go­ing for cash to get things mov­ing.

The biotech said Tues­day night that it is of­fer­ing 8,275,000 shares at $21 a share — to raise a to­tal of $200 mil­lion in a pub­lic of­fer­ing. Tra­vere wrote in SEC doc­u­ments that the funds are planned to go to­wards sparsen­tan’s com­mer­cial launch, along with oth­er “gen­er­al cor­po­rate pur­pos­es” such as clin­i­cal tri­als and man­u­fac­tur­ing.

Brand­ed as Filspari, the can­di­date was ap­proved with a warn­ing of liv­er and fe­tal tox­i­c­i­ty.

The FDA is al­so re­quir­ing Tra­vere to run REMS, a risk eval­u­a­tion and mit­i­ga­tion strat­e­gy, in con­junc­tion with the ap­proval — re­quir­ing doc­tors to get cer­tain kinds of train­ing and hav­ing pa­tients en­tered in­to a mon­i­tor­ing pro­gram.

Shares of $TVTX closed at $22.16 Tues­day af­ter­noon. — Paul Schloess­er

Atea stops work on Dengue to ex­tend cash run­way

Atea said the costs and time as­so­ci­at­ed with run­ning Phase II stud­ies are too much for the biotech. It will fo­cus its mon­ey and time on Covid-19 and HCV, the Boston drug de­vel­op­er said Tues­day af­ter the mar­ket closed.

“We be­lieve AT-752 holds promise for the treat­ment of dengue as an oral di­rect act­ing an­tivi­ral. How­ev­er, it has be­come clear that im­proved di­ag­nos­tics are need­ed to bet­ter iden­ti­fy pa­tients ear­li­er in the course of the dis­ease,” CEO Jean-Pierre Som­ma­dos­si said in a state­ment. “In ad­di­tion, giv­en the high vari­abil­i­ty in both treat­ment and pro­phy­lax­is set­tings, sub­stan­tial­ly larg­er pa­tient sam­ple sizes would be re­quired for fu­ture Phase 2 stud­ies.”

The biotech has run in­to hur­dles be­fore with its Covid-19 pro­gram, lead­ing for­mer part­ner Roche to walk away from an an­tivi­ral. The com­pa­ny moved for­ward with­out the Big Phar­ma, work­ing on a Phase III tri­al test­ing be­m­ni­fos­bu­vir for treat­ment of Covid-19 in pa­tients at high-risk. An in­ter­im analy­sis is due in the sec­ond half of this year, Atea said.

Mean­while, a Phase II of be­m­ni­fos­bu­vir in com­bi­na­tion with ruzasvir will start en­roll­ment this quar­ter and have ini­tial da­ta in the last few months of 2023, Atea said, as it in­ves­ti­gates the pair as a treat­ment for HCV. — Kyle LaHu­cik

Ver­tex lines up ADC deal with Im­muno­gen 

An­ti­body-drug con­ju­gate biotech Im­muno­Gen is let­ting Ver­tex use its ADC tech to find new tar­get­ed con­di­tion­ing agents to be paired with gene edit­ing.

Af­ter a re­search pe­ri­od, Ver­tex can snag the world­wide, ex­clu­sive li­cense for R&D and po­ten­tial com­mer­cial­iza­tion of the con­di­tion­ing agents. Im­muno­Gen gets $15 mil­lion in ex­change to start. An­oth­er $337 mil­lion could come its way via op­tion fees, de­vel­op­ment mile­stones and com­mer­cial biobucks. Tiered roy­al­ties, too.

Ver­tex cell and gene ther­a­pies SVP Mike Cooke said the tech will be in­ves­ti­gat­ed with the bio­phar­ma’s exa-cel, which is be­ing test­ed for treat­ing sick­le cell and trans­fu­sion-de­pen­dent be­ta tha­lassemia. Ver­tex and part­ner CRISPR Ther­a­peu­tics are ask­ing the FDA and oth­er agen­cies to ap­prove the gene-edit­ed ther­a­py. — Kyle LaHu­cik

Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

Roger Perl­mut­ter lines up deals, fresh fund­ing at Eikon; Sec­ond RSV vac­cine ap­proved; Sev­er­al biotechs flash­ing red; 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.

As you come back to our website this weekend for ASCO news, don’t forget to check out our updated event lineup at BIO, which will cover everything from the current state of VC investing in biotech to top pharma R&D chiefs discussing how to make pipeline decisions.

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Peter van de Sande, Synaffix CEO

Lon­za shells out $107M cash to snap up Synaf­fix and its ADC plat­form

After lining up a string of partnerships over the years, Dutch antibody-drug conjugate specialist Synaffix has found a new home: Lonza, the contract development and manufacturing giant.

Lonza is paying about $107 million (€100 million) in cash to acquire Synaffix, with up to $64 million (€60 million) in “additional performance-based consideration” on the table. Synaffix’s ADC tech platform will now become part of Lonza’s offering for biopharma clients, lending its bioconjugate technologies to not just ADCs but also targeted gene therapy, immune cell engagers and other applications.

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Full TIG­IT da­ta from Gilead, Ar­cus show low­er PFS rates than De­cem­ber read­out: #AS­CO23

CHICAGO — Gilead and Arcus unveiled a fuller snapshot of a Phase II study testing their experimental cancer immunotherapy combo that showed lower progression-free survival rates than its previous update, results that are likely to spark further debate over the closely-watched clinical trial.

Last December, the anti-TIGIT/anti-PD-L1 combo, positioned as a first-line treatment for non-small cell lung cancer, recorded data that drew mixed reactions. The latest analysis, presented Saturday afternoon at ASCO, included only a handful more patients than the previous update, but PFS rates fell — in one cohort by nearly three months.

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Bris­tol My­er­s' Op­di­vo keeps can­cer at bay in more lym­phoma pa­tients than Seagen's Ad­cetris in PhI­II: #AS­CO23

CHICAGO — In a study pitting Seagen’s Adcetris against Bristol Myers Squibb’s Opdivo in newly diagnosed patients with advanced classic Hodgkin lymphoma, a greater proportion of those who received Opdivo saw no cancer growth at one year compared to those who got Adcetris.

In addition, patients in the Opdivo arm of the Phase III trial reported reduced toxicities, according to lead investigator Alex Herrera, a hematologist-oncologist at City of Hope’s cancer cancer in Duarte, CA. Notably, the trial included more than 200 children across both arms. Generally, more than half of children with advanced Hodgkin lymphoma receive radiation therapy, but in this trial, dubbed SWOG S1826, only a handful of patients in the two arms received radiotherapy, sparing many children from long-term side effects of radiation.

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Servi­er’s vo­rasi­denib stalls pro­gres­sion of brain can­cer by 61% in piv­otal PhI­II IN­DI­GO study: #AS­CO23

An experimental pill from Servier Pharmaceuticals showed potentially practice-changing results in a narrow group of brain cancer patients, cutting the risk of their cancers progressing by 61%, according to a late-stage clinical trial.

The drug, vorasidenib, is a precision medicine that only works in certain people whose cancer carries mutations in one of two genes called IDH1/2. Doctors hope that the therapy will delay the need for chemotherapy or radiation, which are often used to combat relapses in patients who’ve previously undergone surgery to remove brain tumors.

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As­traZeneca tri­al shows mod­est ben­e­fit in ovar­i­an can­cer, but doc­tors say it's hard to ap­ply find­ings: #AS­CO23

CHICAGO — Adding AstraZeneca’s Imfinzi and Lynparza to the treatment regimen for patients with advanced ovarian cancer and no BRCA mutation extended progression-free survival (PFS) by five months, according to interim data released at the ASCO annual meeting Saturday morning.

However, the design of the Phase III study obscures how much Imfinzi is contributing to the PFS extension, doctors said, making it difficult to apply the findings to clinical practice.

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Keytru­da be­fore and af­ter lung can­cer surgery cuts re­lapse risk by 42%, but doesn’t im­prove sur­vival: #AS­CO23

CHICAGO — Merck has found partial success with its latest effort to more aggressively treat earlier stages of lung cancer.

On Saturday the pharma giant announced results from a large trial in which patients received Merck’s immunotherapy Keytruda plus chemotherapy before surgeons removed their tumors, followed by another course of Keytruda afterward.

The Phase III study, called KEYNOTE-671, enrolled 800 people with the early stages of the most common kind of lung cancer: non-small cell lung cancer, or NSCLC. Everyone got chemo before surgery, and half also got Keytruda before and after. At two years, 62.4% of those who got Keytruda kept their cancer at bay, compared to 40.6% who got a placebo.

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Grail’s blood test charts path for di­ag­nos­ing pa­tients sus­pect­ed of hav­ing can­cer in large study: #AS­CO23

Grail’s vision is simple but bold. The blood testing company has long held that people are often diagnosed with cancer too late. If seemingly healthy people were screened for early signs of the disease before symptoms appear, they may be able to get more effective treatments that nip cancer in the bud.

That premise is the basis of Grail’s commercial blood test, Galleri, which searches for the genetic fingerprints of cancer in the blood. The test, launched in 2021, reaped $55 million in sales last year, but now the company is setting its sights on a new market: patients suspected of having cancer due to symptoms such as abdominal pain, rectal bleeding or unexplained weight loss. Rather than administering expensive scans or conducting invasive biopsies right away, Grail hopes doctors will consider a simple blood test.

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