Bris­tol-My­ers is scout­ing new com­pa­ny star­tups; Ab­b­Vie and Idera team up to eval­u­ate com­bi­na­tion for HN­SCC

Bris­tol-My­ers Squibb has looped in a new part­ner to help scout new com­pa­ny cre­ation op­por­tu­ni­ties. As a lim­it­ed part­ner of Bio­Mo­tiv, the phar­ma gi­ant will work with the drug de­vel­op­ment ac­cel­er­a­tor to fund trans­la­tion­al ven­tures “in dis­ease ar­eas where un­met med­ical needs re­main” — and get first dibs to buy the re­sult­ing star­tups up­on lead iden­ti­fi­ca­tion. The deal gives Bris­tol-My­ers ac­cess to Bio­Mo­tiv’s net­work of aca­d­e­m­ic in­sti­tu­tions and their cut­ting-edge, ear­ly-stage dis­cov­er­ies, said Bruce Car, in­ter­im head of dis­cov­ery re­search.

One of Bio­Mo­tiv’s port­fo­lio com­pa­nies, Dual Ther­a­peu­tics, pre­vi­ous­ly en­ticed Bris­tol-My­ers Squibb to an on­col­o­gy pact that came with $255 mil­lion in mile­stones. The tie-up ap­peared to have fiz­zled, but Bris­tol-My­ers has con­tin­ued to get on the ground floor of biotech when­ev­er it could, for ex­am­ple by spon­sor­ing Bi­o­Lab‘s start­up fa­cil­i­ties.

→ It’s an­oth­er deal for Ger­man phar­ma Boehringer In­gel­heim. Af­ter ink­ing a re­search pact with MD An­der­son ear­li­er last month to cre­ate a “Vir­tu­al Re­search and De­vel­op­ment Cen­ter,” the com­pa­ny and Lupin Lim­it­ed have en­tered in­to a part­ner­ship for Lupin’s MEK in­hibitor com­pound (LNP3794) to be used in com­bi­na­tion with Boehringer’s KRAS in­hibitor for pa­tients with gas­troin­testi­nal and lung can­cers har­bor­ing a broad range of onco­genic KRAS mu­ta­tions.

The com­pa­ny says that “Lupin will re­ceive an up­front pay­ment of $20 mil­lion and po­ten­tial ad­di­tion­al pay­ments for suc­cess­ful achieve­ment of de­fined clin­i­cal, reg­u­la­to­ry and com­mer­cial mile­stones for a to­tal deal val­ue of more than $700 mil­lion.  Ad­di­tion­al­ly, Lupin will be en­ti­tled to re­ceive dou­ble-dig­it roy­al­ties on the sales of the prod­uct.”

→ Af­ter Ab­b­Vie read the last rites to their fi­nal, failed Ro­va-T clin­i­cal tri­al last week, the com­pa­ny is now col­lab­o­rat­ing with Idera Phar­ma to test com­bi­na­tions of an OX40 ag­o­nist (AB­BV-368) and a TLR-9 ag­o­nist (tilso­tolimod) — hop­ing to stim­u­late the im­mune sys­tem re­sult­ing in an­ti-tu­mor re­spons­es — in a Phase Ib study in pa­tients with re­cur­rent or metasta­t­ic head and neck squa­mous cell car­ci­no­ma (HN­SCC)

The study will test three sep­a­rate treat­ment arms: AB­BV-368 plus tilso­tolimod; AB­BV-368 plus tilso­tolimod and chemother­a­py (nab-pa­cli­tax­el); and AB­BV-368 plus tilso­tolimod, nab-pa­cli­tax­el and PD-1 an­tag­o­nist (AB­BV-181).

→ Los An­ge­les-based Puma Ther­a­peu­tics‘ drug Ner­l­ynx has been grant­ed or­phan drug sta­tus by the FDA for the treat­ment of breast can­cer pa­tients with brain metas­tases.

Back in May, in­vestor wor­ries cratered the com­pa­ny’s stock $PBYI when Q1 sales of the drug fell short of Wall Street es­ti­mates — with pa­tient dis­con­tin­u­a­tions run­ning high due to a poor tol­er­a­bil­i­ty pro­file, pre­dom­i­nate­ly re­lat­ing to di­ar­rhea. Ner­l­ynx was in-li­censed by Puma from Pfiz­er and was ap­proved by the FDA in Ju­ly 2017.

→ NICE has backed the NHS use of No­var­tis‘ gene ther­a­py Lux­tur­na for a rare, in­her­it­ed form of blind­ness in Eng­land and Wales. The one-off treat­ment, which was ap­proved in Eu­rope last No­vem­ber, has been sup­port­ed for use in chil­dren and adults who are liv­ing with vi­sion loss due to ge­net­ic mu­ta­tions in both copies of the RPE65 gene. No­var­tis bought the ex-US rights to the drug in 2018 for $170 mil­lion from Spark Ther­a­peu­tics.

→ The FDA has grant­ed re­gen­er­a­tive med­i­cine ad­vance ther­a­py (RMAT) des­ig­na­tion for Cam­bridge, Mass­a­chu­setts-based Ma­gen­ta Ther­a­peu­ticsMG­TA-456, a one-time cell ther­a­py for the treat­ment of mul­ti­ple in­her­it­ed meta­bol­ic dis­or­ders.

“The com­pa­ny is cur­rent­ly study­ing MG­TA-456 in a Phase II clin­i­cal tri­al in pa­tients old­er than 6 months of age with Hurler syn­drome, cere­bral adrenoleukody­s­tro­phy (cALD), metachro­mat­ic leukody­s­tro­phy (MLD) or globoid cell leukody­s­tro­phy (GLD) at four cen­ters of ex­cel­lence: Cincin­nati Chil­dren’s Hos­pi­tal, Duke Uni­ver­si­ty, Emory Uni­ver­si­ty and the Uni­ver­si­ty of Min­neso­ta.”

→ Re­genxbio has inked an op­tion and li­cense agree­ment for ex­clu­sive world­wide rights for Clear­side Bio­med­ical‘s SCS Mi­croin­jec­tor “for the de­liv­ery of RGX-314 to the supra­choroidal space to treat wet age-re­lat­ed mac­u­lar de­gen­er­a­tion (wet AMD), di­a­bet­ic retinopa­thy (DR) and oth­er con­di­tions for which an­ti-vas­cu­lar en­dothe­lial growth fac­tor (an­ti-VEGF) treat­ment is cur­rent­ly the stan­dard of care.”

The deal comes a lit­tle af­ter Clear­side Bio­med­ical an­nounced its pre­dic­tion that the FDA would re­ject its uveitis drug af­ter a re­quest for more da­ta.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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