Roche is bring­ing back gan­tenerum­ab from the dead, tak­ing an­oth­er stab at Alzheimer’s PhI­II

Mar­lies Spro­ll, Mor­phoSys

Alzheimer’s drugs are ex­pen­sive to test and un­like­ly to suc­ceed, but they are al­so hard to kill.

More than two years af­ter gan­tenerum­ab failed de­ci­sive­ly in treat­ing ear­ly-stage Alzheimer’s, Roche is map­ping out an at­tempt­ed come­back through a new, piv­otal Phase III pro­gram that puts them back in­to the late-stage pipeline with their sec­ond ther­a­py.

In­ves­ti­ga­tors stub­born­ly vowed back at an in­ter­na­tion­al Alzheimer’s con­fer­ence in 2015 that if you amped up the dosage of the amy­loid be­ta an­ti­body it would be pos­si­ble to track a re­al treat­ment ef­fect for Alzheimer’s, im­prov­ing cog­ni­tion and func­tion. And now Roche part­ner Mor­phoSys, which con­tributed its plat­form tech in dis­cov­er­ing the drug, says the phar­ma gi­ant is go­ing for it — again.

The Ger­man biotech says that its con­tacts at Roche are plan­ning to launch two Phase III stud­ies in mild to pro­dro­mal pa­tients some­time lat­er in the year, stick­ing to a group of pa­tients who are just be­gin­ning to demon­strate symp­toms of the mem­o­ry-wast­ing ail­ment.

Roche nev­er gave up on gan­tenerum­ab. Roche neu­ro­science de­vel­op­ment chief Paulo Fon­toura tells me they’ve been us­ing two ex­tend­ed stud­ies to see if they can safe­ly use a much, much high­er dose need­ed to have an im­pact on the dis­ease with­out stir­ring up dan­ger­ous lev­els of ARIA-E, or brain swelling.

“We want­ed to find out if 4- or 5-fold (high­er dos­es) would be suc­cess­ful,” Fon­toura tells me, while con­trol­ling any cas­es of ARIA-E. And all in­di­ca­tions,he adds, is that they are on the right track.

Noth­ing has worked in Alzheimer’s R&D over the last 14 years, and gan­tenerum­ab looked like it would join a list of the most promi­nent drugs in the field to wash out of a big Phase III. But re­searchers have al­so been em­bold­ened by bet­ter di­ag­nos­tics to se­lect pa­tients as well as by the ear­ly da­ta from Bio­gen’s ad­u­canum­ab pro­gram which has shown glim­mers of ef­fi­ca­cy. Eli Lil­ly on­ly re­cent­ly wrapped its last piv­otal shot at solanezum­ab, its third straight fail­ure.

Mer­ck has al­so con­tributed to the drum­beat of fail­ures, re­cent­ly con­ced­ing de­feat in the most ad­vanced study of a BACE drug that tried to move up­stream in the dis­ease process, pre­vent­ing the pro­duc­tion of tox­ic lev­els of amy­loid be­ta. And Lund­beck flopped with its three Phase III stud­ies of their 5-HT6 an­tag­o­nist idalopir­dine, leav­ing Ax­o­vant as the last com­pa­ny to test that symp­to­matic ap­proach in a piv­otal study.

Why the ded­i­ca­tion? There are no drugs that can mod­i­fy the pro­gres­sion of Alzheimer’s and a big de­mand for any new symp­to­matic ther­a­pies that can slow the im­pact of the dis­ease, leav­ing the field wide open for a block­buster in­tro­duc­tion. And with every set­back, re­searchers in­sist that the same drugs could work un­der dif­fer­ent cir­cum­stances.

Now Roche will soon have two piv­otal pro­grams for Alzheimer’s back in the clin­ic, with gan­tenerum­ab run­ning along­side crenezum­ab.

“This is great news for Mor­phoSys. We are de­light­ed by the strong com­mit­ment to gan­tenerum­ab as a po­ten­tial new ther­a­py for Alzheimer’s dis­ease”, com­ment­ed Mar­lies Spro­ll, the chief sci­en­tif­ic of­fi­cer of Mor­phoSys AG. “The Hu­CAL-de­rived an­ti­body gan­tenerum­ab has prop­er­ties that we be­lieve make it a promis­ing can­di­date to treat Alzheimer’s dis­ease, and we look for­ward to learn­ing more about these new Phase III tri­als.”

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK; First plas­ma tri­al sug­gests mor­tal­i­ty re­duc­tion

Count NIH director Francis Collins out for any wager that the FDA would authorize a Covid-19 vaccine in October.

The discussion came up during a call with reporters because some states and local governments have been told by the CDC to have vaccination plans ready to go by Oct. 1. Pharma execs, most notably from Pfizer and BioNTech, have raised hopes about a licensure during that month; President Donald Trump last week sounded an optimistic note about having a vaccine on the market “right around” Election Day on Nov. 3 — or possibly before.

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Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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