Eli Lil­ly dish­es out first mile­stone pay­ment for Alzheimer's deal; ADC Ther­a­peu­tics, Viela Bio set terms for IPO

→ With oth­ers shy­ing away from the Alzheimer’s field af­ter the many clin­i­cal im­plo­sions and dis­as­ters, Eli Lil­ly is still chug­ging along with its $2 bil­lion deal with AC Im­mune — de­spite its share of BACE woes. AC Im­mune is set to re­ceive its first mile­stone pay­ment of CHF30 mil­lion, al­most a year af­ter the deal was inked. The com­pa­ny said that the pay­ment “is a recog­ni­tion of progress in the col­lab­o­ra­tion be­tween the two com­pa­nies and fol­lows ini­ti­a­tion in Ju­ly 2019 of the Phase 1 study of ACI-3024, a first-in-class in­ves­ti­ga­tion­al oral small mol­e­cule Tau Mor­phomer in de­vel­op­ment for treat­ment of Alzheimer’s dis­ease (AD) and oth­er neu­rode­gen­er­a­tive dis­or­ders.” A sec­ond mile­stone pay­ment of the same amount is sched­uled for Q1 of 2020.

→ Af­ter sign­ing a pact with Freenome and clos­ing a mon­ster $303 mil­lion E round in the last few months, ADC Ther­a­peu­tics set terms for its IPO on Mon­day. The Swiss com­pa­ny plans to of­fer 8.2 mil­lion shares at a price range of $23 to $26 to raise $200 mil­lion. In­sid­ers in­tend to pur­chase $115 mil­lion worth of shares in the of­fer­ing. At the mid­point of the pro­posed range, ADC would com­mand a mar­ket val­ue of $1.8 bil­lion.

As­traZeneca spin­out Viela Bio has closed in on the price range of its IPO, dis­clos­ing that it plans to of­fer 7.5 mil­lion shares be­tween $19 and $21 each. At the top of the range, the biotech will bring in $157.5 mil­lion to bankroll the de­vel­op­ment and com­mer­cial­iza­tion of its lead au­toim­mune drug, inebi­lizum­ab. The ful­ly di­lut­ed mar­ket val­ue (at the mid­point) could reach $1 bil­lion.

→ Is­raeli-based En­tera Bio has re­leased pos­i­tive da­ta for its oral parathy­roid hor­mone (PTH) drug Oral hPTH(1-34) from its Phase II study in pa­tients with hy­poparathy­roidism. Hav­ing test­ed four times dai­ly (QID) dos­ing in a pre­vi­ous study, the com­pa­ny want­ed to al­so com­pare twice dai­ly (BID) and three times dai­ly (TID) reg­i­mens and dose strengths.

“The re­sults showed that the largest changes in PD end­points were found with QID dos­ing, with mod­er­ate changes in PD end­points gen­er­al­ly found with BID and TID dos­ing,” stat­ed Pro­fes­sor Sofia Ish-Shalom, a prin­ci­pal in­ves­ti­ga­tor at the En­docrine Re­search Cen­ter at Lin Med­ical Cen­ter in Haifa, Is­rael. “In ad­di­tion, the QID dos­ing de­creased urine cal­ci­um over 24-hours, which in­di­cates that it may be able to re­duce uri­nary cal­ci­um in hy­poparathy­roid pa­tients with hy­per­cal­ci­uria dur­ing long-term treat­ment.”

→ In June, teplizum­ab (PRV-031) — which was re­ject­ed by Eli Lil­ly 9 years ago, but picked up by Proven­tion Bio — was shown to de­lay type 1 di­a­betes. Now, Proven­tion an­nounced that “based on writ­ten com­mu­ni­ca­tions from the FDA and the FDA’s des­ig­na­tion of PRV-031 as a break­through ther­a­py, the Com­pa­ny be­lieves that ex­ist­ing clin­i­cal and non-clin­i­cal da­ta for PRV-031 will be suf­fi­cient to sup­port a Bi­o­log­ics Li­cense Ap­pli­ca­tion (BLA) sub­mis­sion for PRV-031 in the fourth quar­ter of 2020 for the at-risk in­di­ca­tion.” The New Jer­sey-based com­pa­ny ex­pects to meet with the FDA in the fourth quar­ter of 2019 to dis­cuss this ex­pe­dit­ed plan.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,900+ biopharma pros reading Endpoints daily — and it's free.

David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,900+ biopharma pros reading Endpoints daily — and it's free.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,900+ biopharma pros reading Endpoints daily — and it's free.

Stephen Hahn (via Senate HELP Committee)

Stephen Hahn gets through Sen­ate’s soft­ball job in­ter­view — but most­ly plays dodge­ball on the is­sues fac­ing the FDA

Anyone looking for fresh insights on what kind of FDA commissioner Stephen Hahn will be got precious few clues during Wednesday’s Senate hearing on the nomination.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Op­di­vo/Yer­voy com­bo for melanoma fails in key pa­tient pop­u­la­tion

Bristol-Myers Squibb’s efforts to expand their checkpoint inhibitor combination have run into another recalcitrant cancer.

The NJ-based pharma announced that a combination of Yervoy and Opdivo didn’t beat out Opdivo alone in patients with resected high-risk melanoma who had very low levels of PD-L1. The drug combo couldn’t improve recurrence-free survival in these post-surgery patients.

Ver­tex's stel­lar quar­ter car­ries on with French re­im­burse­ment deal

Vertex’s golden quarter just got brighter. About a month after the US drugmaker finally clinched a deal with UK authorities to cover its slate of cystic fibrosis (CF) drugs following years of protracted negotiations, the company on Wednesday secured a deal with France for its CF therapy, Orkambi.

After the UK, France has one of the largest CF populations outside the United States. Achieving French reimbursement unlocks an ~7000-patient CF population, around ~2500-3000 of which will likely be eligible to receive (and be reimbursed for) Orkambi, Stifel’s Paul Matteis wrote in a note.

Nello Mainolfi, Kymera via Youtube

Kymera hands the helm to No­var­tis vet — and found­ing CSO — Nel­lo Main­olfi

Kymera Therapeutics is turning to a co-founder to run the company.
The protein degradation specialist with a deep-pocket syndicate behind them has opted to give the helm officially to Nello Mainolfi. The new CEO is a veteran of the Novartis Institutes for Biomedical Research. He joined Atlas Venture in their entrepreneur-in-residence program and helped launch Kymera as the CSO three years ago with Atlas’ Bruce Booth.
The boast at Kymera is that they’re angling to create a new class of protein degraders, a popular field where there’s been a variety of startups. One of its chief advocates is NIBR head Jay Bradner, who launched C4 just ahead of joining Novartis, where he’s also been doing new work in the field.